Boldly
Restoring

Genetic Health

Restoring protein expression by harnessing the body's potential with RNA medicine.

Scientist working in genetics laboratory

Phase 3 Study Now Underway

Our lead program is advancing through global Phase 3 trials — a milestone in our mission to change the trajectory of severe genetic disease.

Visit our Clinical Studies page

Our Platform

PROTON: Restoring
Missing Proteins

Precise RNA-Oriented Targeted Output Normalization, or PROTON, is 24VitalCare's proprietary research platform. Unlike other RNA-based approaches, PROTON aims to restore missing proteins by increasing — or amplifying — protein output from healthy genes, compensating for the non-functioning copy of the gene.

Our initial applications for this technology are diseases where one copy of a gene functions normally and the other is mutated — also called haploinsufficiencies. Our research shows PROTON can increase full-length, fully functional protein expression from a variety of healthy genes, supporting our belief that it may lead to an entirely new way of treating many severe diseases.

Discover PROTON
Parent and child representing Dravet Syndrome families

Lead Disease Area

Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures beginning within the first year of life. The effects go beyond seizures, including intellectual disability and developmental delays. Our goal is to develop the first disease-modifying therapy that addresses the genetic cause.

Learn About Dravet Syndrome

Pipeline

The Future of our Science

We are fearless in our pursuit to realize the full potential of PROTON to help people living with severe diseases. Clinical studies of our lead investigational medicine are ongoing. We've demonstrated promise for a PROTON ASO to treat the underlying cause of autosomal dominant optic atrophy, and continue to explore PROTON's potential across a number of other diseases.

Our Vision

Using Science to Change
People's Lives

24VitalCare was founded on the idea that we could use unique insights in RNA biology to do something that has never been done before: increase protein output from healthy genes using an RNA-based approach. Our team is making steady progress on turning that vision into reality.

Careers

Spark Your Career

We are fired up about science and our ability to make a difference for people living with severe diseases. We are purposefully inclusive, bring our authentic selves to work each day and — despite our serious mission — look to have fun while we're at it.

Careers at 24VitalCare
Team of scientists collaborating in a laboratory

Latest News

Announcements

June 30, 2026

24VitalCare Announces Completion of Enrollment of 162 Patients Into the Phase 3 ASCEND Study for the Treatment of Dravet Syndrome

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June 16, 2026

24VitalCare Announces Inducement Grants Under Nasdaq Listing Rule 5635(C)(4)

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May 28, 2026

24VitalCare to Present at Upcoming Investor Conferences in June

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