Mahzi Therapeutics
Clinical-stage biotechnology company developing precision therapies for rare genetic neurodevelopmental disorders. Focus areas include gene replacement and antisense oligonucleotide therapeutics, preclinical disease modeling (including human brain organoids), and early-stage clinical development conducted in partnership with academic labs, patient organizations, and public funders.
Industries
Nr. of Employees
small (1-50)
Products
AAV9-based TCF4 gene replacement therapy (clinical candidate for TCF4 deficiency)
A central nervous system-targeted gene replacement construct based on an AAV9 expression cassette containing TCF4 isoform B, developed to provide functional copies of TCF4 for patients with TCF4 deficiency.
AAV9-based TCF4 gene replacement therapy (clinical candidate for TCF4 deficiency)
A central nervous system-targeted gene replacement construct based on an AAV9 expression cassette containing TCF4 isoform B, developed to provide functional copies of TCF4 for patients with TCF4 deficiency.
Services
Evaluation and potential provision of investigational therapies outside clinical trials subject to regulatory and ethical approvals, physician-submitted requests, and supply considerations.
Partnerships with clinical trial organizations, academic laboratories, and patient foundations to accelerate clinical development programs.
Evaluation and potential provision of investigational therapies outside clinical trials subject to regulatory and ethical approvals, physician-submitted requests, and supply considerations.
Partnerships with clinical trial organizations, academic laboratories, and patient foundations to accelerate clinical development programs.
Expertise Areas
- Gene replacement therapy development
- Antisense oligonucleotide therapeutics
- Clinical trial management for early‑phase CNS studies
- Regulatory strategy for rare pediatric and orphan diseases
Key Technologies
- AAV9 vector-based gene replacement
- Antisense oligonucleotides (ASO)
- Intracerebroventricular administration
- Human brain organoids
News & Updates
First patient dosed in a Phase 1/2 open-label study evaluating a single administration of an AAV9-based TCF4 gene replacement therapy for Pitt Hopkins syndrome.
U.S. FDA granted Rare Pediatric Disease Designation to the AAV9-based TCF4 gene replacement therapy developed for Pitt Hopkins syndrome.
Discussion of how patient-derived organoids informed discovery and supported development of a regulated AAV gene therapy for a neurodevelopmental disorder; highlights organoids as translational platforms.
Initiation of the first clinical gene therapy trial for Pitt Hopkins syndrome with first patient dosed in Phase 1/2.
Regulatory designation by the U.S. FDA for the clinical candidate targeting a rare pediatric neurogenetic disorder.
First patient dosed in a Phase 1/2 open-label study evaluating a single administration of an AAV9-based TCF4 gene replacement therapy for Pitt Hopkins syndrome.
U.S. FDA granted Rare Pediatric Disease Designation to the AAV9-based TCF4 gene replacement therapy developed for Pitt Hopkins syndrome.
Discussion of how patient-derived organoids informed discovery and supported development of a regulated AAV gene therapy for a neurodevelopmental disorder; highlights organoids as translational platforms.
Initiation of the first clinical gene therapy trial for Pitt Hopkins syndrome with first patient dosed in Phase 1/2.
Regulatory designation by the U.S. FDA for the clinical candidate targeting a rare pediatric neurogenetic disorder.