Join us for a case study on Monday, September 11, at 12:30 pm US EDT to hear about ASO treatment for SCN2A-encephalopathy, allele-specific vs non-specific, with Heather Olson and Elizabeth Berry-Kravis. Register here: bit.ly/487WDhC

For N-of-1/few treatments in rare genetic disease, while antisense oligos may be the initial therapeutic platform of choice, CRISPR holds huge promise for the future.

@UrnovFyodor and Manar Zaghlula from the Innovative Genomics Institute will join the @N1Collaborative Seminar Series on Monday 8/28 at 12:30-1:30p EST to share a framework and recommendations for enabling affordable, accessible bespoke CRISPR treatments.

Join us for a case study on Monday, July 31, at 12:30 pm US EDT to hear about a novel antisense oligonucleotide for CMT2S, a rare subtype of Charcot-Marie-Tooth Disease, with Sandra Smieszek of Vanda Pharmaceuticals.

Join Andrew Lo, @MIT Sloan Professor of Finance, this Monday for a dynamic seminar hosted by the @N1Collaborative. We will discuss the portfolio model in rare disease therapeutics, the role of philanthropy, and reimbursement considerations in the individualized medicine context.

What if coding personalized ASO therapies only required the press of a button? Join us Monday, July 10, at 12:30pm US EDT, and Vanessa Almendro of @DanaherCorp to discuss the paradigm shift needed to make therapeutic interventions for rare genetic diseases accessible to patients.

Join Emil Kakkis, CEO of Ultragenyx & Richard Finkel, MD, Director, Center for Experimental Neurotherapeutics, St. Jude Children's Hospital, at the @N1Collaborative seminar Monday, June 5 at 12:30 PM EDT to discuss how to think about outcome measures for individualized medicines.

Identifying patients who could benefit from individualized ASOs requires consideration of the underlying variant, the disease and the patient.

The @N1Collaborative invites interested investigators to join our Seminar Series on Individualized Medicines this Monday, April 24th, 12:30-1:30pm US ET to discuss Regulatory Submissions and Institutional Implementation.

Margot Cousin from @MayoClinic, Roger Paxton from @ChildrensColo and Catherine Douthwright from @UMassChan will present several illustrated examples including protocols, informed consent, and pre-IND & IND applications.