For Immediate Release
Rarity PBC Secures Exclusive License from UCLA Technology Development Group to Advance a Life-Saving Gene Therapy for ADA-SCID
[Los Angeles, CA] – [Tuesday, October 14, 2025] – Rarity PBC, a pioneering Public Benefit Corporation dedicated to availability of life-changing medical technologies, announced today that it has entered into an exclusive licensing agreement with the UCLA Technology Development Group (TDG). This landmark agreement grants Rarity PBC the rights to a groundbreaking gene therapy for adenosine deaminase severe combined immunodeficiency, or ADA-SCID, a devastating genetic disorder affecting infants.
The licensed therapy offers a profound new hope for babies born with ADA-SCID, a condition often referred to as "bubble baby disease" due to the extreme isolation and protection required for affected children. This innovative approach was invented and developed by UCLA researcher Dr. Donald Kohn in collaboration with researchers at University College London. Approximately 70 children’s lives have been transformed by this curative treatment.
The therapy works by overcoming the genetic mutation that causes ADA-SCID. It involves a highly personalized process: collecting a patient’s own blood-forming stem cells and then using a viral vector to deliver a normal, functional copy of the ADA gene into those cells. Once corrected, the stem cells are reintroduced into the patient’s body, where they can produce all necessary blood cell types, including the immune cells the child needs to fight infections and live a healthy life.
"We are honored and humbled to partner with UCLA to bring this therapy to the world," said Dr. Paul Ayoub, CEO of Rarity PBC. "This is more than just a scientific milestone; it represents a turning point for families affected by ADA-SCID and the possibility of a future with greater certainty. At Rarity PBC, we are committed to responsibly advancing life-saving treatments, guided by urgency, rigor, and a deep focus on patient impact."
“I am so happy that Rarity PBC will be working to move this curative therapy to FDA approval to help make it available to ADA SCID patients across the U.S.,” said Kohn.
As a Public Benefit Corporation, Rarity PBC is a for-profit company with a legally mandated public benefit mission. This unique model allows the company to combine market-driven efficiency with a responsibility for expanding long-term sustainability to rare disease therapies. The exclusive license from UCLA TDG is a critical step in advancing ADA-SCID treatment and reflects Rarity PBC’s role in translating breakthrough science into sustainable patient impact.
About Rarity PBC
Rarity PBC is a Public Benefit Corporation dedicated to advancing and commercializing lifesaving genetic therapies for rare diseases. Guided by a mission to enable responsible development and patient reach, Rarity PBC combines innovation with a sustainable, mission-driven business model to ensure that transformative therapies move beyond development and reach patients worldwide.
About UCLA Technology Development Group (TDG)
UCLA TDG promotes UCLA innovation, research, education and entrepreneurship to benefit society. Working with UCLATDG helps facilitate the translation of UCLA discoveries into new products and services that create economic value to support UCLA’s scholarly and educational missions. The UCLA TDG office manages a large portfolio of technologies and license agreements and has a rich history of startup company formation.
Contact:
Marivi Valcourt, Asst. Director of Marketing & Communications, UCLA TDG
310 569 1764 marivi.valcourt@tdg.ucla.edu