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Resource Navigator
Workgroup Generated Content
Articles & Publications
Workshops
At N1C, we are dedicated to streamlining the development of individualized therapies.
The N1C Resource Navigator guides the community through the end-to-end individualized medicine development process. By leveraging global expertise to create shared resources, we support researchers in advancing individualized medicines safely, rapidly, and accessibly.
If you'd like to contribute or would like to suggest resources, please contact Stefanie@n1collaborative.org
Patient Identification
Preclinical Design
Manufacturing
Institutional Implementation
Clinical Outcomes
Data Coordination
Access & Funding
Seminars & Podcasts
N1C VARIANT Workshop
December 10, 2024 at 10:30 am US EST N1C has recently released the first version of an international consensus guideline for the...
Connecting, Collaborating, Communicating, Challenges: Sharing experiences navigating family conversations in an n-of-1 investigational treatment setting
Monday, March 4, 2024 12:30 pm US EST From an initial email request or conversation at a meeting… to (for some) the possibility of...
Selecting the right candidate: Which disease-causing variants are eligible for N=1 ASOs?
September 18, 2023 12:30-1:30 pm US EST - Individualized genetic treatments provide new opportunities for targeted therapies for rare...
Tailored RNA therapies for ultrarare CNS diseases & mutations: a criteria framework for patient ID
Tailored n-of-1 RNA treatment approaches targeting ultrarare or even private variants have opened up a qualitatively novel realm of...
Workgroup Generated Content
Publications
Workshops
Seminars & Podcasts
Articles & Publications
Fast Track to Final Drug Product: Understanding Costs, Challenges, and Timelines in Personalized Medicine Manufacturing
May 6, 2024 at 12:30 pm US EDT In this webinar, speakers from Berkshire Sterile Manufacturing (BSM) and Argonaut Manufacturing Services...
"Control of starting material quality for oligonucleotide therapeutics" and "To Synthesis and Beyond…Production of oligos for ‘N-of-1’ therapies"
Monday, March 25 2024 12:30 pm US EDT This presentation focuses on the control of starting material (SM) quality in the manufacture of...
CMC Recommendations Draft Guidance
Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...
Workgroup Generated Content
Seminars & Podcasts
Workgroup Generated Content
SNP-selective siRNA for the treatment of Fibrodysplasia Ossificans Progressiva
December 2, 2024 at 12:30 pm US EST Fibrodysplasia Ossificans Progressiva (FOP) is a devastating, ultra-rare condition, where patients...
Preclinical Development for Personalized Therapy Using Disease-Relevant Cell Models
Monday, July 15 2024, 12:30 pm US EDT iXCells Biotechnologies is specialized in providing innovative pre-clinical drug discovery...
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
February 13 - Annemieke Aartsma-Rus, Dutch Center for RNA Therapeutics - “The most important aspects of the guidelines for N=1 exon...
Models for Sustainable Discovery
January, 30 - Jeff Milton, CEO of La Jolla Labs - “A Model for Sustainable Discovery in Rare Disease” Millions of patients world-wide...
Positive and negative controls for ssASOs and Gapmers that can be used for in vitro tox studies
Click below for download: Please feel free to use the ASOs on this list for the toxicity studies in your lab. If you have any ASOs that...
N1C Preclinical Workgroup: Preclinical Modeling Systems
Click below for download
N1C Preclinical Workgroup: Ordering ASO Checklist
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Articles & Publications
A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides
Daniel O'Reilly, Willeke van Roon-Mom, Annemieke Aartsma-Rus; N = 1 Collaborative ( Nucleic Acid Therapeutics ) August 7, 2024...
Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing
Sara Aguti, Shuzhi Cheng, Pierpaolo Ala, Sean Briggs, Francesco Muntoni, Haiyan Zhou ( Molecular Therapy Nucleic Acids ) June 4, 2024...
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
February 13 - Annemieke Aartsma-Rus, Dutch Center for RNA Therapeutics - “The most important aspects of the guidelines for N=1 exon...
Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N=1 Exon Skipping ASOs
Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic opportunities for numerous genetic diseases,...
Nonclinical Testing Draft Guidance
Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...
OTS Rare Disease N-of-1+ Briefing Document, Jan 2020
This briefing document aims to outline the current state of the art of oligonucleotide therapies for those planning to develop...
Workshops
Seminars & Podcasts
Regulatory Pathway: Where We Are Today
Monday, October 16 at 12:30 pm US EDT This session will start with an overview of the contents of an N=1 IND application process from...
Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation
April 24, 2023 12:30-1:30 PM US EST The N=1 Collaborative Institutional Implementation workgroup will present their activities thus far...
Workgroup Generated Content
Illustrated Example for n=1 Clinical Protocol
Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Illustrated Example for n=1 Consent Form
Click below for download: Disclaimer: This document is the work product of the N=1 Collaborative (the “N1C”), and is offered as an...
Administrative and Procedural Draft Guidance
IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for...
Articles & Publications
Clinical Recommendations Draft Guidance
IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
Workshops
Institutional Implementation Workshop
Workgroup Presentation (Institutional implementation)
Seminars & Podcasts
Project Butterfly: from drug development to clinical trial. A personalized antisense oligonucleotide medicine for a rare form of CLN3 Batten Disease
January 13, 2025 The presentation will review our development of a personalized antisense oligonucleotide (ASO) medicine for two children...
Treating the Nano-Rare: Redefining Clinical Management, One Patient at a Time
November 18, 2024 at 12:30 pm US EST This presentation will cover the progress made in the clinical management and treatment of...
