Podcasts about regenxbio

Drug development activities focused on the rare, neuromuscular condition Duchenne muscular dystrophy have translated into functional improvements and an extension of life expectancy. Regenxbio is among several companies pursuing a gene therapy to treat Duchenne. Regenxbio believes both its microdystrophin—a truncated form of the dystrophin gene­ small enough to fit in a vector­—and the vector it is using, give it a competitive advantage over other efforts. We spoke Curran Simpson, CEO of Regenxbio, about the company's platform technology, the advantages he sees with its experimental DMD gene therapy, and how a partnership announced at the start of 2025 focused on a pair of other gene therapies provided a welcome alternative to tapping the capital markets.

President Donald Trump doubled down on tariff threats targeting pharma, saying additional levies on pharmaceuticals will come “at some point,” per CNBC. Meanwhile, Johnson & Johnson became the latest big pharma to respond to Trump's warning of potential tariffs if companies don't reshore their manufacturing, announcing a massive $55 billion U.S. manufacturing and R&D investment. Not all companies are on board, however: AstraZeneca is looking eastward, pumping $2.5 billion into a new research facility in Beijing.    Also on the policy front, Trump nominated acting CDC director Susan Monarez for the top job after pulling his first nominee, Dave Weldon, days before his senate hearing was expected to begin. If confirmed, Monarez would be the first CDC director since 1953 to not have a medical degree; she holds a Ph.D. in microbiology and immunology from the University of Wisconsin.   In weight loss news, Novo Nordisk is paying China-based United Laboratories $200 million upfront to license a triple agonist of the GLP-1, GIP and glucagon receptors that could one day compete with Eli Lilly's retatrutide. And BioSpace examines the next great challenge for GLP-1s: oral formulation manufacturing.    Two more therapeutic spaces in focus last week are Duchenne muscular dystrophy and spinal muscular atrophy, where companies including Dyne Therapeutics, REGENXBIO and Novartis presented new data on their respective candidates. And the Duchenne community continued to react to news of the death of a patient taking Sarepta's approved gene therapy Elevidys.  In cardiovascular news, Alnylam won a much-anticipated approval for Amvuttra as the first RNAi silencer for transthyretin amyloid cardiomyopathy, setting up a three-way race with Pfizer's tafamidis—marketed as Vyndaqel and Vyndama—and BridgeBio's Attruby. Next up is Milestone Therapeutics' CARDAMYST in paroxysmal supraventricular tachycardia, which has a PDUFA date of March 27.   Finally, the saga of Cassava Sciences' Alzheimer's hopeful simufilam is over, as the company announced it has ended development of the controversial candidate.   

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sage has rejected Biogen's unsolicited buyout offer and will seek strategic alternatives. Biogen and Eisai have received approval for a monthly maintenance regimen for Alzheimer's drug Leqembi. Veru's drug has shown promise in sparing lean mass in overweight adults on Wegovy. The Duchenne muscular dystrophy space is on the verge of a pivotal era with several companies developing investigational therapies. Akero has rebounded in a mid-stage trial, Daiichi Sankyo is optimistic for 2025, and Allakos is cutting its workforce. The text discusses the advancements in the Duchenne Muscular Dystrophy (DMD) space as it enters a pivotal era, with companies such as Capricor Therapeutics, Wave Life Sciences, and RegenxBio working on investigational therapies to address unmet needs. It also mentions Daiichi Sankyo's recent success with the approval of Astrazeneca-partnered Dato-DXD. Additionally, new treatments are in development for Achondroplasia to challenge Biomarin's Voxzogo. Novo's obesity drug shows promising results, Merck's Keytruda combo fails in a Phase III trial for GI cancer, and Tris Pharma wins late-stage for a non-opioid painkiller. The text also invites feedback from readers on topics they would like to see covered in the future.

Join us each week as we do a quick review of three compelling stories from the pharma world — one good, one bad and one ugly. Up this week:   The good — FDA grants Regenxbio accelerated approval pathway for Duchenne gene therapy The bad —  Neurogene reports patient death in Rett syndrome gene therapy trial The ugly —  Halozyme withdraws acquisition proposal for Evotec

