OUR GOAL
The Institute is a nonprofit organization that identifies, develops and promotes access to life changing medicines for diseases that are not considered commercially viable.
Tachi Yamada, M.D.
Founder
OUR INSPIRATION
On August 4, 2021, our friend, mentor, and inspiration Tachi Yamada passed away. Tachi served as a co-founder and board member of the Institute for Life Changing Medicines. He was a key visionary behind our launch and the biggest supporter of inclusively bringing life changing medicines to all people suffering. Together, we shared a belief that a healthy life is our most basic human life regardless of how many people had a disease or where in the world they lived.
We are entering into an exciting new phase of disease research where treatments and possibly cures are within reach. Tachi felt that the Institute for Life Changing Medicines could play a critical role in bringing this therapeutic revolution to people suffering from diseases that were less common but still devastating. While Tachi’s unexpected death cut his journey short with us, his lessons and vision will live on through our work and progress each and every day.
OUR PROBLEM
1 in 10 people suffer from rare diseases in the U.S.
The patients left behind
Rare diseases have some of the biggest needs for therapeutic advancements but are often left behind due to the patient population size and profitability.
Complex Development
Rare diseases like cell and gene therapies have enormous therapeutic potential but are still very new in the world of modern medicine and manufacturing. There are a lot of growing pains to work through as far as developing and scaling these novel treatments. Due to the highly specific patient population that they serve, there is no financial incentive for larger companies to commercialize these therapies.
Barriers to Access
Next generation medicines like cell and gene therapies that make it past the R&D stage are incredibly costly to deliver and come with immense barriers to access. Rare diseases such as advanced therapies will not be successful if its therapies can’t reach the patients they intend to treat.
Cost saving breakthroughs are not happening at the pace required to make a significant impact. The rare disease community cannot make meaningful progress on their own. Increasing accessibility through clinical trials, cost and payer structure need to be addressed.
OUR SOLUTION
A non-profit with a for-profit model
The Institute aims to overcome industry challenges in rare disease by monetizing priority review vouchers to help sustainably fund the development of these lifesaving therapies for the patients who need them most.
OUR PIPELINE
Targeting these rare diseases.
Crigler-Najjar Type I
Lesch-Nyhan Syndrome
