"As many as 1 in 10 people in the U.S. has a rare disease, many of which have no known cure. A new program by the California Institute for Regenerative Medicine (CIRM) could change this by funding novel ways to develop multiple genetic therapies for multiple rare diseases at the same time.
The new rare disease funding program approved by the board will allocate $100 million over two years to support multiple platform technologies for rare diseases. These platforms each represent one approach that could ultimately result in treatments for many diseases. That’s good news for those patients and their families who might otherwise have no options. This new approach for developing therapies for rare diseases is known as the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID).
“RAPID is designed to fundamentally reshape how we advance treatments for people with rare diseases,” said Shyam Patel, PhD, CIRM Associate Vice President of Preclinical Development. “By focusing on scalable platform technologies, we’re accelerating individual projects while building an infrastructure that enables faster, more efficient development across entire categories of genetic conditions. These therapies not only have the potential to reduce lifetime healthcare costs but also will streamline the path of therapies to the clinic and ultimately ensure that promising therapies reach patients who otherwise have no options.”