President-elect Donald Trump announced last week that he would nominate prominent anti-vaxxer Robert F. Kennedy Jr. to lead the HHS. While this news caused a dip in biotech stocks, one analyst said the selloff was an overreaction. Meanwhile, fall conference season continues with the American College of Rheumatology Convergence (ACR) and American Heart Association's 2024 Scientific Sessions (AHA). Lupus was the indication du jour at ACR, where BMS, Kyverna, Fate Therapeutics and Cabaletta Bio presented data from early-stage trials of their CAR T candidates, and Biogen and UCB detailed data behind their unexpected late-stage victory for dapirolizumab pegol. This was a much-needed win for Biogen, which has seen its shares decline 36% this year.  Neurogene's stock, meanwhile, tumbled 36% on Monday alone, following the release of details about an adverse event in a trial for its Rett syndrome gene therapy. This followed a 35% fall last week when the adverse event was first announced. And the company decided to drop its gene therapy in Batten disease after the FDA declined to grant Regenerative Medicine Advance Therapy designation to support the program. We also took a look at bluebird bio's cash problem, with the company slated to exhaust its runway to reach a breakeven point before the end of next year. On a more positive note, RegenxBio announced this week it has aligned with the FDA on path to possible accelerated approval for its investigational gene therapy for Duchenne muscular dystrophy, following behind Sarepta's Elevydis, which faced controversy after it received accelerated approval in June 2023, only to miss the primary functional endpoint in its confirmatory trial four months later. And speaking of accelerated approval, we released a special edition of ClinicaSpace Monday focused on this very topic. Sign up to receive it here.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Biopharma company ArsenalBio recently raised $325 million in funding, marking one of the largest biotech funding rounds of the year. This news comes as Biomarin focuses on growth plans centered around their drug for skeletal disorders. However, the gene and cell therapy investment market is currently experiencing a slowdown, with fewer funding rounds closed compared to previous years. On a positive note, a high-dose study of Biogen's spinal muscular atrophy drug showed promising results, while Denali Therapeutics and Regenxbio are moving forward with drug approval applications. In the oncology research sector, advancements in treatments, particularly in cancer immunotherapy and personalized medicine based on genetics, continue to be made. Despite these successes, there are reports of challenges faced by gene therapy companies, such as the closure of Astellas Gene Therapies' manufacturing facility affecting 100 employees and employers hesitant to cover the costs of rare disease treatments.The EU GMP Annex 1 revision has significant impacts on drug manufacturers, as non-compliance puts their ability to create and sell products in impacted markets at risk. Manufacturers must assess components for readiness to comply with the new regulations to avoid manufacturing non-compliance, potential product recalls, and market exclusions. To maintain compliance, staying informed about key considerations and taking steps to mitigate risks is crucial.Mississippi has awarded medicaid contracts to Centene, Molina, and TrueCare. Steward Health Care faced financial challenges with unprofitable deals in Massachusetts, while CEO Ralph de la Torre snubbed a Senate subpoena. Molina appointed its CFO to oversee its medicaid and ACA marketplace businesses. Healthcare providers are addressing burnout among staff, trends in telehealth and provider-payer relationships are being discussed. In California, the Senate passed a bill to regulate private-equity healthcare deals while a judge blocked Ohio from enforcing laws restricting medication abortions.Edwards recently laid off more than 500 employees after selling its critical care division to BD. The FDA has issued warning letters to four companies regarding ozone cleaners for CPAP machines. Abbott has partnered with Beta Bionics to integrate its glucose sensor with an automated insulin delivery system. The European Heart Group recommends renal denervation for some patients with uncontrolled high blood pressure.Biopharma Dive's newsletter on September 5, 2024, highlighted various key topics in the biopharma industry focusing on industry trends and developments. Additionally, it featured sponsored content on AI's role in life sciences and white papers on process analytical technology in the biopharma industry.The pharmaceutical industry faces challenges in reputation and communication with the media despite recent successes such as the development of COVID-19 vaccines. Merck & Co.'s Chief Communications and Public Affairs Officer is promoting openness and transparency in communication to improve the industry's reputation.Cell and gene therapy research is gaining interest from investors and innovators despite facing challenges in keeping up with industry growth. The latest developments focus on overcoming barriers with outside support and potential advancements in CAR-T cell therapy options.Payers can strategically use technology to build a solid data foundation by improving provider data quality through new technology tools.Researchers need to effectively collect social determinants of health (SDoH) data while ensuring privacy and security. A playbook provides insights on utilizing SDoH data effectively for health economics & outcomes research.Industry professionals are invited to join Snowflake Industry Day, a virtual event focusing on how technology leaders use

