N1 Collaborative

Join us at the next N=1 Collaborative seminar led by Marlen Lauffer to discuss recommendations for evaluation of disease causing variants. Register: bit.ly/3Lq6J3I

Individualized medicine provides new opportunities for targeted therapies for rare disease patients, but not all individuals affected by rare disease are eligible for tailored N=1 approaches.

Our N=1 Collaborative seminars are open to everyone and we encourage questions and discussion!

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

The event is open to all, please join us: bit.ly/3KSMpYs

@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.

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.

Our N=1 Collaborative seminars are open to everyone and we encourage questions and discussion! Join Live: bit.ly/3rBemxh

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 us live on July 17, 2023 at 12:30 pm US ET: bit.ly/3DcKwSb

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.

Joining this discussion will be a panel of innovators from Integrated DNA Technologies, Cytiva, and La Jolla Labs Inc. Join Live: bit.ly/46wLhTT

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.

The talk will include novel approaches like the Multi-Domain Responder Index, as well as how to incorporate biomarkers. Join Live: bit.ly/3ITqcJ3 @stopbatten @winstonxyan @timyu

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.

Join us on Monday, May 22, at 12:30pm US EDT, for the next @N1Collaborative seminar to discuss this critical topic with Dr. Matthis Synofzik and Dr. Rebecca Schüle from University of Tuebingen Join live: bit.ly/3BG7j8u

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

Join us today, May 3, at 2pm US EDT, for an @N1Collaborative seminar. Ali Fatemi, MD, MBA of Kennedy Krieger Inst. & Beth McGinn of CureLBSL highlight work toward an N-of-small ASO treatment for #LBSL, an ultrarare neurodegenerative disorder. JOIN LIVE: bit.ly/3oZRxSB

We welcome and encourage your thoughts in the discussion that will follow. Mark your calendars! JOIN LIVE this Monday - bit.ly/41sgTa0

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.

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.