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Thoughts on this? >> Cell and gene therapy approach shows promise for EB children - Epidermolysis Bullosa News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #biotech #pharma #competitivemarketing

Cell and Gene Therapies could be life-changing therapies, but key challenges lie in the complexity of new modalities including product and process, commercial viability, patient access, and compliance with regulatory and quality standards throughout the product development lifecycle.   The FDA recently issued a Draft Guidance Document, "Frequently Asked Questions - Developing Potential Cellular and Gene Therapy Products," offering valuable insights across regulatory review, chemistry, manufacturing, and controls (CMC), as well as non-clinical and clinical considerations.   This draft guidance document includes: - eCTD requirements: Differences between commercial INDs and non-commercial/research INDs. - How to Cross-referencing previous INDs or Master Files using a Letter of Authorization (LoA) - Required Contents of INDs for CMC, nonclinical (pharmacology/toxicology), clinical, and administrative documents such as Form FDA 1571 - Overview of IND process: Submission, Expected timeline and decisions from the FDA - Meeting with FDA: Preclinical stage INTERACT vs. pre-IND meeting and what Type D meeting is. how to request meetings and prepare briefing packages - Expedited programs: Potential expedited paths such as Fast Track, Breakthrough Therapy, or RMAT designation, along with potential rolling reviews - Product development considerations: Donor eligibility for allogeneic therapies, product characterization and release testing, CQA identification and understanding, and potency assurance strategy - Process characterization and validation - Manufacturing changes and comparability - Stability: The stability requirement and how to set acceptance criteria - BLA preparation: Analytical methods and assay performance evaluation throughout product development It is challenging to understand and define critical quality attributes (CQAs) and potency strategies during early product development, as product characteristics relevant to clinical performance may not yet be well understood. Sponsors are advised to evaluate multiple product characteristics early to identify and understand CQAs. While traditional development correlates CQAs with clinical outcomes across multiple lots, rare diseases pose additional challenges due to limited populations and fewer manufactured lots. More to come, I look forward to it!

FDA's November 2024 guidance titled Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products provides developers with answers to frequently asked questions (FAQs) and commonly faced issues that arise during the development of cellular and gene therapy products. The FAQs represent common questions directed to the Agency and span multiple disciplines, including regulatory review, chemistry, manufacturing, and controls, pharmacology/toxicology, clinical, and clinical pharmacology. https://lnkd.in/es726_NG

📢 NEWS - FDA Draft Guidance on FAQs for Cellular and Gene Therapy Products The FDA has released a draft guidance document addressing common questions and challenges faced during the development of cellular and gene therapy products. The guidance covers critical areas such as regulatory review, CMC, pharmacology/toxicology, clinical considerations, and clinical pharmacology. Its goal is to facilitate the creation of safe, effective, and high-quality therapies. The FAQs are based on inquiries from sponsors, feedback following FDA public presentations, and input from stakeholders via CBER. The guidance is open for public comment and may be updated in the future to include additional FAQs. Contact us at info@asphalion.com to learn how we can help you navigate this guidance or other regulatory requirements. #CGT #CellularTherapies #GeneTherapies #RegulatoryAffairs

Thoughts on this? >> Gene therapy treatment increasing body's signal for new blood vessel growth shows promise - Medical Xpress >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #biotech #healthcare #competitivemarketing #pharmaceutical

Thoughts on this? >> SMA gene therapy candidate helps young children to sit up in trial - SMA News Today >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #biotech #pharmaceutical #healthcare #pharma #competitivemarketing

Thoughts on this? >> Cell and gene therapy approach shows promise for EB children - Epidermolysis Bullosa News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #pharmaceutical #biotech #pharma #healthcare

Important quick guide for CGT development

Thoughts on this? >> Gene therapy halts progression of rare genetic condition in young boy - Medical Xpress >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #healthcare #pharma #competitivemarketing #biotech

The recent announcement of the expansion of Regenxbio's Duchenne gene therapy to pivotal stage demonstrates a blurring of boundaries between clinical trial phases, serving as a useful reminder for those involved in the drafting and negotiating of gene therapy licence agreements to carefully consider the wording of milestone triggers. Ellen Lambrix and I discuss useful considerations that parties to such licences may wish to take into account in our article below.