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Steward Health plans to lay off over 1,200 workers in Massachusetts and close two hospitals. Walgreens has reduced its stake in Cencora for $1.1 billion. The cost of healthcare data breaches is nearly $10 million in 2024, making healthcare the most expensive industry for data breaches. Employers are enhancing healthcare benefits despite rising costs, and Medicare has finalized a higher 2.9% inpatient payment rate for 2025. Virtual reality, robotics, and AI are transforming patient engagement, staff burnout, and clinical decision-making in the healthcare industry. Hospitals remain dissatisfied with Medicare's final rule despite the bump in payment rates.On August 5th, J&J launched the Velys spine surgical robotics and navigation platform, while Inspire received FDA approval for obstructive sleep apnea neurostimulator therapy. Philips is suing an independent lab over alleged errors in CPAP foam tests. Compliance hurdles with the FDA's lab developed test rule raise concerns about patient harm. The board of 23andMe rejected CEO Wojcicki's take-private proposal. J&J aims to compete with Stryker and Zimmer Biomet with its new surgical robotics portfolio. Inspire plans to launch its neurostimulator therapy device late in 2024. Philips claims an independent lab overestimated the threat of CPAP foam, leading to unnecessary recall efforts.Bristol Myers has halted Tigit drug research, while Lilly has resolved the shortage of Zepbound and Mounjaro. Biotech IPOs are vital for industry growth, with companies like Os Therapies and Actuate Therapeutics making moves in the market. Cell therapy is advancing in cancer care despite limited uptake, with biotech companies investing in its improvement. Pfizer discontinued late-stage gene therapy development for Duchenne Muscular Dystrophy (DMD), but other companies like RegenxBio and Capricor Therapeutics are making progress with their therapies. Oncology research is introducing new treatments like cell and gene therapies, revolutionizing cancer treatment.BioNTech reported a significant increase in losses in the second quarter of 2024, jumping to nearly $885 million compared to $208.5 million last year. The company is focusing on oncology amidst the impact of COVID-19. Lilly's drugs Mounjaro and Zepbound are no longer in shortage, according to the FDA. Bristol Myers Squibb is backing out of a $1.5 billion deal with Agenus for a bispecific antibody program, while Bayer's finerenone met its primary endpoint in a phase III heart failure trial. The pharmaceutical industry continues to see developments in various therapeutic areas, including Alzheimer's disease and rare diseases.Support the Show.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Several developments in the biopharma industry were reported on March 6. Gilead made a bet on 'trispecifics' in a new cancer drug deal, Alumis secured $259 million for its work on a drug targeting Tyk2, Regenxbio's data showed promise in Duchenne gene therapy, Alnylam and Roche's blood pressure drug performed well in a mid-stage trial, and Sionna raised $182 million to challenge Vertex in cystic fibrosis treatment. The sector saw a surge in biotech IPOs at the beginning of 2024, prompting discussions on whether this trend will continue. The biosimilars market in the US has been growing steadily since its slow start in 2015. Additionally, Brazil declared a state of emergency due to dengue cases, AstraZeneca invested in the UK for pandemic preparedness, and companies rushed to preserve skeletal muscle after the success of obesity drugs.Dexcom has received FDA approval for the first over-the-counter continuous glucose monitor for people with type 2 diabetes. Vicarious continues to focus on launching its soft tissue robot despite financial struggles. Smiths Medical has recalled nearly 86,000 syringe pumps due to a software issue. Haemonetics is set to acquire Attune Medical for $160 million. Medtronic estimates up to 40 job losses in Ireland as part of restructuring efforts. The EU Medical Device Regulation is causing challenges for small and medium-sized medtech companies. Other news includes Philips being sued for milestone payments, a dentistry AI developer receiving a $53 million investment, and CMS rolling out provider flexibilities amid cyberattack fallout.Alumis has raised $259 million in a Series C funding round, making it the largest haul in 2024 for a private biotech company. The California-based biotech plans to use the funding to move its tyk2 inhibitor into phase III for plaque psoriasis, potentially challenging competitors Bristol Myers Squibb and Takeda in the autoimmune market. Other key focuses for investors in 2024 include psychedelics and solid tumors. Sandoz has received FDA approvals for the first two biosimilars for Amgen's denosumab, while Sionna Therapeutics has closed a $182 million Series C round to potentially challenge Vertex Pharmaceuticals in the cystic fibrosis market. Overall, experts anticipate increased interest in the autoimmune and obesity spaces this year, following a trend of heightened investment in oncology and neuroscience in 2023.In 2024, investors are expected to focus on psychedelics, solid tumors, autoimmune, and obesity drugs in the biopharma industry. Pfizer's decision to prioritize biologics over small molecules was influenced by the Inflation Reduction Act (IRA). Researchers suggest that the IRA could potentially discourage drug developers from pursuing subsequent indications for existing drugs, leading to a decrease in new drug approvals. Biopharma mergers and acquisitions are on the rise, with Biospace launching an M&A tracker to keep track of industry transactions. The 10th Annual American Biomanufacturing Summit is set to bring together industry leaders to discuss current trends and best practices. In other news, AstraZeneca lost a court case related to the IRA, the ADC market in oncology is expected to reach $30 billion by 2028, and various biopharma companies have secured substantial funding for drug development. Pfizer is focusing on four main cancer types and aims to launch eight blockbuster drugs by 2030.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/FHQ865. CME credit will be available until December 15, 2024.Retinal Disease in the ED: “See Me Now”- A Primer for Emergency Physicians on the Recognition and Differential Diagnosis of Retinal Vein OcclusionsThis 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 the American College of Emergency Physicians and PVI, PeerView Institute for Medical Education. The American College of Emergency Physicians is accredited by the ACCME to provide continuing medical education for physicians.This activity is developed in collaboration with our educational partner, PVI, PeerView Institute for Medical Education.SupportThis activity is supported by an independent medical education grant from Regeneron Pharmaceuticals, Inc.DisclosuresRobert M. Hughes, DO, has no financial interests/relationships or affiliations in relation to this activity.Christina Y. Weng, MD, MBA, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alcon; Allergan/AbbVie Inc.; Alimera Sciences; Apellis Pharmaceuticals; Dutch Ophthalmic Research Center (DORC); EyePoint Pharmaceuticals, Inc.; Genentech, Inc.; IVERIC bio, Inc.; Novartis Pharmaceuticals Corporation; Regeneron Pharmaceuticals Inc.; and REGENXBIO.Grant/Research Support from AGTC; Alimera Sciences; and DRCR Retina Network.Other Financial or Material Support in the form of royalties from Springer Publishers.Medical DirectorKristin Tomlinson, PhD, has no financial interests/relationships or affiliations in relation to this activity.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/FHQ865. CME credit will be available until December 15, 2024.Retinal Disease in the ED: “See Me Now”- A Primer for Emergency Physicians on the Recognition and Differential Diagnosis of Retinal Vein OcclusionsThis 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 the American College of Emergency Physicians and PVI, PeerView Institute for Medical Education. The American College of Emergency Physicians is accredited by the ACCME to provide continuing medical education for physicians.This activity is developed in collaboration with our educational partner, PVI, PeerView Institute for Medical Education.SupportThis activity is supported by an independent medical education grant from Regeneron Pharmaceuticals, Inc.DisclosuresRobert M. Hughes, DO, has no financial interests/relationships or affiliations in relation to this activity.Christina Y. Weng, MD, MBA, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alcon; Allergan/AbbVie Inc.; Alimera Sciences; Apellis Pharmaceuticals; Dutch Ophthalmic Research Center (DORC); EyePoint Pharmaceuticals, Inc.; Genentech, Inc.; IVERIC bio, Inc.; Novartis Pharmaceuticals Corporation; Regeneron Pharmaceuticals Inc.; and REGENXBIO.Grant/Research Support from AGTC; Alimera Sciences; and DRCR Retina Network.Other Financial or Material Support in the form of royalties from Springer Publishers.Medical DirectorKristin Tomlinson, PhD, has no financial interests/relationships or affiliations in relation to this activity.

RGX-314 (Regenxbio) could be a one-time gene therapy that addresses diabetic retinopathy. How did it perform in the phase 2 ALTITUDE study? Mark Barakat, MD, shares 1-year safety and efficacy findings from the first two dosing levels of RGX-314 in patients with DR. What are the data? And what's coming next?    And Hani Salehi-Had, MD, joins the show to tell us about the findings in the DIAMOND study, a clinical trial assessing the safety and efficacy of OCS-01 (Oculis), an eye drop designed to treat DME. What did he and his study colleagues find? Keep it here to find out. 

Ken Mills, CEO, President, and Director at REGENXBIO joins Erin Harris for this episode of Cell & Gene: The Podcast to discuss the company's progress with its Phase I/II Affinity Duchenne trial of RGX-202 for the treatment of Duchenne Muscular Dystrophy. They discuss the future of AAV gene therapy as well as achieving scalability with consistent yield and product purity in gene therapy manufacturing.

Note: this episode is cross-posted with the Rx for Biotech podcast, hosted by Chris Leidli. Francesca Cook is the Vice President, Pricing and Market Access, at REGENXBIO and past chair of ASGCT's Government Relations Committee. She's attended and helped with ASGCT's annual Policy Summit since its inception five years ago. In this episode, Cook shares her experience and advice around the event—coming up Sept. 18-19 (virtually + in Washington, DC) with its highest-profile line-up yet and more ways to interact with top CGT decision makers. Her interviewer is Chris Leidli, host of the Rx for Biotech podcast and member of ASGCT's Communications Committee. If you work anywhere in the field of cell and gene therapy (CGT)—science, industry, commerce, regulatory—Cook can tell you why you should register for the Policy Summit, a unique opportunity to discuss how your work can be affected by NIH, FDA, patient advocacy groups, private insurers, and more. And if you've already registered, we look forward to seeing you! Cook has some advice for making the most out of the event. Learn more and register at asgct.org/PolicySummitShow your support for ASGCT!: https://asgct.org/membership/donateSee omnystudio.com/listener for privacy information.

