Podcasts about uniqure

On this week's episode, Daphne Zohar, Josh Schimmer, Tim Opler, John Maraganore, and Paul Matteis discuss the biotech market's recent resurgence, and new FDA commissioner Marty Makary's comments during his interview with former Fox News journalist Megyn Kelly. The discussion then shifts to the FDA's reorganization, confusing return-to-work policies, and low morale at the agency, which resulted in slower review times. The conversation moves to BMS' Cobenfy Phase 3 ARISE trial results and uniQure's breakthrough therapy designation for its Huntington's disease gene therapy. The group then revisits a discussion about biotech market updates and short selling. They also shared perspectives on Harvard University's lawsuit against the Trump Administration, the broader issue of academic freedoms, and antisemitism on college campuses. The episode concludes with a discussion on the challenging microenvironments, CEO survival tips, and an overview of how investors are evaluating data. *This episode aired on April 25, 2025.

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.

uniQure Biopharma B.V. v. Pfizer Inc.

This week, GSK and Gilead and Arcellx presented key data at the American Society of Hematology (ASH) annual meeting as they vie for a competitive advantage in multiple myeloma. Meanwhile, Vertex unveiled positive long-term data for its CRISPR Therapeutics–partnered gene therapy Casgevy in sickle cell disease—results BMO Capital Markets analysts said should help Casgevy keep its edge over bluebird bio's Lyfgenia. On that note, Casgevy and Lyfgenia have a new outcomes-based payment model after the Centers for Medicare and Medicaid Service (CMS) said both companies have agreed to participate in a voluntary program to improve access to the gene therapies. Speaking of access, Eli Lilly and Novo Nordisk both announced significant manufacturing investments aimed at shoring up production of their diabetes and weight loss blockbusters tirzepatide and semaglutide. And in related news, the European Commission gave its blessing to Novo Holdings' controversial acquisition of contract manufacturing firm Catalent. Elsewhere, AbbVie got a much-needed win for Cerevel-acquired Parkinson's disease therapy tavapadon—a month after the deal's cornerstone asset emraclidine failed in schizophrenia—while uniQure announced it has aligned with the FDA on “key elements” of the accelerated approval pathway for its investigational gene therapy for Huntington's, AMT-130.

Lauren Holder recently did a webinar with HDYO's Jenna Heilman, Prof. Hugh Rickards, and HDYO Ambassador Molly about breaking down barriers when it comes to access to a potential treatment. You can watch the full video here: https://www.youtube.com/watch?v=y6CrrVGfBTk Breaking Down Barriers is sponsored by Roche, Sage Therapeutics, Teva Pharmaceutical, uniQure, Wave Life Sciences, The Griffin Foundation, Neurocrine Biosciences and PTC Therapeutics.

On this week's episode, hosts Chris Garabedian, Brad Loncar, Tim Opler, Dawn Bell, John Maraganore and Eric Schmidt provide a deep dive into the obesity, diabetes and cardiometabolic space, beginning with an overview of the key findings in Stifel's latest obesity report. The hosts also provide a recap of ADA and EASD abstract highlights and take a look at the next wave of injectable therapies and the trends in consumerization and access. They also discuss Pfizer's update on its once daily oral GLP-1 pill as well as recent reports on patients discontinuing GLP-1 drugs in two years and GLP-1s for obesity-related cancers. Shifting gears, the hosts spotlight a report on the evolving pharma landscape and the future landscape of the blockbuster drug category. In deals of the week, the group discusses Eli Lilly agreeing to buy Morphic for $3.2 billion and Flagship's new $3.6 billion fund. In data news, the hosts cover Roche's setback with its pivotal TIGIT trial, uniQure's positive data for its Huntington's gene therapy and IDEAYA's positive interim phase 2 MAT2a data. *This episode aired on July 12, 2024.

Recap of my visit with UniQure for HD Awareness Month

MedLink Neurology Podcast is delighted to feature selected episodes from BrainWaves, courtesy of James E Siegler MD, its originator and host. BrainWaves is an academic audio podcast whose mission is to educate medical providers through clinical cases and topical reviews in neurology, medicine, and the humanities, and episodes originally aired from 2016 to 2021. Originally released: March 29, 2018 What does a brain look like in a patient with a functional movement disorder? Pretty normal, it turns out. But beneath the normal anatomy is a storm of aberrant signaling. Dr. Mark Hallett, Chief of the Human Motor Control Section of the NIH, describes the underlying neurophysiology in this spectrum of disorders. Produced by James E Siegler. Music by Damiano Baldoni, Kevin MacLeod, Ondrosik, and the Philadelphia String Quartet. Voiceover by Erika Mejia. BrainWaves' podcasts and online content are intended for medical education only and should not be used for clinical decision-making. REFERENCES Maurer CW, LaFaver K, Ameli R, Epstein SA, Hallett M, Horovitz SG. Impaired self-agency in functional movement disorders: a resting-state fMRI study. Neurology 2016;87(6):564-70. PMID 27385746Nahab FB, Kundu P, Gallea C, et al. The neural processes underlying self-agency. Cereb Cortex 2011;21(1):48-55. PMID 20378581Vuilleumier P, Chicherio C, Assal F, Schwartz S, Slosman D, Landis T. Functional neuroanatomical correlates of hysterical sensorimotor loss. Brain 2001;124(Pt 6):1077-90. Erratum in: Brain 2016;139(Pt 5):e29. PMID 11353724 DISCLOSURES Dr. Hallett receives funding from the Intramural program of the National Institute of Neurological Disorders and Stroke. No relevant conflicts of interest are present. He serves as Chair of the Medical Advisory Board for and receives honoraria and funding for travel from the Neurotoxin Institute. He may accrue revenue on US Patent: Immunotoxin (MAB-Ricin) for the treatment of focal movement disorders, and US Patent: Coil for Magnetic Stimulation and methods for using the same (H-coil); in relation to the latter, he has received license fee payments from the NIH (from Brainsway) for licensing of this patent. Supplemental research funds have been granted by BCN Peptides, S.A., for treatment studies of blepharospasm; Medtronics, Inc., for studies of deep brain stimulation; UniQure for a clinical trial of AAV2-GDNF for Parkinson Disease; Merz for treatment studies of focal hand dystonia; and Allergan for studies of methods to inject botulinum toxins. Jim is lucky enough to have no relevant competing financial interests.  We believe that the principles expressed or implied in the podcast remain valid, but certain details may be superseded by evolving knowledge since the episode's original release date.

