Accelerating AAV-based Gene Therapy Development with MS

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.

When large pharmaceutical companies deprioritize drug candidates, smaller developers may license or acquire them, but due diligence, deal structure, and trial planning determine whether a program can move forward.

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.

The FDA approved Roche's satralizumab (Enspryng), an IL-6 receptor-targeting monoclonal antibody, for adults with thyroid eye disease based on the Phase 3 SatraGO-1 and SatraGO-2 trials, giving patients the first at-home subcutaneous treatment option.

EST-201 and EST-101 target the genetic root cause of ADPKD, a disease affecting more than 8 million people worldwide, with trials starting 2027-2028.

Emily Heffernan of Arcadis explains how facility scale, equipment, containment, and quality testing differ across ex vivo cell therapy, viral vector, and mRNA-LNP manufacturing.

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.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.