AGC Biologics, Rarity PBC partner on gene therapy for ‘bubble baby’ disease
AGC Biologics has partnered with Rarity PBC to develop and manufacture RDP-101, a gene therapy designed to treat adenosine deaminase severe combined immunodeficiency disorder (ADA-SCID), also known as bubble baby disease.
RDP-101 uses a patient’s own stem cells to restore immune function, offering a potential cure for a condition that leaves infants highly vulnerable to infections. In early clinical testing, the therapy successfully treated 48 of 50 children. Under the new agreement, AGC Biologics will guide the therapy through development and manufacturing, supporting Rarity’s efforts to bring the treatment to patients. If approved by the FDA, RDP-101 would be the first gene therapy available in the U.S. to reverse ADA-SCID.
The collaboration highlights Seattle-based AGC Biologics’ leadership in advanced therapy manufacturing and its growing role in shaping the future of genetic medicine.