Silence ALS initiative will offer an integrated solution to identify, support and potentially treat nano-rare ALS patients.
n-Lorem foundation and Columbia University receive a founding donation from Target ALS of $400,000 to formalize the Silence ALS initiative and support the development of personalized experimental ASO medicines for nano-rare ALS patients
n-Lorem, a nonprofit foundation, announced today the formation of Silence ALS, an initiative that aligns Columbia University and n-Lorem in support of the characterization of nano-rare amyotrophic lateral sclerosis (ALS) patients and the discovery and development of personalized experimental antisense oligonucleotide (ASO) medicines for these patients for free, for life. The initiative creates an infrastructure that employs n-Lorem’s non-profit model to discover and develop personalized investigational ASO medicines for nano-rare ALS patients. Additionally, the initiative utilizes Columbia University’s resources to conduct a comprehensive clinical and scientific evaluation of pre-symptomatic ALS patients with the ultimate goal of identifying and treating nano-rare ALS patients prior to symptom onset.
ALS is a fatal neurological disorder caused by the degeneration of motor neurons in the brain and spinal cord. People with ALS rapidly lose muscle strength and eventually their ability to move, swallow, and breathe. In at least 10-15% of ALS cases, the disease is caused by a mutation in one of a large and growing number of ALS-associated genes. Silence ALS targets those “nano-rare” ALS patients who have a genetic mutation found in only 1 to 30 affected individuals worldwide.
“The Silence ALS initiative will create the most efficient process to connect the diagnosis and treatment of patients with some of the extremely rare mutations that cause ALS. I am particularly pleased to continue to work closely with Dr. Neil Shneider of Columbia as we bring hope and treatment to patients with the very rare forms of ALS. I am also very grateful to Target ALS for their generous founding donation. As Silence ALS advances, I look forward to working with the broader ALS community to enhance the efficiency of the journey from diagnosis to treatment for many of these very rare ALS patients,” said Stanley Crooke, M.D., Ph.D., Founder and CEO of n-Lorem.”
“Despite great progress in our understanding of the genetics and biology of ALS, no therapy is available today that alters the natural history of the disease in a meaningful way,” said Neil Shneider M.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. “Through our partnership with n-Lorem, Silence ALS aims to have a significant impact on the lives of nano-rare ALS patients, and to generate findings relevant to all patients and families afflicted with this devastating disease.”
“This partnership is an extension of our collaborative approach and part of our vision to find treatments for all forms of ALS. At Target ALS, we are a champion for the power of collaboration that leverages our model to accelerate research and therapeutic development. As such, the Silence ALS initiative aligns our strengths with a world-renowned healthcare institute and a novel non-profit to bring hope and potentially lead to the development of new therapeutics for these patients. As part of our founding donation, we will initially support the initiative by funding a portion of the costs associated with preclinical toxicology for two ALS programs. Over time, we hope to expand our participation to support clinical trial work and to analyze biosamples collected during the course of treatment,” said Manish Raisinghani, M.B.B.S., Ph.D. CEO of Target ALS. “In short, this effort has the potential to make an immediate positive impact for patients with nano-rare forms of ALS while advancing understanding of the disease biology overall.”
“Dr. Shneider leads the ALS Center at Columbia University and is the perfect partner for this initiative. This partnership exemplifies the integration of research focused on the genetics and biology of ALS with therapeutic discovery,” said Sarah Glass., Ph.D., Chief Development Officer of n-Lorem Foundation. “We appreciate Target ALS’ unwavering support and commitment to the development of effective treatments for ALS. Our collective efforts in Silence ALS exemplify how n-Lorem can support a disease-focused approach to identify and treat nano-rare patients by identifying commonalities and introducing efficiencies across the ASO programs.”
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Silence ALS is a newly formed initiative between Columbia University and n-Lorem with Target ALS as a founding donor. Silence ALS plans to establish an infrastructure to enable ASO discovery and development for carriers of nano-rare ALS gene mutations. The collaboration will also involve the comprehensive clinical and scientific evaluation of pre-symptomatic ALS individuals, with the goal of identifying and eventually treating ALS patients in advance of symptom onset or very early in the course of disease. Through the use of n-Lorem’s ASO technology, personalized experimental ASO medicines can be designed to target the underlying genetic cause of disease.
About Target ALS
Target ALS is a 501(c)(3) medical research foundation committed to the search for effective treatments for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. We envision a world in which no one dies of ALS and play a unique role in the battle against this disease. Founded in 2013 by former New York City deputy mayor Dan Doctoroff – who lost both his father and uncle to ALS – our approach is breaking down barriers and silos that previously inhibited research results. We do this through our Target ALS Innovation Ecosystem, which facilitates unparalleled collaboration between researchers from academia and the pharma/biotech industry. The Target ALS Innovation Ecosystem has revolutionized the field in just seven years through collaborations that have resulted in the first potential treatments since ALS was identified in 1869.
To date, the Target ALS Innovation Ecosystem, which launched in 2013 and set the groundwork for the new Target ALS Diagnosis Initiative, has yielded 175+ research projects, 12+ therapeutic targets and six clinical trials, to date. http://www.targetals.org/.
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 130 applications for treatment with more than 50 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, YouTube and Instagram.