Through collaborations with world-renowned academics and clinicians, we are moving science forward to develop life-saving gene therapies for rare and ultra-rare disease patients overlooked and underserved by traditional drug development efforts.
Despite advancements in science and technology, developing a new drug requires an average investment of $2 billion and ten years. This cost and timeline raises challenges for developing therapeutics for rare and ultra-rare disease populations who are fewer in number, yet urgently in need of treatment.
Our collaborative framework reimagines the drug development process to provide an accelerated pathway for the development of life-saving therapeutics that previously did not exist for people with rare and ultra-rare neuromuscular diseases. Moreover, our model offers industry collaborators a mechanism to expand their drug development pipeline.
Characterize patient’s genetic mutation and establish a cell line through a tissue biopsy.
Prototype and optimize therapeutic candidates capable of fixing mutation in the cell line.
Test candidates via tissue modeling and/or mutation-specific mouse model for efficacy.
Manufacture final therapeutic and conduct pivotal study. Submit IND to the FDA for approval.
Administer therapeutic and monitor patient(s) for safety and efficacy signals.
License technology to industry to treat the rest of the population.
The success of our model relies on our extensive network of academic and industry collaborations across the United States, Canada and Europe. As the scope of our research and development grows, so does our need to expand our network of collaborators to advance therapeutics for rare and ultra-rare neuromuscular diseases, speed drug development for orphan diseases, and expand access to treatments to broader populations.Learn More
Our research and development is powered by the generous financial support of families, rare disease communities and organizations unwilling to accept the status quo of drug development practices. As a 501c3 non-profit biotech, 93+% of all donations go directly to the research and development of life-saving therapies.
We offer a number of ways to get involved with moving science forward. Learn More