Synopsis: Faraz Ali is the CEO of Tenaya Therapeutics, a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease. Faraz discusses his non-traditional background as an electrical engineer before he found his passion for applying science and technology to healthcare problems. He talks about his experience working at Genzyme and what aspects from his time there he still carries with him today. Faraz also discusses his perspective on the application of genetic modalities, and his team's work pursuing treatment for both rare and prevalent forms of heart disease. Finally, he talks about what it's like being a first time CEO and his thoughts on team building. Biography: Faraz Ali, MBA, is the CEO of Tenaya Therapeutics, a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease. He was most recently Chief Business Officer at REGENXBIO, where he had accountability for corporate development, commercial planning, portfolio strategy, alliance management and corporate communications. Prior to that, Mr. Ali was a Vice President at bluebird bio, where he was responsible for new product planning, program management, patient advocacy and external affairs. He also held roles of increasing global commercial leadership at Genzyme Corporation, where he helped launch multiple first-in-class enzyme replacement therapies for rare diseases. He started his career at General Electric, including technical roles at GE Healthcare and business assignments at GE Corporate. Mr. Ali received his MBA from Harvard Business School and his B.S. from Stanford University. He was appointed to the Board of Directors for The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, in 2022.

Drs. Akshay Thomas and Priya Vakharia join to discuss the March 2023 issue of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered include gene therapy and immunogenecity, applications in dry AMD, and optogenetics.Relevant Financial Disclosures: Dr. Sridhar has consulted for RegenxBio and Apellis in the past 2 years.You can claim CME credits for prior episodes via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Drs. Safa Rahmani, Kat Talcott, and Sarwar Zahid join to discuss the January 2023 issue of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered include the coding for geographic atrophy injections, private equity perspectives, biomarkers for diabetic retinopathy, and early worsening of diabetic retinopathy with anti-diabetic medications.Relevant Financial Disclosures: Dr. Sridhar is a consultant for Alcon, Allergan, Apellis, DORC, Genentech, Ocuterra, and Regeneron. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Rahmani has received research funding from Regeneron. Dr. Zahid has no relevant disclosures.You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Ken Mills discusses his four key ingredients to building a sustainable life sciences company, and why cultivating employees' skills is critical to the future of life sciences innovation.

Ram Palanki, PharmD, VP of commercial strategy and operations for REGENXBIO, has been involved in the development and launch of at least two groundbreaking treatments: Lucentis (ranibizumab, Genentech/Roche) and Macugen (pegaptanib).If all goes well in clinical trials, Dr. Palanki could soon add one more to his extensive CV: a one-time gene therapy designed to treat age-related macular degeneration and diabetic retinopathy (DR). A master of back-of-the-eye product development and commercialization, Dr. Palanki sits down with OIS podcast host Firas Rahhal, MD, to discuss the history of retinal disease treatments and the potential of gene therapy to address early- and later-stage disease.He hopes REGENXBIO's lead product, RGX-314, helps resolve the adherence issues that plague anti-VEGF drugs, the current standard of care. Currently in Phase III trials, RGX-314 introduces a trans gene that can encode for a therapeutic protein for life—a concept REGENXBIO is exploring for the first time. Clinical trials also show a potential benefit for DR. Interim data show about 50% of patients demonstrated a two-step or greater improvement in visual acuity from baseline at six months after a single dose.            Listen to the podcast today to hear Dr. Palanki and Dr. Rahhal discuss: What it was like to be involved in the groundbreaking Lucentis and Macugen launches.How REGENXBIO plans to solve the sustainability problem that arose with the advent of anti-VEGF therapies.The history, mechanism of action, and route of administration behind RGX-314, REGENXBIO's lead program.The strength of RGX-314's adeno-associated viral vector (AAV) and other novel AAV vectors (NAV) technology platform.The philosophy behind REGENXBIO's DR program, including the potential of treating healthy eyes with RGX-314.How Dr. Palanki's responsibilities differ between development and commercialization. Click “play” to listen.

Drs. Safa Rahmani, Katherine Talcott, and Akshay Thomas join to discuss the October 2022 issue of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered include uveitis clinical trials, diagnostic dilemmas in uveitis, floaters, and cataract surgery timing with macular hole surgery.Relevant Financial Disclosures: Dr. Sridhar is a consultant for Alcon, Apellis, DORC, Genentech, and Regeneron. Dr. Thomas is a consultant for Allergan, Avesis, Bausch and Lomb, and Novartis. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Rahmani has no relevant disclosures.You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Drs. Katherine Talcott and Priya Vakharia join to discuss the August 2022 issue of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered pertain to suprachoroidal therapy for macular degeneration, uveitis, and ocular oncology indications.Relevant Financial Disclosures: Dr. Sridhar is a consultant for Alcon, DORC, Genentech, and Regeneron. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Vakharia is a consultant for Genentech and Bausch and Lomb.You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Drs. Sruthi Arepalli, Avni Finn, and Katherine Talcott join to discuss the June 2022 edition of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered include 27 gauge vitrectomy, heads Up display for retinal surgery, evaluating vitreous floaters, and antithrombotics in retinal surgery.Relevant Financial Disclosures: Dr. Sridhar is a consultant for Alcon, DORC, Genentech, and Regeneron. Dr. Finn is a consultant for Allergan and Genentech. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Arepalli has no relevant disclosures.You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Steve Pakola, MD, Chief Medical Officer for Regenxbio, discusses data from the ongoing gene therapy trials in children with mucopolysaccharidosis type I (MPS I) and mucopolysaccharidosis type II (MPS II). The data was presented at WORLDSymposium 2022.MPS I is an inherited lysosomal storage disorder caused by a deficiency in the enzyme, alpha-L-iduronidase, which is responsible for breaking down glycosaminoglycans (GAGs). These GAGs accumulate in the tissues of MPS I patients, resulting in a diverse clinical profile. In moderate to severe forms of the disease, this accumulation in the central nervous system leads to hydrocephalus, spinal cord compression, and cognitive impairment. Additional symptoms may include clouded corneas; enlarged liver, spleen, and heart; noisy breathing; recurring upper respiratory tract; ear infections; difficulty swallowing; and periodic bowel problems. MPS II is a rare, progressive lysosomal disease caused by deficient activity of iduronate-2-sulfatase, attributable to pathogenic variants of the iduronate-2-sulfatase gene (IDS). This disease has a variable clinical presentation but common signs and symptoms include: developmental decline between 18 and 36 months, followed by progressive loss of skills; coarse facial features; skeletal irregularities; obstructive airway and respiratory complications; joint stiffness; retinal degeneration; and communicating hydrocephalus.Currently, the main treatment option for both MPS I and MPS II is enzyme replacement therapy (ERT). Unfortunately, intravenous iduronidase (for MPS I) and idursulfase (for MPS II) cannot pass the blood-brain barrier which makes it ineffective in treating neurological symptoms associated with severe forms of these lysosomal storage disorders. As Dr. Pakola explains, RGX-111 and RGX-121 are both recombinant adeno-associated virus serotype 9 capsids containing the gene that encodes the alpha-L-iduronidase and iduronate-2-sulfatase enzyme, respectively. When administered to the central nervous system both investigational gene therapies may prevent the progression of cognitive deficits that otherwise occurs in MPS I and MPS II patients.

