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8 results for “AP4M1”

Trials

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

4 participants · First posted Aug 26, 2022

Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

24 participants · First posted Nov 18, 2024

Active, not recruiting

Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50

1 participants · First posted Oct 6, 2023

Drugs

AP4M1 gene therapy (AAV9, hereditary spastic paraplegia 50)

AAV9 vector-based AP4B1 gene therapy (Cure AP-4)

AAV9 vector-based AP4B1 gene therapy (hereditary spastic paraplegia 47)

News

Catalent Partners with Elpida Therapeutics to Advance AAV Gene Therapy for Ultra-Rare SPG50 Disease