More FDA Draft Guidance, Comparing EMA & FDA approaches to CGTs, and Reflecting on a 10-Year Journey Commercializing Two Gene Therapies

TRENDING CONTENT - Catch Up

#1. Understanding FDA's Draft Guidance On Human- And Animal-Derived Materials In The Manufacture Of Cell & Gene Therapy Products

In April 2024, the FDA released draft guidance on the use of human- and animal-derived materials in cellular and gene therapy. The guidance addresses risk assessment, qualification, and cGMP controls, aiming to clarify and consolidate viral testing requirements and improve material management practices for sponsors and manufacturers. By Blake Bergam and Sara Mills , Dark Horse Consulting Group Inc.

#2. How Do Cell & Gene Therapy Requirements Differ Between FDA & EMA?

CGTs are enabling treatment for life-threatening diseases with previously unmet medical needs. However, these innovative products follow nontraditional routes to approval and sponsors face challenges when demonstrating safety and efficacy. Examine the similarities and differences between how the FDA and the European Medicines Agency approach CGTs.

#3. Improving Access And Support For Cell And Gene Therapies

Although cell and gene therapies (CGTs) are potentially transformative, their effectiveness is undermined by difficulties with patient access and support, high costs, and a complex pathway to treatment. Understand the patient access problems that are inherent with CGTs and the benefits of data collection and integrated patient support hubs. Source: Cencora

#4. Cell & Gene Therapies: An Overview Of FDA Regulatory Considerations For 2024

Discover the latest FDA initiatives to accelerate cell and gene therapy (CGT) approvals, including the new Office of Therapeutic Products, enhanced safety monitoring, and expedited pathways. Stay informed about the increasing CGT applications and essential FDA-sponsor interactions shaping the future of innovative therapies. By Emily Marden and Kelly Cho , Sidley Austin LLP

#5. bluebird bio And Lonza Reflect On Their 10-Year Journey Commercializing Two Gene Therapies

Kelly Kral , VP, CMC Strategy & Operations, bluebird bio , and Anthony Basile , Account Management CGT, 龙沙 , share a decade-long story of partnership in bringing two life-changing gene therapies to market successfully in 2022.

#6. FDA's Draft Guidance On Safety Testing Of Human Allogeneic Cells For Use In Cell-Based Therapies

Learn about the FDA 's new draft guidance on safety testing for human allogeneic cells used in cell-based medical products. This article highlights key points, benefits, and limitations of the guidance, and encourages developers to provide feedback to improve regulatory clarity and streamline the development of safe therapies. By Amanda Mack and Alicja Fiedorowicz , Dark Horse Consulting Group Inc.

CELL & GENE: THE PODCAST - Listen Here

“A full-length dystrophin is highly desired for the treatment of Duchenne muscular dystrophy, and it has not yet been delivered.” Elixirgen Therapeutics, Inc. ’s CEO, Aki Ko , joins #CellAndGenePodcast to discuss underserved segments of DMD patients and how they are working to change that.

CELL & GENE LIVE! - Register Now

Join us on Tuesday, June 25 28, 2024 at 11 AM Eastern Daylight Time

With upcoming cell therapy approvals, a variety of products and platforms are essential to ensure manufacturing safety and efficacy, accelerate market entry, and reduce overall costs. While streamlined manufacturing is the aim, challenges remain. Innovations in closed systems, automation, and standardization are crucial to deliver cell therapies to patients promptly.

Join Cell & Gene's Chief Editor, Erin Harris , as she hosts a detailed discussion with Vittoria Biotherapeutics, Inc. 's co-founder and CEO, Dr. Nicholas Siciliano , Ph.D., and cell and gene therapy quality executive, Will Junker . They will explore the next generation of platforms and innovative technologies poised to enhance manufacturing speed and cost efficiency.