New Institute Tackles Rare Diseases with mRNA Therapies, Backed by Moderna
One in 10 Americans lives with a rare disease, yet treatments are often elusive and expensive due to small patient numbers. Now, the Institute for Life Changing Medicines, co-founded by James M. Wilson, MD, PhD, and Alex Karnal, is pushing forward with a unique approach to tackle this challenge, backed by Moderna's support.
The institute aims to champion patients with significant therapeutic needs, often overlooked by traditional pharmaceutical companies. It will identify, acquire, and clinically test advanced treatments for potential commercialization, with profits reinvested into future programs.
One such therapy is mRNA-3351 for Crigler-Najjar syndrome type 1, licensed from Moderna with no upfront fees or downstream payments. The Eva Luise and Horst Köhler Foundation, also working on Crigler-Najjar therapies, has partnered with Moderna. The institute expects to monetize priority review vouchers, estimated at $100 million each, to speed up FDA reviews.
Currently, the institute is developing therapies for Crigler-Najjar, AADC deficiency, and Lesch Nyhan syndrome, demonstrating its commitment to addressing diverse rare diseases.
With Moderna's backing and a novel nonprofit business model, the Institute for Life Changing Medicines is determined to bring hope to patients with rare diseases. By reinvesting profits and leveraging priority review vouchers, the institute is poised to accelerate the development and access to life-changing medicines.
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