TYF-ABCD1

TYF-ABCD1 is an *in vivo* gene therapy designed for the treatment of X-linked adrenoleukodystrophy (X-ALD). It utilizes a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) to deliver a functional copy of the *ABCD1* gene directly to the patient. The *ABCD1* gene encodes the peroxisomal ATP-binding cassette transporter protein, which is responsible for transporting CoA-activated very long-chain fatty acids (VLCFA) into the peroxisomes for degradation. Mutations in this gene lead to the accumulation of VLCFAs, causing the neurological and adrenal symptoms associated with ALD. Unlike traditional treatments that require ex vivo modification of hematopoietic stem cells and myeloablative chemotherapy, TYF-ABCD1 is administered through intrathecal and intravenous injections to directly correct the genetic defect and metabolic pathology. This approach aims to provide a safer and more simplified treatment process for both pediatric and adult patients with symptomatic X-ALD.

Other names

lentiviral vector TYF-ABCD1