Gosset Ownership
Private
Therapeutic Areas
Stage
Preclinical
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Modalities
Mahzi Therapeutics General Information
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.
Contact Information
Drug Pipeline
No pipeline data available
Key Partnerships
Venrock, HealthCap Venture Capital, DROIA Ventures, Ultragenyx Pharmaceutical, HBM Partners—primarily as investors but also strategic partners, Academic partnerships with Muotri Lab at UCSD and others
Mahzi Therapeutics Funding
No funding data available
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