The Future of Medicine is Here
Imagine a world where everyone could access individualized precision therapeutics that could slow or even halt their disease and improve quality of life. At EveryONE Medicines, that is the goal we are working tenaciously toward.
Our story begins with Mila. After a happy and seemingly healthy first few years of childhood, Mila was diagnosed with a rare fatal neurodegenerative disease. In a ground-breaking race against time, Dr. Timothy Yu of Boston Children’s Hospital developed milasen, an antisense oligonucleotide targeting Mila’s unique mutation. Mila became the first person in the world to receive a medicine tailored to just one person. Since then, Mila’s mother, Julia Vitarello, has been on a mission to turn the work for Mila into a new mainstream way of solving the global crisis of rare disease in children. Julia co-founded EveryONE Medicines with the hope of creating a viable business model to bring this approach to all patients who could benefit. Together, we are working toward a future where individualized medicines are routine.
- Mila's Miracle Foundation
- Mila to More short documentary
- Milasen: The drug that went from idea to injection in 10 months
- Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
- Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives
- The quest for the era of personalised medicine
Design, develop, and manufacture individualized antisense oligonucleotide medicines on a global scale to treat life-threatening neurodegenerative diseases in children that currently have no treatment options and secure a new regulatory pathway for our individualized medicines that is sustainable with broad reimbursement from health ministries, insurance plans and health systems.
Individualized Medicines Is a Reality
We have the technology to identify the most unique genetic conditions occurring in children and design medicines to treat them–giving patients and their families a chance at a longer, healthier life.
Generating clinical evidence for individualized medicines presents a paradox because there may only be a few children across the world with a particular genetic variant which is responsible for causing their neurodegenerative disease. With this in mind, we are focused on generating clinical data in a pivotal trial that will include multiple medications treating multiple genetic variants. This is not the traditional pathway of evidence generation for regulatory approval because the traditional approach is not a viable option in a patient population this rare. Therefore, we are working closely with regulatory agencies across Europe to advance our clinical trial strategy and establish a new pathway for marketing authorization of our individualized medicines at scale.
To that end, EveryONE Medicines is honored to be serving as a pilot for the UK’s Rare Therapies Launch Pad (RTLP) working alongside multiple stakeholders, including the MHRA. Their mission is to model a scalable and sustainable system to meet the new era of individualized medicines, focused on putting in place an innovative regulatory and reimbursement end-to-end pathway to allow for rapid access to cutting-edge science by those who could benefit.
