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Our Model

Historically, it could take 10 years and hundreds of millions of dollars to develop a treatment with a 5% chance of success.

Today, we can design, develop, and treat a child with a rare genetic disease in as little as a year.

Our vision is to scale this transformative process, making effective treatments accessible to every child in need. 

Our Strategy.

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    Built Around Patients

    We innovate medicines, technologies, systems, and efficiencies with the speed and urgency needed to maximize patient access to treatments—because every moment matters. Time = Life.

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    Antisense Oligonucleotides

    We utilize a proven genetic medicine platform that is highly targeted, enables rapid development, and supports affordable manufacturing.

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    AI

    We harness the power of AI and machine learning to target the right genes, design better medicines, and reduce the time needed in preclinical and clinical development.