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6 episodes with Ionis
6 episodes with Ionis
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3 episodes with Ionis
6 episodes with Ionis
3 episodes with Ionis
3 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
2 episodes with Ionis
20 episodes with Ionis
2 episodes with Ionis
In this month's episode of the HAE Speaks Podcast, one family shares their journey of living with Hereditary Angioedema (HAE) together. From navigating diagnosis and treatment to supporting one another through the challenges of daily life, they offer an honest conversation about what it means to face HAE as a family. Through their shared experiences, listeners will gain insight into the power of resilience, the importance of understanding each family member's perspective, and the strength that comes from navigating HAE together.Thank you to our 2026 Sponsors: BioCryst, CSL, IONIS, and Takeda
On today's episode, Yaron Werber, John Maraganore, Sam Fazeli, and Matt Gline open with a discussion of biotech market volatility, with Matt noting that it often seems driven more by opaque factor dynamics than company-specific fundamentals. The group then turns to Eli Lilly's continued “Amazonification” and reinventing the pharma business as it acquires a variety of different companies, including this week's $2.8B upfront acquisition of Atai Beckley, a psychedelic-focused company. The conversation turns to BioCentury's reporting on Asia-to-West NewCos, prompting a debate about whether China is uniquely changing the market or simply reflects broader shifts in cheaper, faster development. On policy, the group covers BIO's response to the OMB proposal that could inject political review into federal grant decisions. The co-hosts also debate Kalshi's move to create prediction markets around clinical trial and regulatory outcomes. On pipeline updates, the group discusses Merck's approval of Lipfendra, the first oral PCSK9 drug, and the broader class implications. In CNS, Biogen and Ionis' diranersen tau ASO data spark a discussion of aconfusing dose response, ASO tolerability, tau as a target in Alzheimer's disease, and the promise of alternative modalities from Arrowhead and Alnylam. The episode closes with M&A and financing, including AstraZeneca's licensing deal with Dizal for the EGFR exon 20 inhibitor sunvozertinib andErasca's RAF data plus its $500M financing, which Sam views as a strong market signal despite a volatile biotech backdrop. This episode aired on July 17, 2026.
Biogen's new data, presented at the Alzheimer's Association International Conference, supports a tau-focused approach to the intractable neurodegenerative disease; psychedelics are back in the news with more positive data from Compass Pathways and final guidance from the FDA; and the ATTR-CM space got a major shakeup with the late-stage failure of AstraZeneca and Ionis' antisense therapeutic.
M&A enthusiasm is carrying biotech amid tepid reception for launches. On the latest BioCentury This Week podcast, BioCentury's analysts discuss BioCentury's 3Q26 Public Markets Preview, asking whether M&A momentum can carry biotech through year-end and what happens if the AI trade goes bust?Editor in Chief Simone Fishburn gives her perspective on what incoming Prime Minister Andy Burnham's agenda means for U.K. life sciences. The analysts also discuss Ionis' ATTR-CM miss and Takeda's orexin program. This episode of the BioCentury This Week podcast has been brought to you by CBRE.View full story: https://www.biocentury.com/article/660118#BiotechMA #BiotechInvesting #PublicMarkets #LifeSciences #DrugDevelopment00:01 - Sponsor Message: CBRE01:46 - 3Q Market Preview10:54 - Burnham's Britain19:40 - Ionis' ATTR-CM Miss29:12 - Takeda Orexin ProgramTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text
On this week's episode, Sam Fazeli, Josh Schimmer, Eric Schmidt, Paul Matteis, and special guest STAT's Matt Herper open on the market, noting that despite a wobbly day for biotech, the XBI holds strong gains year-to-date. The conversation turns to FDA transparency, where the co-hosts flag that CRLs haven't been released since April and that the agency may be walking back that practice. The co-hosts debate whether the FDA is swinging too far toward leniency after Agios' mitapivat won priority review in sickle cell disease despite missing its primary endpoint last year. On AI in drug discovery, Matt Herper recaps an interview with Anthropic's CEO about a new research tool, noting that it's plausible the tool helps industry pick better targets and lower the failure rate. In M&A, Vertex's largest-ever deal takes Crinetics for $10 billion net of cash, staking a claim in the specialty rare endocrine space, while Novartis expands its ADC portfolio with Myricx for $1.1 billion upfront plus $400 million in milestones — the second European ADC deal in recent months. On data, AstraZeneca and Ionis' eplontersen trial in ATTR-CM missed its endpoint, showing no benefit or biomarker signal on top of background tafamidis; the group then discusses the readthroughs for BridgeBio, Alnylam, and Pfizer's ATTR therapies. This episode aired on July 10, 2026.
Kun. Virgilijus Poškus ir kun. Arnoldas Valkauskas kalba apie krikščionio santykį su politika, visuomeniniu gyvenimu ir sąžine. Ar tikintis žmogus turi savo tikėjimą palikti tik privačioje erdvėje? Ar krikščionis gali sakyti: „Aš tik pasimelsiu, o viešieji reikalai manęs neliečia“? O gal Evangelija įpareigoja ne tik melstis, bet ir atsakingai veikti pasaulyje? Pokalbyje svarstoma, kuo skiriasi krikščioniškas dalyvavimas visuomenėje nuo susitapatinimo su politinėmis partijomis, ideologijomis ar valdžios interesais. Kunigai remiasi Šventuoju Raštu, Katalikų Bažnyčios Katekizmu, Vatikano II Susirinkimo mokymu ir Bažnyčios istorijos pavyzdžiais. Kalbama apie tai, kad krikščionis yra pasaulyje, bet ne iš pasaulio; jo galutinė viltis nėra politinė sistema, tačiau meilė Dievui ir artimui neleidžia abejingai stebėti neteisybės, melo, žmogaus orumo menkinimo ar sąžinei prieštaraujančių įstatymų. Ši laida kviečia savęs paklausti: ar mano tikėjimas turi poveikį tik mano asmeninei maldai, ar ir mano viešai atsakomybei? Kunigai primena, kad Bažnyčia neturi tapti politine bendruomene, tačiau ji turi teisę ir pareigą kalbėti moraliniais klausimais, kai paliečiamos pagrindinės žmogaus teisės, bendrasis gėris ir sielų išganymas. Krikščioniškas atsakas nėra nei prisitaikėliškas tylėjimas, nei nuolatinis bambėjimas prieš valdžią, bet drąsus, sąžiningas ir Evangelijai ištikimas veikimas. Pokalbyje prisimenami ir konkretūs istorijos pavyzdžiai — nuo ankstyvosios Bažnyčios iki Motiejaus Valančiaus, Antano Mackevičiaus, Lietuvos Katalikų Bažnyčios kronikos, sovietmečio ir kitų laikotarpių, kai tikintieji turėjo rinktis tarp patogaus tylėjimo ir ištikimybės tiesai. Laida kviečia nebijoti atsakomybės, ieškoti bendraminčių, burtis, melstis, veikti ir prašyti Dievo drąsos ten, kur krikščionio sąžinė nebeleidžia likti nuošalyje.
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we explore the dynamic shifts and breakthroughs shaping the industry, from major acquisitions to groundbreaking scientific advancements. Merck KGaA has made headlines with its bold $11.3 billion acquisition of Bio-Techne Corporation. This marks Merck's most significant deal since purchasing Sigma-Aldrich in 2015, reinforcing its strategic focus on expanding its life sciences tools portfolio. The acquisition aims to accelerate innovation in drug development and diagnostics, highlighting Merck's commitment to enhancing its capabilities in biotechnology under the leadership of CEO Kai Beckmann. Such strategic moves underscore a broader trend towards bolstering biotech portfolios through mergers and acquisitions as companies aim to remain competitive in an ever-evolving market landscape. In regulatory news, the FDA has approved a pioneering combination therapy involving Gilead's Trodelvy and Merck & Co.'s Keytruda for the first-line treatment of triple-negative breast cancer (TNBC). This aggressive cancer subtype has historically had limited treatment options, making this approval particularly significant. The combination therapy leverages an antibody-drug conjugate targeting Trop-2 alongside a PD-1 inhibitor, offering a promising new strategy that could substantially improve patient survival outcomes. This development also highlights the growing role of antibody-drug conjugates in oncology, illustrating how innovative therapeutic combinations can enhance treatment efficacy. Meanwhile, Pfizer's Ibrance has received FDA approval for label expansion to treat HR-positive, HER2-positive metastatic breast cancer. As a CDK4/6 inhibitor crucial in cell cycle regulation, Ibrance's expanded use reflects ongoing advancements in targeted therapies that personalize cancer treatment based on specific molecular characteristics. Such expansions demonstrate the importance of continuous clinical evaluation and regulatory engagement in extending the lifecycle and applications of existing drugs. Ionis Pharmaceuticals has gained FDA approval for Tryngolza for severe hypertriglyceridemia, marking a significant milestone for antisense oligonucleotide therapies. By targeting apolipoprotein C-III, Tryngolza offers a novel approach to managing metabolic conditions linked to pancreatitis risks. This approval underscores the growing importance of antisense technology in addressing complex lipid disorders and highlights Ionis' strategic efforts to expand market reach through global partnerships. On the business development front, Boehringer Ingelheim's partnership with Immunai aims to leverage artificial intelligence in T-cell target discovery for cancer and autoimmune diseases. The integration of AI/ML technologies into drug discovery processes is increasingly seen as essential for enhancing precision and efficiency. This collaboration reflects an industry-wide shift towards embracing technology to improve research and development outcomes. In clinical trials, Otsuka's centanafadine shows promise for adults with ADHD and comorbid anxiety following successful Phase 3b trials. As a small molecule reuptake inhibitor, centanafadine could provide dual therapeutic benefits for patients with these overlapping conditions. Such developments highlight ongoing innovation in neuropsychiatric treatments aimed at addressing mental health conditions with greater precision. Financially, Oblenio Bio's $62 million Series B funding round will support advancing its tri-specific autoimmune T-cell engager into trials, potentially offering new solutions for autoimmune diseases through innovative immunotherapy approaches. These financial movements illustrate how companies are strategically positioning themselves to capitalize on emerging therapeutic opportunities. Amid these developments, regulatory dynamics continue to evolve, as seen with the FDA's pilot program aimed at streamlining drug approval processes. Initiatives like these are pivotal in restoring confidence in regulatory frameworks while adapting to new scientific insights and technological advancements. Overall, these developments underscore the pharmaceutical and biotech sectors' dynamic nature, characterized by strategic collaborations, regulatory milestones, and innovative treatment options poised to enhance patient care and strengthen drug development pipelines. The ongoing integration of cutting-edge technologies such as AI signifies an evolution towards more personalized and efficient healthcare solutions. Thank you for tuning into Pharma Daily, where we bring you the latest insights from the forefront of pharmaceutical and biotech innovation. Join us next time as we continue to explore the trends shaping the future of healthcare globally.Support the show
Kun. Virgilijus Poškus ir kun. Arnoldas Valkauskas kalba apie nepatogią krikščioniško gyvenimo temą — pyktį. Ar kiekvienas pyktis yra nuodėmė? Ar krikščionis turi viską tyliai nuryti? O gal matant blogį, melą, neteisybę ir gėrio trūkumą kartais yra būtina reaguoti? Pokalbyje svarstoma, kuo teisėtas pasipiktinimas skiriasi nuo rūstumo, keršto, neapykantos, nuolatinio bambėjimo ir vidinio nepasitenkinimo. Kunigai remiasi Bažnyčios mokymu, Katekizmu, dvasine tradicija, Evangelijos žvilgsniu ir konkrečiais gyvenimo pavyzdžiais. Kalbama apie pyktį kaip stiprią žmogaus prigimties jėgą, kuri gali padėti įveikti baimę ir ginti gėrį, bet gali ir aptemdyti protą, sugriauti santykius, atimti ramybę bei nutolinti nuo meilės. Ši laida kviečia savęs paklausti: ar mano pyktis kyla iš meilės tiesai, Dievui, artimui ir teisingumui, ar iš nesaugumo, puikybės, baimės, keršto ir noro viską kritikuoti? Kunigai primena, kad krikščioniškas atsakas nėra nei bailus tylėjimas, nei nuolatinis piktinimasis. Reikia mokytis atpažinti, kur yra tikras blogis, kalbėti tiesą meilės kalba, nepaversti žmogaus priešu ir neleisti pykčio aistrai užvaldyti širdies.
Join HAEA Director of Health & Research Services, Troyce Venturella, and HAEA Health Services Manager, Mandy Granat, as they discuss important summer safety considerations for individuals and families living with HAE. The conversation is designed to help the HAEA community stay safe, healthy, and prepared while enjoying summer activities with family and friends!Thank you to our 2026 Sponsors: BioCryst, CSL, IONIS, and Takeda
On this week's episode, Sam Fazeli, Josh Schimmer, Eric Schmidt, and Tess Cameron kickoff with deals, highlighting the up to $15.2B Hengrui–BMS partnership and the broader trend of outsourcing early-stage drug development to China. The discussion continues with the co-hosts noting China's edge in speed, quality, and cost-efficiency, while underscoring that the strength of U.S. capital markets remains a key advantage. This week also saw a significant raise, with Isomorphic Labs announcing a $2.1B Series B. In regulatory news, the group described the departure of FDA Commissioner Dr. Marty Makary as creating fresh uncertainty around FDA leadership and direction as they speculated on his exit and who will replace him. On the data front, Regenxbio met the primary endpoint inits Phase 3 trial for Duchenne, though the hosts flagged potential investor skepticism around the side effects, limited data, and FDA uncertainty. Next, they discuss that Biogen and Ionis are advancing their Alzheimer's tau program despite mixed results. The co-hosts also mention Inhibrx's Phase 2 data in head and neck squamous cell carcinoma, as well as Moderna's Hantavirus vaccine research following the recent cruise ship outbreak. The episode concludes with a look ahead to upcoming conferences, including ASCO, ADA, and ATS. *This episode aired on May 15, 2026.
