PrecisionLife and NATA Announce R&D Partnership to Jointly Develop New Motor Neurone Disease / ALS Drug Programme

2024-03-26

寡核苷酸

Leading precision medicine and nucleic acid therapy (NAT) innovators will harness their platforms to develop NAT drug candidates for novel gene targets with strong genetic association to sporadic ALS The programme will be supported by mechanism-based patient stratification biomarkers to identify likely responders and accelerate and derisk clinical development OXFORD and HARWELL, UK – 26 March 2024 – The pioneering precision medicine company, PrecisionLife, and the MRC nucleic acid therapy research unit, NATA, today announce a collaboration to co-develop novel oligonucleotide therapies for sporadic ALS. The collaboration combines NATA’s expertise in NAT design and optimization with PrecisionLife’s deep understanding of complex disease biology and the mechanisms, drug targets and treatments that are relevant to specific ALS patient subgroups. The target and mechanism of the first programme is predicted to have a prevalence amongst ALS patients of 15-30x that of SOD1 (the gene target for tofersen). The partnership builds on PrecisionLife’s unique insights into the mechanisms driving ALS and NATA’s translational research expertise and exploits the teams’ wide range of collaborations with patient charities, academic researchers and the biopharma industry. “The ALS patient community is rightly impatient for a range of new disease modifying approaches for such a devastating disease. Co-development with NATA brings a rapid, clear development path for one such novel drug programme”, said Steve Gardner, CEO of PrecisionLife. “We are particularly excited about the opportunity to explore NATs as modern AAV technologies have shown excellent potential for delivering oligonucleotides across the blood brain barrier”. “We are excited to be working with PrecisionLife, bringing together world-renowned experts in precision neuroscience with unique deep insights into ALS disease biology and design of nucleic acid therapies. This disease needs multiple innovative approaches for different disease mechanisms.”, said Professor Nick Lench, Executive Director, NATA. ENDS About PrecisionLife® PrecisionLife is a pioneering precision medicine company changing the way the world predicts, prevents, and treats complex chronic diseases by finding better, more personalized treatment options for patients across the biopharma, diagnostics, and healthcare value chain. Our combinatorial analytics platform understands disease biology better, identifying disease mechanisms that are relevant to specific patients and the drug targets and treatments that would benefit them. These unique insights enable us to discover new subgroups of patients with mechanism-based patient stratification biomarkers, identify novel treatment opportunities for unmet medical needs, de-risk and accelerate clinical trials, find new applications for existing drug programs, and build highly predictive complementary diagnostics, personalized risk prediction and disease prevention tools. To learn more about our groundbreaking work, please visit precisionlife.com, and follow us on LinkedIn (@PrecisionLifeAI) and X/Twitter (@PrecisionLifeAI). PrecisionLife Media Contact Noah Konig, Marketing Director noah@precisionlife.com About NATA The Nucleic Acid Therapy Accelerator (NATA) is a UK Research & Innovation (UKRI)/Medical Research Council funded research unit, based at the Harwell Science and Innovation Campus. NATA’s mission is to advance the development of nucleic acid therapies and associated technologies through high-quality science, collaborations and interdisciplinary research. NATA works with world-leading academics, clinicians, businesses and charities, bringing together a diverse network of expertise, skilled and innovative technologies to bridge translational gaps in NAT development. For more information, please visit , or follow NATA on LinkedIn (NATA – Nucleic Acid Therapy Accelerator) and Twitter (@NATA_MRC_UK).