FDA, NIH, and 15 private organizations join forces to increase effective gene therapies for rare diseases

The U.S. Food and Drug Administration, the National Institutes of Health, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies.

from Breaking World Pharma News https://ift.tt/2ZtmnGv