The Gene Therapy, RNA, and LNPs stream delivers an integrated look at CMC, analytics, formulation, and manufacturing for RNA, lipid nanoparticles, and viral vectors across early through late development. The program begins with RNA and LNP Production and Formulation, covering standards and raw materials, impurity analysis, scale-up and purification, LNP production, and stability strategies. Next, the Gene Therapy CMC and Analytics meeting advances discussions on emerging CMC challenges, N-of-1 manufacturing, product and process characterization, assay development, formulation, stability, and the unique requirements of in vivo CAR T products. The stream concludes with the Gene Therapy Manufacturing conference, focusing on late-stage readiness, upstream and downstream processing, novel capsid and vector production, and recovery and purification of viral vectors. Collectively, these sessions equip attendees with the technical and regulatory insights needed to accelerate development and prepare for commercial-scale manufacturing.

Conferences Include:

Keynote & Featured Speakers:

GIVE: Distributed RNA Manufacturing and QC to Scale Personalized and Individualized Genetic Medicines
John E. Schiel, PhD, Program Manager, Scalable Solutions, ARPA-H

Innovations in Downstream AAV Purification
Stephen Soltys, PhD, Chief Manufacturing Officer, Primera Genotech

LNP Platform Enabling in vivo CAR T—Update from Stylus Medicine
Srinivas Chollangi, PhD, Executive Director, CMC Tech Ops, Stylus Medicine

CMC for in vivo CAR T Manufacturing: Opportunities, Challenges, and the Road Ahead
Nripen Singh, PhD, Executive Director and Site Head, Process Development, TRD CGT, Novartis

Directed Evolution of New AAV Vectors for Clinical Gene Therapy
David V. Schaffer, PhD, Hubbard Howe Jr. Distinguished Professor, Chemical & Biomolecular Engineering, University of California Berkeley