New Institute Partners with Moderna to Fight Rare Diseases
A new non-profit institute, the Institute for Life Changing Medicines, has partnered with Moderna to advance research and develop new therapies for people with rare diseases. The institute aims to fight for patients with rare diseases and encourage the development of lifesaving therapies, starting with Crigler-Najjar syndrome type 1.
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. The institute's goal is to change this by identifying, acquiring, and clinically testing advanced treatments for potential commercialization, with profits reinvested in future programs. In a novel nonprofit business model, Moderna has provided mRNA-3351 at no cost to support the institute's work. This therapy, licensed from Moderna with no upfront fees and no downstream payments, shows promise in treating Crigler-Najjar syndrome type 1, a rare genetic disorder that leads to severe bilirubin poisoning. The institute is also developing therapies for other rare diseases such as AADC deficiency and Lesch Nyhan syndrome. To speed up the approval process, the institute expects to monetize priority review vouchers, which can shorten FDA review time and are estimated to be worth $100 million each. The institute was co-founded by James M. Wilson, MD, PhD, a gene therapy pioneer, and Alex Karnal, an expert in biotechnological and pharmaceutical investments.
The institute's partnership with Moderna could serve as a model for developing life-changing medicines for rare diseases. By working together, they aim to bring innovative therapies to patients in need, starting with Crigler-Najjar syndrome type 1. The institute's unique business model and dedicated leadership promise a future where treatments for rare diseases are more accessible and affordable.
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