One-by-one treatments

For patients who are more than numbers

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

What We Do

Developing customized therapeutics for those who have been diagnosed with rare, genetic diseases that have no treatment or cures.

What We're DoingWho We Are

Transforming Lives

Harnessing the power of CRISPR gene-editing to address the root cause of degenerative diseases.

Unraveling Puzzles

Eliminating barriers to make cures more accessible for more people.

Aligning Medicine

Advocating to create a supportive ecosystem of regulators and insurers.

A First-of-Kind Collaborative

We asked which of the world’s leading experts could move gene editing from theory to therapy—and then we brought them together to make it happen. 

Meet Our R&D Team

Our Collaborators

Pioneering Progress

1. Characterize mutation

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

2. Prototype and Optimize

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

3. Test efficacy

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

4. Manufacture and test safety

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

5. Clinical trial

Dose patient with therapeutic and conduct long-term follow up.

With demonstrated in-vivo efficacy and a successful FDA pre-IND meeting, we are preparing to administer our first custom therapeutic to treat a patient who has Duchenne muscular dystrophy

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Rare Disease: Not Rare At All

Conditions such as Duchenne muscular dystrophy, cystic fibrosis, and more than 7,000 other rare diseases—including rare cancers —impact more than 300 million people worldwide. Our model could eventually reach all of them.

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Our National Sponsors

Partner With Us

As a 501(c)(3) nonprofit biotech, our model relies on generous financial support from individuals, families, and organizations committed to helping us translate leading-edge science into lifesaving solutions for patients.

Support Our Research