We're in Amsterdam with Sander van Deventer, the CEO of VectorY. We will talk about VectorY and what's missing in gene therapy.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. In today's episode, we have several news updates in the biopharma industry. Let's dive in:## News 1: Uniqure's gene therapy setbackUniqure's gene therapy for Huntington's disease faced a setback as the company's share price fell over 10% following the release of more data from a small study. This news highlights the challenges in developing effective therapies for genetic diseases.## News 2: Novo Nordisk's investment in vaccine researchThe foundation behind Novo Nordisk plans to invest $260 million in vaccine research and development. The initiative will focus on developing vaccines for tuberculosis, influenza, and group A streptococcus. This investment reflects the commitment to improving public health and addressing global health challenges.## News 3: Biogen's market monopoly for TecfideraBiogen's multiple sclerosis medicine, Tecfidera, will maintain its market monopoly in Europe until early 2025 after European regulators revoked approvals for five generic versions of the drug. This decision ensures continued access to this important medication for patients with multiple sclerosis.## News 4: Merck's new pneumococcal vaccineMerck has received an FDA decision date for its new pneumococcal vaccine, which targets 21 strains of pneumococcal bacteria and would compete with Pfizer's Prevner 20 if approved. This development brings hope for improved prevention of pneumococcal infections.## News 5: Fewer biotech companies going publicFewer biotech companies are going public after a record run, and the article discusses how these companies are performing in the market. This trend suggests a shift in the biotech industry and raises questions about future growth and innovation.## News 6: Structure's obesity pill falls shortStructure shares plummeted as results from a phase 2 trial for their obesity pill fell short of expectations. This outcome highlights the challenges of developing effective treatments for obesity, a prevalent and complex health issue.Moving on to our next set of news:## News 7: FTC and DOJ finalize merger guidelinesThe Federal Trade Commission (FTC) and the Department of Justice (DOJ) have finalized merger guidelines that could make it more difficult for healthcare mergers and acquisitions to proceed. These new guidelines aim to ensure fair competition and protect consumers' interests.## News 8: Biden administration's call for action on child Medicaid enrollmentThe Biden administration is urging nine states to address high disenrollment rates in child Medicaid and Children's Health Insurance Program (CHIP). These nine states account for around 60% of the decline in enrollment in these programs between March and September. The administration is taking steps to address this issue and improve access to healthcare for children.## News 9: John Muir Health and Tenet Healthcare deal collapsesThe deal between John Muir Health and Tenet Healthcare for the acquisition of a California medical center has collapsed following a lawsuit from the FTC. The regulators sued to block the deal over concerns that it could lead to increased healthcare costs in the region. This turn of events highlights the importance of regulatory oversight in healthcare mergers and acquisitions.## News 10: Molina Healthcare lowers sale priceMolina Healthcare has agreed to lower the sale price of Bright Health's California Medicare Advantage unit. The original sale price was $510 million, but it has been lowered to $425 million. This adjustment reflects the changing dynamics of

The debut episode hosted by Elena Buglo, PhD features Josh Yoder, PhD, a medical affairs leader with expertise in virology and gene therapy, focusing on hemophilia. Our discussion delves into:

