Our Work

The N=1 Collaborative is an international network of experts working together to bring individualized treatments to rare disease patients as safely and quickly as possible.

 

We are initially focused on the use of antisense oligonucleotides (ASOs), as they are rapidly customizable, cost efficient to manufacture, straightforward to administer, and have a growing safety and efficacy record. Through these efforts we intend to establish a standardized framework for individualized medicine that will extend to other customizable platform technologies such as siRNAs, RNA therapeutics, and CRISPR as well. 

 

We have formed workgroups focused on critical steps in the development of an individualized drug, with an emphasis on data sharing and establishing best practices. Topics include:

  

  • Patient selection criteria 

  • Drug design, validation, and safety assessment

  • Clinical trial design and outcome measures

  • Trial implementation within institutions

  • Ethics, equity and accessibility

What is N=1?

N=1 medicines are shorthand for a new class of customizable treatments targeting the underlying genetic defect in rare diseases — often a specific mutation. Individualized N=1 medicines inherently target a very small number of people, even as few as one.

 

Workshop Resources