April 2023 -
Sharing Papers, Presentations and More
As the emerging field of individualized medicines grows, the N1C is committed to keeping you informed on the latest news. Our hope is that by sharing what we know today on the science, the process and the framing of this new paradigm, more academics, companies, organizations and patients will safely and effectively contribute to the growing ecosystem.
On top of launching our Seminar Series this year to discuss key topics, we also created a “News + Seminars” section of our website to include relevant publications, articles, podcasts and presentations from our members and beyond. We include recordings from our Seminar Series for those who missed them or want to share them with colleagues, papers such as Consensus Guidelines for Splice-Switching ASOs, and articles like the recent one in Nature - Personalized Medicine is Having its Day. Other key documents, such as FDA Guidances and Illustrated Examples of n=1 consent forms and protocols, are housed in our new Collaborative Resources page.
We welcome other papers, presentations or talks that we’ve missed or suggestions for additional resources you would find helpful. Just email Nicole Nolen at firstname.lastname@example.org.
We Need Your Help!
We are excited to announce our first sponsor, iXCells Biotechnologies! The N1C got up and running and now leads 5 fundamental workgroups, twenty seminars and an annual in-person meeting, all thanks to incredible volunteers across academics and industry, together with a lean staff of just two people.
We are now ready to grow our staff and build out our shared database.
We are turning to you to ask for help finding corporate sponsors to allow the N1C to grow as needed to continue serving as the hub for individualized medicines. Collaboration made milasen possible. Now, we need that same team effort to pave this path. If you or a company you know is interested in sponsoring our 501(c)(3), please reach out to Nicole Nolen at email@example.com!
Join us for our seminars coming up this month and next hosted on Mondays, 1-2pm US ET:
May 8 - “Allele-specific ASO treatment for SCN2A-encephaloapthy” - Heather Olson, MD, MS Learn More
May 22 - “Tailored RNA therapies for ultra-rare CNS diseases and mutations: a criteria framework for patient identification” - Matthis Synofizik, MD and Rebecca Schule, MD Learn More
Thank you to the hundreds of N1C members who attended our Seminar Series over the past few months! Below are overviews and recordings of the most recent talks. If you’re interested in presenting topics or (de-identified) cases, reach out to Stef Leonard at firstname.lastname@example.org
The Most Important Aspects of the Guidelines for N=1 Exon Skipping Design and in vitro Development
Approach to Drafting Regulatory Submissions and Challenges of Institutional Implementation
We are encouraged by the increasing participation in our Workgroups and Seminars and thank each of you for playing a critical role in the individualized medicines movement!
- N1C Team
We envision a future where individualized medicine centers around the world routinely offer patients customized treatments targeting their condition’s underlying genetic cause.