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

Julie Douville, Ph.D.

Executive Director, ASO Discovery and 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, M.S.

Director, Foundation and Program Management

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.

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 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.

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

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.

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.

Sr. Director of Clinical Development

Cedrik Ngongang, M.D.

Medical Geneticist

External Physician and Patient Subject Matter Experts

Liz 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.

External Physician and Patient Subject Matter Experts

Eugene Schneider, M.D.

Ionis Pharmaceuticals
Chair

Liz Berry Kravis, M.D., Ph.D.

Rush University Medical Center

Wendy Chung, M.D., Ph.D.

Columbia University Irving Medical Center

Richard Finkel, M.D.

St. Jude Children’s Research Center

Neil Shneider, M.D., Ph.D.

Columbia University Irving Medical Center

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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Join us on our Corps of Discovery of the mind and heart. Help us bring hope and potential help to nano-rare patients today. For free, for life.
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