Founded in 2021 as a non-profit, the N=1 Collaborative is dedicated to advancing the development of individualized medicines for ultra-rare diseases through a global collaborative platform
for knowledge and data sharing.
The annual meeting will serve as a forum for dialogue, information transfer and building of collaborative relationships among industry, academia, health authorities and families.
The Annual Meeting is open to anyone interested or involved in rare disease and individualized medicine.
2025 Agenda at a Glance
Registration Fees
Registration will open Spring of 2025
Trainee scientist (graduate student & post doc)
$85
Academic / Non-Profit / Government Professional
$155
Industry Professional
$300
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Patient / Family Member
Complimentary (but must register)
Emerging Researcher Flash Talks
Flash Talk Presentations (5 min each) will feature patient-focused collaborative research by emerging research scientists. Presentations will be made in short presentation style with Q&A held for the end of session.
Call for Abstracts Opening Spring 2025
2024 Presentations:
(see button below to read abstracts)
Individualized Antisense Oligonucleotide Therapy for a Patient with Posterior Column Ataxia with Retinitis Pigmentosa (PCARP)
Boxun Zhao, Postdoctoral Research Fellow, Manton Center for Orphan Disease Research
Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related
Zellweger Spectrum Disorder (ZSD)
Robert Thompson, Genetics and gene therapy Fellow, Boston Children's Hospital/MGH
Antisense oligonucleotides targeting linked-SNPs provide allele-specific knockdown to a dominant-negative SPTAN1 pathogenic variant in a complex genetic region
Christiana Wang, PhD Student. Baylor College of Medicine
N-of-1 for N-of-Many: comprehensive, scalable development of patient-customized splice modulation ASOs for Ataxia Telangiectasia
Clemens Lochmann, M.Sc., PhD Student, Hertie-Institute for Clinical Brain Research
Individualised Exon Skipping Antisense Oligonucleotide Therapy for CHD2-Related Neurodevelopmental Disorders Jack Morgan, PhD Student Dutch Center for RNA Therapeutics | Leiden University Medical Center
First In Class ASO Targeting IGHMBP2 Cryptic Splice Variant: Efficacy and Safety
Caroline Johnson, Clinical, Vanda Pharmaceuticals Inc.
N1C Partners & Event Sponsors
Organizing Committee
