USHER 2020 FOUNDATION INC

The mission of Usher 2020 Foundation is to stop, slow or reverse the progression of sight-loss caused by Usher Syndrome and Retinitis Pigmentosa (RP). Our goal is to have treatments in clinical trials by the year 2025.

It is our short term goal to bring a drug therapy treatment to clinical trial no later than 2022, and our gene therapy treatment to trial by the year 2025.

Usher 2020 Foundation does this by focusing on funding research and creating opportunities for scientists and clinicians to collaborate, as well as filling in the gaps that exist to move therapies into patients.

Usher 2020 focuses only on moving proven research into clinical trials for patients. By constantly reviewing the latest science and research available, funding the best sources, facilitating collaboration, and expediting treatments, Usher 2020 is moving closer to our goal of slowing the progression of blindness in those with Retinitis Pigmentosa, and stopping the loss of sight in those with a rare form of Usher Syndrome.

1. At LSU, we have advanced the basic science to the point where we are prepared to begin the IND process, and we have provided the researcher with consultation on how to proceed to clinical trial.
2. At MEEI, we have advanced the development of an AAV vector and are supporting the work toward correcting the USH 1C gene.
3. At Iowa, we have supported the advancement of both gene and stem cell therapies toward clinical trials.
4. We have secured Johannes Guttenberg University in Mainz, German, to study the isoform of the USH 1C gene, and to facilitate the creation of a pig model, both needed to bring gene therapy to the clinic.
5. We are supporting work at NEI/NIH for testing the library of drugs available to slow the progression of vision loss in those with Usher Syndrome.
6. We have created a model for all rare diseases to move treatments from the lab into the clinic.
7. We have funded an extension study to bring a drug that will slow the progression of sight loss for those with Retinitis Pigmentosa to clinical trial.
8. We have created a collaborative network of researchers and clinicians to bring a gene therapy treatment for Usher Syndrome to clinical trial.
9. We have supported the design of a clinical trial using a drug therapy to slow the progression of Retinitis Pigmentosa.
10. We have begun testing a gene therapy treatment for a rare form of Usher Syndrome in a large animal model.