Opportunities and Challenges in Developing Stem Cell Therapies

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.

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.

In an eight-week obese non-human primate study, lean mass made up 6% of the weight lost with iBio’s Activin E antibody IBIO-610 plus semaglutide, compared with 22% with semaglutide alone. iBio plans to begin first-in-human studies in the first half of 2027.

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.