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. In the fourth quarter of this year, the FDA is expected to make several important decisions. One of these decisions involves the potential approval of the first CRISPR medicine, which could have significant implications for gene therapy. Additionally, the FDA is considering clearances for companies such as Alnylam, Amgen, and Pfizer, which could lead to the approval of important new therapies.In a major acquisition move, Eli Lilly is set to enter the radiopharmaceutical drug field with a $1.4 billion acquisition of Point Biopharma. This deal will give Lilly access to a pipeline of experimental radioligand therapies and a valuable manufacturing plant. Speaking of leadership changes, Lilly has also announced that Mike Mason will be retiring as the head of its diabetes and obesity division, with Patrik Jonsson taking over.SAS, an analytics company, is encouraging the adoption of open-source technology in the life sciences industry. They emphasize the importance of maintaining security and compliance while utilizing open-source tools.AbbVie has acquired Mitokinin, a Parkinson's drug developer. This acquisition allows AbbVie to expand its portfolio in neurology.Moving on to other news, Amgen has completed its $28 billion acquisition of Horizon after facing challenges from the Federal Trade Commission. However, an FDA panel has found that the data for Amgen's lung cancer drug Lumakras is unreliable, which is a setback for the company. The National Institutes of Health (NIH) has funded research for three experimental drugs for amyotrophic lateral sclerosis (ALS) as part of the ACT for ALS program. Dana-Farber spinout Precede has emerged from stealth with $57 million in funding for its blood test technology.Aetna, the insurance company, may have received overpayments of $25.5 million in Medicare Advantage (MA) for 2015 and 2016. Novant Health has cut 160 jobs due to a challenging healthcare environment. A study published in JAMA Network Open found that physician burnout rates doubled between 2019 and 2021.GE Healthcare Imaging CEO, Jan Makela, discussed the collaboration between GE and Mayo Clinic to advance healthcare through technological innovation. The FDA has called for further tests on recalled devices from Philips due to inadequate testing. A retrospective study by Johnson & Johnson found that catheter ablation is more effective than antiarrhythmic drugs in reducing the risk of heart failure. Northwestern University researchers have found that generative AI models can match the accuracy and quality of radiologist reports.Amgen has successfully completed its acquisition of Horizon and plans to provide updated guidance during its third-quarter earnings call. Mirati Therapeutics is reportedly in acquisition discussions with Sanofi. The Oncologic Drugs Advisory Committee has voted against Amgen's request for full approval of its G12C KRAS inhibitor Lumakras. A study published in JAMA has found that GLP-1 agonists are associated with an increased risk of gastrointestinal adverse events when used for weight loss.In the gene therapy field, Kyowa Kirin is set to acquire Orchard, while uniQure has laid off staff and cut back on research to lower costs. Regeneron and Intellia have expanded their gene editing partnership to target neurological diseases. These developments highlight the ongoing advancements in biopharma and the potential for significant breakthroughs in medicine. Stay tuned for more updates on acquisitions, FDA decisions, cost-cutting measures, and partnerships in the gene therapy field.

On this week's episode of Biotech Hangout, hosts Daphne Zohar, Tim Opler, Chris Garabedian, Mike Yee and Bruce Booth discuss the week's industry news including M&A, regulatory, data and more. They highlight the continued M&A streak with Eli Lilly acquiring Dice Therapeutics. They also discuss PhRMA's lawsuit vs. the U.S. government over IRA Medicare Drug Price Negotiations, following similar suits from Merck & Bristol Myers Squibb. They also talk about several Duchenne news items including Santhera licensing its Duchenne drug to Catalyst and Sarepta's DMD therapy approval. Additional industry news covered includes the SEC's case against biotech executives and investors and conflicts of interest with PBMs and FTC's lawsuit against Amgen. The hosts also discuss what happened to big academic tech transfer deals, Leerink is back, Intercept, Alderya and Arcellx regulatory news, plus Uniqure and Roivant data. *This episode aired on June 23, 2023*

Synopsis: Joel Schneider, Ph.D. and Robert Kotin, Ph.D. are the President & CEO and Founder & Chief Technology Advisor, respectively, of Carbon Biosciences, an emerging leader in the development of novel parvovirus-derived gene therapies. Joel and Robert sit down with host Rahul Chaturvedi to discuss the arc of their careers, how the AAV field has changed over the last decade, and how they approach building out their team at Carbon. They also talk about Carbon's focus on cystic fibrosis and the implications of The Cystic Fibrosis Foundation investing in their Series A. Finally, they both weigh in on what excites them when thinking about the future of gene therapy. Biographies: Dr. Joel Schneider joined Carbon as CEO in 2022, after serving as Chief Operating Officer at Solid Biosciences. As Solid's first employee, he played an instrumental role in building the company's unique disease-focused business model. Dr. Schneider is an accomplished biosciences executive with a track record of achievement in identifying, developing, and financing high potential therapeutic modalities and has diverse leadership experiences across R&D, technical and corporate operations, and corporate development organizations. Dr. Schneider holds a Ph.D. from Rutgers University and an undergraduate degree from Brandeis University and is the author of numerous peer-reviewed articles related to Duchenne and stem cell biology. He completed a postdoctoral fellowship at Harvard University in the Department of Stem Cell and Regenerative Biology, where he characterized and developed the small molecules that enhance skeletal muscle regeneration. Dr. Robert Kotin has been a leader in adeno-associated virus (AAV) research for 35 years, focusing on the molecular biology of the virus's non-structural proteins and then leveraging this understanding to develop novel AAV vectors for somatic cell gene therapy. Beginning as a postdoctoral fellow at Cornell University Medical Center, Dr. Kotin discovered a common integration site for AAV DNA in human chromosome 19, which he designated AAVS1 locus. He spent most of his career in the Intramural Research Program at the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH), first as a tenure-track investigator and then as a tenured senior investigator and Head of the Laboratory of Molecular Biology and Gene Therapy. While at the NIH, Dr. Kotin's laboratory invented and developed a scalable recombinant adeno-associated virus (rAAV) production process in Sf9 cells which was licensed by UniQure, ThermoFisher, Voyager, Biomarin, and others and was used to produce Glybera™, the first rAAV product granted regulatory agency approval for sale. Additional research from the Kotin lab resulted in the discovery of an AAV replicative product that has been described as closed-ended linear duplex DNA (ceDNA) and became the basis of the non-viral gene therapy company Generation Bio (NASDAQ: GBIO). Dr. Kotin served as vice president of virology and gene therapy at Voyager Therapeutics from 2014 to 2016. Since 2016, he has served as an adjunct professor at UMass Medical School, where his research interests include vectorizing and characterizing ancestral parvoviruses based on inferred sequences from endogenous virus elements (EVEs) as novel gene therapy vectors. Dr. Kotin earned his B.A. in biology from the University of California, Santa Cruz, and his doctorate in microbiology from Rutgers University and the University of Medicine and Dentistry of New Jersey (now Robert Wood Johnson Medical School).

