Company intelligence / Profile preview
Mahzi Therapeutics
San Mateo, California, United Statesmahzi.com ↗
- Ownership
- Private
- Development stage
- Preclinical
01
Overview
Mahzi Therapeutics develops genetic therapies for rare neurodevelopmental disorders. Its lead programs are in preclinical development, including an AAV9-based gene replacement therapy targeting TCF4 for Pitt-Hopkins syndrome. The company partners closely with patient advocacy groups and academic labs to advance therapies from discovery through regulatory steps toward clinical trials. No clinical results have been reported yet.
- Therapeutic areas
- Rare DiseasesNeurology
- Modalities
- Gene therapy (including AAV-based gene therapy)Antisense oligonucleotides (ASO)
- Industry
- Biotech
02
Drug pipeline
4 assets3 more assets in the full pipeline.
03
Partnerships
VenrockHealthCap Venture CapitalDROIA VenturesUltragenyx PharmaceuticalHBM Partners—primarily as investors but also strategic partnersAcademic partnerships with Muotri Lab at UCSD and others
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