AGC Biologics has entered into an agreement with Rarity PBC to develop and manufacture RDP-101, a breakthrough gene therapy targeting Adenosine Deaminase Severe Combined Immunodeficiency Disorder (ADA-SCID), also known as “Bubble Baby Disease”. If the therapy receives FDA approval, RDP-101 will become the first commercially available gene therapy in the United States to reverse ADA-SCID, an inherited immune disorder that affects between one and five infants per million births and accounts for about 15% of all SCID cases.
The ex vivo autologous approach leverages patients’ own hematopoietic stem cells, genetically modified using Rarity’s EFS-ADA Lentiviral Vector to restore immune function. Clinical trials have demonstrated dramatic results, with 48 out of 50 children successfully treated. AGC Biologics will provide process development, GMP manufacturing, and regulatory support using its proprietary ProntoLVV™ platform, ensuring robust, scalable production of the therapy for market supply.
“This partnership advances our mission to deliver life-saving therapy to ADA-SCID patients worldwide,” said Rarity PBC CEO Dr. Paul Ayoub. The initiative is backed by substantial support from the California Institute for Regenerative Medicine, highlighting the project’s public benefit and innovation for rare diseases.