Congress is moving quickly to pass legislation before the year ends. Hosts Terry and Bob discuss the health care bills that, if passed, could increase patients' access to affordable and transparent care. From telehealth to generic drug access, learn how this legislation would impact you.And hear from Dan “Dry Dock” Shockley, a Navy veteran living with Attenuated FAP, on his journey from diagnosis to advocacy.Hosts: Terry Wilcox, CEO, Patients RisingDr. Robert Goldberg, “Dr. Bob,” Co-Founder and Vice President of the Center for Medicine in the Public InterestGuests:Dan “Dry Dock” Shockley, retired Navy, Operation Desert Storm; Enduring and Iraqi Freedom veteran and 10 year hereditary colon cancer syndrome (AFAP) virtual international live-case presentationLinks: Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA's finish linePatients Rising Now and 25 Other Patient Groups Send Letter to Hill on Generic DrugsIt's lame-duck time. Here are Congress' health prioritiesWhite House seeks more covid funding in lame-duck session Telehealth policy changes after the COVID-19 public health emergencyWhat's Expected to Happen During Democrats' Lame Duck SessionNeed help?The successful patient is one who can get what they need when they need it. We all know insurance slows us down, so why not take matters into your own hands? Our Navigator is an online tool that allows you to search a massive network of health-related resources using your zip code so you get local results. Get proactive and become a more successful patient right now at PatientsRisingConcierge.orgHave a question or comment about the show, or want to suggest a show topic or share your story as a patient correspondent?Drop us a line: podcast@patientsrising.orgThe views and opinions expressed herein are those of the guest(s)/ author(s) and do not reflect the official policy or position of Patients Rising, nor do the views and opinions stated on this show reflect the opinions of a guest's current or previous employers.

Biogen recently named Christopher Viehbacher its new CEO, ending a monthslong search to replace Michel Vounatsos. Fraiser Kansteiner and Kevin Dunleavy talk about the current state of the company and recent investor reactions to the new hire. Plus, they discuss a string of other biopharma CEO appointments.  And, in this episode, we cover the week's news including what Albert Bourla, Ph.D., said that has him in hot water along with a $3.5 million drug, and we tease a possible major acquisition.  To learn more about topics in this episode: Biogen's incoming CEO Viehbacher gets mixed reviews—and lots of advice—in investor survey  Sporting a $3.5M price tag, CSL and uniQure's hemophilia B gene therapy crosses FDA finish line  Eisai, Biogen rocked by 2nd lecanemab death report ahead of Alzheimer's data reveal  Facing a familiar side effect problem, Eisai makes the case for its next Alzheimer's drug after patient deaths Amgen, J&J and Sanofi size up Horizon as Big Pharma renews interest in rare disease M&A  Pfizer CEO Bourla stung by UK's pharma marketing regulator over kids' COVID vax claims: report  GE Healthcare unveils AI program bundle for simplified MRI scans Philips unveils AI-powered platform to improve diagnoses from imaging scans "The Top Line" is produced by senior multimedia producer Teresa Carey with managing editor Querida Anderson and senior editors Annalee Armstrong, Ben Adams, Conor Hale and Eric Sagonowsky. The sound engineer is Caleb Hodgson. The stories are by all our “Fierce” journalists. Like and subscribe wherever you listen to your podcasts.See omnystudio.com/listener for privacy information.

Vaccine hesitancy, the most expensive gene therapy in the world, a $178M series D, and a $1.3B buyoutFind out more athttps://LifeScienceTodayPodcast.comStory ReferencesSanofi/TakedaUniQure & CSLFog PharmaMerck + Imago About the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry. 

