MELPIDA

MELPIDA is an investigational gene therapy designed to treat hereditary spastic paraplegia type 50 (SPG50), a rare neurodegenerative disorder caused by biallelic loss-of-function mutations in the AP4M1 gene. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a fully functional human AP4M1 cDNA copy directly into neuronal cells via intrathecal injection. This approach aims to restore normal function of the adaptor protein complex 4 subunit mu-1 (AP-4μ), potentially reducing or halting neurodegeneration associated with SPG50. Preclinical studies have demonstrated dose-dependent expression of AP4M1 mRNA in the central nervous system and sustained transgene expression for up to one year post-injection, with acceptable safety profiles in animal models. Early clinical data indicate that MELPIDA is well-tolerated and may stabilize or improve neurological function in treated patients[2][5][6][7][8].

Brand names

MELPIDA

Other names

Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 TransgeneAAV gene therapy for hereditary spastic paraplegia type 50