We love to hear from our listeners. Send us a message. On this week's episode of the Business of Biotech, Brett Monia, Ph.D., CEO at Ionis Pharmaceuticals, talks about how antisense oligonucleotides (ASO) graduated from a late-1980s lab concept to real RNA-targeting medicines, and why Ionis stuck with a difficult modality despite multiple setbacks. Brett describes the company's shift from a partner-first model to building a wholly-owned pipeline, commercial function, and an expanded manufacturing operation, and how he as a founding scientist-turned-CEO bridged a skills gap in finance and investor relations. Access this and hundreds of episodes of the Business of Biotech videocast under the Business of Biotech tab at lifescienceleader.com. Subscribe to our monthly Business of Biotech newsletter. Get in touch with guest and topic suggestions: ben.comer@lifescienceleader.comFind Ben Comer on LinkedIn: https://www.linkedin.com/in/bencomer/
It’s a pivotal year for RNA therapeutics pioneer Ionis Pharmaceuticals. After transitioning from a partnering model to a full-ownership strategy following the 2024 approval of Tryngolza, the company is expecting an FDA decision to expand the drug into severe hypertriglyceridemia as its first non-rare-disease indication for a wholly owned product. Meanwhile, potentially game-changing phase 3 readouts are expected from Ionis’ Novartis-partnered lipoprotein(a) candidate and AstraZeneca-partnered ATTR cardiomyopathy program. In this week’s episode of "The Top Line," Fierce’s Angus Liu interviews Ionis CEO Brett Monia, Ph.D. He discusses the science of antisense oligonucleotides, the significance of the two closely watched upcoming clinical readouts, and what it takes to turn a 30-year-old discovery platform into a full-fledged commercial biotech. To learn more about the topics in this episode: Ionis slashes Tryngolza's price tag by 93% ahead of anticipated label expansion 'It’s going to be huge': The 'diabolical' molecule poised to become biotech's next gold rush AstraZeneca sets sights on 25+ blockbusters by 2030 to fuel $80B revenue ambition As questions swirl around ATTR competition, Alnylam plots path to market leadership for Amvuttra Ionis exec shares method to the Madness after 2026 Drug Name Tournament win Ionis plots FDA filing after rare neurological disease candidate posts phase 3 win See omnystudio.com/listener for privacy information.
This episode covers: Cardiology This Week: A concise summary of recent studies Lp(a) and aortic valve stenosis The truth about climate change and heart disease Snapshots Host: Emer Joyce Guests: JP Carpenter, Borge Nordestgaard, Hugh Montgomery, Stephan Achenbach Want to watch that episode? Go to: https://esc365.escardio.org/event/2548 Want to watch that extended interview on Lp(a) and aortic valve stenosis, go to: https://esc365.escardio.org/event/2548?resource=interview Disclaimer: ESC TV Today is supported by Novartis through an independent funding. The programme has not been influenced in any way by its funding partner. This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. All declarations of interest are listed at the end of the episode. The ESC is not liable for any translated content of this video. The English language always prevails. Declarations of interests: Stephan Achenbach, Yasmina Bououdina and Nicolle Kraenkel have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, BiAL, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, GSK, Lilly, Novartis, Pfizer, Sanofi, Servier, Takeda, Tecnimede. John-Paul Carpenter has declared to have potential conflicts of interest to report: stockholder MyCardium AI. Davide Capodanno has declared to have potential conflicts of interest to report: Abbott Vascular, Bristol Myers Squibb, Daiichi Sankyo, Edwards Lifesciences, Novo Nordisk, Sanofi Aventis, Terumo. Emer Joyce has declared to have potential conflicts of interest to report: Alnylam, Bayer, Pfizer, Fire-1. Konstantinos Koskinas has declared to have potential conflicts of interest to report: honoraria from MSD, Daiichi Sankyo, Sanofi. Felix Mahfoud has declared to have potential conflicts of interest to report: research grants from Deutsche Forschungsgemeinschaft (SFB TRR219), Deutsche Gesellschaft für Kardiologie (DGK), Deutsche Herzstiftung, Ablative Solutions, ReCor Medical. Consulting fees, payment honoraria lectures, presentations, speaker, support travel costs: Ablative Solutions, Astra-Zeneca, Novartis, Inari, Recor Medical, Medtronic, Philips, Merck. Hugh Montgomery has declared to have potential conflicts of interest to report: funded and runs the charity-funded non-profit 'Real Zero'. Unpaid co-chair of the UK Health Alliance on Climate Change, Lancet Countdown on Health and Climate Change. Borge Nordestgaard has declared to have potential conflicts of interest to report: consultancies/talks for AstraZeneca, Sanofi, Ionis, Amgen, Amarin, Novartis, Novo Nordisk, Esperion, Lilly, Arrowhead, Marea, Merck, Torrent, USV – honoraria used for research. Steffen Petersen has declared to have potential conflicts of interest to report: consultancy for Circle Cardiovascular Imaging Inc. Calgary, Alberta, Canada. Emma Svennberg has declared to have potential conflicts of interest to report: Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Johnson & Johnson.
Host: Emer Joyce Guest: Borge Nordestgaard Want to watch that extended interview on Lp(a) and aortic valve stenosis, go to: https://esc365.escardio.org/event/2548?resource=interview Want to watch the full episode? Go to: https://esc365.escardio.org/event/2548 Disclaimer: ESC TV Today is supported by Novartis through an independent funding. The programme has not been influenced in any way by its funding partner. This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. All declarations of interest are listed at the end of the episode. The ESC is not liable for any translated content of this video. The English language always prevails. Declarations of interests: Stephan Achenbach, Yasmina Bououdina and Nicolle Kraenkel have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, BiAL, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, GSK, Lilly, Novartis, Pfizer, Sanofi, Servier, Takeda, Tecnimede. John-Paul Carpenter has declared to have potential conflicts of interest to report: stockholder MyCardium AI. Davide Capodanno has declared to have potential conflicts of interest to report: Abbott Vascular, Bristol Myers Squibb, Daiichi Sankyo, Edwards Lifesciences, Novo Nordisk, Sanofi Aventis, Terumo. Emer Joyce has declared to have potential conflicts of interest to report: Alnylam, Bayer, Pfizer, Fire-1. Konstantinos Koskinas has declared to have potential conflicts of interest to report: honoraria from MSD, Daiichi Sankyo, Sanofi. Felix Mahfoud has declared to have potential conflicts of interest to report: research grants from Deutsche Forschungsgemeinschaft (SFB TRR219), Deutsche Gesellschaft für Kardiologie (DGK), Deutsche Herzstiftung, Ablative Solutions, ReCor Medical. Consulting fees, payment honoraria lectures, presentations, speaker, support travel costs: Ablative Solutions, Astra-Zeneca, Novartis, Inari, Recor Medical, Medtronic, Philips, Merck. Borge Nordestgaard has declared to have potential conflicts of interest to report: consultancies/talks for AstraZeneca, Sanofi, Ionis, Amgen, Amarin, Novartis, Novo Nordisk, Esperion, Lilly, Arrowhead, Marea, Merck, Torrent, USV – honoraria used for research. Steffen Petersen has declared to have potential conflicts of interest to report: consultancy for Circle Cardiovascular Imaging Inc. Calgary, Alberta, Canada. Emma Svennberg has declared to have potential conflicts of interest to report: Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Johnson & Johnson.
Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're delving into the dynamic landscape of scientific breakthroughs, regulatory shifts, and strategic business maneuvers that are shaping the future of healthcare.The pharmaceutical industry is currently abuzz with discussions surrounding Pfizer's leadership, which has taken a firm stance against anti-vaccine narratives, particularly those propagated by figures like Robert F. Kennedy Jr. This critique highlights ongoing tensions between pharmaceutical companies and public health narratives that can significantly impact vaccine distribution and uptake. This situation underscores the crucial role of public trust in the industry's efforts to efficiently manage public health crises and ensure vaccine accessibility.Johnson & Johnson has projected its 2026 revenue to exceed $100 billion, largely driven by the success of its oncology treatment, Darzalex. This strategic focus on cancer therapies not only highlights J&J's commitment to expanding its oncology portfolio but also reflects broader industry trends where targeted cancer therapies are becoming pivotal revenue drivers due to their high efficacy and growing demand.Meanwhile, Takeda is navigating the challenges associated with patent expirations for its depression drug Trintellix. The anticipated patent cliff has led to a reduction of 243 positions within its U.S. neuroscience field force. This scenario emphasizes the ongoing importance of strategic planning around drug lifecycle management as companies strive to sustain revenue streams amidst patent expirations.Curia Global is also undergoing restructuring as it closes its Massachusetts plant, resulting in 81 job cuts. This move aligns with a broader trend in the industry aimed at optimizing operational efficiencies in response to shifting market demands and cost pressures.Advancements in rare disease treatments are making headlines with Intrabio's Aqneursa achieving Phase 3 success for ataxia-telangiectasia and receiving EU approval for Niemann-Pick disease Type C. These milestones underscore the critical role of rare disease research in expanding therapeutic options and highlight the potential for orphan drugs to offer substantial clinical benefits alongside commercial opportunities.In a significant collaboration, GSK has licensed Alteogen's enzyme to develop a subcutaneous form of Jemperli, a promising cancer medication. This partnership exemplifies the industry's focus on enhancing drug delivery technologies to improve patient compliance and therapeutic outcomes.Regulatory developments continue to unfold with notable approvals such as Ionis and Otsuka's hereditary angioedema drug receiving EU approval. Such milestones are indicative of the regulatory momentum that can facilitate market entry and expand access to innovative treatments across different geographies.Novo Nordisk's strategic shift away from cell therapy assets marks another noteworthy development within the industry. By divesting these assets, Novo Nordisk illustrates a broader reevaluation concerning investments in emerging technologies versus more traditional therapeutic areas.Turning our attention to financial strategies, Roche has made headlines with a substantial $2 billion investment in North Carolina aimed at supporting obesity-related drug production. This expansion underscores the critical role of manufacturing infrastructure in meeting rising global demand for innovative therapies—particularly in addressing conditions like obesity.The integration of AI into clinical improvements continues to gain traction in the biotech sector, with reports indicating higher success rates in clinical trials due to enhanced data analytics capabilities. This technological integration represents a paradigm shift in how clinical trials are designed and executedSupport the show
On week's episode, Eric Schmidt, Yaron Werber, Tess Cameron, and Sam Fazeli kick off with biotech M&A news, including Alkermes' $2.37 billion offer for Avadel, following Lundbeck's surprise bid. Next, the co-hosts discuss J&J's $3.05 billion cash acquisition of Halda - the highest-priced deal for a company at this stage. The group also covers Merck's acquisition of Cidara Therapeutics to strengthen its antiviral pipeline. They debate whether biotech M&A is becoming more competitive, what big pharma is prioritizing, and the role M&A is playing in the current biotech rally. The conversation then shifts to Washington policy news, including the CDC website updates suggesting vaccines haven't been proven not to cause autism, alleged tensions between RFK Jr. and Dr. Marty Makary over FDA leadership authority and management-style concerns Next, in regulatory news, the co-hosts overview the FDA's approval of Arrowhead's Redemplo for FCAS, priced at $60K, and its competition with Ionis' Tryngolza. Novo Nordisk's semaglutide price cut and conversations on drug pricing trends and the impact on competition are also mentioned. The episode concludes with an overview of Zymeworks positive data and shifting in business model, Nuvalent's positive data and $500 million raise, and Olema's stock price increase and raise on the back of Roche's breast cancer data. *This episode aired on November 21, 2025.
