Advances in Gene Therapies, Bioreactor Optimization, and FDA Guidance on Therapy Materials

TRENDING CONTENT - Catch Up

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 Understanding FDA's Draft Guidance On Human- And Animal-Derived Materials In The Manufacture Of Cell & Gene Therapy Products

FDA has released the new draft guidance for industry, Considerations for the Use of Human- and Animal-Derived Materials in the Manufacture of Cellular and Gene Therapy and Tissue-Engineered Medical Products. This article provides a summary — and shares what is shocking about the guidance.

#2. The Rising Tide Of Ophthalmologic Gene Therapies

There’s an explosion of clinical activity in the gene therapy space for inherited retinal diseases (IRDs). Atsena Therapeutics ' CMO, Dr. Kenji Fujita , explains what makes IRDs such appealing gene therapy targets and shares the company’s strategic approach to clinical development.

#3. Strategies To Tackle CAR-T Product Challenges

Strategies for scaling up production of CAR-T products, including the hub model, bedside model, innovation of process strategy, and more.

#4. mRNA Delivering Full-length Dystrophin for DMD

Aki Ko , CEO, Elixirgen Therapeutics, Inc. explains that the current set of approved drugs for DMD includes four exon-skipping drugs and one AAV-microdystrophin drug that supplements a missing dystrophin protein with its shortened version.

#5. Growing Happy Cells: Bioreactor Optimization

Successfully maintaining Critical Process Parameters (CPPs) for bioreactors helps to optimize product quality, potency, impurity profiles, total cell density and titer/protein concentration (productivity).

#6. The Path Forward for CLL is Allogeneic

Allogene Therapeutics ' Zachary J Roberts MD PhD explains the elements of allogeneic CAR T that make it an attractive strategy to target relapsed/refractory Chronic lymphocytic leukemia (CLL)?that could also suggest promise in additional scenarios.

CELL & GENE: THE PODCAST - Listen Here

“The problem is there are very few good shared antigens across patients with solid tumors. I think those that are good are very focused in the amount of patients that they can actually address.” CSO, Micah Benson , shares why KSQ Therapeutics, Inc. ' KSQ-001EX, which consists of TILs where the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing, offers the potential to treat solid tumors.

CELL & GENE LIVE! - Watch On-Demand

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With additional cell therapy approvals on the horizon, a diverse array of products and platforms are needed to help ensure safety and efficacy in manufacturing, increase speed to market, and decrease overall CoGS. Streamlined manufacturing is the goal, but challenges persist. Innovations in closed systems, automation, standardization, and more are needed to bring cell therapies to patients as soon as possible.

Cell & Gene's Chief Editor, Erin Harris , welcomed?Vittoria Biotherapeutics, Inc.' s co-founder and CEO, Dr. Nicholas Siciliano , Ph.D. and established executive in cell and gene therapy quality, Will Junker , for a discussion on the next generation of platforms and innovative technologies that may increase speed to market and decrease cost for cell therapy manufacturing.