New Non-Profit Aims to Accelerate Rare Disease Therapies
The Institute for Life Changing Medicines, a new non-profit organisation, has been launched with a mission to accelerate research and develop innovative therapies for patients with rare diseases. Co-founded by James M. Wilson, MD, PhD, who serves as the chief scientific officer, and Alex Karnal, the institute's CEO, it aims to make a significant impact on the lives of those affected by these conditions.
The institute's portfolio currently includes therapies for Crigler-Najjar syndrome type 1, AADC deficiency, and Lesch Nyhan syndrome. Notably, it has acquired the licenses for the mRNA-3351 therapy for Crigler-Najjar Syndrome Type 1 from Regenxbio, demonstrating its commitment to advancing treatments for these rare diseases.
To fund its operations and future programs, the institute plans to sell its products with a margin and reinvest the profits. Additionally, it expects to monetize priority review vouchers, which can significantly reduce the FDA review time from 10 to 6 months and are estimated to be worth around $100 million each. This strategy will help the institute to fast-track its therapeutic programs and bring lifesaving treatments to patients more quickly.
The Institute for Life Changing Medicines is dedicated to identifying, acquiring, and clinically testing advanced treatments for potential commercialization. By doing so, it seeks to encourage and incentivize the development of lifesaving therapies for patients with rare diseases, ultimately improving their quality of life.
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