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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CMC Recommendations Draft Guidance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
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N1C Preclinical Workgroup: Ordering ASO Checklist
Click below for download
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)
