AGC Biologics Partners with Rarity PBC to Advance Life-Saving Gene Therapy for "Bubble Baby Disease"

Collaboration will leverage AGC Biologics’ commercial manufacturing experience and proven platforms to seek regulatory approval for a therapy treating the rare immunodeficiency disorder, ADA-SCID.

AGC Biologics, your friendly CDMO expert, today announced an agreement with Rarity PBC to provide comprehensive development and Good Manufacturing Practice (GMP) manufacturing for Rarity's gene therapy, RDP-101, for Adenosine Deaminase Severe Combined Immunodeficiency Disorder.

If approved by the U.S. Food and Drug Administration, RDP-101 would be the first gene therapy available for commercial use to reverse ADA-SCID in patients within the United States.

ADA-SCID is a rare, life-threatening inherited condition that severely compromises the immune system, leaving infants vulnerable to frequent and severe infections. For every million babies born worldwide, it is estimated that between one and five will have this condition. The ADA-SCID disorder caused by an Adenosine Deaminase deficiency is fatal without treatment and accounts for approximately 15 percent of SCID patients. Rarity’s RDP-101 treatment is an ex vivo autologous gene therapy that modifies a patient’s own hematopoietic stem cells to restore immune function. In clinical trials, this approach has already transformed the lives of patients, successfully treating 48 out of 50 children.

Under the agreement, AGC Biologics will provide end-to-end services for Rarity's gene therapy product, which consists of the EFS-ADA Lentiviral Vector (LVV) and autologous CD34+ hematopoietic stem cells. The scope of work includes process development, GMP manufacturing, and process validation activities required to bring the product to market. The LVV process will utilize AGC Biologics’ proprietary ProntoLVV™ adherent platform, a technology that has already been used to support multiple commercially available products.

“Partnering with AGC Biologics is a critical step in our mission to advance our ADA-SCID gene therapy patients in need,” said Dr. Paul Ayoub, CEO of Rarity PBC. “Their proven commercial manufacturing expertise and collaborative spirit are exactly what we need to navigate the final stages of regulatory approval. This therapy has already transformed lives in the clinic; with AGC Biologics, we’re working to make that benefit reliably available to more families who need it.”

“We needed a collaborator with the technical depth and experience to meet FDA standards for commercial manufacturing,” said Donald B. Kohn, M.D., distinguished professor at the University of California Los Angeles Broad Stem Cell Research Center who invented the lentiviral vector for gene therapy of ADA-SCID. “With AGC Biologics as that collaborator, we can now focus on making this treatment available to patients awaiting a cure and to those who will need it in the years to come.”

Kohn received a $14.7 million grant from the California Institute for Regenerative Medicine in November 2024 to develop a commercial manufacturing protocol for the groundbreaking gene therapy targeting ADA-SCID.

“We are honored to partner with Rarity PBC on such a critical program as part of our mission to support innovators bringing life-changing treatments to patients with rare diseases,” said Luca Alberici, Executive Vice President of AGC Biologics Global Cell & Gene Technologies Division and General Manager of the Milan site. “Our team is leveraging the proven ProntoLVV™ platform and our extensive commercial manufacturing experience to help make this therapy accessible to every child who needs it. As a friendly CDMO expert, we see this as more than a project; it’s a commitment to saving lives and providing relief to families beset by this rare condition.”

This partnership reinforces AGC Biologics’ position as a global leader in cell and gene therapy and the partner of choice for developers aiming for commercial success. The company’s strong presence is demonstrated by multiple FDA-approved therapies already in commercial supply, with additional programs currently under review. This track record makes AGC Biologics an ideal partner for a broad range of organizations, from public benefit corporations to non-profits and institutions funded by public initiatives.

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 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 are made available to patients in need.

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.

About AGC Biologics

AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to delivering the highest standard of service as we work side-by-side with our clients and partners, to provide friendly and expert services. We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cells. Our global network spans the U.S., Europe, and Asia, with locations in Seattle, Washington; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba and Yokohama, Japan. AGC Biologics is a part of AGC Inc.’s Life Science Business. The Life Science Business runs 10+ facilities focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. To learn more, visit www.agcbio.com.

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