AGC Biologics, Rarity PBC partner on gene therapy for ‘bubble baby’ disease

CDMO AGC Biologics will provide comprehensive development and GMP manufacturing for Rarity PBC’s gene therapy, RDP-101, for adenosine deaminase severe combined immunodeficiency disorder (ADA-SCID), also known as bubble baby disease.

If approved by the U.S. FDA, RDP-101 would be the first gene therapy available for commercial use to reverse ADA-SCID in patients in the U.S.

ADA-SCID is a rare, life-threatening inherited condition that severely compromises the immune system, leaving infants vulnerable to frequent and severe infections. 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 a phase 1/2 clinical trial, the therapy successfully treated 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.