Bespoke Gene Therapy Consortium to Fund Research into Improving Standardizing Methods for Preclinical Testing of Gene Therapies.

Gene therapy is a realistic option to treat rare diseases like PFIC, though these therapies are new and require very thorough scientific research and development to minimize risk and get approved. The NIH, FDA, and FIH have unveiled a public-private partnership aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease. There are several gene therapies already available with many more in clinical trial, though there is a lack of standards, access to tools, and economic incentive. This funding and partnership aims to fix this issue to speed up research on gene therapy and bring it to the masses sooner than the projected time period for beneficial therapies.

To read more on the public-private partnership and what it aims to accomplish you can go to the article. In the article there is also more conversation about gene therapies and the clinical trials that may also provide a better understanding on what is happening in the field.

pfic gene therapy