Aldevron’s Post

ICYMI With Biological Safety Levels (BSL), many gene therapy developers and manufacturers aim for BSL I in the manufacturing process. But as Aldevron’s Cody Grasswick points out, there are reasons to consider BSL II when some regulations and controls come into the mix. https://lnkd.in/g5XGFrmt

(Benefit-Risk Management / Gene Therapy / FDA Town Hall CMC Mtg) As gene Rx FDA guidance and precedent regulatory decisions are being actively shared or made publicly accessible, possibly worthwhile if you or your colleagues need to understand the "language" of gene therapy and stay with the conversation (listen to the successes and the challenges). Otherwise, for those that are already in this space, you know this is another one of those essential touchpoints to see what FDA is considering. Enjoy, happy Saturday! #fda #biologics #genomics #genetherapy #cmc #cber #drugmanufacturing #biologicmanufacturing (CMC = chemistry, manufacturing, and controls)

#News: The FDA top #biologics official expects that 2024 will be a “breakout year” for #genetherapy approvals, as a new number of new initiatives take hold to promote the development of these products.   Speaking at the 2024 BioPharma Congress on 26 February, Peter Marks, director the Center for Biologics Evaluation and Research (#CBER), said that the recently established Office of Therapeutic Products (#OTP) is now 75-80 percent staffed.   He also discussed some of the opportunities and some of the agency’s methods for promoting the development of #cellandgenetherapies with moderator Michael McCaughan of Prevision Policy.   “What we are trying to accomplish this year is taking a number of the developments that have started to accelerate over the past year, then taking what we learn and apply it. That means applying the accelerated approval provision and optimizing communications with sponsors and increasing our global cooperation with other regulators.   These initiatives include #FDAs recently finalized guidance stating that the accelerated approval pathway may be used for sponsors developing gene therapy product that incorporate #genomeediting (GE) of #humansomaticcells and the centre's recently announced Support for clinical Trials Advancing Rare disease Therapeutics (#START) communications pilot for rare diseases. ( Read more from Regulatory Affairs Professionals Society (RAPS) 👇🏼

The FDA draft guidance “Potency Assurance for Cellular and Gene Therapy Products” provides recommendations for developing a potency assurance strategy for cellular and gene therapy products. The guidance emphasizes using a multifaceted approach including quality risk management, thorough product and process understanding, potency assays, and compliance with cGMP regulations. It offers guidance on selecting and designing potency assays with desirable characteristics and change control. Overall, the document aims to help industry stakeholders ensure potency through a comprehensive, risk-based strategy incorporating manufacturing design, material control, testing, and lot release assays. At NDA, our team of experts is well-equipped to help you de-risk your advanced therapy development, so you can focus on bringing safe and effective products to market. Schedule a call with us today. Link to the draft guidance: https://loom.ly/awKQEL8 #FDA #ATMP #CTP #GTP #Potency #RegulatoryAffairs #DrugDevelopment

(Benefit-Risk Management / Gene Therapy / CMC Meeting) So you have a gene therapy in your pipeline, or maybe you might in the future, this is likely a pretty good snapshot mtg of what FDA may consider important if you're engaging your BLA for review and/or managing/preparing for CMC inspections. There's a link to last year's mtg on the webpage if interested to see a preview of the topics; likely relevant for the quality folks too! Enjoy. #fda #biologics #bla #genetherapy #genediting #cber #cmc #drugmanfacturing #biologicmanufacturing

It's a new world and gene therapies and nanomedicines are shaping the future of personalized medicine. Battelle scientists have created the HIT SCAN system, a delivery vehicle platform containing nanocarriers that can serve as components of new genetic drug products. But how do we navigate the regulatory pathways to realize their full potential? In this new Inside Battelle blog, Anthony Duong explores this topic in depth. #GeneticNanomedicines #PrecisionMedicine https://okt.to/IF4BZ5

Plasmid DNA (#pDNA) is used in #genetherapies, vaccines, and #RNAtherapeutics. Along each step of development, we need to evaluate potential impurities. In this new Cell & Gene article, Brian Glass of Pharmatech Associates, a USP company delves into potential testing methods for pDNA to evaluate its suitability for therapeutic applications. https://lnkd.in/edA7JPsk #DNA #drugdevelopment #drugmanufacturing #cellandgenetherapies

Thoughts on this? >> FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides - Yahoo Finance >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #healthcare #pharmaceutical #biotech #competitivemarketing

🔗rizochem.com | rizochem.ae Vertex and CRISPR Receive FDA Approval for Gene Therapy for Sickle Cell Disease: The U.S. Food and Drug Administration (FDA) has approved the first gene therapy for sickle cell disease, a hereditary blood disorder. The therapy, called Casgevy, is a one-time treatment that works by correcting a genetic mutation that causes the disease. In clinical trials, Casgevy was shown to significantly reduce the number of painful crises and hospitalizations experienced by patients with sickle cell disease. #pharma #pharmaceutical #pharmacy #pharmaceuticals

Thoughts on this? >> FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides - Yahoo Finance >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #biotech #healthcare #pharmaceutical #pharma