In addition to the now well-known CAR T (chimeric antigen receptor T cells) and HSC (CD34+ hematopoietic stem cells) cell-based gene therapies, there are several other modes of gene modification as well as cell types already approved and on the horizon. CAR T and HSC genetically engineered therapeutics have typically relied upon lentiviral or gamma-retroviral vector transduction to introduce gene(s) of interest but other methods of gene modification can introduce, replace or inactivate genes in the cells of interest. Recently approved CASGEVY is the first approved therapy to rely on CRISPR/Cas9 gene-editing to modify HSCs. More complex genetically engineered cell therapies may require inclusion of multiple modes of gene modification in the same cell to ensure the desired product attributes. Challenges include effective delivery methods, effective targeting, effective cell selection and expansion, and development of appropriate analytical methods to assess more complex and novel genetic engineering approaches.

Session Speakers:

Ingenui-T, a Rapid Autologous Chimeric Antigen Receptor (CAR)-T Manufacturing Solution Using Whole Blood, for Treatment of Autoimmune Disease

Sunetra Biswas, Kyverna Therapeutics, Inc.

CMC Development of PM359, a Prime Edited Hematopoietic Stem Cell Therapy for the Treatment of P47phox-Deficient Chronic Granulomatous Disease (CGD)

Barrett Nehilla, Prime Medicine, Inc.

Enabling the Advancement of Cell Therapies with Epigenetic Editing

Nathan Yee, Tune Therapeutics

Additional Panelist:

Zhaohui Ye, CBER, FDA

Cynthia Riggins

Appearances