Vladimir P. Bermudez, Ph.D., PMP’s Post

As many of you are aware, in late December the FDA announced the availability of a new draft guidance for advanced therapies, which provides recommendations for developing a science- and risk-based strategy to help assure the potency of human cellular therapy or gene therapy products. 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. Understanding a product’s potency from the start is incredibly valuable… ensuring patients receive the full extent of a product's intended therapeutic effect. Laser Force Cytology analytics are a very important piece of an advanced manufacturing strategy due to the technology’s ability to deliver real-time insights, highly precise and reproducible data and all without the need for labels or florescents. Real-time PAT data insights hold the key to improved production and product consistency… it is great to see the increased focus on the need for precision analytics earlier in process development so that through a matrix approach, these data can allow for better understanding and rapid optimization throughout the manufacturing process. #CellTherapy #GeneTherapy #FDA #CGT

AMT Guidance Would Hinder Cell and Gene Therapy Progress, Commentators Charge: The FDA’s draft guidance on its Advanced Manufacturing Technologies (AMT) pathway would be restrictive to manufacturers in the cell and gene therapy spaces, the Alliance for Regenerative Medicine and the American Society of Gene and Cell Therapy said in filed comments. #fda #financial #lifesciences

Reducing manufacturing costs is key to long-term success of gene therapies. CHI’s Gene Therapy Manufacturing conference, August 21-22 in Boston, addresses the practical challenges facing the cost-effective production, purification, and manufacture of viral and non viral-based gene therapies, at scale. Topics include AAV and lentivirus upstream and downstream process development, scaling up, process-related impurities, capture and purification, and manufacturing strategies for clinical and commercial supply. Learn more and reserve your spot today to lock in advance registration rates at https://lnkd.in/gZfq9V7S

The landscape of gene therapy is burgeoning with possibilities, offering promising advancements to patients with genetic disorders. In a revealing conversation with European Pharmaceutical Review Magazine, Roger Palframan, head of U.S. research at UCB, sheds light on the current trends, key developments, and future disruptors in gene therapy. His insights offer a glimpse into the potential of gene therapies. You can read the full interview 👉https://lnkd.in/exhVR3kp

We discussed the differences in #cellandgenetherapy manufacturing and traditional drug manufacturing this week.

Finally, CBER/OTP has released its draft guidance on Potency assurance for gene and cell therapy products. I find this guidance uniquely helpful for the industry as it contextualizes potency assurance beyond just an assay. Per the FDA, this guidance seeks to provide recommendations for developing a science- and risk-based strategy to help assure the potency of a human cellular therapy or gene therapy (CGT) product. This is a much welcome news! https://lnkd.in/e-cvVEiC

Nice to read, the newest draft of “potency assurance strategy for CGT products” 😊😊

best to learn more details about this!

The FDA have issued draft guidance on ‘Potency Assurance for Cellular and Gene Therapy Products’, along with a very informative webinar highlighting key aspects of the guidance. This guidance will replace the 2011 guidance for ‘Potency Tests for Cellular and Gene Therapy Products’ In this new guidance the FDA acknowledge the challenge that industry faces in developing suitable potency assays for CGT and thus recommend a multi-faceted potency assurance strategy following a science and risk-based approach. Key stages of this strategy include: ·      Develop a QTPP ·      Identify potency-related CQAs ·      Conduct a risk-assessment for each of these CQAs ·      Mitigate against identified risks through appropriate design and control of the manufacturing process and materials that impact these CQAs ·      Implement phase-appropriate appropriate release assays The guidance emphasises the possibility of a clinical hold at any stage of product development if an adequate potency assurance strategy is not implemented. FDA welcome feedback on this guidance, with comments due 27 March 2024. Given the importance of potency assurance for CGT products and the challenges we face developing suitable assays it is certainly worth spending time reviewing this comprehensive guidance and submitting any comments and suggestions. #cellandgenetherapy #fda #cber #potency

As a perfect way to end 2023, the FDA released draft guidance on "Potency Assurance for Cellular and Gene Therapy Products." This comprehensive document underscores the pivotal role of potency assessment in ensuring the efficacy and safety of cell and gene therapy products. For those of us who work in the biomanufacturing industry, understanding FDA draft guidance documents is imperative to ensure regulatory compliance, uphold product quality, and navigate the evolving landscape of standards, fostering both innovation and patient safety. I think these are the key highlights: 1️⃣ Precision in Potency Evaluation: The guidance emphasizes the need for precise and standardized methods to assess potency, enhancing the reliability of therapeutic outcomes. 2️⃣ Risk Mitigation Strategies: It outlines strategies to identify and mitigate risks associated with potency variations, fostering robust product development and manufacturing processes. 3️⃣ Patient-Centric Focus: With a focus on patient outcomes, the guidance underscores the importance of consistent potency to optimize therapeutic benefits and minimize risks. In the current landscape of cell and gene therapies, this guidance couldn't come at a more critical time. As the industry witnesses unprecedented growth and innovation, the document provides a crucial roadmap to ensure the potency and effectiveness of emerging therapies. Do the reading! https://lnkd.in/euCydsvT #FDA #celltherapy #genetherapy #innovation #healthcareadvancements