Watch every episode ad-free & uncensored on Patreon: https://patreon.com/dannyjones Matthew LaCroix is an author and ancient history expert who has extensively studied the secrets of lost civilizations, megaliths, earth cycles and ancient texts for two decades. SPONSORS https://www.amentara.com/go/dj - Use code DJ22 for 22% off your first order. https://www.irestore.com/dannyjones - Use code DANNYJONES & unlock HUGE savings. https://clean.ver.so/danny - Use code DANNY for 15% off your order. https://whiterabbitenergy.com/?ref=DJP - Use code DJP for 20% off EPISODE LINKS Matt's YouTube: @MatthewLaCroix https://thestageoftime.com FOLLOW DANNY JONES https://www.instagram.com/dannyjones https://twitter.com/jonesdanny OUTLINE 00:00 - Merging academia & alternative history 09:06 - The history of humanity is wrong 22:12 - Lake Van in Turkey 30:51 - The ancient climate apocalypse 39:23 - Serapeum boxes & disrespectful Egyptians 46:23 - Largest underground cities in the world 52:33 - How real are the ancient Sumerian stories? 58:05 - The depth of Lake Van 01:08:03 - The moon is impossible 01:19:29 - Our society is growing less advanced 01:23:32 - Secret societies & suppressing ancient knowledge 01:35:56 - The controllers of our reality 01:45:13 - The ancient Egypt coverup 01:53:39 - Ancient tablets & magical civilizations 02:03:10 - Inconsistencies in religious texts 02:05:58 - Ancient Greek vs Hebrew 02:08:33 - The Ionis temple 02:18:58 - Dating Lake Van ruins - older than Gobekli Tepe 02:32:44 - The Trimurti 02:39:13 - The Anunnaki story & what Zecharia Sitchin got wrong 02:48:59 - The Myth of Adapa - oldest text ever written 02:57:26 - UFOs: ancient tech vs. government tech 03:04:58 - Plutarch & proof of Atlantis Learn more about your ad choices. Visit podcastchoices.com/adchoices
This episode covers: Cardiology This Week: A concise summary of recent studies Arrhythmias in cardiac amyloidosis Taking the 'O' out of HOCM: managing LVOT obstruction Snapshots Host: Susanna Price Guests: Carlos Aguiar, Stephanie Schwarting, Ahmad Masri Want to watch that episode? Go to: https://esc365.escardio.org/event/2176 Want to watch that extended interview on Arrhythmias in Cardiac Amyloidosis? Go to: https://esc365.escardio.org/event/2176?resource=interview Disclaimer: ESC TV Today is supported by Bristol Myers Squibb and Novartis through an independent funding. The programme has not been influenced in any way by its funding partners. This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. The ESC is not liable for any translated content of this video. The English language always prevails. Declarations of interests: Stephan Achenbach, Yasmina Bououdina, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, BiAL, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, GSK, Lilly, Novartis, Pfizer, Sanofi, Servier, Takeda, Tecnimede. John-Paul Carpenter has declared to have potential conflicts of interest to report: stockholder Mycardium AI. Davide Capodanno has declared to have potential conflicts of interest to report: Bristol Myers Squibb, Daiichi Sankyo, Sanofi Aventis, Novo Nordisk, Terumo. Konstantinos Koskinas has declared to have potential conflicts of interest to report: honoraria from MSD, Daiichi Sankyo, Sanofi. Ahmad Masri has declared to have potential conflicts of interest to report: research grants from Pfizer, Ionis, Attralus, Cytokinetics and Janssen. Consulting fees from Cytokinetics, BMS, BridgeBio, Pfizer, Ionis, Lexicon, Attralus, Alnylam, Haya, Alexion, Akros, Edgewise, Rocket, Lexeo, Prothena, BioMarin, AstraZeneca, Avidity, Neurimmune, and Tenaya. Steffen Petersen has declared to have potential conflicts of interest to report: consultancy for Circle Cardiovascular Imaging Inc. Calgary, Alberta, Canada. Stephanie Schwarting has declared to have potential conflicts of interest to report: advisory board for Alnylam, Bayer, Pfizer; principal investigator in trials sponsored by Alexion, Novo Nordisk and Intellia. Emma Svennberg has declared to have potential conflicts of interest to report: Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Johnson & Johnson.
On this week's episode, Tess Cameron, Brian Skorney, Sam Fazeli, Yaron Werber, and Luba Greenwood, kick off with a pop quiz on the last time the $XBI hit 105 (spoiler, it was 2021) driven by recent positive news. The co-hosts highlight a steady rate of M&A activity, including Novo Nordisk's acquisition of Akero Therapeutics highlighting continued interest in metabolic conditions and BMS' acquisition of Orbital Therapeutics reflecting growing momentum around in vivo CAR-T delivery platforms. The LB Pharma and MapLight IPOs are also mentioned. The conversation shifts to AI pharma deals, spotlighting AstraZeneca's partnership with Algen Biotechnologies and Sanofi's collaboration with BenchSci, both designed to accelerate discovery and target identification. In other financing news, the co-hosts cover Nilo Therapeutics' $101 million Series A financing and the debut of Ascenta's $325 million biotech fund. In data news, the group covers Arcus' HIF-2a monotherapy data in kidney cancer, Dyne Therapeutics' encouraging results in DM1, and Ionis' pipeline and platform updates presented at its Innovation Day. The episode concludes with Lexeo Therapeutics' regulatory updates for its Freidreich's ataxis gene therapy and discussion on Peter Marks' transition from the head of CBER to Eli Lilly, noting the pharma-agency “revolving door.” *This episode aired on October 10, 2025.
Šiluvoje vykstančius atlaidus aplankė JAV žurnalistai – komanda „Miracle hunters“.Kas sunkiausia mokantis lietuvių kalbos buvo Osakoje gyvenančiam gidui Kotaro Hisada?Kauno IX forto muziejaus projektas „Birželio sodas“ atrinktas tarp 25 geriausių Tarptautinės muziejų asociacijos projektų.Kodėl keikiamasi viešai prieinamose vaizdo įrašuose ir ką suteikia keiksmažodžiai turiniui?Naujausiame ciklo „Čiurlioniai“ epizode lankysimės Sofijos Kymantaitės-Čiurlionienės namuose Kaune.Sostinėje atidaromi Vilniaus fotografijos namai.Vilniaus senajame teatre pristatomas spektaklis keturiomis kalbomis „Klausau, Jūsų Didenybe!“ pagal Januszo Korczako pasaką „Karalius Motiejukas Pirmasis“.Ved. Marius Eidukonis
Audio roundup of selected biopharma industry content from Scrip over the business week ended September 5, 2025. In this episode: the US MASH market after Wegovy's approval; AstraZeneca and Mineralys in close hypertension race; United Therapeutics' Phase III win in IPF; Corsera aims to predict and prevent cardiovascular disease; and Ionis's RNA-targeted win in severe hypertriglyceridemia. https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-RKP4E4CFGFGGXBRVXTI73V27U4/ This episode was produced with the help of AI text-to-voice and voice emulation tools. Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things
On this week's episode, hosts Paul Matteis, Sam Fazeli, John Maraganore, and Graig Suvannaveijh kick off the discussion with a more positive look at the sector and some of the fundamental factors at play. The group then shares an overview of Sanofi's 10% stock fall on the back of positive Phase 3 eczema data with worries about the Dupixent patent expiry. The discussion then turns to Insmed, a company that has had a monster year with the stock up 100% and a market cap of over $30 billion market. On the data front, the group highlights ApoC3 data from Ionis and Arrowhead at the European Society of Cardiology Congress 2025, which leads into a discussion around ASO versus RNAi. With multiple obesity readouts in recent weeks, the group theorizes on whether obesity is a zero sum game. It's a big year ahead for AD readouts and the hosts summarizes some important catalysts on the horizon. Other discussion topics include the launch of Corsera Health for cardiovascular prevention, Trump's Truth Social post on vaccine data, RFK Jr in front of congress, and public concern around access to vaccines. This episode aired on September 5, 2025.
00:00 – netikėtas „Žalgirio“ sprendimas 34:50 – ar turės dar naujokų „Žalgiris“ 39:20 – trys klaustukai rinktinėje 41:10 – kritika LKF 44:58 – „Ryto“ darbai ir LKL ėjimai 57:32 – įspūdingas moterų čempionato finalas 1:03:15 – lietuvių pasirodymas 1:19:06 – K.Jakučionis Majamyje
The best conversations about the Heat The Joe Rose Show, Tobin & Leroy, and Hochman, Crowder & Solana. Reacting to the Heat missing out on Kevin Durant, who was sent to the Rockets instead. How hard do we think Miami tried to land the future Hall of Famer? Plus, diving into the draft, which saw them select Kasparas Jakučionis. That and more this week on Best of Heat!
The best conversations about the Heat The Joe Rose Show, Tobin & Leroy, and Hochman, Crowder & Solana. Reacting to the Heat missing out on Kevin Durant, who was sent to the Rockets instead. How hard do we think Miami tried to land the future Hall of Famer? Plus, diving into the draft, which saw them select Kasparas Jakučionis. That and more this week on Best of Heat!
The best conversations about the Heat The Joe Rose Show, Tobin & Leroy, and Hochman, Crowder & Solana. Reacting to the Heat missing out on Kevin Durant, who was sent to the Rockets instead. How hard do we think Miami tried to land the future Hall of Famer? Plus, diving into the draft, which saw them select Kasparas Jakučionis. That and more this week on Best of Heat!
This CME program provides information on best practices to manage children with lysosomal disorders who have been identified by newborn screening. WIth the wide range of symptoms and severities that present for these rare conditions, it is not always certain when the best time to start treatment is in these patients.Continuing Education InformationThis continuing education activity is provided by AffinityCE and the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC). This activity provides continuing education credit for physicians. A statement of participation is available to other attendees.To obtain credit, visit https://checkrare.com/learning/p-transforming-clinical-outcomes-with-early-treatment-of-lysosomal-disorders/ Faculty and DisclosuresAffinityCE staff, LDRTC staff, planners, and reviewers, have no relevant financial relationships with ineligible companies to disclose. Faculty disclosures, listed below, will also be disclosed at the beginning of the Program.Ozlem Goker-Alpan MDFounder and CMO, Lysosomal & Rare Disorders Research & Treatment CentersDr. Goker-Alpan is on the Advisory Board/Consultant for Chiesi, Takeda, Sanofi, Prevail/Lilly, Sparks Therapeutics, Uniqure, Exegenesis, Astellas, Freeline, Team Sanfilippo. She receives grants/research support from Chiesi, Sanofi, Takeda, Prevail/Lilly, Spark Therapeutics, Amicus, Freeline, Sangamo, Cyclo, Odorsia, DMT, Homology, Protaliz. She is on the speaker bureau for Sanofi, Takeda, Amicus, ChiesiDavid F. Kronn MDAssociate Professor of Pathology and Pediatrics New York Medical CollegeDr. Kronn is on the Advisory Board for Sanofi. He is also on the speaker bureau for Sanofi. He receives research funding from Sanofi.Uma Ramaswami FRCPCH, MDRoyal Free London Hospitals & Genetics and Genomic Medicine, University College LondonDr. Ramaswami is on the Advisory Board for Amicus, Chiesi, Sanofi and Takeda. She receives research grants from Chiesi and Intabio.Liz Jalazo MDAssistant Professor of Pediatrics and GeneticsUniversity of North Carolina at Chapel HillDr. Jalazo is on the Advisory Board for Sanofi and Ionis. Lindsay Torrice MSN, CPNP-PC MDAssistant Professor of PediatricsUniversity of North Carolina at Chapel HillMs. Torrice has no financial relationships to disclose.Mitigation of Relevant Financial RelationshipsAffinityCE adheres to the ACCME's Standards for Integrity and Independence in Accredited Continuing Education. Any individuals in a position to control the content of a CME activity, including faculty, planners, reviewers, or others, are required to disclose all relevant financial relationships with ineligible companies. All relevant financial relationships for faculty were mitigated by the peer review of content by non-conflicted reviewers before the commencement of the activity.Learning ObjectivesAt the end of this activity, participants should be able to:• Cite the importance of early diagnosis and treatment of lysosomal storage disorders• List the guidelines for the early treatment of LDs and enhanced integration of newborn screening programs• Identify key research gaps and priorities and strengthen collaboration among researchers and healthcare professionals• List the educational resources and support programs for familiesPhysiciansThis activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of AffinityCE and the LDRTC. AffinityCE is accredited by the ACCME to provide continuing medical education for physicians.AffinityCE designates this enduring activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.Other ProfessionalsAll other healthcare professionals completing this continuing education activity will be issued a statement of participation indicating the number of hours of continuing education credit. This may be used for professional education CE credit. Please consult your accrediting organization or licensing board for their acceptance of this CE activity.Commercial SupportThis activity was supported by educational grants from Takeda, Sanofi, and Chiesi.Participation CostsThere is no cost to participate in this activity. CME InquiriesFor all CME policy-related inquiries, please contact us at ce@affinityced.com.Send customer support requests to cds_support+ldrtc@affinityced.com.
Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.AstraZeneca has recently made a significant investment of up to $1 billion in cell therapy through the acquisition of esobiotec. This move is part of AstraZeneca's larger strategy to expand in the cell therapy space, positioning itself to be a major player in the market. Dyne is also looking to file for accelerated approval for its Duchenne exon skipping oligomer, while Taiho Pharmaceutical has acquired ADC partner Araris for up to $1.1 billion. Alnylam is expected to enter the transthyretin amyloid cardiomyopathy market, which is projected to reach $11.2 billion by 2030.Sino Biological has developed reagents for the 2025-2026 influenza vaccine strains, offering a range of recombinant proteins for vaccine development. The pharmaceutical industry is seeing significant activity in various therapy areas, with companies making strategic investments and advancements in research and development.Alnylam is awaiting approval for their drug Amvuttra in the transthyretin amyloid cardiomyopathy market, which is currently dominated by Pfizer and BridgeBio. The obesity drug market is becoming increasingly competitive, with companies focusing on overall health benefits rather than just weight loss. Biopharma companies are now exploring the use of CAR T cell therapies for autoimmune disorders, with several readouts expected this year. Ionis and Ultragenyx are competing to develop treatments for Angelman syndrome, while Neuren is trying to catch up.Overall health outcomes are becoming more important in the obesity drug market, with successful studies in therapeutic areas like cardiovascular and sleep apnea providing a market advantage. Other news includes flu vaccine recommendations from the FDA and updates on drugs for alcohol use disorder, plaque psoriasis, breast cancer, and weight loss. The biopharma industry continues to evolve, with readers encouraged to provide suggestions for future coverage topics.
