Apodi Limited’s Post

Interesting case study where a client requested support to bring an AAV gene therapy for a rare neurogenerative disease into the clinic for a Phase 1b study in patients. Click here to learn how our experts were able to successfully guide this CGT trial: https://lnkd.in/g8jd8qNk #clinicaltrials #drugdevelopment #CNS #raredisease #pharmacology #cellandgenetherapy

With the steady growth in the number of cell and gene therapies (CGTs) over the past few years, access and reimbursement for these products remains a top concern among population-based decision makers. Strategies to manage the cost of these potentially multimillion-dollar products took a large focus at this year’s AMCP, as stakeholders shared organizational perspectives and predictions for the evolving landscape. Read our most recent Access Talk POV by Zachary to learn more about key trends in CGT access and reimbursement, and what this means for market access marketers. https://lnkd.in/eFhxizjX #MarketAccess #MosaicGroup #IPGHealth #AccessReimbursement

The European Medicines Agency (EMA) has updated its guidelines on advanced therapy medicinal products (ATMPs), emphasizing a collaborative approach with stakeholders. Key takeaways include: 🧬 Separation of guidelines for Gene Therapy Medicinal Products (GTMPs) and Cell Based Medicinal Products (CBMPs) was suggested but not implemented; 🧬 Updates on stage-appropriate CMC expectations to alleviate high validation demands; 🧬 Clarification on potency assay validation and process evaluation/validation for clinical trials. Check out the full article by Pam Dhadda, Associate Director at PharmaLex, for an in-depth analysis of the changes and their impact on ATMP development in Europe. Read more: https://lnkd.in/dDcaNFVE #PharmaNews #ATMP #GeneTherapy #CellTherapy #PharmaLex #EMA

Check out my latest POV on cell and gene therapies, and how the access landscape continues to evolve!

The European Medicines Agency (EMA) has updated its guidelines on advanced therapy medicinal products (ATMPs), emphasizing a collaborative approach with stakeholders. Key takeaways include: 🧬 Separation of guidelines for Gene Therapy Medicinal Products (GTMPs) and Cell Based Medicinal Products (CBMPs) was suggested but not implemented; 🧬 Updates on stage-appropriate CMC expectations to alleviate high validation demands; 🧬 Clarification on potency assay validation and process evaluation/validation for clinical trials. Check out the full article by Pam Dhadda, Associate Director at PharmaLex, for an in-depth analysis of the changes and their impact on ATMP development in Europe. Read more: https://lnkd.in/geBbj4_i #PharmaNews #ATMP #GeneTherapy #CellTherapy #PharmaLex #EMA

Lizette Janse van Rensburg presents on how recently the manufacturing of Cell and Gene Therapeutics (CGT) has been a subject of discussion among various organizations. Although both CGT therapies have been in existence for several years, the key focus now lies in their advantages and widespread acceptance at a manufacturing scale. This is crucial to ensure that these technologies can be made accessible to a larger population. The flexibility of the industries involved will play a vital role in meeting the growing demand for these therapies. #ISPE #ISPEAseptic #aseptic #asepticprocessing #asepticfilling #annex1

The European Medicines Agency (EMA) has updated its guidelines on advanced therapy medicinal products (ATMPs), emphasizing a collaborative approach with stakeholders. Key takeaways include: 🧬 Separation of guidelines for Gene Therapy Medicinal Products (GTMPs) and Cell Based Medicinal Products (CBMPs) was suggested but not implemented; 🧬 Updates on stage-appropriate CMC expectations to alleviate high validation demands; 🧬 Clarification on potency assay validation and process evaluation/validation for clinical trials. Check out the full article by Pam Dhadda, Associate Director at PharmaLex, for an in-depth analysis of the changes and their impact on ATMP development in Europe. Read more: https://lnkd.in/geBbj4_i #PharmaNews #ATMP #GeneTherapy #CellTherapy #PharmaLex #EMA

🌟 Sangamo has secured alignment with the U.S. FDA on an Accelerated Approval pathway for its gene therapy targeting Fabry disease. This groundbreaking decision means we could see approval up to three years earlier than anticipated, with a Biologics License Application (BLA) submission expected in late 2025. Using data from the ongoing Phase 1/2 STAAR study, the FDA has agreed that improvements in kidney function (eGFR slope) can serve as the primary basis for approval. This marks a significant step forward in delivering life-changing treatments to patients with Fabry disease, a rare and devastating genetic condition. Sangamo is committed to making a difference in the lives of patients with unmet medical needs, and this achievement moves us closer to fulfilling that mission. Learn more: https://lnkd.in/gwDVg8_P #GeneTherapy #FabryDisease #FDAApproval #AcceleratedApproval #SangamoTherapeutics

Access barriers for cell and gene therapies differ from digital therapeutics. In this infographic, see the primary barriers for top areas of clinical advancement and learn how pharma companies can solve access issues. https://ow.ly/r6sQ50SqhcH

Cellular and Gene Therapy Approvals are on the Rise! Recent data from the EMA and FDA show a significant increase in approvals over the past six years, highlighting major advancements in these therapies. What's driving this increase?  One of the key factors is regulatory progress. The FDA and EMA have developed specialized regulations specifically for CGTs, including the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation and the EMA’s Priority Medicines (PRIME) scheme. Thanks to early engagement with these regulatory agencies, developers receive comprehensive guidance and support, speeding up the entire development process and approvals. As we continue to see remarkable progress in CGT, we must now turn our attention to a crucial question: How can we improve patient's access to these innovative treatments and enhance their  journey? #CellTherapy #GeneTherapy #RegulatoryAdvancements #MedicalInnovation