Newest members bolster scientific and financial expertise as the Foundation expands to meet an increasing demand from the nano-rare community

n-Lorem a nonprofit Foundation, discovering, developing and providing experimental antisense oligonucleotides (ASOs) medicines to nano-rare patients (1 to 30 patients with the same single gene mutation worldwide) for free for life, today announced the newest additions to its Board of Directors – Paul Compton and Neil Shneider, M.D. Ph.D.

“I am pleased to be joining the Board of Directors for n-Lorem Foundation. n-Lorem’s approach combines three decades of expertise in ASO drug discovery and development, a staunch commitment to quality throughout the entire process and professional management of clinical exposure to create a novel non-profit model that, for the first time, provides a personalized approach to treat patients who would otherwise go overlooked in today’s healthcare system,” said Paul Compton, Global Head of the Corporate and Investment Bank, and President of Barclays Bank PLC. “I look forward to working with n-Lorem and supporting the directors as we help n-Lorem establish financial sustainability while continuing to expand to meet the needs of more and more patients.”

“ASO technology has enabled researchers and physicians to rethink how we approach rare genetic diseases. For patients with nano-rare mutations, a gene-based therapy may be the most promising way to change the trajectory of their disease. However, while ASO development is a rational and efficient process, assuring that each patient is treated with an optimal ASO is a complex challenge. Dr. Crooke led the creation of ASO technology and has decades of experience in drug development, and I am confident that he and the leadership of n-Lorem will make sure that each patient treated with an experimental medicine is managed professionally,” said Neil Shneider M.D. Ph.D., director of the Eleanor and Lou Gehrig ALS Center and the Claire Tow Associate Professor of Motor Neuron Disorders in the Department of Neurology at Columbia University, Vagelos College of Physicians and Surgeons. “The opportunity to contribute to n-Lorem not only as a collaborating physician but also as a director is an extraordinary opportunity to help my nano-rare ALS patients, as well as nano-rare patients around the world.”

“I am pleased to welcome Paul and Neil to our team of dedicated directors who are integral to our efforts to change the lives of nano-rare patients and their families. While we focus on one patient at a time, we anticipate treating thousands of patients over the course of the next ten years,” said Stanley T. Crooke, M.D., Ph.D., Founder, chairman and CEO, n-Lorem Foundation. “Our senior leadership, scientists, support staff, and our Board believes in the extraordinary task ahead and feels the urgency to reach and provide help to nano-rare patients. The focused support and guidance on our Board will continue to provide substantial value to n-Lorem as we scale up to meet the tremendous demand.”

Learn more about n-Lorem’s mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these needy patients and families.

• Subscribe: n-Lorem Patient Empowerment Program Podcast Series
• Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
• Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time

About Paul Compton

Mr. Compton joins n-Lorem with a wealth of finance and investment expertise. Paul Compton serves as the Global Head of the Corporate and Investment Bank, and President of Barclays Bank PLC (BBPLC), and is a member of the Group Executive Committee of Barclays. Mr. Compton leads Barclay's provision of funding, financing, strategic and risk management for money managers, corporate clients, financial institutions, governments and supranational organizations. Before joining Barclays in 2016, he served for nearly three decades in a variety of senior roles at JPMorgan Chase and Ernst & Young. Mr. Compton holds a Bachelor of Commerce and a Bachelor of Economics from the University of Queensland, Australia. He received post-graduate degrees from the Institute of Chartered Accountants in Australia and the Securities Institute of Australia.

About Neil Shneider

Dr. Shneider serves as the Claire Tow Associate Professor of Motor Neuron Disorders and the Director of the Eleanor and Lou Gehrig ALS Center at Columbia University. He is an investigator in the Center for Motor Neuron Biology and Disease where his lab focuses on the study of models and mechanisms of ALS and the discovery and development of novel therapeutics for ALS and related disorders. Dr. Shneider worked with Ionis Pharmaceuticals to develop ION363 (jacifusen), an anti-sense oligonucleotide (ASO) for ALS patients with rare mutations in the FUsed in Sarcoma (FUS) gene. Dr. Shneider is a graduate of Harvard College and earned his M.D. and Ph.D. degrees at the Columbia University College of Physicians and Surgeons.

In partnership with n-Lorem and Columbia University, Dr. Shneider founded Silence ALS, an initiative to develop ASOs for ALS patients with nano-rare, pathogenic mutations in ALS genes. Dr. Shneider was co-chair of the Translating Fundamental Research into Potential ALS Therapies Working Group for the NIH ALS Strategic Planning Workshop.

About n-Lorem

n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 160 applications for treatment with more than 80 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.

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