RDP-101

RDP-101 is an **ex vivo autologous gene therapy** being developed to treat **Adenosine Deaminase Severe Combined Immunodeficiency Disorder (ADA-SCID)**, also known as "bubble baby disease". This investigational gene therapy works by modifying a patient’s own hematopoietic stem cells outside the body to introduce a functional copy of the adenosine deaminase gene, addressing the underlying genetic cause of the disorder. In clinical studies, this approach has demonstrated restoration of immune function, with success reported in a majority of treated children. If approved, RDP-101 would be the first gene therapy available in the United States to reverse ADA-SCID. The therapy is being developed by Rarity PBC, with manufacturing support from AGC Biologics[5][7][10][11][12].

Brand names

RDP-101RDP101RDP 101

Other names

RDP-101RDP101RDP 101