Hereditary angioedema (HAE) is a rare condition often due to reduced levels C1-inhibitor, which is a protein involved in various physiological processes in plasma, most notably with the complement system. C1-inhibitor also binds and inhibits plasma kallikrein and factor XIa, thereby affecting bradykinin production. It is believed that the disruptions of these processes cause fluid to leak from the blood to connective tissue, leading to HAE attacks. Owing to its rarity, HAE is often poorly recognized, leading to misdiagnoses and significant diagnostic delays. Being aware of the early signs and symptoms of this condition can lead to faster diagnosis and the use of effective therapies.This program is supported by independent medical education grants from Takeda. To earn CME credit please visit https://checkrare.com/learning/p-consider-rare-suspecting-and-diagnosing-hereditary-angioedema/lessons/consider-rare-suspecting-and-diagnosing-hereditary-angioedema-module/ Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in primary care, pediatrics, emergency care, otolaryngology, gastroenterology, and dermatology .Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to:- Describe the early symptoms of HAE and its clinical relevance.- Apply best practices to diagnose HAE more efficiently to reduce diagnostic delays. Faculty Jonathan A Bernstein, MDProfessor of MedicineUniversity of Cincinnati Department of Internal MedicineDivision of Immunology, Allergy SectionPartner Advanced Allergy Services, LLCPartner Bernstein Clinical Research Center Disclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Dr. Bernstein discloses the following relevant financial relationships with ineligible companies:Advisory Board Consultant: Takeda/Shire, CSL Behring, KalVista, Pharming, Biocryst, Ionis, Intellia, Pharvaris, Astria and BiomarinGrant/Research Support: Takeda/Shire, CSL Behring, KalVista, Pharming, Biocryst, Ionis, Intellia, Pharvaris, Astria and BiomariSpeaker's Bureau: PharmingPlanners for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPsOther members of the care team will receive a certificate of participation.There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you within 30 days.ContactFor any questions, please contact: CEServices@academycme.orgCopyright© 2025. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).
In the week since Donald Trump took office, he's caused quite the stir with healthcare-centered moves that include ordering the Department of Health and Human Services to stop communications, hiring and travel and announcing that he would withdraw the U.S. from the World Health Organization. Wednesday, the U.S. Senate Finance Committee convenes to vote on the controversial nomination of Robert F. Kennedy, Jr. for health secretary—a vote that Jefferies analysts said they expect to be “close.” Biogen continues to grab headlines this month, as the latest chapter in the Sage saga saw the smaller biotech rejecting its neuro partner's unsolicited buyout offer. Meanwhile, Biogen laid off an undisclosed number of employees from its research unit, just as a higher dose of its Ionis-partnered spinal muscular atrophy therapy Spinraza was accepted for review by both the FDA and EMA. Elsewhere, the weight loss space continues to click on all cylinders, with Versant Ventures debuting its newest obesity biotech Helicore Biopharma on Tuesday with $65 million in series A funds, and two obesity-focused companies, Aardvark Therapeutics and Metsera, seeking entry to the public markets. These up-and-comers will have to compete against the likes of Eli Lilly and Novo Nordisk, the latter of which reported data last week showing that its next-gen obesity drug amycretin could elicit up to 22% weight-loss. And Veru announced that its enobosarm could significantly improve the quality of weight loss in seniors also taking Novo's Wegovy. Another busy therapeutic space is Duchenne muscular dystrophy, where analysts predict a lot of action in the next couple of years, with a number of data readouts and regulatory submissions. And finally, Annalee Armstrong caught up at JPM with Novavax CEO John Jacobs, who said the vaccine maker is at a pivot point.
Hour one of DJ & PK for January 24, 2025: Utah Hockey Club game recap Jeremy Werner, 24/7 Sports Reno Mahe, Former BYU Cougar and Philadelpia Eagle
Hour two of DJ & PK for January 23, 2025: What is Trending Justin Werner, Illini Inquirer Will the Utah Jazz find a Shai Gilgeous-Alexander?
Justin Werner from Illini Inquirer joined DJ & PK to talk about Kasparas Jakučionis and what he would offer to a team like the Utah Jazz if they took him in the NBA Draft.
Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Novo Nordisk's weight loss drug, cagrisema, failed to meet investor expectations of achieving 25% weight loss, causing the stock to plummet. Galectin's stock also tumbled after its lead asset missed the primary endpoint in a phase IIb/III trial. On a positive note, Ionis' Tryngolza became the first FDA-approved therapy for familial chylomicronemia syndrome. GSK posted mixed survival data for the Jemperli/Zejula combo in first-line ovarian cancer treatment. Intelligencia AI accurately predicted high-potential biotechs before the ASH conference, showcasing the reliability of its AI-driven methodology. Overall, the biopharma industry is seeing a mix of successes and setbacks in various drug developments and approvals.
On this episode of Biotalk, Geoff Meyerson, CEO and Co-founder of Locust Walk, speaks with Sudhir Agrawal, Founder and President of ARNAY Sciences and a pioneer in RNA therapeutics. Sudhir's extensive career spans over three decades, during which he has made groundbreaking contributions to the field, including developing gapmer antisense technology, immune modulation approaches, and, in recent years, designing cyclic structures of DNA and RNA for therapeutic applications. In their conversation, Sudhir recounts his journey from his early days in nucleic acid research to becoming a leading innovator in RNA therapeutics. He delves into the origins of the gapmer technology, its impact on antisense drug development, and the lessons learned from his time Idera Pharmaceuticals. He also shares insights into his latest work on cyclic RNA/DNA structures and discusses his vision for the future of nucleic acid therapeutics. He concludes by offering practical advice to entrepreneurs aspiring to break into the biotech space and sharing his thoughts on the future of RNA science. Join us for a deep dive into the world of RNA therapeutics with one of its most influential figures.Subscribe or follow Biotalk on Apple Podcasts | Spotify. Timestamps: 1:34 What inspired you to focus specifically on RNA therapeutics, and how did your whole journey here begin? 3:04 Can you provide more details about the initial insight behind the development of the gapmer technology, which has been transformational for the space? 5:34 What lessons did you learn from your experience at Ionis (formerly Isis) that you would apply to your current work? 13:54 How are you thinking about commercializing the cyclic RNA/DNA structures you have developed at your company RNA Sciences? 23:40 Beyond the cyclic structures, what other innovations or areas of focus do you see on the horizon for nucleic acid therapeutics? 23:23 What advice would you give your early self starting out as an entrepreneur and innovator in the biotech space?
Host James Oxford (@AotGScouting) is joined by Chris Filer (@UCLA_Chris) and Finn Vandergriff (@FinnDraft) to discuss early season play from Khaman Maluach, Dylan Harper, Kasparas Jakucionis, and Egor Demin.
Tuesday, October 22, 2024 First Principles Genetic disease means that gene broken since conception. Novel medicines are possible ways to fix the gene - Genetic Therapies (ASO &/or AAV), this is recent, before now, kids with these diseases were a “go home and love them” situation. These are delivered via spinal tap or directly to the brain in leading medical centers. First though, regulators must approve. Our job Develop medicines or get industry to - This is happening see Pipeline Get regulators to approve trials Get medical centers up to speed on SYNGAP1-Related Disorders (SRD) What we are building on CHOP ENDD funded externally (see #S10e92) and replicating what was built for STXBP1, check last week's webinar https://curesyngap1.org/resources/webinars/93-endd-chop-2024-syngap1/ Rare-X platform for PRO collection Regulatory pathway being made clearer every day by Stoke (Dravet), Praxis (SCN2A), Ionis (many) all of whom are working on SYNGAP1 as well. What we are asking for We need to raise at least $500k (3rd site), preferably $1.13M (ProMMiS) Make your largest gift ever to SRF Fundraise with friends and family ACES is now ProMMiS, who knew ACE meant Adverse Childhood Event, not us. Key slides: S1 Path to Treatment | 2024 (09.27.24) 1. Why Now? Why is it time to go from bench to bedside (research to clinical)? At least 10 companies on our pipeline not to mention multiple small molecule efforts We have limited resources – so the focus has to transition, clinical funding first. CHOP Gift is 1 year down… 2. Why NHS?Understand SYNGAP1 better, go beyond Vlaskamp 2019 and Wiltrout 2024, see #S10e105 FYI at CHOP, as I shared in #S10e151, at year 1, we are at – 86 (Visits) + 10 (new scheduled) + 19 (2nd) + 4 (3rd) + 22 (follow up) Learn what to measure in clinical trials for SRD, remember our seizures are challenging Ideally we develop a Synthetic Control Arm if we use GCP Why top shelf? We need institutions the FDA will take seriously and our children are very complex requiring experienced clinicians. 3. Why Multidisciplinary. Neuro, Psych, Genetics, PT, ST, OT, GI, Sleep, ENT, Ortho. Beyond the sheer burden of getting our kids out and about for multiple appointments the coordination by a parent is almost impossible. 4. Why Multisite/3 sites?Replicable/scalable required by regulators Accessibility (not primary reason) Establish more locations where trials will be managed Laying a foundation for a national self-sustaining network 3 is the minimum, look at STARR or Angelman, both had/ve 4. 5. How and why so fast? Because we can. Time is Brain. Following a well trodden path SMA, Rett, Angelman, Dravet, but we are moving FASTER. 6. Does the industry really care? We are next there are so so many behind us, eager to take the resources we have access to today. Market size (Per our Census 425 US/1500 global is tip of iceberg) Multiple players reassuring each other Relatively strong amount of scientific and clinical research Haploinsufficiency (like Dravet – STOKE) – so relatively easy 7. Expensive? No. Clinical Research is more expensive than basic scientific research. Leveraging CHOP and Rare-X, setting up required networks to prepare for clinical trials. It's time. 8. Why Bother/Help? Now is the time for SYNGAP1, we miss it at our peril. Sure, once in these places we will still see our patients, but the study, the support and the focus may pass. Our kids don't die, regardless of patient age, what we are doing can change their future and that of their loved ones and caregivers. If not us, then who? It is a rare exception when a non-family member gives a gift, and it is always because a family member asked. We must ask. 9. What can I do? Donate to, share, join our Coast2Coast Clinics Challenge – two SYNGAP1 Squads in West and East – it's critical $500k goal by end of 2024; more than $1M needed just for the SYNGAP1ProMMiS. So far, donations from $25 to $25,000 – each and every contribution matters. This requires our entire S1 network to solicit family, friends, work colleagues, companies, etc. to contribute. Many causes out there – why not ours? Syngap.Fund/C2C https://Syngap.Fund/C2C > https://secure.givelively.org/donate/syngap-research-fund-incorporated/coast2coast-clinics-challenge Two teams: https://Syngap.Fund/West & https://Syngap.Fund/East
Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Florida has awarded additional Medicaid contracts to CVS, UnitedHealth, and Molina, after they were excluded from the first round of contracts in April. Meanwhile, Steward Health failed to attract qualified bidders during the first round of hospital sales. In other news, CMS has tightened rules for ACA brokers to prevent unauthorized plan switching. Commure is set to acquire AI scribe Augmedix in a $139 million deal to expand its documentation products. The healthcare industry is increasingly using AI to transform unstructured data and improve patient care. Additionally, online tools are being utilized to address gaps in mental healthcare services. AI and machine learning are also playing a crucial role in improving clinical trials by retaining participants, enhancing key performance indicators, and boosting query quality.The text discusses various updates in the medical technology industry on July 22, 2024. The FDA sent warning letters to Chinese syringe manufacturers, Embecta, a diabetes device firm, is considering a sale, Truvian raised $74 million for a blood test instrument, and a CrowdStrike outage affected US hospitals. Medtronic executive Stacey Churchwell spoke about the use of artificial intelligence in reducing false positives in cardiac monitors. Intuitive's Da Vinci 5 robot launch exceeded Wall Street expectations. The text also includes information on real-time vital signs data, Biden's healthcare legacy, and suggestions for further reading.Private biotech M&A activity has increased significantly, with acquisitions of private biotechs outpacing public offerings. Ionis is moving forward with plans for an Angelman drug that Biogen passed on, with a late-stage study set to begin next year. Meanwhile, J&J is seeking expanded approval for its antidepressant Spravato. The advanced therapeutic medicinal product (ATMP) sector is at the forefront of personalized medicine, with over 1,000 trials in development and potential disruption through manufacturing automation. In the oncology research space, there is fierce competition in the GLP-1 drug race as companies vie for a share of the obesity treatment market expected to surpass $100 billion by the end of the decade.Innovent's Phase III trial for their diabetes drug in China has been successful, bringing them closer to potential approval in the country. The drug, Mazdutide, is also being developed for weight management. Meanwhile, Lilly has received approval in China for their drug Tirzepatide for obesity, following Novo's approval for Semaglutide. Samsung Bioepis has received FDA approval for a biosimilar to AstraZeneca's Soliris. Novo and Lilly are looking to expand their GLP-1 pipelines beyond treating diabetes and obesity. Other news in the biopharma industry includes startups competing in the obesity space, GSK plotting Blenrep's return to market, and a procurement deal between IGA and the US government.Researchers in China conducted a small study using an "off the shelf" CAR-T cell therapy to treat patients with autoimmune diseases. The therapy, made with engineered cells from a healthy donor, resulted in deep remission of immune diseases and a reversal of inflammation and fibrosis in three patients after six months. This study marks a potential breakthrough in allogeneic CAR-T cell therapy for autoimmune diseases, offering a potentially cheaper and easier-to-manufacture alternative to approved autologous therapies. However, despite this progress, payment barriers need to be addressed to make these therapies accessible to patients. The study highlights the need for outside support to overcome barriers in the development and commercialization of cell and gene therapies. Additionally, the research landscape for cancer treatments continues to grow, with oncology being a significant area of growth for pharma drug development. The stud
After deciphering the first part of the prophecy revealed by Sara Benthem, the adventurers journey to Briarcleft Forest with the assistance of R&R Brigade ally Ionis. Following a lead given to them by Professor Roundland, they seek out the Gem of Fire. Featuring special guest Sam Richardson! Learn more about your ad choices. Visit megaphone.fm/adchoices
00:00:00 Surf's Up, Season 5 Episode 20Naim Alkhouri and Mazen Noureddin join co-hosts Jörn Schattenberg and Roger Green to discuss major drug development stories from the EASL Congress 2024. They pay tribute to Stephen Harrison and proceed to groundbreakers.00:08:16 - Presentations on ResmetiromMazen shares a paper he presented looking at resmetirom dose-response. The 100mg dose performed better than the 80mg dose. Jörn notes that the most important element of the study might be the demonstrated 36-month effect. 00:14:19- Implications of Different Analytical Modes Naim compares different modes of analysis. Intent to Treat, which treats all non-completers as failures, will produce lower rates of response than a "completer" or "modified Intent to Treat" analysis, which either eliminates non-completers or assigns them placebo-level response. 00:16:12 - The "Twin-Cretins"Jörn coined the name "Twin-cretins" for agents with GLP-1 agonism plus another incretin effect. Tirzepatide is a GLP/GIP while survodutide, pemvuditide, and efinopegdutide, are GLP/glucagons. Naim reviews the tirzepatide late-breaker presentation, which showed high rates of MASH resolution, but, as a completer analysis, might not have proven whether a GLP/GIP can achieve significant fibrosis regression. Naim notes that Stephen doubted strongly this was possible, but that bariatric surgery's effect creates a paradox. Mazen concurs, and states he is more confident in the GLP/glucagon agents. Jörn agrees. 00:23:09 - Implications of widespread GLP useRoger asks how widespread prior patient use of incretins will affect prescribing. Naim states that many of his Rezdiffra MASH patients have been on GLP-1 already. He describes a key difference between the three GLP/glucagon agents in development. 00:24:20 - The previous GLP/glucagon studiesNaim expresses his excitement about glucagon agents in general. He mentions that the efinopegdutide study included a semaglutide cell, which showed a further reduction in liver fat compared to the GLP-1 agent. 00:26:44 - Safety of survodutide in cirrhosisJörn comments on the high level of tolerability in the survodutide trials. Mazen shares his high hopes for GLP/glucagons and the triple agents. He reviews survodutide data from the NEJM article. 00:29:41 - FGF-21s, starting with efrux data Focus shifts to FGF-21s. Mazen shares data from the efruxifermin late-breaker, a 96-week, triple biopsy study showing dramatic, sustained one- and two-level fibrosis improvement. Jörn notes the design demonstrates durability, which Roger finds particularly encouraging given the pegbelfermin experience. Naim adds that a sustainable two-stage drop in fibrosis might position FGF-21 as induction therapy before long-term oral maintenance. 00:35:28 - Is a drug-based MASH "cure" possible? Mazen wonders whether sustained FGF-21 efficacy might return livers to their "normal" state, almost like a "cure." Jörn briefly discusses pegozafermin, and Mazen notes that a third FGF-21 is in development. 00:42:22 - Wrap-up The panel covers three other agents: the FASN inhibitor, denifenstat, Ionis's DGAT-2 antisense inhibitor, ION224, and Takeda's TAK-227, a TG2 inhibitor, plus the results of SPECIAL, a study evaluating the effect of bariatric surgery on people with cirrhosis. After these, Roger asks Mazen and Jörn what they consider likely to be the biggest story at AASLD in November. 00:53:03 - Question of the Week Given this discussion, Roger asks what are likely to be the three most prescribed medications for MASH five years from now. 00:53:36 - Business Report The next EASL Congress review episodes, how to attend recording sessions live, and a vault discussion from last year's EASL Congress wrap-up.