Drs. Greg Lee, Kat Talcott, and Akshay Thomas join to discuss the May 2022 edition of Retinal Physician, found online at http://www.retinalphysician.com. Topics covered include patient and physician Perceptions of intravitreal injection therapy, retinal vein occlusion Clinical Trials, laser for diabetic retinopathy, and uveitis pearls. Relevant Financial Disclosures: Dr. Sridhar is a consultant for Genentech and Regeneron. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Thomas is a consultant for Allergan, Avesis, Bausch and Lomb, and Novartis. Dr. Lee is a consultant for Allergan, Alimera, Apellis, and Eyepoint.You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

Drs. Marianeli Rodriguez, Katherine Talcott, and Akshay Thomas join for a traditional journal club discussion covering four recent publications in major ophthalmology journals:Retinal Detachment Trends During COVID-19 (https://www.ophthalmologyretina.org/article/S2468-6530(22)00046-X/fulltext)Racial Disparities in DME Trials (https://www.ophthalmologyretina.org/article/S2468-6530(22)00040-9/fulltext)Department Gender Diversity Influence on Residency Makeup (https://www.ajo.com/article/S0002-9394(22)00043-5/fulltext)Ghost and Honorary Authorship (https://www.ajo.com/article/S0002-9394(22)00067-8/fulltext)Financial Disclosures: Dr. Rodriguez has no relevant disclosures. Dr. Sridhar is a consultant for Alcon, Allergan, Dorc, Genentech, and Regeneron. Dr. Talcott is a consultant for Genentech, Regenxbio, and Zeiss. Dr. Thomas is a consultant for Allergan, Avesis, Bausch and Lomb, and Novartis.

The idea behind gene therapy is to introduce, remove, or change a portion of person's genetic code in order to cure a disease. Several gene therapy products are currently under development and there are two FDA-approved in vivo products, both using adeno-associated virus (AAV) vector gene delivery. Both of these treatments provide a functional copy of a gene that is not functioning properly in order to provide the body with the instructions for making the appropriate gene product or protein. One is for inherited retinal dystrophy and was approved in 2017, the other is for spinal muscular atrophy and was approved in 2019. In this podcast Dr. Nicholas Buss from REGENXBIO will discuss the science behind the nonclinical development of gene therapy products using AAVs.

REGENXBIO's VP of Regulatory and Science Policy, Nina Hunter, Ph.D., shares potential solutions to gene therapies' regulatory roadblocks and why the accelerated approval pathway is so pertinent. 

Episode 331: Journal Club Including Discussion of Vitreoretinal Procedure Reimbursement, Outcomes of Retinal Detachments with Multiple Surgeries, Post-Hoc Analysis of LEAVO study for retinal vein occlusion, PALADIN study for intravitreal fluocinolone implant,Drs. Shriji Patel and Katherine Talcott join to discuss four recent publications in major ophthalmology journals:Reimbursement of Vitreoretinal Procedures (https://www.ophthalmologyretina.org/article/S2468-6530(21)00379-1/fulltext)Outcomes of Eyes Undergoing Multiple Retinal Detachment Repair (https://www.ophthalmologyretina.org/article/S2468-6530(22)00019-7/fulltext)Visual Outcomes in the LEAVO Study (https://jamanetwork.com/journals/jamaophthalmology/article-abstract/2787670)PALADIN study for fluocinolone implant for diabetic macular edema (https://www.aaojournal.org/article/S0161-6420(22)00067-7/fulltext)Financial Disclosures: Dr. Sridhar is a consultant for Alcon, Allergan, Dorc, Genentech, and Regeneron. Dr. Talcott is a consultant for Genentech and has received grant funding from Regenxbio and Zeiss. Dr. Patel has no disclosures. You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audi

The pipeline in retina is bursting! Which datasets shared at the ASRS 2021 meeting provided insights into the future of retina? Mark Barakat, MD, joins the podcast to discuss topline results from a study examining the suprachoroidal delivery of RGX-314 (RegenxBio). And Nancy Holekamp, MD, stops by to educate us on the potential for targeting C1q in the complement cascade in patients with geographic atrophy. What's the latest with the phase 2 ARCHER study? Find out on this episode.

Most people think of gene therapies as a way to replace a mutated gene with a copy that functions properly. But gene therapies are also being developed as a way to get the body to produce therapeutic proteins. AbbVie in September announced a strategic partnership with RegenxBio to develop and commercialize the company's experimental gene therapy for wet age-related macular degeneration and other eye conditions. The one-time treatment, delivered to the eye, encodes for an antibody fragment designed to inhibit VEGF like the antibodies ophthalmologists regularly inject into the eyes of patients to treat the condition. We spoke to Ken Mills, CEO of RegenxBio, about wet-age macular degeneration, the company's collaboration with AbbVie, and the potential to use gene therapy to alter the way patients with this and other eye conditions are treated.

