AGC Biologics Partners with Rarity PBC to Advance First Commercial Gene Therapy for ADA-SCID "Bubble Baby Disease"
Key Insights
AGC Biologics has partnered with Rarity PBC (search) to provide comprehensive development and GMP manufacturing for RDP-101 (search), a gene therapy targeting ADA-SCID (search) that could become the first FDA-approved commercial treatment for this rare immunodeficiency disorder in the United States.
The therapy has demonstrated remarkable clinical success, successfully treating 48 out of 50 children in trials by modifying patients' own hematopoietic stem cells to restore immune function.
ADA-SCID (search) affects one to five babies per million births worldwide and is fatal without treatment, accounting for approximately 15 percent of all SCID (search) patients.
AGC Biologics announced a strategic partnership with Rarity PBC (search) to provide comprehensive development and Good Manufacturing Practice (GMP) manufacturing for RDP-101 (search), a gene therapy targeting Adenosine Deaminase Severe Combined Immunodeficiency Disorder (search) (ADA-SCID (search)). If approved by the U.S. Food and Drug Administration, RDP-101 would become the first gene therapy available for commercial use to reverse ADA-SCID in patients within the United States.
Addressing a Critical Unmet Medical Need
ADA-SCID (search), commonly known as "bubble baby disease," is a rare, life-threatening inherited condition that severely compromises the immune system, leaving infants vulnerable to frequent and severe infections. The disorder affects between one and five babies per million births worldwide and is caused by an Adenosine Deaminase (search) deficiency. Without treatment, ADA-SCID is fatal and accounts for approximately 15 percent of all SCID (search) patients.
Rarity's RDP-101 (search) treatment represents a breakthrough approach as an ex vivo autologous gene therapy that modifies a patient's own hematopoietic stem cells to restore immune function. The therapy has already demonstrated remarkable clinical success, successfully treating 48 out of 50 children in clinical trials.
Comprehensive Manufacturing Partnership
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 (search) (LVV) and autologous CD34+ (search) 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. This proven platform provides the technical foundation necessary to meet FDA standards for commercial manufacturing.
"Partnering with AGC Biologics is a critical step in our mission to advance our ADA-SCID (search) gene therapy patients in need," said Dr. Paul Ayoub, CEO of Rarity PBC (search). "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."
Scientific Leadership and Funding Support
The gene therapy development is led by Donald B. Kohn, M.D., distinguished professor at the University of California Los Angeles (search) Broad Stem Cell Research Center, who invented the lentiviral vector for gene therapy of ADA-SCID (search). Kohn received a $14.7 million grant from the California Institute for Regenerative Medicine (search) in November 2024 to develop a commercial manufacturing protocol for the groundbreaking gene therapy.
"We needed a collaborator with the technical depth and experience to meet FDA standards for commercial manufacturing," said Kohn. "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."
Strategic Positioning in Cell and Gene Therapy
The partnership reinforces AGC Biologics' position as a global leader in cell and gene therapy manufacturing. The company's track record includes multiple FDA-approved therapies already in commercial supply, with additional programs currently under review.
"We are honored to partner with Rarity PBC (search) 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."
The project has been made possible in part by funding from the California Institute for Regenerative Medicine (search) (CIRM), a state agency that funds regenerative medicine, stem cell, and gene therapy research, through Grant Numbers CLIN2-09339 and CLIN2-17078.