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
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Scientist @Garuda Therapeutics | Computational Biologist I Bioinformatics Scientist I NGS I WGS I GWAS I Python I R
🤔💡Vertex Pharmaceuticals and CRISPR Therapeutics announce the availability of their sickle cell gene therapy, Casgevy, in the U.S. at a list price of $2.2 million, while Bluebird bio sets the list price for Lyfgenia, its treatment for the same condition, at $3.1 million, following their approval by FDA. The exorbitant pricing seems to prioritize profit over patients, potentially limiting access to those in dire need. A critical reflection on the balance between advancing medical science and ensuring affordability for widespread patient benefit is imperative. 💔🏥 #HealthcareEthics #GeneTherapy #AffordableAccess #MedicalInnovation https://lnkd.in/eKADtGCg.
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We are excited to be working with Elpida Therapeutics (www.elpidatx.com) as their real-world data capture platform for upcoming clinical trials on SPG50. SPG50 (Hereditary Spastic Paraplegia type 50) is a rare disease afflicting the son of Elpida's Founder, Terry Pirovolakis. Within a few short years, Terry and his team have created MELPIDA for SPG50, an AAv9 Gene Therapy which is now in human trials (Phase II) for FDA approval. Elpida Therapeutics hope to expand to a pipeline of four other conditions, including CMT4J very soon. Elpida Therapeutics - Bringing hope to rare disease families noink (www.noink.me ) is a real-world data capture platform that brings structured data to life sciences at scale and provides a complete health tracking system for individuals and their families. noink bridges the efficacy gap within healthcare currently by providing a platform that is scientific enough for Life Science’s data requirements while simultaneously meeting the needs of users to manage their and their family’s health. noink - Real World Data for Life Science, Complete Health Tracking for Individuals #melpida #noink #spg50 #realworlddata #elpidatx #CMT4J
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With 27 FDA-approved cell and #genetherapy products to date, #plasmidDNA (pDNA) continues to play a crucial function within advanced therapies medicinal product (ATMP) development. In this webinar, Andrew Frazer (Associate Director, Scientific Solutions, Gene Therapy CDMO Services, Charles River Laboratories) will explore common manufacturing and quality standards for pDNA and how they align with regulatory guidance. Watch this #webinar to learn about: • Manufacturing strategies and mitigation plans to streamline #ATMP program • The role of #pDNA as a critical starting material • Case studies demonstrating expedited development and manufacture of advanced therapies Watch this webinar at you convenience below:
Expediting development and manufacture of advanced therapies: critical starting materials case study
insights.bio
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#Skyreg #FDA update #Cell_and_gene_therapy **Just released** Considerations for the Use of Human- and Animal-Derived Materials in the Manufacture of Cellular and Gene Therapy and Tissue-Engineered Medical Products This guidance supplements the following two final guidances: 1- “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Guidance for Industry” dated January 2020 (Gene Therapy CMC Guidance) (Ref. 2) 2- “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs)” dated April 2008 (Cell Therapy CMC Guidance) (Ref. 3).
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In June 2023, The FDA approved BioMarin's Roctavian #genetherapy for severe haemophilia A along with a #companiontest that might help determine whether #patients might benefit from the #medication. The test has reportedly become the first companion #diagnostic immunoassay approved by the FDA for use with a gene therapy as a result of the FDA's approval for use with #Roctavian, which is also the first gene therapy approved to treat severe #haemophilia A. The test, known as #AAV5DetectCDx, was created through a partnership between BioMarin Pharmaceutical Inc. and ARUP Laboratories. BioMarin employed the test for several of its Roctavian clinical trials. In the #UnitedStates, ARUP will be the exclusive lab testing supplier for the #diagnosis. For more info:https://bit.ly/3sXSTz7 #hemophilia #HemophiliaTreatment #HemophiliaResearch #HemophiliaFoundation #medicalresearch #prophecy #marketresearch #prophecymarketinsights
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In the last five years, the FDA approved 12 novel cell and gene therapies. Compare that figure to 3,000 CAGT assets in the global pipeline at the end of 2022. Navigating these constricting pipelines will require careful strategic planning and multidisciplinary expertise. Learn how Parexel can maximize your chances of market success:
Cell and Gene Therapies
parexel.com
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What will the “good news” be for the life sciences sector in 2024? ❓Will the XBI Biotech Index recover at least 20%? ❓Will the uptick in M&A activity continue and be sustained through 2024 at a 30% higher level? ❓Will more than 2% of all U.S. patients with sickle cell disease get access to treatment with new approved cell-based gene therapies? Institute Executive Director @Murray Aitken gives his insights on these questions and others in his latest blog, Outlook for 2024: 7 Key Indicators of Progress in the Life Sciences Sector. Read more here: https://bit.ly/3S2gkAx #Lifesciences #biotech #pharma #globalhealth
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Check out this webinar on June 11th to learn about analysis of AAV Genome Integrity using a quadplex dPCR assay!
💡 Are you developing cell and gene therapy products? Then you probably recognize the need for product-specific analytics to ensure consistent drug potency and safety. One key factor to look out for? Heterogeneity of packaged genomes in vectors. Join our BioInsights-hosted webinar on June 11 to learn why and how Ryan Massopust, a Scientist at Asklepios BioPharmaceutical, Inc. (AskBio), uses quadruplex dPCR to assess the genome integrity and heterogeneity of target-based AAV genome variants. ▶ https://lnkd.in/g-nrzMy9 #GeneTherapy #AAV #DigitalPCR #dPCR
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Cell & Gene Therapies: An Overview Of FDA Regulatory Considerations For 2024 https://lnkd.in/egjpc2AU Emily Marden and Kelly Cho from Sidley Austin LLP outline key regulatory developments relevant to sponsors seeking regulatory approval of CGTs for commercialization in the U.S. #cellandgene #FDA #regulatory
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The FDA has just published draft guidance which provides recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. A potency assurance strategy is a multifaceted approach that reduces risks to the potency of a product through manufacturing process design, manufacturing process control, material control, in-process testing, and potency lot release assays. The goal of a potency assurance strategy is to ensure that every lot of a product released will have the specific ability or capacity to achieve the intended therapeutic effect. This guidance document includes recommendations for helping to assure the potency of CGT products at all stages of the product lifecycle. For investigational products, the policy describes how to progressively implement a strategy for potency assurance during product development, and provides additional considerations for assuring the potency of products that are undergoing rapid clinical development. #cellandgenetherapy #celltherapy #genetherapy #advancedtherapies
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