AGC Biologics Collaborates with Rarity PBC to Advance Gene Therapy for Bubble Baby Disease
AGC Biologics, a friendly, dedicated CDMO partner, has announced a collaboration with Rarity PBC to provide comprehensive development and Good Manufacturing Practice (GMP) manufacturing of Rarity PBC’s gene therapy drug, RDP-101, for the treatment of severe combined immunodeficiency caused by adenosine deaminase deficiency.
If approved by the U.S. Food and Drug Administration, RDP-101 would be the first commercially available gene therapy for ADA-SCID patients in the United States.
ADA-SCID is a life-threatening, rare genetic disorder that severely weakens infants’ immune systems, making them susceptible to repeated severe infections. It is estimated that one to five of every one million infants born worldwide have the disease. Caused by a deficiency in adenosine deaminase, ADA-SCID is life-threatening without treatment and accounts for approximately 15% of SCID patients. Rarity’s RDP-101 treatment is an ex vivo autologous gene therapy that restores immune function through genetic modification of a patient’s own hematopoietic stem cells. In clinical trials, this approach has already transformed patients’ lives, successfully treating 48 of 50 children.
Under the agreement, AGC Biologics will provide end-to-end services for Rarity’s gene therapy product, which consists of EFS-ADA lentiviral vector (LVV) and autologous CD34+ hematopoietic stem cells . The collaboration will include process development, GMP manufacturing, process validation, and other activities required for commercialization. The LVV process will utilize AGC Biologics’ proprietary ProntoLVV™ adherent platform, a technology already used to support multiple commercial products.
Our collaboration with AGC Biologics marks an important step in our mission to advance gene therapy for ADA-SCID patients in need,” said Paul Ayoub, Ph.D., CEO of Rarity PBC . “Their proven commercial manufacturing expertise and collaborative spirit is exactly what we needed to get us through the final stages of regulatory approval. This treatment has already had life-changing success in the clinic, and together with AGC Biologics, we are working to ensure it reaches even more families in need.
To meet FDA standards for commercial manufacturing, we needed a collaborator with deep technical expertise and experience,” said Donald B. Cone, MD, 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. “Our collaboration with AGC Biologics allows us to focus on bringing this therapy to patients who are desperately waiting for it now and those who will need it in the future.
In November 2024, Cohn received a $14.7 million grant from the California Institute for Regenerative Medicine to develop a commercial manufacturing protocol for its groundbreaking gene therapy for ADA-SCID .
We are honored to partner with Rarity PBC on such an important program as part of our mission to support innovators who bring life-changing treatments to patients with rare diseases,” said Luca Alberici, Executive Vice President, Global Cell & Gene Technology and General Manager, Milan, AGC Biologics. “Leveraging our proven ProntoLVV™ platform and extensive commercial manufacturing experience, our team is committed to bringing this treatment to every child who needs it. As a trusted CDMO specialist partner, we see this commitment as going beyond the project itself: to saving the lives of families and patients affected by this rare disease.
This collaboration strengthens AGC Biologics’ position as a global leader in cell and gene therapy and the partner of choice for developers striving for commercial success. The strength of the company’s presence is demonstrated by multiple FDA-approved therapies already in commercial supply, and additional programs currently awaiting approval. This track record makes AGC Biologics an ideal partner for a wide range of organizations, from public benefit corporations to nonprofit organizations and public initiative funded institutions.
Acknowledgment for CIRM Support
This project was made possible in part through funding from the California Institute for Regenerative Medicine (CIRM), a California state agency that funds regenerative medicine, stem cell, and gene therapy research (grant numbers: CLIN2-09339, CLIN2-17078).
About Rarity PBC
Rarity PBC is a public benefit corporation advancing the development and commercialization of life-saving gene therapies for rare diseases. Driven by its mission to ensure responsible development and patient access, Rarity PBC combines innovation with a sustainable, mission-driven business model to ensure groundbreaking therapies move through development and into the hands of patients who need them.
About California Institute for Regenerative Medicine (CIRM)
The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to advance regenerative medicine research to bring therapies to patients with unmet medical needs. Established in 2004 by Proposition 71 and expanded in 2020 by Proposition 14, CIRM has provided billions of dollars in funding to support stem cell and genetic research and development programs. Through research, infrastructure, and education, CIRM strives to transform regenerative medicine and improve lives in 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), working hand-in-hand with clients and partners to provide friendly, professional service and committed to the highest standards of service. We offer world-class development and manufacturing services for mammalian and microbial-derived therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically modified cells. Our global network spans the United States, Europe, and Asia, with locations in Seattle (Washington), Copenhagen (Denmark), Heidelberg (Germany), Milan (Italy), Chiba, and Yokohama (Japan). AGC Biologics is part of AGC Inc.’s Life Sciences business, which operates more than 10 facilities specializing in biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. For more information, visit www.agcbio.com .
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