Top Medical Startups in Gene Therapy

We focus on cellular aging as this is the leading risk factor for most major pathology. We are developing therapies to regenerate muscle and tissue. We believe everyone has the right to access therapies.

DNAlite Therapeutics committed to bringing the next-generation of drugs to organs protected by mucus barriers. Diseases such as FAP, colorectal cancer, and Crohn's disease are currently treated with complete or partial removal of the colon and/or rectum. Our aim is to prevent, rather than manage the progression of these diseases.

Meira Gene Therapy is Targeting inherited retinal diseases with therapies designed to halt vision loss.

Bluebird Bio offers products based on the transformative potential of gene therapy for patients with genetic and orphan diseases.

Genprex is a clinical stage gene therapy company developing molecular therapies to better target lung cancer cells. Their new approach to treating cancer is based upon our novel proprietary technology platform, including our initial product candidate, Oncoprex immunogene therapy, or Oncoprex.

We design transformational gene therapies that aim to enhance the body's own defenses to defeat disease.

Deep Genomics brings together world-leading expertise in machine learning and genome biology. We’re inventing a new generation of computational technologies that predict what will happen within a cell when DNA is altered by genetic variation, whether natural or therapeutic. Our systems predict the molecular effects of genetic variation, opening a new and exciting path to discovery for disease diagnostics and therapies.

Orgenesis is a development stage company with a novel therapeutic approach in the treatment of diabetes by correcting malfunctioning organs with new functional tissues created from the patient’s own existing organs. Orgenesis employs a molecular and cellular approach directed at converting liver cells into functional insulin-producing cells as a treatment for diabetes.

Akouos is a new biotechnology company focused on restoring and preserving hearing.