Gene Therapy, Gene editing platform, delivery vector platform

HuidaGene Therapeutics is at the forefront of innovative gene therapy technologies, primarily utilizing advanced CRISPR systems, specifically CRISPR-Cas13 and CRISPR-Cas12, for RNA and DNA editing. These technologies enable precise modifications to genetic material, which are crucial for addressing various genetic disorders. The company focuses on developing therapies for conditions in neurology, such as Huntington's Disease and Amyotrophic Lateral Sclerosis, as well as ophthalmological diseases like RPE65 Mutation-Associated Inherited Retinal Dystrophies. Their pipeline reflects a commitment to utilizing these cutting-edge gene editing techniques to create effective treatments for a range of inherited and acquired diseases.

In addition to CRISPR technologies, HuidaGene employs the HG-AAV platform, which leverages adeno-associated viruses (AAV) as vectors for gene delivery. AAVs are recognized for their safety and efficiency in transducing target cells, making them ideal for gene replacement and genome editing therapies. The company is actively enhancing the AAV platform by developing novel capsids with improved transduction efficacy and safety profiles, as well as optimizing production systems to increase yield. This dual approach of combining CRISPR editing with advanced AAV technology positions HuidaGene Therapeutics to make significant strides in the field of gene therapy, particularly for challenging conditions in neurology and ophthalmology.

China

HuidaGene Biotechnology Co., Ltd (辉大基因) is a global biotechnology company focusing on the discovering, engineering, and developing CRISPR-based genetic medicine to rewrite the future of genomic medicine.

Based in Shanghai and New Jersey, HuidaGene is committed to addressing patient's needs globally with various preclinical therapeutic programs covering ophthalmology, otology, myology, and neurology.

Company's CRISPR-based therapeutics offer the potential to cure patients with life-threatening conditions by repairing the cause of their disease. We are committed to transform the future of genome-editing medicine.

Digestive System Diseases

Eye Diseases

Genetic Diseases

Infectious Diseases

Metabolic Diseases

Musculoskeletal Diseases

Nervous System Diseases

Pathological Conditions

HuidaGene Therapeutics

Leber congenital amaurosis

HG004

Biologics

Gene Therapy

Age-related macular degeneration

HG202

Retinitis Pigmentosa

HG301

Stargardt disease

HuidaGene Therapeutics Drug #1

Rett syndrome

HG204

Amyotrophic lateral sclerosis

HG2A2103

Huntington's disease

HG2A2019

Hearing loss

HG205

Duchenne muscular dystrophy

HG302

Myotonic Dystrophy Type 1

HuidaGene Therapeutics Drug #2

Transthyretin amyloidosis

HuidaGene Therapeutics Drug #3

Dyslipidemia

HuidaGene Therapeutics Drug #4

Hepatitis B

HuidaGene Therapeutics Drug #5

Adeno-associated viral vector (AAV)

Antiviral Therapies

Gene Editing/CRISPR

Therapeutic

HuidaGene Therapeutics

Company Details