Clinical Trial Update: Melpida: Recombinant Adeno-associated Virus (serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt) [Phase 1, Phase 2]

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Summary

Phase 1/2 trial evaluating Melpida, a recombinant AAV9 gene therapy delivering codon-optimized AP4M1 (hAP4M1opt) for SPG50-related hereditary spastic paraplegia…

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Diseases / indications

• Growth Retardation
• Hereditary Spastic Paraplegia Type 50
• Intellectual Disability
• Microcephaly

Drugs mentioned

• Melpida (AAV9-hAP4M1opt gene therapy)

Snippet

Sponsor: Elpida Therapeutics SPC | Conditions: Spasticity, Muscle, Microcephaly | Phase 1, Phase 2 | Status: RECRUITING