We are very grateful to the supporters of the 31st International Symposium on ALS/MND, held virtually on 9-11 December 2020:
Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing, and commercializing first-in-class muscle activators and best-in-class muscle inhibitors as potential treatments for people with debilitating diseases in which muscle performance is compromised and/or declining. A leader in muscle biology research Cytokinetics is developing small molecule drug candidates specifically engineered to impact muscle function and contractility.
Amylyx is a Cambridge-based pharmaceutical company dedicated to the development of therapeutics for the treatment of neurodegenerative disorders. Guided by our core values, we incorporate unconventional approaches through strong partnerships with industry leaders, scientists, doctors and organizations. We work collaboratively across everything we do to positively impact the lives of patients and their families.
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases.
Brainstorm Cell Therapeutics
Brainstorm Cell Therapeutics is a leading developer of adult stem cell therapies for neurodegenerative diseases.
The company’s innovative investigational cell therapy platform, NurOwn®, uses a patient’s own bone-marrow derived mesenchymal stem cells and modifies the cells to deliver high levels of neuronal repair and immunomodulatory molecules directly into the neurodegenerative disease cellular environment to modify known disease pathways. BrainStorm has recently completed its Phase 3 clinical trial in ALS, and has fully enrolled its Phase 2 clinical trial in progressive MS.
Mitsubishi Tanabe Pharma America / Mitsubishi Tanabe Pharma Canada
Mitsubishi Tanabe Pharma America (MTPA) and Mitsubishi Tanabe Pharma Canada (MTP-CA) are relentlessly focused on the goal of providing therapies for some of the most difficult-to-treat diseases, including amyotrophic lateral sclerosis (ALS). Our teams work diligently to develop smart options that will enable healthcare providers to offer safe and effective treatments for devastating illnesses. We thrive on solving complex problems in science and medicine and strive to make a real difference in the lives of people struggling with debilitating diseases.
Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). This therapy is now approved in the U.S., Japan, EU, Brazil and Israel, and additional registrations are being pursued in close to three dozen countries, with regulatory decisions anticipated in Switzerland, Canada, Australia, Argentina and South Korea in late 2020 or early 2021. Our robust AAV-based pipeline is advancing treatments for Rett syndrome and Friedreich’s ataxia, along with additional undisclosed programs. We are powered by the world’s largest gene therapy manufacturing footprint of more than one million square feet, enabling us to bring gene therapy to patients around the world at quality and scale.
Orphazyme is a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases. The company is harnessing amplification of Heat-Shock Proteins (or HSPs) in order to develop and commercialize novel therapeutics for diseases caused by protein misfolding, protein aggregation, and lysosomal dysfunction, including lysosomal storage diseases and neuromuscular degenerative diseases. Our product candidate is in clinical development for four orphan diseases: Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), Inclusion Body Myositis (IBM) and Gaucher disease.
QurAlis is bringing hope to the ALS community by developing breakthrough precision medicines for this devastating disease. Our stem cell technologies generate proprietary human neuronal models that enable us to more effectively discover and develop innovative therapies for genetically validated targets. We are advancing three antisense and small molecule programs addressing sub-forms of the disease that account for the majority of patients. Together with a world-class network of thought leaders, drug developers and patient advocates, our team is rising to the challenge of conquering ALS.
UCB is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. We are Inspired by Patients. Driven by Science. Follow us on Twitter: @UCB_news