News

The Amplo Biotechnology gene therapy showed positive findings in mouse models, suggestive of the therapy’s development for use in a clinical setting. The AAV therapy delivers DOK7 gene, which has shown benefit in neuromuscular disorders.

Data from the HOPE-2-OLE were presented in a late-breaking session at the 2023 MDA Conference.

All children included in the analysis achieved swallowing, oral nutrition, and airway protection outcomes.

Investigators plan to present full results from the OS analysis of the phase 3 ZUMA-7 trial at an upcoming medical meeting.

Presented at MDA 2023, the gene therapy (also known as delandistrogene moxeparvovec) showed significant improvements on NSAA total score, 10-meter walk/run scores, and time to rise scores relative to an external control cohort.

The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.

Individual items on RULM revealed meaningful improvements in participants from the SHINE and CHERISH studies.

New data from a single-site, first-in-human study were presented at the 2023 MDA Conference.

The director of the Center for Biologics Evaluation and Research at the FDA discussed his keynote speech at the 2023 MDA Conference.

Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.

The barrier is an essential part of keeping the brain safe, but it also prevents the immune system from eliminating brain cancer tumors.

Ahead of the May 29, 2023, action date, the FDA is now seeking to hold an advisory committee meeting on Sarepta’s investigational agent SRP-9001 (also known as delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy.

Patients in the UK with r/r multiple myeloma may still be able to access the CAR-T through ongoing clinical trials, which are not impacted by the decision.

Review top news and interview highlights from the week ending March 17, 2023.

Intergalactic Therapeutics’ IG-002 utilizes a proprietary non-viral delivery method.

Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, discussed the potential use of microdystrophin as an end point for clinical evaluations of gene therapies for DMD.

Nirav N. Shah, MD, associate professor at the Medical College of Wisconsin in Milwaukee, discussed the evaluation of LV20.19 CAR in MCL, as well as key efficacy and safety data on the therapy to date.

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

The announcement comes in light of serious adverse events reported in the phase 1/2 clinical trials for BPX-601.

The investigator-initiated, single-arm, open-label trial will follow 20 patients with high-risk LBCL for 2 years, with a primary outcome measure of complete response rate per Lugano classification.

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.

GenSight Biologics reported that 73% of patients treated bilaterally with Lumevoq improved at least +15 ETDRS letters relative to their worst recorded BCVA.

The full dataset, published in the New England Journal of Medicine, reveals the newly approved hemophilia B gene therapy reduced annualized bleeding rates and improved the need for factor IX infusions.

Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.

Catch up with some of CGTLive’s top conversations from the annual ASTCT and CIBMTR meeting.

Alison Betof Warner, MD, PhD, assistant attending physician, Memorial Sloan Kettering Cancer Center, discussed the solid tumor session she chaired at the 2023 Tandem Meetings.

Serious adverse events of reduced visual acuity and eye inflammation were observed in 2 clinical trials.

Review top news and interview highlights from the week ending March 10, 2023.

The study of VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy, is already ongoing in Canada. It is expected to kick off in the United States in the first half of 2023.