Rarity PBC is advancing a one-time autologous hematopoietic stem cell gene therapy designed to correct the genetic mutation that causes adenosine deaminase severe combined immunodeficiency (ADA-SCID). The therapy, licensed exclusively from UCLA, has demonstrated robust and durable immune reconstitution in 59 of 62 children across published trials, and more than 70 children have been treated to date. As a Public Benefit Corporation, the company combines market discipline with a legal commitment to ensure broad patient access to rare-disease treatments. Proceeds from its recent seed financing will be used to establish commercial-grade manufacturing, finalize regulatory work for FDA approval, and build the infrastructure for sustainable global delivery. Rarity’s model is bolstered by prior non-dilutive funding from the California Institute for Regenerative Medicine (CIRM) and a public-benefit mandate that prioritizes affordability and availability. The company was co-founded by UCLA scientists and clinicians Donald Kohn, MD, Roger Hollis, PhD, Paul Ayoub, PhD, MBA (CEO), and Katelyn Masiuk, MD, PhD (CSO). Financially, the business has secured $4.6 million in seed capital alongside CIRM grants to advance its lead therapy toward commercialization.

Total raised

$5M

Funding rounds

1

Latest round

Seed

Latest activity

Oct 2025