SPOTLIGHT -
AT845 Shows Promising Efficacy for Late-Onset Pompe Disease in Phase 1/2 Trial
Three of 4 patients withdrew from their previous enzyme replacement therapy following treatment with AT845 gene therapy.
First Patient Dosed With Descartes-08 in Phase 2b Trial for Myasthenia Gravis
Descartes-08, an investigational autologous RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T) has been dosed with the first participant in phase 2b randomized controlled trial.
REGENXBIO's Clinical Trial for Duchenne Therapy RGX-202 Begins Patient Recruitment
Recruitment has begun for REGENXBIO's AFFINITY BEYOND clinical trial assessing the prevalence of AAV8 antibodies in male patients with Duchenne muscular dystrophy.
Gene Therapy SRP-9001 Performs Well in Duchenne Muscular Dystrophy, Multiple Findings Show
In addition to meeting primary end point of change in North Star Ambulatory Assessment, treatment with SRP-9001 resulted in statistically significant improvements in timed function tests.
