SHERIDAN, WYOMING - December 3, 2025 - AGC Biologics is partnering with public benefit company Rarity PBC to bring a potentially life-saving gene therapy for Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) through commercial manufacturing and toward FDA approval, underscoring how CDMO collaborations are becoming central to translating academic breakthroughs into scalable rare disease treatments.
From transformational trials to a U.S. commercial pathway
Under the new agreement, AGC Biologics will provide end-to-end development and GMP manufacturing for Rarity's ex vivo autologous gene therapy RDP-101. If approved, RDP-101 would become the first gene therapy commercially available in the United States to reverse ADA-SCID, a rare inherited immunodeficiency that leaves infants dangerously exposed to severe, recurrent infections.
ADA-SCID, often called "bubble baby disease," affects an estimated one to five babies per million births worldwide and is fatal without effective treatment. Rarity's approach modifies a patient's own CD34+ hematopoietic stem cells using an EFS-ADA lentiviral vector to restore functional immune responses. In clinical trials, this strategy has already "successfully treating 48 out of 50 children," highlighting both the medical impact and the urgency of securing a robust commercial supply chain.
Harnessing ProntoLVV™ and global GMP expertise
AGC Biologics will manufacture both components of the product-EFS-ADA lentiviral vector and autologous CD34+ cells-covering process development, GMP production and validation activities required ahead of a BLA filing. The lentiviral vector program will use AGC's proprietary ProntoLVV™ adherent platform, which has already supported multiple commercially available gene therapies, providing regulators with a familiar and industrially proven framework.
"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."
For Rarity, the Milan-based AGC site and the wider global network across the U.S., Europe and Japan provide both capacity and regulatory experience, lowering risk as the program moves from single-center trials toward routine clinical use.
De-risking the last mile to FDA approval
The collaboration also formalizes the manufacturing strategy behind academic work led by Donald B. Kohn, M.D., at UCLA's Broad Stem Cell Research Center, who invented the lentiviral vector for ADA-SCID. "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."
Kohn's team previously secured a $14.7 million grant from the California Institute for Regenerative Medicine (CIRM) in November 2024 to develop a commercial manufacturing protocol for the therapy, laying the foundation for today's CDMO partnership. The CIRM funding, alongside Rarity's public benefit model, illustrates how public, academic and private actors are aligning to push curative therapies for ultra-rare diseases across the regulatory finish line.
A CDMO partnership built around patient access and reliability
For AGC Biologics, the Rarity agreement strengthens its position as a preferred partner for cell and gene therapy sponsors pursuing commercial scale. "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."
With multiple FDA-approved therapies already in commercial supply and additional programs under review, AGC Biologics is using its established regulatory track record to help Rarity move from bespoke clinical production to reproducible, inspection-ready manufacturing that can withstand the scrutiny applied to first-in-class gene therapies.
Aligning public benefit, philanthropy and industrial scale in gene therapy
Rarity PBC's status as a public benefit corporation puts patient access and long-term sustainability at the center of its business model. Combined with CIRM support and AGC Biologics' global manufacturing infrastructure, the partnership offers a blueprint for how high-complexity genetic medicines can move beyond academic excellence toward broad, equitable availability.
As regulatory discussions advance, the success of RDP-101 will be closely watched as a test case for how industry, public funders and mission-driven companies can work together to deliver durable cures for rare pediatric diseases that traditional commercial models often overlook.
To learn more about AGC Biologics and its global cell and gene therapy manufacturing capabilities, visit www.agcbio.com.