RP-L401

RP-L401 is an ex vivo lentiviral vector-based gene therapy developed for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare and life-threatening autosomal recessive bone disease. The therapy utilizes a lentiviral vector to deliver a functional copy of the *TCIRG1* gene into a patient's own hematopoietic stem and progenitor cells (HSPCs). Mutations in the *TCIRG1* gene lead to dysfunctional osteoclasts that cannot properly resorb bone, resulting in excessive bone density and marrow failure. By restoring TCIRG1 expression, RP-L401 aims to re-establish normal osteoclast function and skeletal homeostasis. The program was originally in-licensed by Rocket Pharmaceuticals from Lund University and Medizinische Hochschule Hannover and received support from the California Institute for Regenerative Medicine (CIRM). However, in December 2021, Rocket Pharmaceuticals discontinued its sponsored clinical evaluation of RP-L401 to focus resources on other pipeline programs, returning the asset to its academic innovators.

Other names

TCIRG1 gene therapyTCIRG-1 gene therapyTCIRG 1 gene therapy