In this podcast, Thomas Czech, Distinguished Professor at the University of Colorado, Boulder, with a lineage of remarkable contributions on RNA, ribozyme, and telomeres, discuss why RNA is so incredibly versatile.Video snippet from our conversation. Full videos of all Ground Truths podcasts can be seen on YouTube here. The audios are also available on Apple and Spotify.Transcript with links to the audio and external linksEric Topol (00:07):Well, hello, this is Eric Topol from Ground Truths, and it's really a delight for me to welcome Tom Cech who just wrote a book, the Catalyst, and who is a Nobel laureate for his work in RNA. And is at the University of Colorado Boulder as an extraordinary chemist and welcome Tom.Tom Cech (00:32):Eric, I'm really pleased to be here.The RNA GuyEric Topol (00:35):Well, I just thoroughly enjoyed your book, and I wanted to start out, if I could, with a quote, which gets us right off the story here, and let me just get to it here. You say, “the DNA guy would need to become an RNA guy. Though I didn't realize it at the time, jumping ship would turn out to be the most momentous decision in my life.” Can you elaborate a bit on that?Tom Cech (01:09):As a graduate student at Berkeley, I was studying DNA and chromosomes. I thought that DNA was king and really somewhat belittled the people in the lab next door who were working on RNA, I thought it was real sort of second fiddle material. Of course, when RNA is acting just as a message, which is an important function, a critical function in all life on earth, but still, it's a function that's subservient to DNA. It's just copying the message that's already written in the playbook of DNA. But little did I know that the wonders of RNA were going to excite me and really the whole world in unimaginable ways.Eric Topol (02:00):Well, they sure have, and you've lit up the world well before you had your Nobel Prize in 1989 was Sid Altman with ribozyme. And I think one of the things that struck me, which are so compelling in the book as I think people might know, it's divided in two sections. The first is much more on the biology, and the second is much more on the applications and how it's changing the world. We'll get into it particularly in medicine, but the interesting differentiation from DNA, which is the one trick pony, as you said, all it does is store stuff. And then the incredible versatility of RNA as you discovered as a catalyst, that challenging dogma, that proteins are supposed to be the only enzymes. And here you found RNA was one, but also so much more with respect to genome editing and what we're going to get into here. So I thought what we might get into is the fact that you kind of went into the scum of the pond with this organism, which by the way, you make a great case for the importance of basic science towards the end of the book. But can you tell us about how you, and then of course, many others got into the Tetrahymena thermophila, which I don't know that much about that organism.Tom Cech (03:34):Yeah, it's related to Tetrahymena is related to paramecium, which is probably more commonly known because it's an even larger single celled animal. And therefore, in an inexpensive grade school microscope, kids can look through and see these ciliated protozoa swimming around on a glass slide. But I first learned about them when I was a postdoc at MIT and I would drive down to Joe Gall's lab at Yale University where Liz Blackburn was a postdoc at the time, and they were all studying Tetrahymena. It has the remarkable feature that it has 10,000 identical copies of a particular gene and for a higher organism, one that has its DNA in the nucleus and does its protein synthesis in the cytoplasm. Typically, each gene's present in two copies, one from mom, one from dad. And if you're a biochemist, which I am having lots of stuff is a real advantage. So 10,000 copies of a particular gene pumping out RNA copies all the time was a huge experimental advantage. And that's what I started working on when I started my own lab at Boulder.Eric Topol (04:59):Well, and that's where, I guess the title of the book, the Catalyst ultimately, that grew into your discovery, right?Tom Cech (05:08):Well, at one level, yes, but I also think that the catalyst in a more general conversational sense means just facilitating life in this case. So RNA does much more than just serve as a biocatalyst or a message, and we'll get into that with genome editing and with telomerase as well.The Big Bang and 11 Nobel Prizes on RNA since 2000Eric Topol (05:32):Yes, and I should note that as you did early in the book, that there's been an 11 Nobel prize awardees since 2000 for RNA work. And in fact, we just had Venki who I know you know very well as our last podcast. And prior to that, Kati Karikó, Jennifer Doudna who worked in your lab, and the long list of people working RNA in the younger crowd like David Liu and Fyodor Urnov and just so many others, we need to have an RNA series because it's just exploding. And that one makes me take you back for a moment to 2007. And when I was reading the book, it came back to me about the Economist cover. You may recall almost exactly 17 years ago. It was called the Biology's Big Bang – Unravelling the secrets of RNA. And in that, there was a notable quote from that article. Let me just get to that. And it says, “it is probably no exaggeration to say that biology is now undergoing its neutron moment.”(06:52):This is 17 years ago. “For more than half a century the fundamental story of living things has been a tale of the interplay between genes, in the form of DNA, and proteins, which is genes encode and which do the donkey work of keeping living organisms living. The past couple of years, 17 years ago, however, has seen the rise and rise of a third type of molecule, called RNA.” Okay, so that was 2007. It's pretty extraordinary. And now of course we're talking about the century of biology. So can you kind of put these last 17 years in perspective and where we're headed?Tom Cech (07:34):Well, Eric, of course, this didn't all happen in one moment. It wasn't just one big bang. And the scientific community has been really entranced with the wonders of RNA since the 1960s when everyone was trying to figure out how messenger RNA stored the genetic code. But the general public has been really kept in the dark about this, I think. And as scientists, were partially to blame for not reaching out and sharing what we have found with them in a way that's more understandable. The DNA, the general public's very comfortable with, it's the stuff of our heredity. We know about genetic diseases, about tracing our ancestry, about solving crimes with DNA evidence. We even say things like it's in my DNA to mean that it's really fundamental to us. But I think that RNA has been sort of kept in the closet, and now with the mRNA vaccines against Covid-19, at least everyone's heard of RNA. And I think that that sort of allowed me to put my foot in the door and say, hey, if you were curious about the mRNA vaccines, I have some more stories for you that you might be really interested in.RNA vs RNAEric Topol (09:02):Yeah, well, we'll get to that. Maybe we should get to that now because it is so striking the RNA versus RNA chapter in your book, and basically the story of how this RNA virus SARS-CoV-2 led to a pandemic and it was fought largely through the first at scale mRNA nanoparticle vaccine package. Now, that takes us back to some seminal work of being able to find, giving an mRNA to a person without inciting massive amount of inflammation and the substitution of pseudouridine or uridine in order to do that. Does that really get rid of all the inflammation? Because obviously, as you know, there's been some negativism about mRNA vaccines for that and also for the potential of not having as much immune cell long term activation. Maybe you could speak to that.Tom Cech (10:03):Sure. So the discovery by Kati Karikó and Drew Weissman of the pseudouridine substitution certainly went a long way towards damping down the immune response, the inflammatory response that one naturally gets with an RNA injection. And the reason for that is that our bodies are tuned to be on the lookout for foreign RNA because so many viruses don't even mess with DNA at all. They just have a genome made of RNA. And so, RNA replicating itself is a danger sign. It means that our immune system should be on the lookout for this. And so, in the case of the vaccination, it's really very useful to dampen this down. A lot of people thought that this might make the mRNA vaccines strange or foreign or sort of a drug rather than a natural substance. But in fact, modified nucleotides, nucleotides being the building blocks of RNA, so these modified building blocks such as pseudoU, are in fact found in natural RNAs more in some than in others. And there are about 200 modified versions of the RNA building blocks found in cells. So it's really not an unusual modification or something that's all that foreign, but it was very useful for the vaccines. Now your other question Eric had to do with the, what was your other question, Eric?Eric Topol (11:51):No, when you use mRNA, which is such an extraordinary way to get the spike protein in a controlled way, exposed without the virus to people, and it saved millions of lives throughout the pandemic. But the other question is compared to other vaccine constructs, there's a question of does it give us long term protective immunity, particularly with T cells, both CD8 cytotoxic, maybe also CD4, as I know immunology is not your main area of interest, but that's been a rub that's been put out there, that it isn't just a weaning of immunity from the virus, but also perhaps that the vaccines themselves are not as good for that purpose. Any thoughts on that?Tom Cech (12:43):Well, so my main thought on that is that this is a property of the virus more than of the vaccine. And respiratory viruses are notoriously hard to get long-term immunity. I mean, look at the flu virus. We have to have annual flu shots. If this were like measles, which is a very different kind of virus, one flu shot would protect you against at least that strain of flu for the rest of your life. So I think the bad rap here is not the vaccine's fault nearly as much as it's the nature of respiratory viruses.RNA And Aging Eric Topol (13:27):No, that's extremely helpful. Now, let me switch to an area that's really fascinating, and you've worked quite a bit on the telomerase story because this is, as you know, being pursued quite a bit, has thought, not just because telomeres might indicate something about biologic aging, but maybe they could help us get to an anti-aging remedy or whatever you want to call it. I'm not sure if you call it a treatment, but tell us about this important enzyme, the role of the RNA building telomeres. And maybe you could also connect that with what a lot of people might not be familiar with, at least from years ago when they learned about it, the Hayflick limit.Tom Cech (14:22):Yes. Well, Liz Blackburn and Carol Greider got the Nobel Prize for the discovery of telomerase along with Jack Szostak who did important initial work on that system. And what it does is, is it uses an RNA as a template to extend the ends of human chromosomes, and this allows the cell to keep dividing without end. It gives the cell immortality. Now, when I say immortality, people get very excited, but I'm talking about immortality at the cellular level, not for the whole organism. And in the absence of a mechanism to build out the ends of our chromosomes, the telomeres being the end of the chromosome are incompletely replicated with each cell division. And so, they shrink over time, and when they get critically short, they signal the cell to stop dividing. This is what is called the Hayflick limit, first discovered by Leonard Hayflick in Philadelphia.(15:43):And he, through his careful observations on cells, growing human cells growing in Petri dishes, saw that they could divide about 50 times and then they wouldn't die. They would just enter a state called senescence. They would change shape, they would change their metabolism, but they would importantly quit dividing. And so, we now see this as a useful feature of human biology that this protects us from getting cancer because one of the hallmarks of cancer is immortality of the tumor cells. And so, if you're wishing for your telomeres to be long and your cells to keep dividing, you have to a little bit be careful what you wish for because this is one foot in the door for cancer formation.Eric Topol (16:45):Yeah, I mean, the point is that it seems like the body and the cell is smart to put these cells into the senescent state so they can't divide anymore. And one of the points you made in the book that I think is worth noting is that 90% of cancers have the telomerase, how do you say it?Tom Cech (17:07):Telomerase.Eric Topol (17:08):Yeah, reactivate.Tom Cech (17:09):Right.Eric Topol (17:10):That's not a good sign.Tom Cech (17:12):Right. And there are efforts to try to target telomerase enzyme for therapeutic purposes, although again, it's tricky because we do have stem cells in our bodies, which are the exception to the Hayflick limit rule. They do still have telomerase, they still have to keep dividing, maybe not as rapidly as a cancer cell, but they still keep dividing. And this is critical for the replenishment of certain worn out tissues in our such as skin cells, such as many of our blood cells, which may live only 30 days before they poop out. That's a scientific term for needing to be replenished, right?Eric Topol (18:07):Yeah. Well, that gets me to the everybody's, now I got the buzz about anti-aging, and whether it's senolytics to get rid of these senescent cells or whether it's to rejuvenate the stem cells that are exhausted or work on telomeres, all of these seem to connect with a potential or higher risk of cancer. I wonder what your thoughts are as we go forward using these various biologic constructs to be able to influence the whole organism, the whole human body aging process.Tom Cech (18:47):Yes. My view, and others may disagree is that aging is not an affliction. It's not a disease. It's not something that we should try to cure, but what we should work on is having a healthy life into our senior years. And perhaps you and I are two examples of people who are at that stage of our life. And what we would really like is to achieve, is to be able to be active and useful to society and to our families for a long period of time. So using the information about telomerase, for example, to help our stem cells stay healthy until we are, until we're ready to cash it in. And for that matter on the other side of the coin, to try to inhibit