ALS Investment Fund

Verge Genomics is a biotechnology company focused on transforming drug discovery for neurodegenerative diseases through the application of artificial intelligence. Founded by experts in machine learning and seasoned drug developers in neuroscience, the company aims to leverage advances in computational genomics and insights into brain science to identify promising therapeutic targets. Its drug discovery platform utilizes machine learning algorithms to analyze large datasets, enabling the identification of effective drugs and accelerating the development of life-saving treatments. By improving the efficiency of the drug development process, Verge Genomics seeks to enhance patient outcomes and reduce the overall costs associated with pharmaceutical development.

Bloom Science, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing neuroprotective medicines that leverage the microbiome to address epilepsy and other neurological conditions. Established in 2017, the company employs its proprietary IrisRx™ discovery platform, which utilizes insights into the Gut-Brain Axis to create therapeutics targeting rare and complex neurological disorders. This innovative approach aims to translate the therapeutic benefits of the ketogenic diet into effective medications, enhancing safety and efficacy. Bloom Science is advancing a diverse pipeline of therapeutic candidates aimed at conditions such as Dravet syndrome, other rare epilepsies, Amyotrophic Lateral Sclerosis (ALS), and various neurodegenerative and cognitive disorders, with a commitment to providing improved treatment options for patients facing serious, life-altering illnesses.

Prilenia is a clinical-stage biotechnology company focused on developing innovative treatments for neurodegenerative and neurodevelopmental disorders. Founded in 2018, the company operates from locations in Israel, the Netherlands, and Boston. Its primary asset is Pridopidine, an oral drug candidate that has demonstrated an established safety profile and shows promise in treating various movement disorders and neurodegenerative diseases in both adults and children. Prilenia aims to enhance the functional capacity of patients, particularly those undergoing early hemodialysis, by addressing the underlying challenges posed by these conditions.

Apic Bio, Inc. is a gene therapy company based in Cambridge, Massachusetts, focused on developing innovative treatment options for rare and undertreated neurological and liver diseases. Established in 2017, the company is advancing several novel gene therapies, particularly targeting Alpha-1 Antitrypsin Deficiency, genetic Amyotrophic Lateral Sclerosis, and other central nervous system disorders. Apic Bio utilizes a gene therapy platform that employs a Dual Function Vector to silence the Z-AAT protein while augmenting the M-AAT protein, thereby addressing the underlying genetic issues. Through its targeted approach, Apic Bio aims to provide effective solutions for patients suffering from these challenging conditions.

Verge Genomics is a biotechnology company focused on transforming drug discovery for neurodegenerative diseases through the application of artificial intelligence. Founded by experts in machine learning and seasoned drug developers in neuroscience, the company aims to leverage advances in computational genomics and insights into brain science to identify promising therapeutic targets. Its drug discovery platform utilizes machine learning algorithms to analyze large datasets, enabling the identification of effective drugs and accelerating the development of life-saving treatments. By improving the efficiency of the drug development process, Verge Genomics seeks to enhance patient outcomes and reduce the overall costs associated with pharmaceutical development.

Emulate, Inc. specializes in organ-on-chip technology that simulates human biology to enhance the understanding of how diseases, medications, chemicals, and food impact human health. The company develops organ-chips for various organs, including the lung, intestine, liver, and skin, which replicate normal functions and disease conditions. Emulate's innovative technology offers researchers a more accurate alternative to traditional experimental methods, such as cell cultures and animal testing, by providing detailed insights into human biological responses. Additionally, the company supplies drug development and instrumentation software to facilitate research and development workflows for its clients. Emulate serves a diverse range of sectors, including biotechnology, pharmaceuticals, consumer health, cosmetics, chemicals, food, agrochemicals, government agencies, and academic institutions. Founded in 2013 and headquartered in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. until its name change in 2014.

Orphazyme is a Copenhagen-based biopharmaceutical company focused on developing innovative therapies for lysosomal storage diseases, a group of serious genetic disorders. The company was founded on research conducted by Professor Marja Jäättela and Thomas Kirkegaard Jensen at the Danish Cancer Society. Orphazyme is engaged in biopharmaceutical research and development, and it has established collaborations with leading academic institutions in Europe and the United States. The company has also entered into a strategic partnership with CombiGene AB, further enhancing its capabilities in the field.

Amylyx Pharmaceuticals develops innovative therapies for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's primary product, AMX0035, is an investigational therapy that combines two active compounds, sodium phenylbutyrate and Taurursodiol. This fixed-dose formulation aims to address mitochondrial energy crises and the accumulation of toxic proteins in the endoplasmic reticulum, which contribute to neurological decline. In addition to ALS, Amylyx's research pipeline includes potential treatments for Wolfram syndrome and Alzheimer's disease. Founded in 2013 and based in Cambridge, Massachusetts, Amylyx Pharmaceuticals is dedicated to advancing solutions for serious brain disorders.