A new biotechnology company will take over human trials of two gene therapies that could offer one-time treatments for a form of childhood blindness and hemophilia B.
The gene therapies were developed by researchers at the Children’s Hospital of Philadelphia, which has committed $50 million to the new company called Spark Therapeutics. The launch is the latest hint that after decades of research and some early setbacks, gene therapy may be on its way to realizing its potential as a powerful treatment for inherited disease.
Spark has a chance to be the first gene-therapy company to see FDA approval.
Read the full, original story here: New Gene Therapy Company Launches