Insights into CAR-T Manufacturing, TILs for Solid Tumors, and the Future of RNA in Therapeutics
Cell & Gene
The most valuable resource for professionals who make their living in the cell & gene therapies industry.
TRENDING CONTENT - Catch Up
Manufacturing chimeric antigen receptor (CAR) T-cell therapies is a costly and complicated process. Interius BioTherapeutics is on a mission to reduce this complexity and get these therapies to patients faster — by manufacturing CAR T/NK cells in patients' bodies.
Gene therapy is revolutionizing treatment for genetic diseases, with companies like Novartis and CSL Behring B.V. leading the way in developing AAV-based therapies. As more of these therapies gain FDA and EMA approval, the challenge of accurately measuring potency becomes crucial. This article delves into how companies and regulators are addressing these challenges to ensure the safety and efficacy of these groundbreaking treatments.
Do you have access to all the relevant technical documents to avoid a process delay? West Pharmaceutical Services has created a technical documentation package (TDP) to support your navigation of new document requests. The TDP provides customized information, which helps you easily locate and extract the information needed to support your EU MDR filing process.
KSQ Therapeutics, Inc. 's CSO, Micah Benson , Ph.D., joins Erin Harris to discuss how TILs as a treatment modality have the potential to treat a variety of solid tumor types. Benson explains KSQ's Phase 1/2 clinical study, KSQ-001EX, which consists of TILs in which the SOCS1 gene is inactivated by CRISPR/Cas9 gene editing.
Therapeutic RNAs come with a set of challenges stretching through development and production, including the need for sufficient stability in vivo. However, multiple strategies have been developed for RNA protection, transport, and delivery to the intended target. Source: Bio-Rad Laboratories
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Rocket Pharmaceuticals takes a modality-agnostic approach to rare diseases, prioritizing unmet medical needs over specific technologies. Dr. Kinnari Patel, PharmD, MBA , Rocket's president and COO, highlights their focus on using the best available gene therapy platforms, such as lentiviral or AAV, to treat monogenic diseases. Despite challenges, Rocket is advancing treatments for conditions like leukocyte adhesion deficiency-I, with a vision to expand gene therapy's potential to more common diseases.
CELL & GENE: THE PODCAST - Listen Here
“It’s still a challenge for CMC development and cell and gene therapy to keep pace with the rapid clinical development. Particularly in rare disease.” Dark Horse Consulting LLC 's Kimberly Benton shares how resource allocation and FDA discussions can help companies make the case to get to BLA readiness.
CELL & GENE LIVE! - Available On-Demand
Since the first CAR-T cell therapy approval in 2017, the industry has exploded with companies and candidates aiming to be the best. With each CAR-T that enters the clinic (and subsequently, the market), the modality matures — best practices are established, lessons are learned (sometimes the hard way), and technology evolves, challenging the industry’s idea of what’s possible. As CAR-T cell therapies evolve alongside technology, so does the way these therapies are manufactured. While centralized CAR-T manufacturing is the industry standard, some folks believe the future of CAR-T manufacturing will be decentralized, taking place at or near patients’ point of care (POC). Others think POC manufacturing won’t ever fully replace centralized manufacturing — that it’s not scalable and is laden with quality control and regulatory challenges.
Our speakers (Tyler Menichiello, Jason Bock , Emily English , & Tal Salz ) met on Cell & Gene Live on 8/27 and discussed the ways CAR-T manufacturing has advanced in recent years, as well as where it’s going. Register to gain access to the event on-demand.