Insights on Purity and Intactness of AAV Gene Therapy Vectors

Opus Genetics reports sustained vision gains with gene therapy, the FDA approves Roche’s Enspryng for at-home treatment of thyroid eye disease, and Estuary Biotherapeutics launches with $103 million to develop gene therapies for polycystic kidney disease.

Interim phase 1/2 data presented at Retina in Rio 2026 showed sustained visual improvements with Opus Genetics’ AAV8 gene therapy OPGx-LCA5 and improved retinal sensitivity with OPGx-BEST1 in inherited retinal diseases.

FDA expands Anktiva’s bladder cancer indication, Alvotech and Advanz submit a proposed Eylea 8 mg biosimilar in Europe, and Lilly reports new remission data for Omvoh in inflammatory bowel disease.

The FDA launches a new rare disease development initiative, BioNTech’s BNT113 misses a progression-free survival endpoint, and Viking Therapeutics begins Phase III trials of its oral obesity candidate.

FDA reviews a proposed Keytruda biosimilar, Roche expands its obesity pipeline with an RNAi deal, and guselkumab shows promise for fistula remission in Crohn disease.

Clinical results reveal new opportunities and setbacks in rare disease and cancer care, shaping treatment strategies and future regulatory decisions.

New clinical data highlight improved disease control, reduced eye bulging, and weight-loss benefits, potentially broadening options for patients with unmet needs.

New clinical milestones, manufacturing investments, and regulatory incentives could help developers overcome barriers to delivering treatments for patients with rare diseases.