GC311

GC311 is an adeno-associated virus (AAV) gene therapy designed to treat X-linked adrenoleukodystrophy (X-ALD), a rare genetic disorder caused by mutations in the *ABCD1* gene. The therapy utilizes an AAV vector to deliver a functional copy of the *ABCD1* gene, which encodes the adrenoleukodystrophy protein (ALDP). ALDP is essential for the transport of very-long-chain fatty acids (VLCFAs) into peroxisomes for degradation; its deficiency leads to VLCFA accumulation, causing progressive demyelination in the central nervous system and adrenal insufficiency. Developed by GeneCradle, GC311 aims to restore ALDP function and halt disease progression. It is currently in Phase 1/2 clinical development for cerebral X-linked adrenoleukodystrophy (CALD).

Other names

AAV-ABCD1 gene therapyAAV-ABCD-1 gene therapyAAV-ABCD 1 gene therapyGeneCradle X-ALD program