Company intelligence / Profile preview
Elpida Therapeutics
Los Angeles, California, United Stateselpidatx.com ↗
- Ownership
- Private
- Development stage
- Preclinical
01
Overview
Elpida Therapeutics develops gene therapies targeting ultra‐rare pediatric neurological diseases. Its lead candidate, Melpida, an AAV9/AP4M1 gene therapy for SPG50, has completed Phase I/II and will enter a Phase III trial in August 2024. The company also has a preclinical program for CMT4J using an AAV9/FIG4 vector and additional pipeline assets targeting other rare CNS disorders. Elpida’s model focuses on programs deprioritized by traditional biotech due to small patient populations but with strong scientific rationale. Early results show promise in animal models and initial human studies[2][3][5][6].
- Therapeutic areas
- Neurology
- Modalities
- AAV-based gene therapy (AAV9)Antisense oligonucleotides (ASO)
- Industry
- Biotech
02
Drug pipeline
7 assets6 more assets in the full pipeline.
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Partnerships
Viralgen Vector Core – manufacturing partner for clinical trial material[9]Cure CMT4J Foundation – advocacy/research partnership[4]University of Texas Southwestern Medical Center – academic collaboration on Melpida development[6]California Institute for Regenerative Medicine – grant funding/collaboration[3]
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