orphan-disease
(2)
GEMMA Biotherapeutics
GEMMA Biotherapeutics (GEMMABio) is a Philadelphia-based gene therapy company spun out of the University of Pennsylvania and founded by AAV pioneer James M. Wilson. Launched in October 2024 with $34 million in seed funding, GEMMABio builds the research, manufacturing, and product-development infrastructure to move adeno-associated virus (AAV) gene therapies from the bench to the bedside faster and more affordably - with a focus on rare and ultra-orphan inherited diseases and a stated commitment to global patient access.
Noema Pharma
Noema Pharma is a Basel-based clinical-stage biotech building first-in-disease oral small-molecule therapeutics for central nervous system disorders. Founded in 2019 by Sofinnova Partners around four mid-stage assets in-licensed from Roche, the company targets overlooked neurological conditions - from seizures in tuberous sclerosis complex and pain in trigeminal neuralgia to Tourette syndrome and CNS-mediated symptoms of menopause. It has raised roughly CHF 130 million (about USD 147 million) in Series B financing from a syndicate including Forbion, Jeito Capital, Sofinnova, EQT Life Sciences and UPMC Enterprises.