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 – Chief Technology Officer at n-Lorem and Chief Scientific Officer at Ionis Pharmaceuticals
Jeff Carroll, Ph.D.
Scientific Advisor at n-Lorem
Tracy Cole, Ph.D.
Senior Director of Research at n-Lorem
Julie Douville, Ph.D.
Senior Director of Pre-clinical Development at n-Lorem
Sarah Glass, Ph.D.
Chief Operating Officer at n-Lorem
Joseph G. Gleeson, M.D.
Chief Medical Officer at n-Lorem and Rady Professor, Neurosciences and Pediatrics, University of California, San Diego
Megan Knutsen, MBA
Director of Development Operations at n-Lorem
Cedrik Ngongang, M.D.
Senior Director, Medical Geneticist at n-Lorem
Amy Williford, Ph.D.
Senior Director of Communications and Donor Relations at n-Lorem
Wei Zhang, Ph.D.
Senior Scientist at n-Lorem
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 Lab
Shyamanga Borooah, M.B.B.S., Ph.D.
Assistant Clinical Professor of Ophthalmology at the Shiley Eye Institute at UC San Diego Health
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.
Vice President, Head of the Neuromuscular Development Unit at Biogen
Richard Finkel, M.D.
Professor of Neurology at the University of Central Florida School of Medicine, and Chief, Division of Neurology at Nemours Children’s Hospital in Orlando
Kenneth ‘Kurt’ Fischbeck, M.D.
NIH Distinguished Investigator, Neurogenetics Branch
Matthew Might, Ph.D.
Director at Hugh Kaul Precision Medicine Institute at University of Alabama at Birmingham
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.
Associate Professor of Neurology in the Neuromuscular Division at Columbia University, Vagelos College of Physicians and Surgeons
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 at n-Lorem
Joseph G. Gleeson, M.D.
Chief Medical Officer
Chair
Laurence Mignon, Ph.D.
Sr. Director of Clinical Development
Cedrik Ngongang, Ph.D.
Medical Geneticist
External Physician and Patient Subject Matter Experts
Liz Berry Kravis, M.D., Ph.D.
Rush University Medical Center
Co-Chair
Terry Jo Bichell, Ph.D.
COMBINEDBrain
Wendy Chung, M.D., Ph.D.
Columbia University Irving Medical Center
Richard Finkel, Ph.D.
St. Jude Children’s Research Center
Eugene Schneider, M.D.
Ionis Pharmaceuticals
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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