News

According to Federal Register notices, the guidance documents are updated versions of drafts originally published in March 2022.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed his research career with muscular dystrophies.

The company has received clearance from the FDA for a phase 1 clinical trial for its allogeneic CAR-T in patients with lupus nephritis.

The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy.

The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the latest results announced from the phase 2 portion of the PRISM study.

The committee is set to discuss idecabtagene vicleucel’s sBLA on March 15, 2024.

The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the need to set standard guidelines regarding potential fertility issues for CAR-T treatment.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the hypothesis behind research on the use of CAR-T for multiple sclerosis.

The professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

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 the significant challenges in this field, but also expressed optimism for the future.

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed talks from a session on dysfunction and trafficking at the 2024 MDA Conference.

The lead of preclinical research at Amylyx Pharmaceuticals discussed the literature supporting AMX0114’s target and its upcoming trial design.

Cohort 1, which originally was set to enroll only 5 patients, has been expanded to allow for the inclusion of 8 patients in total.

Taysha Gene Therapies also announced additional updates on the TSHA-102 program for both adolescent adult patients and pediatric patients.

The postdoctoral scholar at University of California – Irvine discussed his research looking at TDP-43 and alternative polyadenylation in neuronal cells.

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

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed investigations into liver toxicity and deaths after AT132 gene therapy.

The data comes from 7 patients treated in the phase 1/2 IGNITE-DMD study (NCT03368742).

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed initiatives including the BGTC and the platform vector gene therapy project.

The real-world data, which includes patients who received Zolgensma before and after tracheostomy, comes from the noninterventional RESTORE registry (NCT04174157).

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed the role of organelle transport in neuromuscular diseases such as CMT.

The phase 3b SMART trial included 24 patients in total who each weighed from 8.5 kg to 21 kg at the time of treatment.

RGX-202 delivers a slightly larger form of microdystrophin than other gene therapies, including the CT domain of dystrophin.

In observance of World Hearing Day, held annually on March 3, CGTLive® took a closer look at the clinical evaluation of hearing loss gene therapy DB-OTO.

The lead scientist at Percheron Therapeutics discussed how ATL1102 could target inflammation as a secondary cause of muscle damage in DMD.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.