Alison Rodriguez

Results of a Phase 1/2a gene therapy clinical trial in children with Duchenne muscular dystrophy (DMD) revealed a significant decrease in levels of serum creatine kinase, an enzyme biomarker associated with muscle damage caused by DMD.

The new immunotherapy of chimeric antigen receptor (CAR)-T cells has demonstrated the ability to increase clinical remission in multiple myeloma patients by targeting the B-cell maturation protein that participates in disease progression.

Novartis’ chimeric antigen receptor T-cell (CAR-T) therapy for treating pediatric leukemia is on the cusp of being the first FDA-approved gene therapy, which will lead to new developments and utilizations of CAR-T therapy for treating other advanced blood cancers.

Sarepta Therapeutics has announced a new collaboration with Genethon for gene therapy research in efforts to develop new Duchenne muscular dystrophy (DMD) treatments.