the telomerase in cancer because cancer, as we all know, is a disease of aging, right? There are young people who get cancer, but if you look at the statistics, it's really heavily weighted towards people who've been around a long time because mutations accumulate and other damage to cells that would normally protect against cancer accumulates. And so, we have to target both the degradation of our stem cells, but also the occurrence of cancer, particularly in the more senior population. And knowing more about RNA is really helpful in that regard.RNA DrugsEric Topol (20:29):Yeah. Well, one of the things that comes across throughout the book is versatility of RNA. In fact, you only I think, mentioned somewhere around 12 or 14 of these different RNAs that have a million different shapes, and there's so many other names of different types of RNAs. It's really quite extraordinary. But one of the big classes of RNAs has really hit it. In fact, this week there are two new interfering RNAs that are having extraordinary effects reported in the New England Journal on all the lipids, abnormal triglycerides and LDL cholesterol, APOC3. And can you talk to us about this interfering the small interfering RNAs and how they become, you've mentioned in the book over 400 RNAs are in the clinic now.Tom Cech (21:21):Yeah, so the 400 of course is beyond just the siRNAs, but these, again, a wonderful story about how fundamental science done just to understand how nature works without any particular expectation of a medical spinoff, often can have the most phenomenal and transformative effects on medicine. And this is one of those examples. It came from a roundworm, which is about the size of an eyelash, which a scientist named Sydney Brenner in England had suggested would be a great experimental organism because the entire animal has only about a thousand cells, and it's transparent so we can look at, see where the cells are, we can watch the worm develop. And what Andy Fire and Craig Mello found in this experimental worm was that double-stranded RNA, you think about DNA is being double-stranded and RNA as being single stranded. But in this case, it was an unusual case where the RNA was forming a double helix, and these little pieces of double helical RNA could turn off the expression of genes in the worm.(22:54):And that seemed remarkable and powerful. But as often happens in biology, at least for those of us who believe in evolution, what goes for the worm goes for the human as well. So a number of scientists quickly found that the same process was going on in the human body as a natural way of regulating the expression of our genes, which means how much of a particular gene product is actually going to be made in a particular cell. But not only was it a natural process, but you could introduce chemically synthesized double helical RNAs. There are only 23 base pairs, 23 units of RNA long, so they're pretty easy to chemically synthesize. And that once these are introduced into a human, the machinery that's already there grabs hold of them and can be used to turn off the expression of a disease causing RNA or the gene makes a messenger RNA, and then this double-stranded RNA can suppress its action. So this has become the main company that is known for doing this is Alnylam in Boston, Cambridge. And they have made quite a few successful products based on this technology.Eric Topol (24:33):Oh, absolutely. Not just for amyloidosis, but as I mentioned these, they even have a drug that's being tested now, as you know that you could take once or twice a year to manage your blood pressure. Wouldn't that be something instead of a pill every day? And then of course, all these others that are not just from Alnylam, but other companies I wasn't even familiar with for managing lipids, which is taking us well beyond statins and these, so-called PCSK9 monoclonal antibodies, so it's really blossoming. Now, the other group of RNA drugs are antisense drugs, and it seemed like they took forever to warm up, and then finally they hit. And can you distinguish the antisense versus the siRNA therapeutics?Tom Cech (25:21):Yes, in a real general sense, there's some similarity as well as some differences, but the antisense, what are called oligonucleotides, whoa, that's a big word, but oligo just means a few, right? And nucleotides is just the building blocks of nucleic acid. So you have a string of a few of these. And again, it's the power of RNA that it is so good at specifically base pairing only with matching sequences. So if you want to match with a G in a target messenger RNA, you put a C in the antisense because G pairs with C, if you want to put an A, if want to match with an A, you put a U in the antisense because A and U form a base pair U is the RNA equivalent of T and DNA, but they have the same coding capacity. So any school kid can write out on a notepad or on their laptop what the sequence would have to be of an antisense RNA to specifically pair with a particular mRNA.(26:43):And this has been, there's a company in your neck of the woods in the San Diego area. It started out with the name Isis that turned out to be the wrong Egyptian God to name your company after, so they're now known as Ionis. Hopefully that name will be around for a while. But they've been very successful in modifying these antisense RNAs or nucleic acids so that they are stable in the body long enough so that they can pair with and thereby inhibit the expression of particular target RNAs. So it has both similarities and differences from the siRNAs, but the common denominator is RNA is great stuff.RNA and Genome EditingEric Topol (27:39):Well, you have taken that to in catalyst, the catalyst, you've proven that without a doubt and you and so many other extraordinary scientists over the years, cumulatively. Now, another way to interfere with genes is editing. And of course, you have a whole chapter devoted to not just well CRISPR, but the whole genome editing field. And by the way, I should note that I forgot because I had read the Codebreaker and we recently spoke Jennifer Doudna and I, that she was in your lab as a postdoc and you made some wonderful comments about her. I don't know if you want to reflect about having Jennifer, did you know that she was going to do some great things in her career?Tom Cech (28:24):Oh, there was no question about it, Eric. She had been a star graduate student at Harvard, had published a series of breathtaking papers in magazines such as Science and Nature already as a graduate student. She won a Markey fellowship to come to Colorado. She chose a very ambitious project trying to determine the molecular structures of folded RNA molecules. We only had one example at the time, and that was the transfer RNA, which is involved in protein synthesis. And here she was trying these catalytic RNAs, which we had discovered, which were much larger than tRNA and was making great progress, which she finished off as an assistant professor at Yale. So what the general public may not know was that in scientific, in the scientific realm, she was already highly appreciated and much awarded before she even heard anything about CRISPR.Eric Topol (29:38):Right. No, it was a great line you have describing her, “she had an uncanny talent for designing just the right experiment to test any hypothesis, and she possessed more energy and drive than any scientist I'd ever met.” That's pretty powerful. Now getting into CRISPR, the one thing, it's amazing in just a decade to see basically the discovery of this natural system to then be approved by FDA for sickle cell disease and beta thalassemia. However, the way it exists today, it's very primitive. It's not actually fixing the gene that's responsible, it's doing a workaround plan. It's got double strand breaks in the DNA. And obviously there's better ways of editing, which are going to obviously involve RNA epigenetic editing, if you will as well. What is your sense about the future of genome editing?Tom Cech (30:36):Yeah, absolutely, Eric. It is primitive right now. These initial therapies are way too expensive as well to make them broadly applicable to the entire, even in a relatively wealthy country like the United States, we need to drive the cost down. We need to get them to work, we need to get the process of introducing them into the CRISPR machinery into the human body to be less tedious and less time consuming. But you've got to start somewhere. And considering that the Charpentier and Doudna Nobel Prize winning discovery was in 2012, which is only a dozen years ago, this is remarkable progress. More typically, it takes 30 years from a basic science discovery to get a medical product with about a 1% chance of it ever happening. And so, this is clearly a robust RNA driven machine. And so, I think the future is bright. We can talk about that some more, but I don't want to leave RNA out of this conversation, Eric. So what's cool about CRISPR is its incredible specificity. Think of the human genome as a million pages of text file on your computer, a million page PDF, and now CRISPR can find one sentence out of that million pages that matches, and that's because it's using RNA, again, the power of RNA to form AU and GC base pairs to locate just one site in our whole DNA, sit down there and direct this Cas9 enzyme to cut the DNA at that site and start the repair process that actually does the gene editing.Eric Topol (32:41):Yeah, it's pretty remarkable. And the fact that it can be so precise and it's going to get even more precise over time in terms of the repair efforts that are needed to get it back to an ideal state. Now, the other thing I wanted to get into with you a bit is on the ribosome, because that applies to antibiotics and as you call it, the mothership. And I love this metaphor that you had about the ribosome, and in the book, “the ribosome is your turntable, the mRNA is the vinyl LP record, and the protein is the music you hear when you lower the needle.” Tell us more about the ribosome and the role of antibiotics.Tom Cech (33:35):So do you think today's young people will understand that metaphor?Eric Topol (33:40):Oh, they probably will. They're making a comeback. These records are making a comeback.Tom Cech (33:44):Okay. Yes, so this is a good analogy in that the ribosome is so versatile it's able to play any music that you feed at the right messenger RNA to make the music being the protein. So you can have in the human body, we have tens of thousands of different messenger RNAs. Each one threads through the same ribosome and spills out the production of whatever protein matches that mRNA. And so that's pretty remarkable. And what Harry Noller at UC Santa Cruz and later the crystallographers Venki Ramakrishnan, Tom Steitz, Ada Yonath proved really through their studies was that this is an RNA machine. It was hard to figure that out because the ribosome has three RNAs and it has dozens of proteins as well. So for a long time people thought it must be one of those proteins that was the heart and soul of the record player, so to speak.RNA and Antibiotics(34:57):And it turned out that it was the RNA. And so, when therefore these scientists, including Venki who you just talked to, looked at where these antibiotics docked on the ribosome, they found that they were blocking the key functional parts of the RNA. So it was really, the antibiotics knew what they were doing long before we knew what they were doing. They were talking to and obstructing the action of the ribosomal RNA. Why is this a good thing for us? Because bacterial ribosomes are just enough different from human ribosomes that there are drugs that will dock to the bacterial ribosomal RNA, throw a monkey wrench into the machine, prevent it from working, but the human ribosomes go on pretty much unfazed.Eric Topol (36:00):Yeah, no, the backbone of our antibiotics relies on this. So I think people need to understand about the two subunits, the large and the small and this mothership, and you illuminate that so really well in the book. That also brings me to phage bacteria phage, and we haven't seen that really enter the clinic in a significant way, but there seems to be a great opportunity. What's your view about that?Tom Cech (36:30):This is an idea that goes way back because since bacteria have their own viruses which do not infect human cells, why not repurpose those into little therapeutic entities that could kill, for example, what would we want to kill? Well, maybe tuberculosis has been very resistant to drugs, right? There are drug resistant strains of TB, yes, of TB, tuberculosis, and especially in immunocompromised individuals, this bug runs rampant. And so, I don't know the status of that. It's been challenging, and this is the way that biomedicine works, is that for every 10 good ideas, and I would say phage therapy for bacterial disease is a good idea. For every 10 such ideas, one of them ends up being practical. And the other nine, maybe somebody else will come along and find a way to make it work, but it hasn't been a big breakthrough yet.RNA, Aptamers and ProteinsEric Topol (37:54):Yeah, no, it's really interesting. And we'll see. It may still be in store. What about aptamers? Tell us a little bit more about those, because they have been getting used a lot in sorting out the important plasma proteins as therapies. What are aptamers and what do you see as the future in that regard?Tom Cech (38:17):Right. Well, in fact, aptamers are a big deal in Boulder because Larry Gold in town was one of the discoverers has a company making aptamers to recognize proteins. Jack Szostak now at University of Chicago has played a big role. And also at your own institution, Jerry Joyce, your president is a big aptamer guy. And you can evolution, normally we think about it as happening out in the environment, but it turns out you can also make it work in the laboratory. You can make it work much faster in the laboratory because you can set up test tube experiments where molecules are being challenged to perform a particular task, like for example, binding to a protein to inactivate it. And if you make a large community of RNA molecules randomly, 99.999% of them aren't going to know how to do this. What are the odds? Very low.