ELP-001

ELP-001 is an adeno-associated virus serotype 9 (AAV9) vector-based gene therapy designed to treat Spastic Paraplegia Type 50 (SPG50), a rare neurodegenerative disorder caused by mutations in the AP4B1 gene. The therapy delivers a functional copy of the AP4B1 gene to the central nervous system via intrathecal administration. By restoring the production of the AP-4 complex subunit beta-1 protein, the treatment aims to halt or reverse the progression of the disease, which typically manifests as developmental delay and progressive spasticity. It was developed by Elpida Therapeutics, a company founded to advance treatments for ultra-rare diseases, following initial development by the CureSPG50 foundation.

Other names

SPG50 gene therapySPG-50 gene therapySPG 50 gene therapyAAV9-AP4B1AAV-9-AP4B1AAV 9-AP4B1