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

8:00 – 9:00 | Registration & Networking Coffee

Registration & Networking Coffee
9:00 – 9:10 | Welcome & Opening Remarks

N1C Team Welcome
9:15 – 10:00 | Parent Perspective Panel

Participants to be announced
10:00 – 10:45 | Keynote Presentation

Keynote Presentation: To be announced
10:45 – 11:15 | Networking Break

Networking Break
11:15 – 12:15 | Community Updates

Get to know the community! Updates will be shared by organizations in the ecosystem of individualized medicine. Please come prepared with questions. 2025 Presenters to be announced.
12:15 – 1:30 | Networking Lunch

Meet up with colleagues old and new and get to know each other, connect with the N1C Team and workgroup members!
1:30 – 2:00 | Emerging Researcher Talks

"Flashtalk Presentations (5 min each) will feature patient-focused collaborative research by emerging trainee scientists. These talks will be selected from abstract submissions. Call for Abstracts to open Spring 2025
2:00 – 4:00 | Best Practices Talks

This Best Practices session will explore current strategies and approaches used by scientists and clinicians navigating the N-of-1 and N-of-few therapeutics space. 2025 Speakers to be announced
4:00 - 4:30 | Refreshment Break

Refreshment Break
4:30 – 5:15 | Strategy Presentations

In the N1C Meeting Strategy Session, we'll engage with rare disease thought leaders to brainstorm how to scale N=1 therapeutics for more families, emphasizing collaboration and a patient-centered approach. The session will cover various disciplines, including genetic testing, scalable models, and multiple modalities to address all phases of the individualized medicines pipeline. Presenters to be announced
5:15 – 6:15 | Strategy Panel Discussion

Panel Discussion will include Q&A participation from the delegates.
6:10 – 6:15 | Closing Remarks

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6:15 – 8:00 | Networking Reception

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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.