Throughout his career, Ramin Valian, Vice President of International Glaucoma, Reimbursement Pipeline, for Allergan, has stayed by the side of his mentors.Fresh out of college, where he earned degrees in Middle Eastern studies and Russian, he shifted his goals from working at the U.S. Department of State to joining his older sister at Roche. After learning the pharmaceutical industry ropes as a sales representative, he followed his supervisor, industry leader Joseph Schultz, to Johnson & Johnson and then Allergan.Holding various leadership roles over the past 15-plus years, Ramin has helped launch every one of Allergan's glaucoma products. With host Ehsan Sadri, MD, Ramin discusses his tenure at Allergan, the acquisition by AbbVie, and his ability to adapt to change.When you listen to this podcast, you'll also hear Ramin's take on:The new frontier for presbyopia: the latest on AGN-190584, AbbVie/Allergan's drop.The AbbVie/Allergan innovation pipeline, including a collaboration with REGENXBIO for gene therapy in the retina space.The four pieces of advice he gives to entrepreneurs and mentees.How he's navigated a long career through mergers, buyouts, and other external pressures.

REGENXBIO ($RGNX) presents data using the Clearside Bio ($CLSD) SCS injector to treat wet AMD and diabetic retinopathy patients with RGX-314. Since existing treatments are on the market for these indications, it is very important that safety signals in these studies are reduced to a minimum. It is also critical that efficacy remains non-inferior to Eylea or Lucentis. In this video, I go through both readouts and discuss the potential for the therapy moving forward. 4D Molecular Therapeutics ($FDMT) announces positive early interim data in their intravitreal treatment of 4D-125 in patients with X-linked Retinitis Pigmentosa. With only a small patient population to analyze, it is difficult to derive much conclusion, but so far the efficacy and safety is hopeful. Patients advanced in their disease and also had an extensive prophylactic regimen, making it difficult to assess the treatment's success. In this video, I go through the details of the space and talk about what's next for FDMT. Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email matthewlepoire@gmail.com www.breakingbiotech.com #breakingbiotech Disclaimer: All opinions expressed by Matt (or his guests) in this podcast are solely his (their) opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt's opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

REGENXBIO and Abbvie announce a collaboration to co-develop RGX-314 for the treatment of wet AMD, diabetic retinopathy and other chronic retinal diseases. This massive announcement puts a lot of resources behind the commercialization efforts of RGX-314 in anticipation of an eventual approval. In the short-term, $RGNX benefits from a large upfront payment as well as upcoming milestone payments. $RGNX/$CLSD are about to announce Cohort 1 data from their SCS wet AMD trial, which if positive, will be very bullish for the company. I talk about what I'm looking forward to here. As we approach Q4, a number of interesting oncology companies are set to provide updates in their clinical programs. Companies I have my eye on are: $ONCT, $CRIS, $BTAI and $PDSB and in this video, I discuss where they are in their programs and how I feel about them as potential investments. $RGNX/$CLSD/$ABBV - 1:05 $APLS - 7:30 $ONCT - 15:20 $CRIS - 21:28 $BTAI - 26:00 $PDSB - 32:29 Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email matthewlepoire@gmail.com www.breakingbiotech.com #breakingbiotech Disclaimer: All opinions expressed by Matt (or his guests) in this podcast are solely his (their) opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt's opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

In this episode editors Virginia Li, Michael Flanagan and Simon King discuss AbbVie's deal to in-license an investigational gene therapy for age related macular degeneration from Regenxbio, an increasing focus on mRNA vaccines and therapeutics among Chinese biotech companies and why a new academic study may pose further challenges for Biogen's Aduhelm.

The pipeline of wet AMD treatments is inspiring. What can retina specialists around the world expect when it comes to wet AMD therapy candidates KSI-301 (Kodiak Sciences), RGX-314 (RegenxBio), and ADVM-022 (Adverum Biotechnologies)? Join moderator Arshad M. Khanani, MD, MA (United States), and panelists Justus G. Garweg, MD (Switzerland), Seenu M. Hariprasad, MD (United States), and Peter Kertes, MD, FRCSC (Canada), as they review early-phase data from some of the possible wet AMD drugs of the future. 

Various companies in the Ophthalmology gene therapy space saw upsets from either an efficacy, safety or collaboration standpoint. However, three related companies have upcoming catalysts in the second half of 2021 that could turn sentiment around. They are: 4D Molecular Therapeutics, REGENXBIO and Clearside Biomedical. In this episode, I talk about eye diseases caused by neovascularization, discuss the sector as it stands today and explain my investment theses for each of those 3 companies. I also talk about the troublesome position Biogen has found themselves in and what my plan is for the position I have in the company. Check out our NEW sponsor: BiopharmIQ, which is the best place to get consolidated information about small-mid cap biotechs. Use my link here to let them know that I sent you: https://bit.ly/3iwVGbu Presentation slides: https://drive.google.com/drive/folders/1MENe3kFzFosR6-ALPoY9ovpXXcERH_j3?usp=sharing Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech Follow me on twitter @matthewlepoire Send me an email matthewlepoire@gmail.com www.breakingbiotech.com #breakingbiotech Disclaimer: All opinions expressed by Matt (or his guests) in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt's opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

Join me this week as I talk to one of my clients, Jenna Burke about putting yourself first and making powerful changes in your career and your life.Jenna works in clinical operations in the biotechnology industry (REGENXBIO).  When we started working together she was looking to make big changes in her career.  She learned who she really was, stopped comparing herself to others, got happy in her career and says she doesn't even recognize the person she was.Listen in to hear her journey, lessons, and advice for anyone who feels they want more in their career and their life.To apply for coaching, go to www.melissamlawrence.com/apply.

