ILCM Launches to Accelerate Rare Disease Therapies, Licenses mRNA-3351 from Moderna
The Institute for Life Changing Medicines (ILCM) has been co-founded by James M. Wilson, MD, PhD, and Alex Karnal with a mission to accelerate the development of therapies for patients with rare diseases. The institute is currently focusing on Crigler-Najjar syndrome type 1, AADC deficiency, and Lesch Nyhan syndrome.
ILCM has licensed mRNA-3351, a potential therapy for Crigler-Najjar syndrome, from Moderna without any upfront fees or downstream payments. This therapy was provided by Moderna to support ILCM's novel nonprofit business model. The institute aims to monetize priority review vouchers, which can significantly shorten FDA review times and are estimated to be worth around $100 million each. ILCM will identify, acquire, and clinically test advanced treatments for potential commercialization, with profits reinvested in future programs.
One in 10 people in the U.S. has a rare disease, but treatments are often costly and difficult to access due to small patient populations. ILCM's partnership with Moderna could serve as a model for developing life-changing medicines for those living with rare diseases.
The Institute for Life Changing Medicines is dedicated to fighting for patients with the largest therapeutic needs and encouraging the development of lifesaving therapies. By licensing mRNA-3351 from Moderna and pursuing other advanced treatments, ILCM is committed to improving the lives of people with rare diseases.
