2022 Year in Review: Top Conversations on Cell and Gene Therapy

2022 has been a year of improvement and expansion for cell and gene therapies, with new gene therapy approvals in rare diseases and hematological disorders and CAR T-cell therapies being investigated in earlier lines and novel indications. With a number of first-in-human and pivotal trials underway, 2023 is sure to bring exciting new data and regulatory decisions on these novel therapies.

CGTLive has spoken to clinicians, researchers, biotech companies, and advocacy groups about new data and technologies that have come up this year. Let’s review some of our most-viewed interviews on cell and gene therapy innovations below:

1. Treating Autoimmune Diseases With Smarter Cell Therapies

Karen Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies. She also discussed challenges in the space and how Kyverna is aiming to meet them.

2. Merit Cudkowicz, MD, on Evaluating Mesenchymal Cell Therapy for ALS

The chief, neurology, and director, Sean M. Healey & AMG Center for ALS, and Julieanne Dorn Professor of Neurology, Harvard Medical School, discussed NurOwn and its potential advantages in treating ALS after BrainStorm Cell Therapeutics decided to submit a BLA following an erratum to a phase 3 trial.

3. TJ Cradick, PhD, on A Potential Cure for HIV With EBT-101

The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy. He discussed the potential of a 1-time infusion to change the HIV treatment landscape.

4. Michael Leek, PhD, on Manufacturing Challenges in Cell Therapy

The co-founder and executive chairman of TC BioPharm discussed his thoughts on the regulatory, logistical, and quality hurdles of manufacturing cell therapies. He emphasized the importance of having control over the manufacturing process and of developing universal standards for the industry.

5. Craig M. McDonald, MD, on Developing Gene Therapy for Different Populations of DMD

The principal investigator of the HOPE-2 trial discussed the results of the trial in the non-ambulatory population of patients with Duchenne muscular dystrophy. He discussed the importance of assessing therapies in the non-ambulatory population of patients with DMD.