This week on the OIS podcast, host Firas Rahhal, MD, Partner at ExSight Ventures & Retina Vitreous Associates Medical Group facilitates an illuminating discussion on the topic of retina start-ups with a focus on providing tips to anyone interested in joining the retina space. Our guests include Andrew Stewart, AVP, US Commercial Retina of Allergan; Jay Duker, MD, Chief Strategic Scientific Officer, EyePoint Pharmaceuticals; Paul Hallen, VP & Global Head, Retina & Glaucoma, Alcon; and Ram Palanki, PharmD, SVP, Commercial Strategy & Operations, REGENXBIO.Listen in as this group shares advice on the topics of talent, asset quality, company strategies, and product development across the board. Learn what companies are looking for and where there is room for start-ups to flourish!

This NRR miniseries “The Art of Drug Choice: Wet AMD and the Latest Data” continues as Dr. Khanani and the roundtable participants Drs. Fuller, London, and Weng review the wet AMD pipeline with a focus on anti-Ang-2 therapy (faricimab, Roche/Genentech) and gene therapy (RGX-314, RegenxBio and ADVM-022, Adverum Biotechnologies). After the break, Dr. London shares the case of a patient with wet AMD who experienced a ceiling of response to anti-VEGF therapy. Did a switch to brolucizumab (Beovu, Novartis) result a change? 

This NRR miniseries "The Art of Drug Choice: Wet AMD and the Latest Data" continues as Dr. Khanani and the roundtable participants Drs. Fuller, London, and Weng review the wet AMD pipeline with a focus on anti-Ang-2 therapy (faricimab, Roche/Genentech) and gene therapy (RGX-314, RegenxBio and ADVM-022, Adverum Biotechnologies). After the break, Dr. London shares the case of a patient with wet AMD who experienced a ceiling of response to anti-VEGF therapy. Did a switch to brolucizumab (Beovu, Novartis) result a change?

The economics of developing gene therapies can make it unattractive for biopharmaceutical companies to invest in bringing a gene therapy through development and to the market for ultra-rare conditions. But researchers at the National Center for Advancing Translational Sciences are working to develop a set of gene therapy vectors that can be used in multiple indications and eliminate the time and cost of preclinical development for a range of conditions through its Platform Vector Gene Therapy, or PaVe-GT program. In this fourth and final part of our gene therapy series, we spoke to P.J. Brooks, program director in the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences, about the PaVe-GT program, the potential for developing a toolkit of plug-and-play vectors, and how this can alter the cost of developing gene therapies for ultra-rare and individual patients. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

Though there are only a handful of gene therapies on the market today, there is a robust and growing pipeline of these transformative medicines advancing toward market. In this third part of our gene therapy series we spoke to Janet Lambert, CEO of the Alliance for Regenerative Medicine, about the state of the gene therapy industry, the challenges developers face in advancing therapies to the market, and emerging pricing approaches to make them accessible to the patients who need them. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

Genetic counselors play a unique role in the medical life of a person with a rare disease. They can serve as guide, translator, and trusted advisor. In this first part of our four part series on gene therapies, we spoke to genetic counselor Stephanie Gandomi, a Global Genes advocacy resource, about gene therapies, the role genetic counselors play in working with rare disease patients, and the considerations rare disease patients may make about whether to pursue a gene therapy through a clinical trial. This series is made possible through support from BioMarin, Pfizer, Retrophin, Novartis Gene Therapies (formerly AveXis), UCB Inc., Genentech, Ultragenyx, Novartis, RegenxBIO, and Sangamo Therapeutics.

On this week’s OIS Retina podcast, we have the privilege of talking to Steve Pakola, MD. Steve is an industry veteran who has held key leadership roles in preclinical and clinical development, regulatory affairs, and medical affairs. Currently, he serves as Chief Medical Officer at REGENXBIO, a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.

In this episode, John S. Pollack, MD, discusses how he got started with the American Society of Retina Specialists, gives the scoop on the PAT survey and provides career advice for young retina specialists. Intro :15 In this episode :18 About Dr. Pollack :24 The interview :45 Who is Dr. Pollack? 1:10 What’s your favorite part about the field of retina? 2:37 How did you get involved with the ASRS? 3:58 Who write the questions for the PAT survey? 5:54 How should community retina specialists utilize and view the PAT survey? 7:04 Are you currently taking responses? 7:50 What are the top issues ASRS will be dealing with this year? 8:12 Indications for surgery for epiretinal membranes: visual acuity or visual function? 8:50 What will have the biggest impact on retina for years to come? 15:28 Career advice for young retina specialists; academic vs. private practice? 18:49 Do you factor health care policy/reform into this decision? In 5 years, will health care be the same? 21:32 The Winning Pitch Challenge 22:51 How an idea in clinic turned into a product 26:45 Thanks for listening! 28:00 John S. Pollack, MD, is past president of the American Society of Retina Specialists; partner at Illinois Retina Associates, Rush University Medical Center in Chicago. Disclosures: Mali reports he is founder and CEO of Mali Enterprises; retina medical director at Macular Degeneration Association; grant/research support from Alimera Sciences, Allergan/AbbVie, Chengdu Kanghong Biotechnology, Genentech, Notal Vision, Regeneron and Santen; consulting for Alimera Sciences, Allergan/AbbVie, Eyepoint Pharmaceuticals, Genentech, Kala Pharmaceuticals, Macular Degeneration Association, Notal Vision, Novartis, Regeneron and Sun Pharmaceuticals; speaker bureau for Alimera Sciences, Genentech, Kala Pharmaceuticals, Macular Degeneration Association, Notal Vision, Novartis and Sun Pharmaceuticals; and stock/shareholder with AbbVie and Regeneron. Pollack reports grant/research support from Aldeyra; consulting for Allergan, Dutch Ophthalmic Research Company, Graybug, Regeneron, REGENXBIO and Santen; founder and stock at Covalent Medical; grant support and consulting from Genentech; BOD, stock and consultant for Notal Vision; and founder and stock at Vestrum Health. We’d love to hear from you! Send your comments/questions to Dr. Mali at eyecareinsider@healio.com. Follow us on Twitter @Healio_OSN

