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.