N=1 Collaborative

Today, individualized medicines are possible.

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

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

Upcoming Seminars

Splice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa

Monday, June 23, 2025 at 12:30 pm US EDT Transposon insertions represent a significant yet understudied source of disease-causing...

Preclinical development of personalized ASO therapeutics using patient-derived organoid systems

October 6, 2025 at 12:30 pm US EDT This presentation will cover our laboratory's ongoing efforts in the areas of personalized and...

Towards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders

June 2, 2025 at 12:30 pm US EDT Featured in the News: A recent Endpoints News article spotlighted the groundbreaking work of Kiran...

Resources & Articles

'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

Ryan Cross ( Endpoint News ) May 8, 2025 Fyodor Urnov had given a version of his speech dozens of times before. Yet as he leaned into the...

Articles & Podcasts

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

Jillian Belgrad , Erin McConnell , Stef Leonard , Nicole Nolen , Marlen C Lauffer , Jonathan K Watts , Timothy Yu , Winston X Yan ,...

Publications

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments

David Cheerie , Margaret M. Meserve , Danique Beijer , Charu Kaiwar , Logan Newton , Ana Lisa Taylor Tavares , Aubrie Soucy Verran ,...

Publications

N=1 Collaborative

Splice-Altering Transposon Insertions in Neurodegenerative Diseases and Its Application in an Individualized ASO Therapy for Retinitis Pigmentosa

Preclinical development of personalized ASO therapeutics using patient-derived organoid systems

Towards a platform for in vivo corrective editing of ultra-rare and N-of-1 metabolic disorders

'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments

Join Our Community List

Contact Us