News

Promising data on OriCAR-017 were previously published in the Lancet Haematology in 2023.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed how far the field has come since founding the organization/

Funding from ACGT will help Chiocca perform FDA-required studies on mice to show the oncolytic virus is safe and can be manufactured for humans. It is the second such grant awarded to Chiocca from ACGT.

The assistant professor at Mayo Clinic School of Medicine discussed the design of the phase 1 trial.

The clinical research director of the UCSF Multiple Sclerosis Center also discussed the high expense of producing individualized therapies like CAR-T.

Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, discussed clinical trial design considerations for this emerging field.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

Chris McDonald, senior vice president and global head of technical operations, Kite Pharma, discussed axi-cel's FDA approved manufacturing changes.

Iovance’s tumor infiltrating lymphocyte (TIL) therapy is the first cellular therapy to earn this indication, with the phase 3, confirmatory TILVANCE-301 trial set to verify its clinical benefit.

The Prescription Drug User Fee Act date for the FDA’s decision on the supplement has been set for June 21, 2024.

Review top news and interview highlights from the week ending February 16, 2024.

David Porter, MD, the director of cell therapy and transplant at Penn Medicine, discussed the importance of sharing knowledge in the emerging field of cell therapy for autoimmune disease.

The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed challenges on the horizon in this rapidly emerging field.

The first 3 participants in the MUSIC-HFpEF trial have now been dosed with the AAV vector-based gene therapy.

Ascidian Therapeutics presented positive preclinical data at the 2023 ASGCT meeting.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the importance of rigorous clinical trial design for determining whether CAR-T will truly be of benefit in autoimmune disease.

The approval follows the separate US approvals in December 2023 and January 2024.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham pointed out access to a sickle cell specialist as one such priority.

Rocket submitted additional CMC data in response to FDA information requests.

The phase 3 trial of tivanisiran failed its primary endpoint.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed what he views as the next horizon in the field of gene therapy.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

Hemogenyx noted that the agency confirmed all issues pointed out in the initial clinical hold letter from June 2023 had been sufficiently addressed by the company.

The BAFFR-CAR T-cell therapy is being investigated in a phase 1 single-center and phase 1 multicenter trial in B-ALL and B-NHL.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the current standard of care for B-cell driven autoimmune diseases and how CAR-T could address unmet needs.

The associate professor of otolaryngology from Harvard Medical School also shared other big takeaways from the landmark trial for the gene therapy field.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed his research on a new application of efgartigimod alfa (Vyvgart).

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.