Gene therapy, long seen as too scary to invest money has had a big comeback over the past year. Below are some of the best gene therapy stocks that could make you serious amounts of money if these companies become successful in their gene therapy research over the next 10 years.
Celladon (stock symbol: cldn) – Working on a cure to treat advanced heart failure.
Celladon Corporation, a clinical-stage biotechnology company, focuses on developing cardiovascular gene therapy and calcium dysregulation. Celladon’s lead product candidate includes MYDICAR that uses genetic enzyme replacement therapy to correct the Sarco/endoplasmic reticulum Ca 2+ -ATPase 2a enzyme deficiency in heart failure patients that results in inadequate pumping of the heart. Its MYDICAR product is used to treat patients with systolic heart failure, diastolic heart failure, and advanced heart failure, as well as to treat pulmonary arterial hypertension and arteriovenous fistula maturation failure. The company is also developing a membrane-bound form of Stem Cell Factor for the treatment of cardiac ischemic damage and compounds for the treatment of diabetes and neurodegenerative diseases. Celladon Corporation was founded in the year 2000 and is headquartered in beautiful San Diego, Southern California.
Gensight Biologics – Working on a cure to treat genetic blindness.
Bluebird Bio (stock symbol: blue) – Working on a cure to treat Lorenzo’s Oil disease.
Audentes Therapeutics – Working on a cure to treat X-Linked Myotubular Myopathy.
Spark Therapeutics (stock symbol: once) – Working on a cure to treat genetic blindness.
Dimension Therapeutics – Working on a cure to treat Hemophilia.
Juno Therapeutics – Working on a cure to treat Cancer.
Uniqure (stock symbol: qure) – Working on a cure to treat Lipoprotein lipase deficiency.
Voyager Therapeutics – Working on a cure to treat Parkinson’s disease.
Nightstarx – Working on a cure to treat Retinal Dystrophies.