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
Trainee scientist (graduate student & post doc)
$120
Academic / Non-Profit / Government Professional
$180
Industry Professional
$295
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Patient / Family Member
Complimentary (but must register)
Emerging Researcher Flash Talks
N1C seeks to encourage and support trainees working in rare disease therapeutics through our Annual Meeting Emerging Researcher Flash Talks. These flash talks recognize the accomplishments and potential of emerging scientists dedicated to advancing rare disease research through collaboration.
Trainees are invited to submit abstracts to info@n1collaborative.org by August 1, 2025, for evaluation by the N1C team and will be evaluated based on their patient focus and collaboration. In your email, please identify if you would like to be considered for the Flash Talks or if you prefer a poster presentation.
Any abstract submitted for a flash talk will be automatically considered for a poster presentation if not selected for the Emerging Researchers Session. Flash talk invitations and poster presentation decisions will be sent via email by September 2, 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.
New Voices
We’re excited to introduce a new segment in our agenda, New Voices, which invites community members involved in rare disease initiatives to share their projects.
Would you be interested in presenting on behalf of your organization? If so, please provide your organization’s name and a brief, three-sentence summary of your topic or content to info@n1collaborative.org by August 1, 2025. Selected presenters will be notified via email by September 2, 2025.
Community Posters
We are thrilled to welcome poster presentations from any community, advocacy, or research team to this year’s N1C Annual Meeting. Please email your poster abstract for the Community Poster Session to info@n1collaborative.org by August 1, 2025. Confirmation of poster acceptance will be emailed by September 2, 2025.
N1C Partners & Event Sponsors
Organizing Committee
