One promising aspect of the future of healthcare is gene therapy. Gene therapy corrects genetic disorders by transplanting normal genes into cells in place of missing or defective ones. Spark Therapeutics (ONCE) is getting closer to achieving an FDA approval for voretigene neparvovec, a one-time treatment for inherited retinal disease (IRD) caused by mutations in the RPE65 gene. To put that in layman’s terms, it’s a treatment that a patient would take only once to restore functional vision from a genetic disorder that causes blindness.
Spark Therapeutics was created from the knowledge and technology that was accumulated over two decades at the Children’s Hospital of Philadelphia (CHOP). The company aims to change the lives of patients where either no current therapies exist or where only palliative therapies exist. The company’s ticker symbol is ONCE, meaning that Spark’s treatments are designed to be one-time only treatments, as opposed to drugs that have to be taken on an ongoing basis.
