N=1 Collaborative

The challenges of developing individualized medicines aren’t just scientific—N-of-1 clinical trials also come with complex institutional and logistical hurdles. Join the N=1 Collaborative Institutional Implementation Work Group’s Open Forum, a space for those with experience or interest in N-of-1 trials to engage in candid discussions, identify challenges, and share strategies for advancing individualized treatments safely, efficiently, and equitably. Have specific questions you’d like addressed? Send them in advance to [email protected]. We hope you’ll be part of the conversation: https://wix.to/zyUMZ6A

Thank you Jeff Milton for your kind words and for recognizing the importance of collaboration in individualized medicine. It was an honor to host this discussion on scaling n-of-1 therapies with you alongside Steven Ringel, Scott Demarest, Marlen Lauffer, and Annemieke Aartsma-Rus—a group truly dedicated to turning vision into action. Excited to share this conversation with our community! https://lnkd.in/eu36XSFy

Join us in welcoming Robert Thompson, Claudia Lentucci, and Didem Demirbas, Ph.D. to the next N=1 Collaborative seminar highlighting Personalized Splice-modulating Antisense Oligonucleotide Therapy for PEX1-related Zellweger Spectrum Disorder. You're invited to an N1C seminar https://wix.to/wqvMRDS

Progress in rare disease isn’t just about innovation—it’s about collaboration. We’re grateful for Charles River Laboratories, and all of our additional Donated Resource Center contributors, as well as the broader N1C community for sharing their resources, knowledge, and time to help ultra-rare patients.

Save the Date for the 2025 N=1 Collaborative Annual Meeting! This year in Budapest, Hungary, the N1C Annual Meeting:?Individual Journeys, Shared Discoveries,?will unite academic and industry researchers, patients, families, regulators, and community partners to discuss challenges, share insights, and advance solutions for n-of-few therapeutic development and patient access. Stay tuned—event registration opens in April 2025!

Thank you for hosting us on Sounds of Science! It was a pleasure for N=1 Collaborative Executive Director, Hugh Hempel, to share his vision for the future of individualized medicine and the mission of the N1C. It’s always a privilege to collaborate with Charles River Laboratories in advancing innovation for rare disease patients.

Join us in welcoming Christoph Grünig to the next N=1 Collaborative seminar. This presentation will review the traditional analytical life cycle for quality control of drugs with a discussion of the possibilities and challenges of adapting it to N-of-1-treatments. You're invited to an N1C seminar https://wix.to/Md7HE91

The session will begin with a presentation from our guest speaker, Jason Gill, providing an overview of the science and key lessons from bacteriophage development. In the second half, we’ll highlight parallels between bacteriophages and other platform-based approaches, shifting to a collaborative discussion on the challenges and opportunities in regulatory pathways, scalability, and the future of platform-based individualized medicines. Whether you’re a developer of ASOs, bacteriophages, or other innovative individualized modalities, this seminar offers a chance to share your perspective, learn from others, and contribute to shaping the future of these complex therapeutic platforms. You're invited to an N=1 Collaborative seminar https://wix.to/LlUtWpP

As we welcome 2025, we’re excited to recognize?ADViRNA?for joining The N=1 Collaborative Donated Resource Center! ADViRNA is contributing?scientific advisory consultations and Modified Oligonucleotide Design Services?for up to 2 unique transcripts.?N1C members also gain access to exclusive discounts on additional services. ADViRNA joins a growing community of companies—including Argonaut Manufacturing Services Inc., Hongene Biotech Corporation, 3billion, Charles River Laboratories, Jeeva Clinical Trials Inc., ChemGenes, and Microsynth AG—united in the mission to advance individualized treatments. This collaborative model connects academia, industry, and patients, paving the way for broader access to life-changing therapies. Looking to make an impact in 2025? Contact Nicole Nolen to learn how your company can contribute to the Donated Resource Center. Developing an individualized therapy? Explore the Donated Resource Center and apply today! https://lnkd.in/ew-Y3K_Z