ReCode Therapeutics’ Post

Tonight on Biztech's Health & Wellness Show discover the Future of Healthcare with Raman Singh, CEO of Juniper Biologics. Key takeaways from our conversation: Gene therapy for knee pain, innovations in rare disease treatments, and the changing landscape of biotech. Join us on this transformative journey toward a healthier world. 🌍💉 #HealthcareInnovation #JuniperBiologics #MedicalInnovations #HealthcareSolutions #FutureOfMedicine #BiotechLeadership #PatientCare #GlobalHealth #InnovationInHealthcare #MedicalAdvancements #HealthTech #BiotechSuccess #HealthierFuture #RamanSingh #CEOInsights #HealthcareLeadership #BiotechInvestment #MedTech #InnovativeTherapies Catch all this and much more on https://www.biztech.asia/

💚 EAP WEBINAR ALERT 💚 Join us for our next webinar hosted by my dear team member - Karlijn Doorn, PhD, Head of Pharma Partnerships & Customer Success at myTomorrows, and Anke Friedetzky, an experienced Medical Affairs Expert in Cell and Gene Therapies. They will share their expertise in designing and launching successful Expanded Access Programs outside the US. 📅 Date: May 23, 2024 ⏰ Time: 12:00 - 12:45 EST / 18:00 – 18:45 CEST Register through this link --> https://lnkd.in/ePA5MgMz Why should you attend? - and what will you will learn! ·     Recognize the 3 critical regulatory frameworks essential for EAPs and learn strategies for navigating and complying with these regulations across different jurisdictions. ·     Develop robust mitigation plans for handling supply chain issues and tackling specific cell and rare disease therapies-related challenges. ·     Discover tactics for collecting fit-for-purpose RWD in your EAP program through real-world case study examples. ·     Design a sustainable EAP that aligns with organizational objectives, patient needs, and exit strategies—ensuring a smooth transition post-program. Looking forward to seeing you there! . #ExpandedAccess #ManagedAccess #CompassionateUse #RWD

Generation Bio has revealed the development of a novel form of DNA, known as "immune-quiet DNA" (iqDNA), which is derived from its closed-ended DNA (ceDNA). iqDNA has shown cytokine levels and tolerability comparable to mRNA in studies involving mice and primates. This development could lead to more effective genetic medicines. Generation Bio plans to use iqDNA in their treatments, including one for hemophilia A. Their goal is to make genetic medicines that are safe and can help many people. Read more: https://lnkd.in/dbu2ATgS #dna #therapeutics #medicine #drugdiscovery #medicalscience

Wilson Bryan MD, former FDA Director of Office of Tissues and Advanced Therapies (OTAT) from 2016-2023 spoke at Jefferies Cell and Genetic Medicine conference yesterday. His talk highlighted some of the regulatory uncertainties that are contributing to the lull in cell and gene therapy funding and enthusiasm. Top 3 takeaways: ·       FDA Reorg/Leadership - For years, OTAT’s “workload far outpaced” staffing. This was alleviated, in some part, by the reorganization of OTAT into the first Super Office at CBER, the Office of Therapeutic Products (OTP). The bid for increased efficiencies was further cemented by the appointment of Nicole Verdun MD in July of 2023. All of this reorganizing, expanding and onboarding of supplemental staff has led to a little discordance and uncertainty. ·       Accelerated Approval - Accelerated Approvals have been based on surrogate endpoints that are “reasonably likely” to predict clinical benefit. Sarepta’s recent accelerated approval for Elevidys led to a discussion of what percentage of likelihood “reasonably likely” represents. And analysts and hedge funders alike want real precision “Would you say that’s 65% likely, or more like 70%? Or is it 68.3”  More uncertainty, more risk. ·       Changing Minds - Finally, the recent change of guidance for Intellia’s in vivo CRISPR HAE product led Intellia to start their trial abroad. When combined with a last-minute change in Ad Com guidance for BioMarin back in November and this spring for Sarepta, we see how these mixed signals from the agency cause confusion and uncertainty. #genetherapy #cellandgenetherapies #atmp #advancedtherapies #celltherapy #luxturna #zolgensma #zynteglo #roctavian #vyjuvek #skysona #bluebirdbio #vertex #CRISPR #biomarin #libmeldy #orchard #hemgenix #krystal #vyjuvek #EB #asgct #meetingonthemesa

Technology is poised to impact healthcare in revolutionary ways in the next few years.  Telemedicine, AI-driven diagnostics, and gene therapies are already making waves.  However, these advancements come with significant costs. At ParetoHealth, we stay in front of healthcare trends so our Members can stay in front of the costs that come with them. These trends are what inform the strategies we develop with our Members, which include cost-saving prescription drug plans and data-driven solutions.  How can I assist you and your clients in staying ahead of the curve? Reach out today and discover the difference. #ParetoHealth #ProudlyContrarian #HealthcareTrends

This week’s Medical Science & Tech Digest highlights more fascinating articles. The first publication is an announcement from the U.S. Food and Drug Administration which recently approved Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). https://lnkd.in/epcpJtrT The next publication looks at a common anti-clotting drug called heparin, derived from pig intestines, which risks contaminations and infections. A safer, synthetic version has been developed but it requires scaling up for commercial manufacturing. https://lnkd.in/gFB_EE3s The last publication focuses on an MIT professor who has decoded fundamental aspects of Huntington’s disease and glioblastoma, and now uses computation to better understand amyotrophic lateral sclerosis. https://lnkd.in/eTkxfsTN #manhattanlife #medicalscience #healthtechnology #medicaltechnology

📢 REGULATORY UPDATE - The FDA webinar on Human Gene Therapy Products Incorporating Human Genome Editing guidance dives into the key points covering product development, CMC, nonclinical, and clinical recommendations. Watch webinar: https://lnkd.in/gPnJYQHt At ERGOMED, we're committed to staying ahead in regulatory affairs. With our expertise and dedication to compliance, we're here to help you navigate the ever-evolving regulatory landscape. #FDA #GeneTherapy #RegulatoryAffairs #Compliance #ErgomedExpertise

It was great to lead the first practical session of the 'Genetics in Drug Discovery' course at the MRC Biostatistics Unit University of Cambridge. Today, we covered examples including IL-6 inhibitors and drugs for Alzheimer's disease. https://bit.ly/3uD6ddm #genetics #drugdiscovery #bigdata #clinicalpharmacology #R

Generation Bio has revealed the development of a novel form of DNA, known as "immune-quiet DNA" (iqDNA), which is derived from its closed-ended DNA (ceDNA). iqDNA has shown cytokine levels and tolerability comparable to mRNA in studies involving mice and primates. This development could lead to more effective genetic medicines. Generation Bio plans to use iqDNA in their treatments, including one for hemophilia A. Their goal is to make genetic medicines that are safe and can help many people. Read more: https://lnkd.in/dbu2ATgS #dna #therapeutics #medicine #drugdiscovery #medicalscience

Say goodbye to lengthy genetic testing. 👋 Our Aptegra Platform reduces genetic stability testing time by 66% using next-generation sequencing technology. That means clients spend less time waiting for test results and, ultimately, helps new therapies get to patients faster. Crucial for patient safety and regulatory approval, watch how we are speeding up genetic testing.