orphan-disease
(3)
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.
Ramon Salsas Escat
Ramon Salsas Escat is the Chief Business Officer of GEMMA Biotherapeutics (GemmaBio), the Philadelphia gene-therapy company spun out of Jim Wilson's lab to widen global access to AAV therapies for rare inherited diseases. A chemist by training who earned a PhD in computational and systems biology at MIT and an MBA at IESE, Salsas has led or co-led more than 20 transactions across AAV gene therapies, CNS disorders, oncology and manufacturing platforms, with cumulative deal value north of $4 billion in upfront payments and milestones. He helped build the business engine at Penn's Gene Therapy Program, served as SVP of Business Development at G2 Bio, and now runs business development, alliance management, legal and IP at GemmaBio, where he played a pivotal role in the company's founding and its $34 million seed round.
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.