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Data Sharing & an Open Letter

February 27 - Scott Demarest MD MSCS, Clinical Director Precision Medicine, Children's Hospital Colorado, Associate Professor, Departments of Pediatrics and Neurology, University of Colorado School of Medicine

Jonathan Watts, Professor, RNA Therapeutics Institute, UMass Chan Medical School

Nina Green, senior advisor to BCHs Technology Innovation and Development Office

This talk will feature the N=1 Collaborative’s stance on data sharing. Scott Demarest, MD and Jon Watts, PhD will present a Data Sharing Pledge for Orphan Therapies - an open letter from the N1C that highlights the need for transparent data sharing to unlock the full potential of genetic therapy for orphan diseases. This presentation will describe the pledge and commitments made by the N1C and other members of the community who sign, as well as actionable next steps we can all take to help ensure we foster a community of learning that advances the field of individualized medicines.

Following this, the N=1 Collaborative Clinical Data and Safety workgroup will present their activities thus far in catalyzing the Data Sharing Pledge to action. Nina Green and Ashley Kuniholm will provide a brief overview of the Preclinical Data Sharing Agreement (DSA) that has been executed amongst six participating N1C institutions and their ongoing work to increase the number of DSA signatories as well as construct a pilot database of clinical trial data from current and previously treated N=1 patients.

Dr. Scott Demarest is an associate professor in the Department of Pediatrics, Division of Neurology. He is board certified in Child Neurology and Epilepsy. His clinical practice and research focus on the evaluation and treatment of neurogenetic conditions. This includes clinical trials for novel therapeutics, natural history studies and the development of improved outcome measures for neurogenetic conditions. He is the Clinical Director of Precision Medicine at Children's Hospital Colorado, co-director of the neurology complex drug program and medical director of the Batten and Neurogenetic Multi-disciplinary Clinics. He is also the director for the International CDKL5 Clinical Research Network.

Jonathan Watts is Professor at the RNA Therapeutics Institute of UMass Medical School (Worcester, MA). He has a PhD in chemistry from McGill University and did postdoctoral work in biochemistry and pharmacology at UT Southwestern Medical Center. Current work in the Watts lab is mainly focused on the optimization of oligonucleotide therapeutics for use in the brain and lung, and on advancing genome editing toward therapeutic use. The Watts group works on both platform technology and disease applications, and has contributed to the development of two drugs that have reached patients on a compassionate use basis.

Nina Green is an experienced technology transfer professional working chiefly in academic settings. She began her career at Harvard Medical School and in 2001 became the director of Tufts University’s licensing office. After leaving Tufts in 2014, Nina began consulting to Boston-area academic medical centers and research institutions, including Mass General Brigham, Beth Israel Deaconess, The Broad Institute of MIT and Harvard and Boston Children’s Hospital, where she currently serves as Senior Advisor to the Vice President, Technology Innovation and Development Office. Nina has significant experience negotiating research and license agreements, and has helped launch numerous research alliances between research institutions and biopharmaceutical companies. She has worked closely with venture capital investors and start-up companies to license early stage innovations, some of which have reached the clinic. Nina’s work with the N1C to enable data sharing among participants is consonant with her passion to help innovators find cures that will benefit patients.


We envision a future where individualized medicine centers around the world routinely offer patients customized treatments targeting their condition’s underlying genetic cause.



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