New Institute Aims to Accelerate Rare Disease Therapies
A new non-profit institute, the Institute for Life Changing Medicines, has been established to accelerate research and develop therapies for people with rare diseases. The institute, co-founded by gene therapy pioneer James M. Wilson, MD, PhD, and biotech investment expert Alex Karnal, aims to make a significant impact on the lives of millions affected by rare conditions.
The institute's primary focus is on advancing treatments for Crigler-Najjar syndrome type 1, AADC deficiency, and Lesch Nyhan syndrome. It has licensed mRNA-3351, a therapy for Crigler-Najjar, from Moderna with no upfront fees or downstream payments. This aligns with Moderna's values and brings new hope to patients and their families.
The institute plans to monetize priority review vouchers, worth an estimated $100 million each, to shorten FDA review times. This strategy will help expedite the approval process for these much-needed therapies. Both Karnal and Wilson praised Moderna's support of the institute's innovative nonprofit business model.
In addition to its therapeutic pipeline, the institute aims to identify, acquire, and clinically test advanced treatments for potential commercialization. Profits generated will be reinvested in future programs, ensuring a sustainable and continuous effort in rare disease research.
With one in ten people in the U.S. affected by a rare disease, the Institute for Life Changing Medicines is poised to make a substantial difference. By advancing therapies, reinvesting profits, and expediting approval processes, the institute is committed to improving the lives of millions of people living with rare diseases.
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