(39:30):But just by luck, there will be an occasional molecule of RNA that folds up into a shape that actually fits into the proteins active sighting throws a monkey wrench into the works. Okay, so now that's one in a billion. How are you going to find that guy? Well, this is where the polymerase chain reaction, the same one we use for the COVID-19 tests for infection comes into play. Because if you can now isolate this needle in a haystack and use PCR to amplify it and make a whole handful of it, now you've got a whole handful of molecules which are much better at binding this protein than the starting molecule. And now you can go through this cycle several times to enrich for these, maybe mutagen it a little bit more to give it a little more diversity. We all know diversity is good, so you put a little more diversity into the population and now you find some guy that's really good at recognizing some disease causing protein. So this is the, so-called aptamer story, and they have been used therapeutically with some success, but diagnostically certainly they are extremely useful. And it's another area where we've had success and the future could hold even more success.Eric Topol (41:06):I think what you're bringing up is so important because the ability to screen that tens of thousands of plasma proteins in a person and coming up with as Tony Wyss-Coray did with the organ clocks, and this is using the SomaLogic technology, and so much is going on now to get us not just the polygenic risk scores, but also these proteomic scores to compliment that at our orthogonal, if you will, to understand risk of people for diseases so we can prevent them, which is fulfilling a dream we've never actually achieved so far.Tom Cech (41:44):Eric, just for full disclosure, I'm on the scientific advisory board of SomaLogic in Boulder. I should disclose that.Eric Topol (41:50):Well, that was smart. They needed to have you, so thank you for mentioning that. Now, before I wrap up, well, another area that is a favorite of mine is citizen science. And you mentioned in the book a project because the million shapes of RNA and how it can fold with all hairpin terms turns and double stranded and whatever you name it, that there was this project eteRNA that was using citizen scientists to characterize and understand folding of RNA. Can you tell us about that?RNA Folding and Citizen ScienceTom Cech (42:27):So my friend Rhiju Das, who's a professor at Stanford University, sort of adopted what had been done with protein folding by one of his former mentors, David Baker in Seattle, and had repurposed this for RNA folding. So the idea is to come up with a goal, a target for the community. Can you design an RNA that will fold up to look like a four pointed cross or a five pointed star? And it turned out that, so they made it into a contest and they had tens of thousands of people playing these games and coming up with some remarkable solutions. But then they got a little bit more practical, said, okay, that was fun, but can we have the community design something like a mRNA for the SARS-CoV-2 spike protein to make maybe a more stable vaccine? And quite remarkably, the community of many of whom are just gamers who really don't know much about what RNA does, were able to find some solutions. They weren't enormous breakthroughs, but they got a several fold, several hundred percent increase in stability of the RNA by making it fold more tightly. So I just find it to be a fascinating approach to science. Somebody of my generation would never think of this, but I think for today's generation, it's great when citizens can become involved in research at that level.Eric Topol (44:19):Oh, I think it's extraordinary. And of course, there are other projects folded and others that have exemplified this ability for people with no background in science to contribute in a meaningful way, and they really enjoy, it's like solving a puzzle. The last point is kind of the beginning, the origin of life, and you make a pretty strong case, Tom, that it was RNA. You don't say it definitively, but maybe you can say it here.RNA and the Origin of LifeTom Cech (44:50):Well, Eric, the origin of life happening almost 4 billion years ago on our primitive planet is sort of a historical question. I mean, if you really want to know what happened then, well, we don't have any video surveillance of those moments. So scientists hate to ever say never, but it's hard to sort of believe how we would ever know for sure. So what Leslie Orgel at the Salk Institute next to you taught me when I was a starting assistant professor is even though we'll never know for sure, if we can recapitulate in the laboratory plausible events that could have happened, and if they make sense chemically and biologically, then that's pretty satisfying, even if we can never be absolutely sure. That's what a number of scientists have done in this field is to show that RNA is sort of a, that all the chemistry sort of points to RNA as being something that could have been made under prebiotic conditions and could have folded up into a way that could solve the greatest of all chicken and egg problems, which came first, the informational molecule to pass down to the next generation or the active molecule that could copy that information.(46:32):So now that we know that RNA has both of those abilities, maybe at the beginning there was just this RNA world RNA copying itself, and then proteins came along later, and then DNA probably much more recently as a useful but a little bit boring of genetic information, right?Eric Topol (46:59):Yeah. Well, that goes back to that cover of the Economist 17 years ago, the Big Bang, and you got me convinced that this is a pretty strong story and candidate. Now what a fun chance to discuss all this with you in an extraordinary book, Tom. Did I miss anything that you want to bring up?Tom Cech (47:21):Eric, I just wanted to say that I not only appreciate our conversation, but I also appreciate all you are doing to bring science to the non-scientist public. I think people like me who have taught a lot of freshmen in chemistry, general chemistry, sort of think that that's the level that we need to aim at. But I think that those kids have had science in high school year after year. We need to aim at the parents of those college freshmen who are intelligent, who are intellectually curious, but have not had science courses in a long time. And so, I'm really joining with you in trying to avoid jargon as much as possible. Use simple language, use analogies and metaphors, and try to share the excitement of what we're doing in the laboratory with the populace.Eric Topol (48:25):Well, you sure did that it was palpable. And I thought about it when I read the book about how lucky it would be to be a freshman at the University of Boulder and be having you as the professor. My goodness. Well, thank you so much. This has been so much fun, Tom, and I hope everybody's going to get out there and read the Catalyst to get all the things that we didn't even get a chance to dive into. But this has been great and look forward to future interactions with you.Tom Cech (48:53):Take care, Eric.*********************Thanks for listening or reading this edition of Ground Truths.Please share this podcast with your friends and network. That tells me you found it informative and makes the effort in doing these worthwhile.All Ground Truths newsletters and podcast are free. Voluntary paid subscriptions all go to support Scripps Research. Many thanks for that—they greatly helped fund our summer internship programs for 2023 and 2024.Thanks to my producer Jessica Nguyen and Sinjun Balabanoff for audio and video support at Scripps Research.Note: you can select preferences to receive emails about newsletters, podcasts, or all I don't want to bother you with an email for content that you're not interested in. Get full access to Ground Truths at erictopol.substack.com/subscribe
JOIN SRF LT on Thursday: Volunteer Info session with Leadership Team is this week: https://syngap.fund/LT Thursday 5/23 at 5:30 Pacific. MDBR is 3 weeks away! https://Syngap.Fund/Unite $5k match https://x.com/phalliburton/status/1792288377049415835 It's all about therapies. Precision Genetic and Repurposed. Conferences are where we engage professional communities around SYNGAP1 & SRF. - Last week I was at Milken Global. All diseases are talking about biomarkers & endpoints. https://milkeninstitute.org/events/global-conference-2024/program - This week I was at the #Ultragenyx Bootcamp with our CSO https://www.ultragenyx.com/video-this-bootcamp-helps-parents-advance-rare-disease-research/ So good to see Kathryn energized by this role. - ASGCT was last week and that means announcements… - Kathryn and I are off to BIO in June in San Diego. Ionis for Angelman https://www.linkedin.com/posts/cureangelman_exciting-news-for-the-angelman-syndrome-community-activity-7196872264976322563-_rvX Capsida for STX https://www.linkedin.com/posts/graglia_capsida-biotherapeutics-presents-new-preclinical-activity-7194004214635716608-M01Y Encoded for STX https://www.linkedin.com/posts/stxbp1-foundation_encoded-therapeutics-provides-pipeline-updates-activity-7196942568859787265-leKO Nasha at FOXG1 https://www.youtube.com/watch?v=ELKijSx0uwQ Repurposed therapies are just as important. Cost effective. Globally available. They are here now. We must act, the suffering is immense. They show us what is improvable and therefore inform clinical trial design. They are not compromising other trials. And to even suggest that is unethical if it suggests people should hold off on helping patients. How about we just diagnose more kids? Or think harder about which kids go to which trials? Precision Genetic Therapies are going to be more effective than repurposed drugs, people will happily participate in trials. Review of repurposed drugs: RAVICTI Rx- 10+ patients with Dr. Grinspan, not all but a few have had significant seizure reduction. I remain worried about cost and look to STXBP1 and SLC6A1 for guidance here. NORTRIPTYLINE Rx - Has helped me, a handful of families are getting a Rx. I am hopeful that some researcher does an investigator led trial. But until then, ask your Neuro. ACETYL-LEUCINE is a Nutraceutical - Update 1 has really been noticed and Update 2 will share a few potential mechanisms of action. Many are trying this drug from https://bit.ly/tanganil24 Encouraging… no, updating. Please share data with us if you are trying. We are collecting case studies for Update 3. Thank you to the team here. NEWLY DIAGNOSED? New families have resources here! https://syngap.fund/Resources SOCIAL MATTERS - AMPLIFY SRF TO MAKE SURE FAMILIES FIND US - 1,010 YouTube. https://www.youtube.com/@CureSYNGAP1 - 9,900 Twitter https://twitter.com/cureSYNGAP1 - 3,560 LinkedIn. https://www.linkedin.com/company/curesyngap1/ - 49k TikTok https://www.instagram.com/curesyngap1/ Podcasts, give all of these a five star review! SRF Channel - https://podcasts.apple.com/us/channel/syngap1-podcasts-by-srf/id6464522917 Episode 141 of #Syngap10 - May 20, 2024 #epilepsy #autism #intellectualdisability #id #anxiety #raredisease #epilepsyawareness #autismawareness #rarediseaseresearch #SynGAPResearchFund #CareAboutRare #PatientAdvocacy #GCchat #Neurology #GeneChat #F78A1 #CureSYNGAP1
This week on “The Top Line,” we're joined by Fierce Biotech Senior Editor Annalee Armstrong and Fierce Pharma Marketing Senior Editor Ben Adams. We're diving into our recent spin on March Madness at Fierce. This time around, Fierce Biotech kicked off its inaugural Best Biotech Name tournament, while over at Fierce Pharma Marketing, we held our biannual Drug Names tournament. The editors discuss what makes a company or drug name tick, what to steer clear of, and how the entire competition played out. To learn more about the topics in this episode: #FierceMadness: AstraZeneca and Ionis cruise to victory as Wainua trounces Lilly's Mounjaro for the tournament win #FierceMadness: The Best Biotech Name Tournament—A champion is CROWNED See omnystudio.com/listener for privacy information.
Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA has posted guidance on cybersecurity for medical device companies seeking authorization for new devices. Mass General Brigham is collaborating with the FDA to create a brain-computer interface group to address challenges facing developers of such devices. Moody's predicts that AI will expand medtech portfolios and revenue streams in the next two years. Abbott has recalled its HeartMate LVAD communication system due to reported injuries linked to the device malfunctioning. 3M has named William Brown as CEO, replacing Michael Roman. The medical device industry is seeing a trend towards incorporating AI and digital health technologies.The FDA has raised safety concerns about early patient deaths in those treated with Bristol Myers Squibb's Abecma and Johnson & Johnson's Carvykti ahead of an advisory committee meeting. In other news, the Alabama Supreme Court's ruling on in vitro fertilization has caused political blowback and uncertainty in the market. Additionally, Lilly has made its weight-loss drug Zepbound available through Amazon Pharmacy, and Ionis' antisense molecule has shown positive results in a Phase II trial for liver disease. Novartis has acquired IFM Due in a deal potentially worth $835 million, and the FDA is questioning the benefit-risk profile of Geron's MDS candidate.Heinz is launching a campaign to promote gravy as more than just a holiday condiment, leveraging its iconic status to encourage consumers to use gravy like they use ketchup. "Godzilla x Kong" has released Roblox's first immersive movie trailer, allowing players to be swept into a virtual game after watching a 2D teaser. Meta announced updates to Advantage+ and shopping ads, while Barkley and OKRP have merged to create a "big indie" agency. Marketing Dive provides insights into news and trends shaping the marketing and advertising industry, covering topics such as marketing technology, social media, video marketing, analytics, and more.
ESC TV Today brings you concise analysis from the world's leading experts, so you can stay on top of what's happening in your field quickly. This episode covers: Cardiology This Week: A concise summary of recent studies Management of cardiac amyloidosis e-cigarettes and cardiovascular disease Statistics Made Easy: The Hazard Ratio Host: Susanna Price Guests: Carlos Aguiar, Julian Gillmore, Maryam Kavousi Want to watch that episode? Go to: https://esc365.escardio.org/event/1144 Disclaimer This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC. Declarations of interests Stephan Achenbach, Maryam Kavousi, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, Lilly, Novartis, Pfizer, Sanofi, Servier, Tecnimede. Davide Capodanno has declared to have potential conflicts of interest to report: Sanofi, Novo Nordisk, Terumo, Medtronic. Julian Gillmore has declared to have potential conflicts of interest to report: consultancy for Alnylam, ATTRalus, AstraZeneca, Bridgebio, Intellia, Ionis, Lycia. Emma Svennberg has declared to have potential conflicts of interest to report: institutional research grants from Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Boehringer-Ingelheim, Johnson & Johnson, Merck Sharp & Dohme.