Paula Soteropoulos brings to Ensoma more than 30 years of experience in the biopharma industry in areas of drug development, manufacturing, business development, global commercialization with P&L accountability, company building and organizational development. Her leadership throughout her career spans a broad range of therapeutic areas, including rare disease, cardiovascular and metabolism, infectious disease, renal, and transplant and oncology. In addition to Ensoma, Paula is a strategic advisor to 5AM Ventures and a member of the Board of Directors of uniQure and Rallybio. Previously, Paula served as the founding CEO and board member of rare disease therapeutic developer Akcea Therapeutics. There, she led the company through its IPO, as well as significant growth — employing over 270 staff in 13 countries and overseeing six drugs in its portfolio with two rare disease drug approvals and commercial launches. Prior, Paula served as senior vice president and general manager, cardiometabolic and rare disease businesses and strategic alliances at Moderna Therapeutics. She also spent more than 20 years at Genzyme Corporation, most recently as vice president and general manager, cardiovascular, rare diseases. Paula holds B.S. and M.S. degrees in chemical and biochemical engineering from Tufts University and an executive management certificate from the Darden School of Business, University of Virginia. Paula serves on the advisory board for the Chemical and Biological Engineering Department of Tufts University.

In 2020, CSL Behring entered into a global license and commercialization agreement with UniQure for the company's experimental hemophilia B gene therapy. The expected, one-and-done treatment carries the potential to free people with the genetic bleeding disease from reliance on regular infusions of clotting factor IX for which they are deficient. We spoke to Steve Pascoe, senior vice president and head of therapeutic areas and development strategy for R&D at CSL about hemophilia B, how the gene therapy fits into CSL's broader therapeutic offerings, and the encouraging results from the recent pivotal study.

Joe Liberman, Associate  Director, Medical Affairs at UniQure discusses MSL insights and how they can help your career.   Joe shares…  

Patrick and Amy catch up on some recent community news then speak with leukemia survivor and bone marrow transplant recipient Bob Falkenberg as he’s set to kick off his 10th Lifeblood cycling ride to support Be The Match.   Notes: PRESENTING SPONSOR: Takeda - bit.ly/TakedaBDsite   SUBSCRIBE to BloodStream on Apple Podcasts: bit.ly/BSPAP CHECK OUT all our stuff:  bit.ly/AllBloodStreamStuff   BloodStream LIVE May 26th - Event link: http://bit.ly/BloodStreamIsBack   Bob’s Team Lifeblood: http://bihttps://bit.ly/3yaPj2U NHF’s Community Voices in Research: http://bit.ly/CVRNHF CSL and uniQure close agreement: http://bit.ly/CSLuni Experimental SubQ Inhibitor Treatment: http://bit.ly/SubQinhib

No one is immune to stress and the effects it can have on the body and mind. Unfortunately, the COVID-19 pandemic has been a major stressor for a lot of people, leading to a sharp increase in employee burnout as the lines between work and home have blurred. As both individuals and companies are beginning to pay closer attention to wellness, it's imperative we discuss the importance of wellness -- both physical and mental -- and how to improve it. To provide her insight into wellness and share some things you can do to improve it, Danielle Day joins Jennifer and the Survive & Thrive podcast. Danielle holds a PhD in Integrative Physiology, is a former collegiate gymnast and aspiring weightlifter, and the founder of C-Health; giving her a unique perspective on the importance of wellness in business and our personal lives. Act 1: The beginning Danielle's background and experience (1:44) Jennifer and Danielle share some of the highlights from her career. The leadup to the COVID-19 pandemic shutdown (3:47) Still working for uniQure, Danielle was in a meeting when the shutdown started. Despite a job that involved a lot of travel, Danielle suddenly had to get accustomed to working from home. Realizing the new reality (5:19) As the weeks of lockdown stretched on with no clear end in sight, a new reality set in for Danielle. She discusses what it was like dealing with the launch of a new and innovative treatment option for hemophilia when they couldn't actually meet with anyone.  How are you? (9:56) During the pandemic, Danielle asked her employees how they were doing instead of focusing solely on deliverables and work metrics. As many previous guests have found, taking an interest in you and your staff's personal lives led to a new understanding of the difficulties many people faced balancing work and home life during the pandemic. Act 2: Finding new purpose A new perspective (12:25) So many people used the pandemic as a time of reflection and growth, and Danielle is no different, starting several new ventures including starting her own company. Danielle reflects how the coronavirus pandemic shaped her perspective on purpose. Better handling high-stress situations (19:39) Danielle's company, C-Health, seeks to provide holistic self-care and boundary-setting strategies to maintain mental and physical health practices during high-stress periods. The pandemic was certainly one of those times. Danielle discusses some of the issues that plague people, especially women, and lead to employee burnout Act 3: Using wellness to decrease employee burnout Supporting employee wellness (24:41) As companies begin focusing more attention on their employees' mental and physical wellness beyond just work itself, finding a way to properly support that can be difficult. What does it actually look like? As Danielle notes, it doesn't have to mean needing a big block of time. Taking small moments to stretch and change your routine can lead to feeling reenergized.  How to implement and sustain wellness programs (29:37) The lines between work and home life have blurred during the pandemic, which has led to an increase in employee burnout. Danielle shares some of the ways companies can improve employee buy-in and sustainability in their wellness programs. Act 4: Taking time Taking small breaks (38:34) A recent study has found taking even small breaks can significantly decrease your brain's stress levels. Danielle discusses the importance of incentivizing taking small breaks. Reading, reacting, and learning (40:05) Danielle believes it's important to be open to what's happening around us and focus on developing the skills necessary to cope well to that increase in stress.   Sources: BMJ - Physical inactivity associated with higher risk for severe COVID-19 outcomes Morning Brew - Microsoft study shows need for breaks in between video meetings Microsoft WorkLab - Research proves your brain needs breaks...

