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.

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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.

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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.

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Upcoming Seminars

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 An actively practicing cardiologist and committed teacher, Kiran Musunuru, M.D., Ph.D., M.P.H., M.L.,...

Resources & Articles

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

The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK

Daniel J. O’Connor , Parker Moss , Matthew Wood , Martin Murphy , Michael Parker , Nicola Blackwood , Matthew A. Brown , Deb Lancaster ,...

Publications

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

Anneliene H. Jonker, Elena-Alexandra Tataru, Holm Graessner, David Dimmock , Adam Jaffe, Gareth Baynam, James Davies, Shruti Mitkus,...

Publications

N=1 Collaborative

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

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

The Rare Therapies Launchpad: a pilot program for individualized medicines in the UK

The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

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