Tapk KALBA MAMOS bendruomenės dalimi ir klausyk pilnų pokalbių, dalyvauk mamų rytmečiuose, Discord'o pokalbiuose, Knygų klube bei kitose tik bendruomenei skirtose veiklose. Visą informaciją rasi www.kalbamamos.lt. Nori tik klausytis pokalbių? Prenumeruok tinklalaidę KALBA MAMOS „Spotify“ platformoje. Pokalbiui apie tikslus ir įpročius pakviečiau gydytoją psichoterapeutą Dainių Jakučionį, kuris jau daugiau nei 15 metų gilinasi į su žmogaus kūnu ir protu susijusius klausimus, o pastaruosius 10 metų aktyviai konsultuoja kaip psichoterapeutas. Jis veda mokymus ir skaito pranešimus įvairiose konferencijose, susijusiose su psichologine gerove ir psichine sveikata, o taip taip pat yra programėlės „Sensa“, padedančios susidoroti su psichologiniais sunkumais, bendraautorius. Dainius Jakučionis socialiniuose tinkluose dalinasi psichologinėmis įžvalgomis, skirtomis asmeninei saviugdai. Man atrodo, kad sausio tema apie tikslus ir įpročius yra tam tikras mūsų gruodžio temos tęsinys, kai kvietėme stabtelėti ir peržvelgti prabėgusius metus, paklausti savęs, kas mums patiko, praturtino, kokias pamokas išsinešame į kitus metus, ką norėtume tęsti, o ko – ne. Dabar man norisi kviesti pokalbiui ne apie tai, kaip nuversti kalnus, bet kaip iš tiesų išgirsti, suprasti, ko aš noriu, dėl ko dega mano širdis ir kokius žingsnius, nors ir labai mažus, galiu kasdien žengti to tikslo ar tikslų link? Šiame pokalbyje kalbėsime apie: kodėl ši, tikslų, tema mus galbūt erzina, o gal net ir žmonės, kurie kelia sau kažkokius tikslus ir viešai apie tai dalinasi? Ar verta susirūpinti, jei neturime šiuo metu jokių tikslų? Kodėl svarbu atskirti norus, svajones nuo tikslų ir pasidžiaugti kiekvienu žingsniu, kurį žengiame tikslo link. Apie baimes. Kodėl bijome kelti tikslus ir iš kur tos baimės atsiranda? Apie nusivylimus. Kodėl neverta nusivilti, bet verta bandyti vėl ir vėl iš naujo. Apie perfekcionizmą. Kodėl daug vertingiau pastovumas darant nors ir labai mažus žingsnelius, bet daryti, o ne sustoti, nes neišeina pasiekti tobulo rezultato. Pokalbiui baigiantis dar turėjau pilną kišenę klausimų, bet laiko yra tiek, kiek yra, sąlygos tokios, kokios yra, ir paleidžiu šį pokalbį su viltimi, kad bet kokiu atveju išsinešite iš jo gerų, įkvepiančių, padrąsinančių minčių. Gero klausymo! Marija
*Note, we aware part of the video froze; that's life! RESEARCH! - Dr. Helbig gave a killer presentation today… at Stanford. It was so cool to see SYNGAP1 mentioned. “Deciphering the Epilepsy Phenome – Understanding Longitudinal disease trajectories and outcomes” it showed how much data you can get form EHRs, ciitizen and NHS. We need them all. Sign up for Ciitizen! https://www.ciitizen.com/SYNGAP1/ - SRF Supported work out of Huganir's Lab is on BioRx! https://www.biorxiv.org/content/10.1101/2023.08.06.552111v1.full.pdf - Grants due on 9/1, already seeing good stuff. https://www.syngapresearchfund.org/professionals/grants/how-to-apply VOLUNTEER SITE IS UP - https://www.syngapresearchfund.org/volunteer-with-syngap-research-fund WEBINARS ARE A VALUABLE RESOURCE - https://www.syngapresearchfund.org/families/resources/all-webinars/webinars - Yesterday, Dr. Underbakk, Previously with Dr. Courtney NPR ARTICLE INCLUDING JACKIE KANCIR - https://wpln.org/post/episodes/the-cost-of-care-for-disabilities-and-chronic-illness SYNGAP STORIES Ashley Frye is killing it. Suzanne Jones just did an episode. The CHOP team listens, so do clinicians. Call Ashley and share your story. COMPANY UPDATES - Stoke had great news - https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-reports-second-quarter-financial-results-2 - Praxis also having good results - https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-9 - Have not heard from RegEl or Ionis. EVENTS - Getting ready for the Soiree - August 26th. - Cannonball set for October 4-6 - SRF SYNGAP1 CONFERENCE - Register, book etc. https://www.syngapresearchfund.org/professionals/syngap1-roundtable-2023-syngap-research-fund - Watch #s10e109 https://youtu.be/to8SAwdzCmg BIOMARKERS NEED BIOSAMPLES AND EEGS - Combined Brain collections are great Dr. TJB met our very own Pavel this weekend! Plasma, plasma, plasma. - UCLA Study is moving forward for EEG collection. Contact Declan via study page. Upcoming times and places to do biosamples, thanks you Corey we are at 18 Syngapians and 21 Siblings. KEEP GOING - GLUT1 Deficiency, August 26th, 9am-5pm (Embassy Suites, 13700 Conference Center Drive South, Noblesville, IN 46060) - IRF2BPL Foundation, Sept 22nd-23rd 2023 (Home 2 Suites by Hilton, 7145 Liberty Centre Drive, Liberty Township, OH 45069, 513-644-2207) - KCNQ2 Cure Alliance Conference, Sept 29th-30th 2023 (Hilton Hotel Chicago 300 E Ohio St, Chicago, IL 60611) - NARS1 Conference, October 1st, 9am-5pm (13550 Commerce Blvd Rogers, MN 55374) - Prader-Willi Syndrome/USP7 Foundation, October 5-7 (1672 Lawrence St, Denver, CO 80202) - TBRS Community, Oct 12th-14th 2023 (Morgan's Wonderland, 5223 David Edwards Dr, San Antonio, TX 78233) - COMBINEDBrain Meeting, Oct 15th-16th 2023 (Washington DC) - FAM177A1, Oct 29th 2023, 9am-5pm (2737 77th Ave Se Suite 101 Mercer Island, WA 98040) - ADNP Syndrome, Oct 30th-Nov 1st 2023 (Los Angeles, CA) - SYNGAP1 Research Fund, Dec 1st-3rd 2023 (8978 International Drive Orlando, FL, 32819) —--- CONFERENCE Registration link: https://Syngap.Fund/Orlando Hotels ASAP: https://Syngap.Fund/2023hotel Shirts https://www.bonfire.com/srf-syngap1-conference-2023/ Volunteer with SRF! Info@SyngapResearchFund.org This is a podcast: subscribe to and rate this 10 minute #podcast #SYNGAP10 here - https://www.syngapresearchfund.org/syngap10-podcast Apple podcasts: https://podcasts.apple.com/us/podcast/syngap10-weekly-10-minute-updates-on-syngap1-video/id1560389818 Episode 111 of #Syngap10 - August 12, 2023 #epilepsy #autism #intellectualdisability #id #anxiety #raredisease #epilepsyawareness #autismawareness #rarediseaseresearch #SynGAPResearchFund #CareAboutRare #PatientAdvocacy #GCchat #Neurology #GeneChat
*Note, we are aware part of the video froze; that's life! RESEARCH! - Dr. Helbig gave a killer presentation today… at Stanford. It was so cool to see SYNGAP1 mentioned. “Deciphering the Epilepsy Phenome – Understanding Longitudinal disease trajectories and outcomes” it showed how much data you can get form EHRs, ciitizen and NHS. We need them all. Sign up for Ciitizen! https://www.ciitizen.com/SYNGAP1/ - SRF Supported work out of Huganir's Lab is on BioRx! https://www.biorxiv.org/content/10.1101/2023.08.06.552111v1.full.pdf - Grants due on 9/1, already seeing good stuff. https://www.syngapresearchfund.org/professionals/grants/how-to-apply VOLUNTEER SITE IS UP - https://www.syngapresearchfund.org/volunteer-with-syngap-research-fund WEBINARS ARE A VALUABLE RESOURCE - https://www.syngapresearchfund.org/families/resources/all-webinars/webinars - Yesterday, Dr. Underbakk, Previously with Dr. Courtney NPR ARTICLE INCLUDING JACKIE KANCIR - https://wpln.org/post/episodes/the-cost-of-care-for-disabilities-and-chronic-illness SYNGAP STORIESAshley Frye is killing it. Suzanne Jones just did an episode. The CHOP team listens, so do clinicians. Call Ashley and share your story. COMPANY UPDATES - Stoke had great news - https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-reports-second-quarter-financial-results-2 - Praxis also having good results - https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-9 - Have not heard from RegEl or Ionis. EVENTS - Getting ready for the Soiree - August 26th. - Cannonball set for October 4-6 - SRF SYNGAP1 CONFERENCE - Register, book etc. https://www.syngapresearchfund.org/professionals/syngap1-roundtable-2023-syngap-research-fund - Watch #s10e109 https://youtu.be/to8SAwdzCmg BIOMARKERS NEED BIOSAMPLES AND EEGS - Combined Brain collections are great Dr. TJB met our very own Pavel this weekend! Plasma, plasma, plasma. - UCLA Study is moving forward for EEG collection. Contact Declan via study page. Upcoming times and places to do biosamples, thanks you Corey we are at 18 Syngapians and 21 Siblings. KEEP GOING - GLUT1 Deficiency, August 26th, 9am-5pm (Embassy Suites, 13700 Conference Center Drive South, Noblesville, IN 46060) - IRF2BPL Foundation, Sept 22nd-23rd 2023 (Home 2 Suites by Hilton, 7145 Liberty Centre Drive, Liberty Township, OH 45069, 513-644-2207) - KCNQ2 Cure Alliance Conference, Sept 29th-30th 2023 (Hilton Hotel Chicago 300 E Ohio St, Chicago, IL 60611) - NARS1 Conference, October 1st, 9am-5pm (13550 Commerce Blvd Rogers, MN 55374) - Prader-Willi Syndrome/USP7 Foundation, October 5-7 (1672 Lawrence St, Denver, CO 80202) - TBRS Community, Oct 12th-14th 2023 (Morgan's Wonderland, 5223 David Edwards Dr, San Antonio, TX 78233) - COMBINEDBrain Meeting, Oct 15th-16th 2023 (Washington DC) - FAM177A1, Oct 29th 2023, 9am-5pm (2737 77th Ave Se Suite 101 Mercer Island, WA 98040) - ADNP Syndrome, Oct 30th-Nov 1st 2023 (Los Angeles, CA) - SYNGAP1 Research Fund, Dec 1st-3rd 2023 (8978 International Drive Orlando, FL, 32819) —--- CONFERENCE Registration link: https://Syngap.Fund/Orlando Hotels ASAP: https://Syngap.Fund/2023hotel Shirts https://www.bonfire.com/srf-syngap1-conference-2023/ Volunteer with SRF! Info@SyngapResearchFund.org This is a podcast: subscribe to and rate this 10 minute #podcast #SYNGAP10 here - https://www.syngapresearchfund.org/syngap10-podcast Apple podcasts: https://podcasts.apple.com/us/podcast/syngap10-weekly-10-minute-updates-on-syngap1-video/id1560389818 Episode 111 of #Syngap10 - August 12, 2023 #epilepsy #autism #intellectualdisability #id #anxiety #raredisease #epilepsyawareness #autismawareness #rarediseaseresearch #SynGAPResearchFund #CareAboutRare #PatientAdvocacy #GCchat #Neurology #GeneChat --- Send in a voice message: https://podcasters.spotify.com/pod/show/syngap10/message
This week, Jeremy talks to Dr. Paul Larkin, director of research at The ALS Association, and Dr. Frank Bennett, Chief Scientific Officer at Ionis and a pioneer in the field of antisense oligonucleotide research, about the FDA's approval of tofersen and the future of ALS drug development. To learn more about why the FDA's decision on tofersen matters to everyone, check out: https://www.als.org/blog/tofersen-approved-sod1-als For more information on antisense technology go to: https://www.als.org/research/research-we-fund/scientific-focus-areas/genetics/antisense-therapy-for-alsThis episode is brought to you The ALS Association in partnership with CitizenRacecar.
ONCE UPON A GENE - EPISODE 175 Every Patient Matters - Discovering, Developing, and Providing Experimental ASO Treatments to Nano-Rare Patients for Free with n-Lorem Founder and CEO Stan Crooke Stan Crooke is the Founder and CEO of the n-Lorem Foundation. He's also the Founder and former CEO of Ionis Pharmaceuticals, the leader in RNA-targeted therapeutics. Under his leadership at Ionis, he pioneered development of the revolutionary antisense technology platform and created one of the largest, most advanced pipelines in the biotechnology industry. His foundation is now using this technology to discover, develop and provide personalized, experimental antisense oligonucleotide medicines to nano-rare patients for free, for life. EPISODE HIGHLIGHTS What do you do at n-Lorem? With antisense oligonucleotides (ASOs), we design chemically modified pieces of genetic information and those pieces of genetic information allow us to target a specific RNA, which is the molecule that gets transcribed from your DNA to make the protein that makes cells work. Because we now understand that code very well, and because we have a lot of experience, we can be very rational, efficient and inexpensive compared to traditional drug development. In a nano-rare patient, a patient with a unique mutation in a single gene, we begin with this incredible advantage. We can create a genetic medicine for many of these genes and mutations and do that very rapidly and inexpensively and provide medicines for free to patients. What criteria does a family need to meet to become a n-Lorem candidate? The patient has to have a disease with a mutation that is present in no more than 30 patients in the world. By definition, our patients are truly unique and extremely rare. Patients also have to be genotypically and phenotypically characterized fully. A patient has to have a qualified research physician who can do all the work to understand what's wrong with the patient and then treat the patient with an experimental medicine after we make it. How are some of your patients doing on the drug created for them? The first patient that was treated with a personalized ASO that we were involved in was an 18 year old German named Anna who has a rare form of ALS. It manifests itself typically in early teens, and untreated is extremely rapidly fatal. Anna was desperately ill when the ASO treatment was started and we weren't sure if we'd even get the ASO to her before in enough time. She recovered and did remarkably well. She's now walking up multiple flights of stairs on her own with no ventilator. She has a speaking tube so her mom can hear her talk for the first time in almost three years. She's also planning to go to school. Anna even wrote a personal note to me, and for a patient with ALS to have the fine motor coordination to do that is really quite astonishing. Where do you think we're going be in 5 to 10 years with personalized medicine? We look forward to being able to treat thousands of patients, which means we'll also be learning incredible amounts from each of these patients. We are committed to sharing what we learn and we'll have our first annual meeting of investigators, patients, and parents this year. As we learn more, then I think there will be more interest in this space, and as we create a model of quality, others can follow. I hope that as n-Lorem succeeds, we serve as a model for others to follow, and we drive policy changes that will enable more people, more technologies and more organizations to come to the aid of patients who are unserved. LINKS & RESOURCES MENTIONED https://www.mi-reporter.com/opinion/show-your-stripes-to-honor-rare-disease-day-on-feb-28/ https://www.nlorem.org/ https://www.ionispharma.com/ https://twodisableddudes.com/ https://www.ultragenyx.com/rare-entrepreneur-bootcamp/ https://open.spotify.com/episode/14RO0pNneHdpYxSqEzk1l7
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