Mahzi Therapeutics
Clinical-stage biotechnology company developing genetic medicines for rare pediatric neurodevelopmental disorders.
South San Francisco, California
2020
14
$60M
$87.9M pre-money
What They Do
Mahzi develops disease-specific gene therapies and antisense oligonucleotides for rare genetic neurodevelopmental conditions with few or no approved treatments. Its lead asset, MZ-1866, is an AAV9 gene-replacement therapy in a Phase 1/2 trial for Pitt Hopkins syndrome, while earlier programs target WWOX-related epileptic encephalopathy and CHD2 deficiency. The model pairs internal drug development with academic labs and patient foundations.
Competitive Position
Mahzi competes for rare-neurology talent, trial sites, and capital with genetic-medicine developers such as Ultragenyx, Encoded Therapeutics, Taysha Gene Therapies, and Stoke Therapeutics. It differentiates by selecting the modality for each genetically defined disorder and by formally involving disease foundations and families in program formation and financing.
Funding Rounds
No funding rounds on record for this company.