Top Gene Therapy Experts

Experts providing access of best of their services to people in affordable manner, diagnosing and curing variety of diseases.

Dimension Therapeutics is developing novel, liver-directed gene therapy treatments for severe, rare genetic disorders. Dimension’s lead programs address significant medical needs for new therapies and include a wholly owned program in hemophilia B.

Generation Bio is a biotechnology company developing a breakthrough class of genetic medicines to enable a new generation of people unaffected by inherited disease.

Spark is a leader in the field of gene therapy, seeking to transform the lives of patients by developing potential one-time, life-altering treatments for debilitating genetic diseases. We initially are applying our integrated platform to treat rare diseases where no, or only palliative, therapies exist.

Mirna is a clinical-stage biopharmaceutical company developing a broad pipeline of leading microRNA-based oncology therapeutics, the first to demonstrate clinical proof of concept for microRNA replacement therapy.

Edge Therapeutics is a clinical-stage biotechnology company that discovers, develops and seeks to commercialize novel, hospital-based therapies capable of transforming treatment paradigms in the management of acute, life-threatening neurological conditions.

4D Molecular Therapeutics is unlocking the full potential of the gene therapy field. Our customized AAV vectors can deliver genes to any cell in the body to eradicate disease.

Intellia is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics utilizing a recently developed biological tool known as the CRISPR/Cas9 system.

UniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients
 with severe disorders.