Mahzi Therapeutics Announces FDA Rare Pediatric Disease Designation for MZ-1866 ...

Mahzi Therapeutics' investigational gene therapy MZ-1866 for Pitt Hopkins syndrome received the FDA Rare Pediatric Disease Designation, enabling eligibility for a valuable Priority Review Voucher upon approval. The ongoing Phase 1/2 UNITE study is over 50% enrolled, supported by state funding and collaborations with academic institutions.

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