AGC Biologics Partners with Rarity PBC to Commercialize Breakthrough Gene Therapy

AGC Biologics 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 US 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. 

Under the agreement, AGC Biologics will provide end-to-end services for Rarity's gene therapy product. 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 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 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.”