Next-generation therapeutics

Advancing treatments for rare neuromuscular diseases
We’re accelerating tailored, life-saving therapies for people with rare diseases who need them—now.
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What We Do

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.

Dollars invested into R&D
Potential lives impacted
Number of indications in development

How We Do It

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.

Our Science

Pioneering Progress

Stage 1

Characterize patient’s genetic mutation and establish a cell line through a tissue biopsy.

Stage 2
Prototype and Optimize

Prototype and optimize therapeutic candidates capable of fixing mutation in the cell line.

Stage 3

Test candidates via tissue modeling and/or mutation-specific mouse model for efficacy.

Stage 4
Manufacture and
Test Safety

Manufacture final therapeutic and conduct pivotal study. Submit IND to the FDA for approval.

Stage 5

Administer therapeutic and monitor patient(s) for safety and efficacy signals.

Stage 6
License to

License technology to industry to treat the rest of the population.

Work With Us

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.

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