Pitt Hopkins syndrome gene therapy - MAHZI Therapeutics
Latest Information Update: 31 Jul 2023
At a glance
- Originator MAHZI Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Pitt-Hopkins syndrome
Most Recent Events
- 13 Jul 2023 Preclinical trials in Pitt-Hopkins syndrome in USA (Parenteral) before July 2023 (MAHZI Therapeutics pipeline, July 2023)