New Non-Profit Fights Rare Diseases, Seeks Profit for Future Treatments
The Institute for Life Changing Medicines, a newly launched non-profit based in Amsterdam, Netherlands, has set its sights on fighting for patients with rare diseases. Co-founded by James M. Wilson, MD, PhD, who serves as the institute's chief scientific officer, and Alex Karnal, who acts as CEO, the organisation aims to encourage the development of lifesaving therapies.
Currently, the institute is engaged in developing therapies for several rare diseases. These include Crigler-Najjar syndrome type 1, AADC deficiency, and Lesch Nyhan syndrome. The institute's strategy involves identifying, acquiring, and clinically testing advanced treatments for potential commercialization. Once sold, the institute plans to reinvest the profits, which will be made with a margin, into future programs.
To further accelerate its therapeutic programs, the institute expects to monetize priority review vouchers. These can be worth up to $100 million each and provide a faster review process for new drugs by the FDA. The institute's mission is particularly important given that one in 10 people in the U.S. has a rare disease, yet treatments are often costly and difficult to access.
The Institute for Life Changing Medicines, founded in 2018, is dedicated to improving the lives of those affected by rare diseases. With a focus on developing and commercializing lifesaving therapies, the institute is committed to reinvesting its profits to advance its mission. By leveraging priority review vouchers and working towards a margin on its products, the institute aims to make a significant impact on the rare disease community.