Regenxbio Inc is a biotechnology company focused on advancing the field of gene therapy for the treatment of rare and debilitating diseases. The company specializes in developing innovative therapies that utilize its proprietary AAV (adeno-associated virus) gene delivery technology, which aims to correct genetic defects and restore normal function at the cellular level. Regenxbio is committed to addressing unmet medical needs through its robust pipeline of product candidates, targeting various conditions, including inherited retinal diseases and neurodegenerative disorders. By harnessing the power of gene therapy, Regenxbio seeks to transform treatment options and improve the quality of life for patients.
Clearside Biomedical pioneers suprachoroidal drug delivery for precise, effective treatment of retinal diseases. Oppenheimer rates CLSD Outperform with a $5 target, citing its innovative SCS Microinjector and strategic partnerships.
The FDA has approved Sarepta Therapeutics' Elevidys for treating Duchenne muscular dystrophy in patients aged 4 and older with specific DMD gene mutations.
Latest updates on safety and efficacy in the Phase 1/2 AFFINITY DUCHENNE trial for Duchenne muscular dystrophy. Positive results, no serious adverse events. Expect pivotal dose determination by mid-2024.
