AMP-101 Shows Pharmacologic and Safety Promise for Neuromuscular Disorders in Preclinical StudyMarch 23rd 2023
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.
Sarepta’s SRP-9001 Shows Functional Improvements in DMD Integrated AnalysisMarch 22nd 2023
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.
FDA Revisits Need for Advisory Committee on Sarepta’s DMD Gene TherapyMarch 18th 2023
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.
Microdystrophin Proposed as a Surrogate End Point in DMDMarch 16th 2023
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.
Mantle Cell Lymphoma Outcomes May Improve With Bispecific CD20/CD19-targeted CAR T-cell TherapiesMarch 16th 2023
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.
JW Therapeutics Announces New Phase 2 Trial of Relma-Cel in Large B-cell LymphomaMarch 15th 2023
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.
Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness MonthMarch 14th 2023
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.