Our Work
The N=1 Collaborative is an international network of experts working together to bring individualized treatments to rare disease patients. We are initially focused on the use of antisense oligonucleotides (ASOs) as they are rapidly customizable. Through our efforts with ASOs, we are establishing a standardized framework for individualized medicine that will extend to other customizable platform technologies such as siRNAs, RNA therapeutics, and CRISPR as well.
Multidisciplinary collaboration was vital to making the earliest individualized medicines – milasen, jacifusen, and afinersen – possible. This collaborative ethos continues to be necessary in order to learn and improve.
Case Conferences & Seminar Series
The N1C Seminar Series will host leading experts in the field to advance important conversations in the personalized medicine space. We feature speakers from academia, industry, and people directly affected by rare disease. The format varies from research talks, to case conferences, to panel discussions. The talks are always open to the entire N1C community, with time allotted for Q&A. If you have ideas or requests for additional topics, please reach out to Erin McConnell at info@n1collaborative.org.
See our Upcoming and Past series here.
Workgroups
Special focus workgroups have been formed to address important topics critical to the development of individualized medicines. The current workgroups are listed below, with more to come.
Patient Identification:
We are developing and disseminating guidance on identifying patients that may be amenable to experimental individualized ASO intervention.
View Collaborative Resources or watch the Workshop Recording
Preclinical Design:
We are promoting standards and advancing the science of ASO design and testing.
View Collaborative Resources or watch the Workshop Recording
Data Coordination:
We are establishing databases to make sharing of preclinical and clinical learnings across individualized ASO treatments more efficient. By reducing the barriers to this type of sharing, we can learn and improve.
Clinical Outcome Measures:
We are developing unified approaches to capturing clinical outcome measures and safety signals.
Watch the Workshop Recording
Institutional Implementation:
We are sharing experiences and creating standardized templates and resources helpful to academic investigators and medical centers running individualized clinical trials.
View Collaborative Resources or watch the Workshop Recording
Manufacturing:
We are establishing shared resources, strategies, and standards dedicated to the Chemistry, Manufacturing, and Controls (CMC) aspects of consistently developing individualized medicines. Our aim is to streamline and share processes that ensure the quality of these therapeutics throughout the entire development cycle.
Access:
We are identifying accessible, sustainable, and equitable funding models for research and implementation of individualized genetic medicines. We bring together thought leaders and key stakeholders to rethink the economics of the drug development process.