Sangamo Therapeutics is clinical-stage biotech company looking to commercialize a number of assets for the treatment of disease. Their furthest along program is a gene therapy treatment for Hemophilia A. The sector has seen some recent upsets with the FDA issuing a CRL to Biomarin for their HemA gene therapy, as well as some safety concerns with Uniqure and Bluebird Bio. Sangamo also has a number Phase 1/2 programs that will have readouts in late 2021 as well as a large preclinical pipeline. They have been able to leverage a number of partnerships with Pharmaceutical companies that has led to significant cash payments and equity purchases. In this episode, I share an email exchange I had with the SVP, Head of Business Development at Sangamo, Melita Sun Jung. I also discuss updates from BioXcel and Bluebird Bio. Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll your pet for stem cell banking. 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 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

The Series Podcast Objectives are:  Understand how the medical affairs strategic planning process can drive decision making throughout the year  Gain insight into the value medical affairs bring across an organization when an effective, collaborative, aligned medical strategy plan is developed  Obtain a working knowledge of how cross-functional teams within medical affairs can reference strategic plans to inform decision making and assess impact of efforts  Moderator: Monica DeAbadal, MD, MBA/HSA,VP, Medical Excellence, Ipsen Speaker: Peter Piliero, MD, VP & Head, Medical Affairs Americas, GSK Consumer Healthcare Speaker: Eileen K Sawyer PhD, Vice-President, Global Medical Affairs, UniQure , Speaker: Len Valentino, MD, President and CEO, National Hemophilia FoundationSpeaker: Anna Walz, CEO, MedEvoke

The Series Podcast Objectives are:  Understand how the medical affairs strategic planning process can drive decision making throughout the year  Gain insight into the value medical affairs bring across an organization when an effective, collaborative, aligned medical strategy plan is developed  Obtain a working knowledge of how cross-functional teams within medical affairs can reference strategic plans to inform decision making and assess impact of efforts  Monica DeAbadal, MD, MBA/HSA, Vice President, Medical Excellence at Ipsen and MAPS Medical Strategy & Launch Excellence co-lead, culled out highlights from the Medical Strategy Strategy Planning live session at the March 2020 Annual MAPS Conference.The session speakers who provided these critical intellectual components of Building the Medical Affairs Strategic Plan are: Peter Piliero, MD, Vice President & Head, Medical Affairs Americas at GSK Consumer Healthcare; Eileen K Sawyer, PhD, Vice-President, Global Medical Affairs, UniQure; Len Valentino, MD, President and CEO, National Hemophilia Foundation; and, Anna Walz, CEO of MedEvoke. 

The Series Podcast Objectives are:  Understand how the medical affairs strategic planning process can drive decision making throughout the year  Gain insight into the value medical affairs bring across an organization when an effective, collaborative, aligned medical strategy plan is developed  Obtain a working knowledge of how cross-functional teams within medical affairs can reference strategic plans to inform decision making and assess impact of efforts  Monica DeAbadal, MD, MBA/HSA, Vice President, Medical Excellence at Ipsen and MAPS Medical Strategy & Launch Excellence co-lead, culled out highlights from the Medical Strategy Strategy Planning live session at the March 2020 Annual MAPS Conference.The session speakers who provided these critical intellectual components of Building the Medical Affairs Strategic Plan are: Peter Piliero, MD, Vice President & Head, Medical Affairs Americas at GSK Consumer Healthcare; Eileen K Sawyer, PhD, Vice-President, Global Medical Affairs, UniQure; and, Anna Walz, CEO of MedEvoke. 

Matt Kapusta from UniQure joins us to provide an update.

Uniqure’s stock fell 21% today because they had a licensing deal with CSL rather than getting a buyout offer. Listen to hear why it’s bullish. *This episode is not financial advice. All content should be considered opinionated. We’re not responsible for any gains and losses. And host and the people working in Dissecting the Markets are neither licensed nor registered financial experts. Please see a financial advisor before making any investment decisions. --- This episode is sponsored by · Anchor: The easiest way to make a podcast. https://anchor.fm/app --- Send in a voice message: https://anchor.fm/hardwoodtile/message Support this podcast: https://anchor.fm/hardwoodtile/support

In our second episode, HD Insights Podcast welcomed Dr. Joseph Higgins, Vice President, Clinical Development, CNS at uniQure. Over the past summer, uniQure announced in a formal letter to the community that enrollment in the first clinical trial for AMT-130 was starting. We spoke with Dr. Higgins about the development effort around this novel treatment for Huntington's disease, the reaction from the HD community, and his early career experiences in clinical research. Dr. Higgins shares some interesting stories about being activated for wartime duty while and the NIH, and a personal connection he has to George Huntington, the man who discovered and for whom HD is named after.

