Regenxbio

REGENXBIO's RGX-121 shows sustained efficacy in reducing heparan sulfate levels in CSF, potentially allowing patients to remain off ERT, and is well-tolerated. The company plans to submit a BLA for accelerated approval using CSF D2S6 as a surrogate end point.

The global Retinal Gene Therapy market, valued at US$119.15 million in 2024, is projected to grow at a CAGR of 9.6% through 2034. Driven by advancements in gene therapy technologies and clinical successes, it offers hope for treating inherited retinal diseases like retinitis pigmentosa and Leber congenital amaurosis. Key players include Novartis AG and Spark Therapeutics, with recent developments focusing on expanding treatment options and manufacturing capabilities.

The global Retinal Gene Therapy market, valued at US$119.15 million in 2024, is projected to grow at a CAGR of 9.6% through 2034. Driven by advancements in gene therapy technologies and clinical successes, it offers hope for treating inherited retinal diseases. Opportunities include diversifying targeted diseases and leveraging gene editing technologies like CRISPR-Cas9. Major players include Novartis AG and Spark Therapeutics, with recent developments highlighting strategic collaborations and manufacturing expansions.

2023 saw significant progress in DMD treatment with Sarepta Therapeutics’ Elevidys approval, yet it benefits only a small patient group. Challenges remain in therapy's long-term efficacy, safety, and expanding access. Research continues for broader, more effective treatments, emphasizing the need for innovative strategies and precise outcome measures.