Rapid development of an n-of-few gene therapy for SPG50 through duantless collaboration
June 17, 2024 at 12:30 pm US EDT Steven Gray and Terry Pirovolakis will share the story of the development of a gene therapy treatment...
Stopping the clock on KIF1A associated neurological disorder
September 16, 2024, 12:30 pm US EDT KIF1A associated neurological disorder is a rare neurodegenerative neurological disorder associated...
PRAX-222 Therapy for a Child: Prenatal Onset SCN2A Developmental and Epileptic Encephalopathy in a Named Patient Setting
Monday, April 22 2024 12:30 pm US EDT Early onset SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is caused by...
Potential Treatment: CMT2S Caused By IGHMBP2 Cryptic Splice Site Variant With ASO Based Therapeutic
October 2, 2023 12:30-1:30 pm US EDT VCA-894A is a novel antisense oligonucleotide (ASO)
Articles & Publications
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases
Olivia Kim-McManus, Joseph G. Gleeson, Laurence Mignon, Amena Smith Fine, Winston Yan, Nicole Nolen, Scott Demarest, Elizabeth...
N-of-1 Studies in an Era of Precision Medicine
Erika Augustine, Timothy Yu, Richard Finkel ( JAMA Network ) September 25, 2024 Genetically individualized N-of-1 trials have recently...
A Multistakeholder Perspective on Advancing Individualized Therapeutics
Michael Pacanowski, Julia Vitarello, Insoo Hyun, Timothy Yu, Issam Zineh August 24, 2023 Precision medicine has evolved from the...
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Jinkuk Kim, Ph.D., Chunguang Hu, M.D., Ph.D., Christelle Moufawad El Achkar, M.D., Lauren E. Black, Ph.D., Julie Douville, Ph.D., Austin...
Year End Review and Outcome Measures Workshop
Year-end Recap: Mission/Vision/Values, Workgroup Updates, Clinical Outcomes
Workshop
Seminars & Podcasts
Data Sharing & an Open Letter
February 27 - Scott Demarest MD MSCS, Clinical Director Precision Medicine, Children's Hospital Colorado, Associate Professor,...
Seminars & Podcasts
Treating the Nano-Rare: Redefining Clinical Management, One Patient at a Time
November 18, 2024 at 12:30 pm US EST This presentation will cover the progress made in the clinical management and treatment of...
The challenges and opportunities in rare disease
November 4, 2024 at 4:00 pm US EST Four hundred million people world wide have a rare disease and yet still less than 5% of patients have...
Transforming Patient Access to High-Cost, One-Time Treatments through Financial Engineering Innovation
August 26, 2024 at 12:30 pm US EDT Self-insured employers collectively cover over 120 million lives in the United States. Many of these...
Stopping the clock on KIF1A associated neurological disorder
September 16, 2024, 12:30 pm US EDT KIF1A associated neurological disorder is a rare neurodegenerative neurological disorder associated...
Regulatory: The Future Pathway to Access
Monday, November 6, 2023 at 12:30 pm US EDT - Join us for an informative and engaging webinar where we delve into the complex regulatory...
Regulatory Pathway: Where We Are Today
Monday, October 16 at 12:30 pm US EDT This session will start with an overview of the contents of an N=1 IND application process from...
Articles & Publications
Personalized medicine for rare neurogenetic disorders: can we make it happen?
Agnies M van Eeghen , Hilgo Bruining , Nicole I Wolf , Arthur A Bergen , Riekelt H Houtkooper , Mieke M van Haelst , Clara D van...
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics
Annemieke Aartsma-Rus, Rob W.J. Collin, Ype Elgersma, Marlen C. Lauffer, Willeke van Roon-Mom ( Therapeutic Advances in Rare Disease )...
Employers Don't Want to Pay for Life-Saving Cures for Rare Diseases
Gerry Smith ( Bloomberg ) September 5, 2024 As a wave of gene therapies with multimillion-dollar price tags hit the market, many...
How to pay for individualized genetic medicines
Julia M. Y. Pian, Nana Owusu, Julia Vitarello, Winston X. Yan, Andrew W. Lo & Timothy W. Yu (Nature Medicine) June 19, 2024 For precision...
A Multistakeholder Perspective on Advancing Individualized Therapeutics
Michael Pacanowski, Julia Vitarello, Insoo Hyun, Timothy Yu, Issam Zineh August 24, 2023 Precision medicine has evolved from the...
‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
In 2014, the National Institutes of Health posted an estimate that at the current pace of drug development and approval, it would take...
Clinical Recommendations Draft Guidance
IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases:...
CMC Recommendations Draft Guidance
Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating...
Nonclinical Testing Draft Guidance
Nonclinical Testing of Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases...
Administrative and Procedural Draft Guidance
IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for...
Regulatory Guidances
Community Presentations
Discovery Forum 2022 | Julia Vitarello & Timothy Yu | From Mila to millions
We envision a future where individualized medicine centers around the world routinely offer patients customized treatments targeting...
Discovery Forum 2022
From Mila to millions: opportunities for individualised medicines
Julia Vitarello and Timothy Yu
RARE-X Exchange - The Future is Now: Realizing the Possibilities of Patient Driven Research
Wendy Erler - Alexion AstraZeneca, RARE-X Board Member, Betsy Bogard - FOP Advocate, Ensoma, RARE-X Board Member, and Julia Vitarello -...
Bold Predictions for Human Genomics by 2030: Session 10
NHGRI hosts a seminar series on the “Bold Predictions for Human Genomics by 2030” that are described in NHGRI’s “Strategic Vision for...
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