Re-air of the UniQure show from February 2019

Dr. Higgins is the Vice President of Clinical Development at uniQure. He is responsible for the early development of clinical gene therapy trials for neurological disorders. Prior to joining uniQure in 2018, Dr. Higgins was responsible for all neurological diagnostic testing at Quest Diagnostics and was the federal CLIA-license holder for a specialty laboratory. He was formerly a Professor at Weill Cornell Medical College where he was the principal investigator on several private foundation and NIH research grants focused on clinical neurogenetics. He held senior leadership positions in genomic medicine at the federal and state governments including the National Institute of Health (NIH) and the New York State Department of Health (NYSDOH). At NIH he was part of the Human Genome Project and teams that initiated the first-in-human enzyme replacement and gene therapy clinical trials. He led the Human Genetic Initiative at the NYSDOH. He discovered the genes or loci for several diseases including Parkinson disease, essential tremor, ataxia, intellectual disability (ID), and metabolic disorders. He is a fellow of the American Academy of Neurology with board certifications in Pediatrics by the American Board of Pediatrics and Neurology with Special Qualification in Child Neurology by the American Board of Psychiatry and Neurology. He has authored more than 100 peer-reviewed publications, book chapters, and reviews articles in the field of neurogenetics. He is licensed to practice medicine in MA and NY. Dr. Higgins earned his bachelor’s degree in biology from Marist College and medical degree from New York University. He completed residences in Pediatrics at Children’s Hospital/Harvard Medical School; Adult Neurology at NYU/Bellevue; and Child Neurology at Children’s National Medical Center/George Washington University. He completed a 3-year fellowship program at NIH in neurogenetics and metabolic neurology.

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What does a brain look like in a patient with a functional movement disorder? Pretty normal, it turns out. But beneath the normal anatomy is a storm of aberrant signaling. Dr. Mark Hallett, Chief of the Human Motor Control Section of the NIH, describes the underlying neurophysiology in this spectrum of disorders. Produced by James E. Siegler. Music by Damiano Baldoni, Kevin MacLeod, Ondrosik, and the Philadelphia String Quartet. Voiceover by Erika Mejia. BrainWaves' podcasts and online content are intended for medical education only and should not be used for clinical decision making. REFERENCES Maurer CW, LaFaver K, Ameli R, Epstein SA, Hallett M and Horovitz SG. Impaired self-agency in functional movement disorders: A resting-state fMRI study. Neurology. 2016;87:564-70. Vuilleumier P, Chicherio C, Assal F, Schwartz S, Slosman D and Landis T. Functional neuroanatomical correlates of hysterical sensorimotor loss. Brain. 2001;124:1077-90. Nahab FB, Kundu P, Gallea C, Kakareka J, Pursley R, Pohida T, Miletta N, Friedman J and Hallett M. The neural processes underlying self-agency. Cereb Cortex. 2011;21:48-55. DISCLOSURES Dr. Hallett receives funding from the Intramural program of the National Institute of Neurological Disorders and Stroke. No relevant conflicts of interest are present. He serves as Chair of the Medical Advisory Board for and receives honoraria and funding for travel from the Neurotoxin Institute. He may accrue revenue on US Patent: Immunotoxin (MAB-Ricin) for the treatment of focal movement disorders, and US Patent: Coil for Magnetic Stimulation and methods for using the same (H-coil); in relation to the latter, he has received license fee payments from the NIH (from Brainsway) for licensing of this patent. Supplemental research funds have been granted by BCN Peptides, S.A., for treatment studies of blepharospasm; Medtronics, Inc., for studies of deep brain stimulation; UniQure for a clinical trial of AAV2-GDNF for Parkinson Disease; Merz for treatment studies of focal hand dystonia; and Allergan for studies of methods to inject botulinum toxins. Jim is lucky enough to have no relevant competing financial interests.

uniQure achieved a milestone when it won approval in Europe for Glybera, the first gene therapy approved in the Western World, but it later pulled it from the market because it wasn’t commercially viable. Now, it is advancing a gene therapy for the rare genetic clotting disorder hemophilia B through development. We spoke to Steve Zelenkofske, chief medical officer at uniQure, about what the company has learned from its experience with Glybera, what the data from its hemophilia B gene therapy has shown, and how its expecting to alter that therapy to make a more effective version that may provide an even greater response.

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: EMA, Merck, Chiesi, Uniqure, LabCorp, Chiltern, Covance, Avara International, Pfizer, QuintilesIMS, FDA, Celgene, Agios Pharmaceuticals.Persone: Tommasina Iorno (UNIAMO FIMR).Nuove terapie: AMT-060, Idhifa. Patologie: emofilia B, leucemia mieloide acuta.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: EMA, Merck, Chiesi, Uniqure, LabCorp, Chiltern, Covance, Avara International, Pfizer, QuintilesIMS, FDA, Celgene, Agios Pharmaceuticals.Persone: Tommasina Iorno (UNIAMO FIMR).Nuove terapie: AMT-060, Idhifa. Patologie: emofilia B, leucemia mieloide acuta.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/Hai un dispositivo Apple? Seguici e abbonati al podcast tramite la app iPod http://nelfarmaceutico.link/pharma-apple

