Individualized Medicines, Accessible at Scale
We’re pioneering a scalable solution to connect children with life-threatening neurodegenerative disease to individualized therapies that target their specific genetic variant.
different rare diseases globally
of ultra-rare diseases have a genetic cause
present in childhood
lack approved treatments
children impacted
We're creating a future where individualized treatment is not just possible—it's scaled, sustainable and accessible.
We’re building a system solution to address every part of the patient treatment journey, from drug development to regulatory approval, reimbursement and connectivity to a library of ASO solutions which address the rarest of neurodegenerative diseases impacting children globally.
Creating a Treatment Library
We are focused on antisense oligonucleotides: ASOs
ASOs are designed to specifically target and modify RNA transcripts to slow down or halt rare genetic disease progression.
Our proprietary AI-driven design and development process allows us to create individualized medicines to correct unique genetic variants causing devastating diseases. With each new therapy we develop, our ASO Library will grow. We're creating a future where ASO-amenable genetic variants affecting children with neurodegenerative diseases can be instantly matched to an ASO we have developed in our Library of medicines. We can also design and develop a custom therapy for newly discovered genetic mutations which have amenability to antisense oligonucleotide treatment.
Pioneering a System Solution
Regulatory agencies are recognizing the need to create new pathways for approval of medicines treating unique, rare conditions. Especially ultra-rare diseases where the traditional preclinical studies and clinical trial models are not possible in a traditional setting. Unfortunately, children with life-threatening neurodegenerative disease do not always have time to wait for the lengthy clinical trial process to be completed.
A new pathway to regulatory approval needs to be created. EveryONE Medicines will be the changemaker leading the catalyzation of a scalable and sustainable solution by working early and often with regulatory agencies, payers, treatment centers, diagnostic firms and patient advocates to build this new pathway.
A System Designed for Speed
Time is the enemy for children suffering from neurodegenerative disease. Every day counts, and progression of disease can limit any benefit an ASO treatment can provide.
By connecting treatment centers and diagnostic databases with our Library of ASO medicines, we can speed up the time to treatment. With AI-enabled sequencing and design, we believe it’s possible to develop a new ASO in less than 12 months safely and efficiently.
The Story Behind EveryONE Medicines
Inspired by Mila’s journey, we are committed to developing medicines to give as many children the chance at life that she had.