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

Article

Review some of our most-viewed interviews with clinicians, researchers, and biotech companies about expanding applications of cell and gene therapies.

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.

Recent Videos
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
Related Content
© 2025 MJH Life Sciences

All rights reserved.