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Sanofi, Allergan, Novartis, Uniqure, Horizon Pharma, Biogen, Recordati, Alexion Pharmaceuticals, AIFA, EMA, Diasorin, Kedrion Biopharma.Persone: David Nicholson (Allergan), Pierluigi Petrone (Global Virus Network), Massimo Galli (SIMIT), Sandra Horning (Roche), Riccardo Perfetti (Sanofi), Alessandro Segato (Aip), Carlo Agostini (Ospedale di Padova).Nuove terapie: emicizumab, insulina glargine.Patologie: Steatoepatite non alcolica, deficit familiare di lipoproteina lipasi, disordini del ciclo dell’urea, atrofia muscolare spinale, Malattia di Pompe, malattia granulomatosa cronica, osteopetrosi maligna severa, emoglobinuria parossistica notturna, Sindrome emolitico-uremica atipica, carcinoma uterino, emofilia A, diabete, immunodeficienze primitive.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Sanofi, Allergan, Novartis, Uniqure, Horizon Pharma, Biogen, Recordati, Alexion Pharmaceuticals, AIFA, EMA, Diasorin, Kedrion Biopharma.Persone: David Nicholson (Allergan), Pierluigi Petrone (Global Virus Network), Massimo Galli (SIMIT), Sandra Horning (Roche), Riccardo Perfetti (Sanofi), Alessandro Segato (Aip), Carlo Agostini (Ospedale di Padova).Nuove terapie: emicizumab, insulina glargine.Patologie: Steatoepatite non alcolica, deficit familiare di lipoproteina lipasi, disordini del ciclo dell’urea, atrofia muscolare spinale, Malattia di Pompe, malattia granulomatosa cronica, osteopetrosi maligna severa, emoglobinuria parossistica notturna, Sindrome emolitico-uremica atipica, carcinoma uterino, emofilia A, diabete, immunodeficienze primitive.Ogni mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/

In this episode, we take a look back at a historic summer that saw the National Hemophilia Foundation's (NHF) Annual Meeting and the World Federation of America's (WFH) World Congress convene in Orlando for the largest gathering ever of people with bleeding disorders. In addition to featuring some conference related content, our Like Share Comment segment highlight a story on Elijah Warren, a high school swimming star with hemophilia as well as a recent article from John Hopkins University on the power of humor in healthcare. Finally, we close this month's episode with a remembrance of Matt Stinger, a community member and advocate who passed unexpectedly this month at 33 years old. Headlines Novo Nordisk’s B-HERO-S study has found, to the surprise of some, that many patients with mild or moderate hemophilia B in the United States experience their condition negatively impacting their career, education, and recreational activities- something that can be overlooked in those affected by mild or moderate hemophilia as opposed to severe. Link. High schooler Federico Parres is one of five team members on the VEX Robotics World Championship Team F, responsible for creating Caerus-7, a ball-flinging robot. Federico lives with severe hemophilia A and plans on becoming an engineer one day. Link. Bayers’ Hemophilia Awards Program recently announced 16 physicians and researchers from 11 countries who will receives grant money totaling about two million dollars, including 6 individuals from the United States. This is the fourteenth year Bayer has run this program. Link A study published in the European journal Haematologica examines a new potential model for how to treatment patients with hemophilia A who undergo surgery. Something I personally find interesting given our ongoing need to learn more about aging with hemophilia. Link The FDA has approved a new needleless reconstitution device called Baxject III for Shire’s long-lasting factor FVIII therapy Adynovate. For those familiar, it is a very similar device to that which is found in Advate. Link Two medical advisory updates have been announced by the National Hemophilia Foundation regarding existing products: NHF Medical Advisory 419 (four-one-nine) states that Bayer has announced a voluntary recall of two lots of Kogenate FS for a loss of potency. The lot numbers are 270TN1C and 270R978. For more information or to arrange for an exchange please call the Recall Processing Center at 855-838-5782. Link And NHF Medical Advisory 420 (four-two-zero) states that CSL Behring has announced a voluntary recall of two lots of Helixate FS for a loss of potency. Those lot numbers are 270TN1G and 270R979. For more information or to arrange an exchange for these products, call CSL Behring’s Customer Service at 1800-683-1288. Link Product Related Headlines (not mentioned in the show) Genentech/Roche has released encouraging Phase 1/2 extension results for their investigational subcutaneous medicine, emicizumab-previously referred to as ACE910. Link The Netherlands-based uniQure, a company self-described as a leader in human gene therapy, announced encouraging clinical data from their ongoing Phase 1/2 trail of AMT-060, an investigational gene therapy for patients with hemophilia B. Link​ BioMarin has presented positive interim data from a Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for people severe hemophilia A. The company has announced intent to begin another study with an eye toward regulatory approval in both the US and Europe. Link CSL Behring’s hemophilia B therapy Idelvion shows promise after encouraging Phase 3 trial results. Link The company also posted positive results from a Phase III study from its FDA approved, hemophilia A treatment AFSTYLA, unique for being the first and only single-chain recombinant product available in the US. Spark Therapeutics and Pfizer received Breakthrough Therapy Status from the FDA for expedited development of a hemophilia B gene therapy treatment SPK-9001. The treatment is currently also undergoing a Phase 1/2 clinical trial. Link. Alnylam Pharmaceuticals announced positive results of a Phase 1 trial for their subcutaneous therapy fitusiran, designed to treat patients with both hemophilia A and B, with or without inhibitors. Link. Like Share Comment Segment NHF Daily, a digital recap of Annual Meeting. Link WFH The Congress Daily. Link High school swimming star Elijah Warren. Link John Hopkins University article on therapeutic humor. Link BloodStream on Social BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.

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