Jim Wilson’s rethink of funding model underscores ultrarare headwinds

Despite FDA’s attempts to ease the regulatory path for ultrarare disease therapies, venture capital remains largely uninterested. That’s the reality AAV gene therapy pioneer Jim Wilson confronted as he set out to develop and fund gene therapies for ultra-orphan indications, but the hurdles he encountered during the first year prompted him to overhaul the funding model to keep the mission alive.

Jim Wilson left his academic post at the University of Pennsylvania’s Orphan Disease Center in 2024 to found Gemma Biotherapeutics Inc., an AAV gene therapy company that intended to take on what most others have avoided: developing one-time treatments for rare diseases, even those affecting only handful of patients...