Mahzi Therapeutics
Mahzi is focused on treating under-served rare genetic neurodevelopmental disorders. The company aims to unite patient and family groups, academic researchers, industry members, and its internal team of experts to develop therapies for these serious diseases. With a team experienced in biopharmaceutical research, development, and manufacturing, Mahzi is dedicated to making a difference in the lives of patients with rare genetic diseases.
Industries
Nr. of Employees
small (1-50)
Products
Gene replacement therapy using AAV9 for WWOX-related disorders (WOREE/SCAR12)
A gene replacement program using AAV9 vector delivery targeting WWOX deficiency-associated neurodevelopmental disorders, advanced through preclinical development in collaboration with an academic laboratory.
Gene replacement therapy using AAV9 for TCF4 deficiency (Pitt Hopkins)
A gene replacement program using AAV9 vector delivery targeting TCF4 deficiency (Pitt Hopkins syndrome), progressed through preclinical development in collaboration with an academic laboratory.
Antisense oligonucleotide program for CHD2 deficiency
Development of an ASO-based therapeutic approach targeting CHD2 deficiency, including preclinical model evaluation and translational studies in collaboration with academic researchers.
Gene replacement therapy using AAV9 for WWOX-related disorders (WOREE/SCAR12)
A gene replacement program using AAV9 vector delivery targeting WWOX deficiency-associated neurodevelopmental disorders, advanced through preclinical development in collaboration with an academic laboratory.
Gene replacement therapy using AAV9 for TCF4 deficiency (Pitt Hopkins)
A gene replacement program using AAV9 vector delivery targeting TCF4 deficiency (Pitt Hopkins syndrome), progressed through preclinical development in collaboration with an academic laboratory.
Antisense oligonucleotide program for CHD2 deficiency
Development of an ASO-based therapeutic approach targeting CHD2 deficiency, including preclinical model evaluation and translational studies in collaboration with academic researchers.
Services
Collaborative research partnerships
Joint development collaborations with academic laboratories and external research groups to advance target validation, preclinical studies, and therapeutic development for rare genetic neurodevelopmental disorders.
Collaborative research partnerships
Joint development collaborations with academic laboratories and external research groups to advance target validation, preclinical studies, and therapeutic development for rare genetic neurodevelopmental disorders.
Expertise Areas
- Rare disease drug discovery and development
- Gene therapy development
- Antisense oligonucleotide therapeutics
- Preclinical translational research and animal models
Key Technologies
- AAV-based gene delivery (AAV9)
- Antisense oligonucleotides (ASOs)
- In vivo animal disease models
- Preclinical efficacy and safety studies