Gene therapy for rare genetic neurodevelopmental disorders

Mahzi Therapeutics is dedicated to addressing rare genetic neurodevelopmental disorders, which often lack effective treatment options. The company employs a collaborative approach, bringing together patient and family groups, academic researchers, and industry experts to develop targeted therapies. Their research focuses on specific conditions such as WOREE/SCAR12, Pitt Hopkins syndrome, and CHD2 deficiency, each characterized by distinct genetic mutations and clinical presentations. By leveraging the expertise of their team and partners, Mahzi aims to identify the most suitable therapeutic strategies for these complex diseases.

To advance their therapeutic goals, Mahzi is utilizing innovative gene therapy techniques. For WOREE/SCAR12 and Pitt Hopkins syndrome, the company is developing an AAV9-based gene replacement approach in collaboration with leading academic labs. This method aims to address the underlying genetic causes of these disorders by delivering functional copies of the affected genes. Additionally, for CHD2 deficiency, Mahzi is exploring an antisense oligonucleotide (ASO) approach, which targets the specific genetic mutations associated with the condition. Through these advanced technologies, Mahzi Therapeutics is committed to rapidly progressing potential treatments for patients suffering from these serious neurodevelopmental disorders.

United States

The comapanies' research and development efforts are focused exclusively on rare genetic neurodevelopmental disorders, a group of serious diseases with few or no treatment options.

Genetic Diseases

Nervous System Diseases

Psychological Diseases

Skin Diseases

Mahzi Therapeutics

Developmental and Epileptic Encephalopathy

Mahzi Therapeutics Drugs #1

Biologics

Gene Therapy

Pitt-Hopkins Syndrome

Mahzi Therapeutics Drug #2

Autism

Epilepsy

Photosensitivity

Mahzi Therapeutics Drug #3

California - North

California

Adeno-associated viral vector (AAV)

Antisense Oligonucleotides

Oligonucleotides

Therapeutic

Mahzi Therapeutics

Company Details