Open Innovation: Transforming the Model of Biopharmaceutical R&D

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.

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.

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.

Mirikizumab (Omvoh) achieved comprehensive disease control in 27.7% of UC patients and deep remission in 20.4% with Crohn disease at 1 year.

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.

As ArsenalBio, TScan, Bristol Myers Squibb, and others abandon ex vivo programs for in vivo engineering, early clinical data and platform differences point to both the promise and the unresolved safety, manufacturing, and regulatory questions of generating CAR-T cells inside the body.