Lisa Carlton, PhD, understands rare childhood diseases both professionally and personally. She was already a scientist specializing in rare diseases when her daughter was diagnosed with one: tuberous sclerosis. As VP of Global Regulatory Affairs for a biotech drug developer she is involved in development of treatments for other rare diseases too. This makes her an ideal person to co-chair an industry-patient-advocacy group called the Working Group on Regulatory Science, a part of the EveryLife Foundation for Rare Disorders that seeks to advance the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.Tuberous sclerosis, a rare genetic disorder, affects one in every 6,000 newborns in the United States. It is a disorder that causes benign growth in almost any tissue; it’s of particular concern when it occurs in the brain, lungs or kidneys. Some people who have tuberous sclerosis may have learning problems or difficult-to-control seizures. Her daughter—one of a pair of twins—was diagnosed in utero. She’s doing well, although the speech and language disorders associated with the condition have made it hard to access the curriculum in school. Dr. Carlton talks about the challenges of developing new treatments for rare diseases and the approaches taken to try to overcome them. She also talks about her daughter’s disease, and how families can bring their best to their journey as they adapt to support their child. Based on her experience as a parent and advocate—as well as her experience in the industry—she has this advice for families: “Take a breath.” Drug development is slow, especially in rare diseases. “You are in for a marathon of supporting your child.” When possible, connect with other parents and advocacy groups. Or build your own tribe—work, friends, family, etc. “You have to be healthy and rested enough to do this. You have to be at your best.” She’s often asked how she manages to keep going. “I don’t know how I wouldn’t do it. You’ve been given this gift of a special child. Now it’s time to find your tribe and push ahead.”Her advice for a biotech company considering launch of a clinical trial for a rare disease? Nobody knows more about a patient’s journey, the patient’s experience, than the patient—or a parent or caregiver. “Just listen to what patients are saying. You are going to learn a lot.”Lisa Carlton, PhD, has previously worked in regulatory roles at the National Institutes of Health, Otsuka Pharmaceuticals and International Partnership for Microbicides. She is currently serving as Vice President of Global Regulatory Affairs at REGENXBIO, a gene therapy-focused biotech company with headquarters in Rockville, MD. Lisa received her PhD in Pharmaceutics and MS in Medicinal Chemistry from the University of North Carolina in Chapel Hill and a BS in Biochemistry from North Carolina State University.

Pfizer's latest phase 3 trial results put its promising eczema drug on a path to challenge Regeneron and Sanofi's $2 billion-per-year drug, Dupixent. Also, how Regenxbio and Adverum Biotechnologies hope to change how a we treat a common cause of vision loss in seniors, and why United Healthcare's latest quarterly results sent shares soaring. Check out more of our content here: TMF's podcast portal YouTube Twitter Join Our Motley Fool Podcast Facebook Group LinkedIn StockUp, The Motley Fool's weekly email newsletter

Here's why post-approval setbacks have caused Puma Biotechnology, Clovis Oncology, and Regenxbio shares to tumble, and if any of these stocks are finally worth buying. Stocks: PBYI, CLVS, RGNX Check out more of our content here: TMF's podcast portal YouTube Twitter Join Our Motley Fool Podcast Facebook Group LinkedIn StockUp, The Motley Fool's weekly email newsletter

ay is joined by Dr. Amy Schefler, Dr. Chirag Shah, and Dr. Nathan Steinle for a panel discussion regarding how to best approach maximizing retina clinic efficiency and other issues new retina attendings in practice may encounter. You can now claim CME credits via the AAO website. Visit https://www.aao.org/browse-multimedia?filter=Audio&sub=ONE.ContentTypes.Audio. Financial Disclosures: Dr. Sridhar: Consultant for Alcon, Alimera Sciences, and Thrombogenics. Dr. Schefler: Consultant for Allergan, Aura Biosciences, Castle Biosciences, Genentech, and Regeneron. Receives research support from Aura Biosciences, Castle Biosciences, Genentech, and Regeneron. Dr. Shah: Consultant for Regeneron. Receives research support from NIH, Alcon, Genentech, Novartis, Regeneron, Apellis, and Ellex. Dr. Steinle: Consultant for Apellis Pharmaceuticals, Alimera Sciences, Regeneron Pharmaceuticals, Genentech, Regenxbio, Carl Zeiss Meditec, Regenerative Patch Technologies, Novartis, and Vortex Surgery. Speaker’s Bureau for Alimera Sciences, Genentech, Regeneron Pharmaceuticals, NotalVision; and Novartis. Receives research support from Carl Zeiss Meditec, Genentech, Regeneron Pharmaceuticals, and Novartis. Investor in Vortex Surgical and Garbha Health.

Today, I talk about REGENXBIO and their in-house gene therapy candidates. Their most important asset in early clinical trials is RGX-314, indicated for wet AMD (and hopefuily Diabetic Retinopathy). This gene therapy could replace blockbuster drugs that treat millions patients, but is it a buy today? Follow me @matthewlepoire www.breakingbiotech.comthis is not investment adviceSupport Breaking Biotech by donating to the tip jar: https://tips.pinecast.com/jar/breaking-biotech

Here's why Roche's acquisition of gene therapy pioneer Spark Therapeutics and Biogen's acquisition of gene therapy upstart Nightstar Therapeutics could put Regenxbio in play. RHHBY, ONCE, BIIB, NITE, RGNX Check out more of our content here: TMF's podcast portal YouTube Twitter Join Our Motley Fool Podcast Facebook Group LinkedIn StockUp, The Motley Fool's weekly email newsletter

The U.S. Food and Drug Administration recently granted gene therapy developer RegenxBio Orphan Drug designation for its experimental gene therapy to treat CLN2 disease, a form of Batten disease, a rare and deadly condition that progressively robs children of their abilities until they die. The gene therapy is one of 12 clinical programs and 20 partnered programs RegenxBio is advancing. We spoke to Ken Mills, CEO of RegenxBio, about CLN2 disease, the company’s next-generation gene therapy platform, and why he believes it offers advantages over other AAV gene therapies.

REGENXBIO founding CEO Ken Mills reviews the company’s recent positive Phase I results for RGX-314, a potentially game-changing gene therapy for wet AMD. Mills also shares the responsibility he feels leading a pioneer in the blockbuster gene therapy field.