Committees
Every patient deserves expert advice about treatment. At n-Lorem, we have a number of committees that provide expert advice across all key steps.
Access to Treatment
Committee (ATTC)
The Access to Treatment Committee is made up of pharmaceutical executives, physicians, academic professors, patient advocates, and a bioethicist. This committee will review applications from physicians or organizations, like the UDN, to treat diagnosed, genetically confirmed nano-rare disease patients. Applications will be approved and prioritized based on criteria such as: feasibility of developing an ASO treatment for the genetic cause of the disease, degree of potential benefit of treatment, practicality of treatment, availability of physician and institution to treat patient and conduct necessary studies, and other complexities of the condition.
At the Foundation we understand that there are seriously ill patients with immediate needs of treatment for their condition. We are sympathetic to patients and all who are involved. The Committee will fully review all applications received before a decision is made. We plan to do all we can to make sure we are as transparent as possible. Due to the complex nature of treating patients with nano-rare diseases, unfortunately not all can be approved for treatment.
n-Lorem Representatives:
Frank Bennett, Ph.D.
Co-Chairman – Executive Vice President, Chief Scientific Officer of Ionis Pharmaceuticals
Julie Douville, Ph.D.
Vice President, ASO Discovery and Development
Sarah Glass, Ph.D.
Chief Operating Officer
Joseph G. Gleeson, M.D.
Chief Medical Officer at n-Lorem and Rady Professor, Neurosciences and Pediatrics, University of California, San Diego
Co-Chair
Megan Knutsen, M.S.
Director, Clinical Operations and Project Management
Laurence Mignon, Ph.D.
Executive Director, Clinical Development
Co-Chair
Konstantina Skourti-Stathaki, Ph.D.
Senior Director, Discovery and Research
Amy Williford, Ph.D.
Executive Director, Communications and Donor Relations
Wei Zhang, Ph.D.
Assistant Director of Research
External Experts
Allison Bateman-House, M.D., Ph.D.
Assistant Professor of Population Health at NYU Grossman School of Medicine
Alan Beggs, Ph.D.
Professor of Pediatrics at Boston Children’s Hospital
Lauren Black, Ph.D.
Distinguished Scientist at Charles River Laboratories
Shyamanga Borooah, M.B.B.S., Ph.D.
Assistant Clinical Professor of Ophthalmology at the Shiley Eye Institute at UC San Diego Health
Jeff Carroll, Ph.D.
Associate Professor in the Department of Neurology at the University of Washington
Darryl De Vivo, M.D.
Sidney Carter Professor of Neurology, Professor of Pediatrics, and Director Emeritus (1979-2000) of the Child Neurology Service at Columbia University Irving Medical Center, New York City
Toby Ferguson, M.D., Ph.D.
Chief Medical Officer, Voyager Therapeutics
Richard Finkel, M.D.
Director, Experimental Neuroscience Program, and Endowed Chair in Neurotherapeutics at St. Jude Children’s Research Hospital
Kenneth ‘Kurt’ Fischbeck, M.D.
NIH Distinguished Investigator, Neurogenetics Branch
Jeffrey Noebels, M.D., Ph.D.
Cullen Chair in Neurogenetics and Professor of Neurology, Neuroscience, and Molecular and Human Genetics at Baylor College of Medicine
Alfred Sandrock, Jr, M.D., Ph.D.
Executive Vice President, Research & Development and Chief Medical Officer at Biogen
Francis Sessions Cole, III, M.D.
Professor of Pediatrics at Washington University School of Medicine in St. Louis
Neil Shneider, M.D., Ph.D.
Claire Tow Associate Professor of Motor Neuron Disorders and Director of the Eleanor and Lou Gehrig ALS Center at Columbia University
STAR Committee (Study Treatment and Assessment Review)
The STAR Committee (Study Treatment and Assessment Review) is composed of experts in clinical trial design, outcome measures, and quantitative assessments, representing medical disciplines covering the range of clinical presentations and endpoints that n-Lorem hopes to achieve with individualized ASO medicines. Committee membership is by invitation, and may include ad hoc guests who can bring unique perspectives. The STAR committee provides n-Lorem and the patient’s physicians guidance on the development of treatment goals and clinical outcome assessments for our diverse patient populations.
n-Lorem Representatives
Sarah Glass, Ph.D.
Chief Operating Officer
Joseph G. Gleeson, M.D.
Chief Medical Officer
Chair
Laurence Mignon, Ph.D.
Executive Director, Clinical Development
External Physician and Patient Subject Matter Experts
Elizabeth M. Berry Kravis, M.D., Ph.D.
Rush University Medical Center
Co-Chair
Wendy Chung, M.D., Ph.D.
Columbia University Irving Medical Center
Richard Finkel, M.D.
St. Jude Children’s Research Center
Eugene Schneider, M.D.
Ionis Pharmaceuticals
DSMB (Data Safety Monitoring Board)
The DSMB (Data Safety Monitoring Board) is composed of highly recognized independent experts in ASO technology, in conducting and evaluating clinical trials and in drug development, in addition to being dedicated clinicians treating patients with rare diseases. This team of experts will provide unbiased safety oversight through interim monitoring of accumulating data from all of the investigator-initiated studies evaluating n-Lorem ASO medicines.
n-Lorem Representatives
Joseph G. Gleeson, M.D.
Chief Medical Officer
External Physician and Patient Subject Matter Experts
Eugene Schneider, M.D.
Ionis Pharmaceuticals
Chair
Elizabeth M. Berry Kravis, M.D., Ph.D.
Rush University Medical Center
Wendy Chung, M.D., Ph.D.
Boston Children’s Hospital
Richard Finkel, M.D.
St. Jude Children’s Research Center
Lisa R. Grillone, Ph.D.
PharmaQuest Associates
Francis Sessions Cole, III, M.D.
Washington University School of Medicine in St. Louis
We cannot do
this alone
Together we are changing the world—
one patient at a time
We hope that you join us on this journey to discover, develop and provide individualized antisense medicines for free for life for nano-rare patients. The ultimate personalized medicine approach – for free, for life.
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