We’ve screened 60+ family-founded programs. Each program we back targets Rare Pediatric Disease Designation and PRV eligibility.
A family-founded program in GMPPA-CDG. Full details will be shared once the family approves public disclosure.
Pipeline: Discovery, Rare Pediatric Disease Designation complete. Current stage: N-of-1 (IND Application), underway. Next: NDA-enabling studies (Phase 1/2/3).
A family-founded program in Leigh syndrome. Full details will be shared once the family approves public disclosure.
Pipeline: Discovery, Rare Pediatric Disease Designation complete. Current stage: N-of-1 (IND Application), underway. Next: NDA-enabling studies (Phase 1/2/3).
More programs announced soon — names, diseases and stages are shared as each family approves disclosure.
Our intake starts with a simple introductory call about your program.