N=1 Collaborative

Today, individualized medicines are possible.

We are on a mission to make them safe and rapidly accessible to patients worldwide.

Upcoming Seminars

Potential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic

October 2, 2023 12:30-1:30 pm US EDT VCA-894A is a novel antisense oligonucleotide (ASO)

Our Story

What started as a race against time for one little girl has turned into a movement that could help solve the global health crisis of rare genetic diseases.

LEARN MORE

Our Work

We are advancing critical steps in the development of individualized medicines, keeping the sharing of knowledge, data, safety and experience at the core.

LEARN MORE

Who We Are

We are a group of internationally acclaimed doctors, researchers, patients and companies from across the globe who are committed to expanding the field of individualized medicine.

LEARN MORE

Resources & Articles

Publications

A framework for individualized splice-switching oligonucleotide therapy

July 12, 2023 Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of individuals with genetic diseases1,...

Articles & Podcasts

A young girl’s custom gene therapy hints at a framework for tailored rare disease treatments

Bree Iskandar (STAT) July 12, 2023 When Timothy Yu developed milasen, a custom drug for a young girl named Mila with Batten disease, he...

Articles & Podcasts

They Created a Drug for Susannah. What About Millions of Other Patients?

By Erika Check Hayden (New York Times) Photographs by Brittainy Newman Scientists have made rapid progress in customizing drugs for...

N=1 Collaborative

Join Our List

Contact Us