GeneTherapyLive’s Weekly Rewind – November 5, 2021

Welcome to GeneTherapyLive’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.

1. Rare Disease Consortium to Focus on Optimizing AAV Gene Therapies

The NIH, the FDA, 10 pharmaceutical companies, and 5 non-profit organizations are partnering to launch the Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership program, to accelerate the development of gene therapies for the treatment of rare diseases.

2. Developing Safer, Next-Generation CAR T-Cell Therapies

Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s lead programs in development, BPX-603 and BPX-601, as well as future plans the company has for these programs.

3. Cell Therapy Cancer Vaccine Shows Promise for Progression-Free Survival in Metastatic Pancreatic Cancer

KiroVax (BSK01; Kiromic BioPharma) was efficacious in treating metastatic pancreatic cancer in 1 patient dosed in a phase 1 clinical trial of the cell therapy cancer vaccine candidate, with the patient experiencing a progression-free survival (PFS) of 7 months after treatment with KiroVax and chemotherapy compared with a PFS of 3.9 months with standard of care.

4. Efficient Gene Editing in Monogenic Diseases

André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies. He talked about the company’s new .HEAL platform and its potential in creating efficient candidates to treat monogenic diseases. He also discussed future research the company is conducting with TALGlobinb01.

5. Improving Cell Therapy Manufacturing Through Strategic Partnerships

GeneTherapyLive spoke with Paul Lammers, MD, MSc, president and chief executive officer, Triumvira Immunologics, to learn more about their techniques in cell manufacturing and their recent partnership with Lonza.