Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 million seed financing led by biotech investor Steve Oliveira, Head of Nemean Asset Management. Proceeds will accelerate development of Rarity’s lead program: a one-time autologous hematopoietic stem cell gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID).

The financing follows Rarity’s exclusive licensing agreement with the UCLA Technology Development Group (TDG), granting Rarity the rights to the ADA-SCID gene therapy invented and developed by Dr. Donald B. Kohn at UCLA in collaboration with researchers at University College London. In published trials and long-term follow-up studies, the approach achieved robust and durable immune reconstitution in 59 of 62 children treated, and more than 70 children have now benefited from treatment to date.

“This investment is about turning proven science into sustainable patient reach,” said Paul Ayoub, PhD, MBA, Co-Founder and Chief Executive Officer of Rarity PBC. “With support from Nemean Asset Management, we’re moving faster to establish commercial-grade manufacturing, complete the work needed for FDA approval, and ensure that every child with ADA-SCID can benefit from this therapy, not just those in clinical studies.” Ayoub co-founded Rarity alongside Roger Hollis, PhD (UCLA), and Katelyn Masiuk, MD, PhD, who serves as Chief Scientific Officer. Having worked closely with Dr. Kohn at UCLA, where the therapy was first developed, the team now leads the effort to bring it to patients.

“We back mission-driven teams building therapies that truly change the standard of care,” said Steve Oliveira, Head of Nemean Asset Management. “Rarity’s model, pairing a clinically validated, curative therapy with a public benefit mandate, has the potential to redefine how rare disease treatments reach patients.”

“This additional support builds directly upon the critical funding we’ve received from the California Institute for Regenerative Medicine (CIRM),” said Donald B. Kohn, MD, Co-Founder of Rarity PBC and Distinguished Professor at UCLA. “The partnership between CIRM, UCLA, and Rarity exemplifies how collaborative models can accelerate the translation of science into approved treatments.”

Use of Proceeds

The proceeds from this financing will support Rarity’s continued advancement of its ADA-SCID gene therapy toward commercial readiness. This includes strengthening manufacturing capabilities, advancing regulatory preparations for FDA approval, and building the foundation for sustainable delivery once the therapy is approved.

About the ADA-SCID Gene Therapy

The therapy corrects the genetic mutation causing ADA-SCID by harvesting a child’s own blood-forming stem cells, inserting a functional ADA gene using a viral vector, and reinfusing the corrected cells so they can produce all blood and immune cell types necessary to fight infections. Children with ADA-SCID are extremely vulnerable to life-threatening infections; without effective treatment, most do not survive beyond early childhood.

Rarity’s Public Benefit Mission

As a Public Benefit Corporation, Rarity couples market discipline with a legal commitment to public impact, prioritizing long-term availability and sustainability of rare disease therapies.

Recognition of CIRM Support

This project has been made possible in part by funding from the California Institute for Regenerative Medicine (CIRM), a state of California agency that funds regenerative medicine, stem cell, and gene therapy research. (Grant Numbers CLIN2-09339 and CLIN2-17078).

About Rarity PBC

Rarity PBC is a Public Benefit Corporation advancing and commercializing life-saving genetic therapies for rare diseases. Guided by a mission to enable responsible development and broad patient reach, Rarity integrates scientific rigor with sustainable, mission-driven operations to deliver therapies beyond development and into the hands of patients worldwide. Rarity was co-founded by Donald Kohn, MD (UCLA), Roger Hollis, PhD (UCLA), Paul Ayoub, PhD, MBA (CEO), and Katelyn Masiuk, MD, PhD (CSO).

About UCLA Technology Development Group (TDG)

UCLA TDG promotes UCLA innovation, research, education and entrepreneurship to benefit society. Working with UCLA TDG 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.

About the California Institute for Regenerative Medicine (CIRM)

The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through Proposition 71 and expanded in 2020 through Proposition 14, CIRM has provided billions in funding to support stem cell and genetic research and development programs. Through research, infrastructure, and education, CIRM aims to transform regenerative medicine and improve lives across diverse communities. For more information, visit cirm.ca.gov.

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“This investment is about turning proven science into sustainable patient reach,” said Paul Ayoub, PhD, MBA, Co-Founder and CEO of Rarity PBC.