ALS Investment Fund

Verge Genomics focuses on accelerating drug discovery for neurodegenerative diseases through the use of artificial intelligence and genomic data. The company has developed a platform that leverages machine learning to analyze large datasets, identifying genetic targets and predicting effective therapeutics. By mapping genes associated with various neurodegenerative conditions, Verge aims to discover drugs that can simultaneously target multiple genetic factors. Founded by experts in machine learning and neuroscience, Verge Genomics seeks to improve patient outcomes and reduce the costs associated with pharmaceutical development. The company is based in Mountain View, California, and has formed a strategic alliance with Datavant to enhance its data-driven approach.

Bloom Science, Inc. is a biotechnology company focused on developing neuroprotective medicines that leverage the microbiome to address epilepsy and other neurological disorders. Founded in 2017 and based in San Diego, California, the company aims to create genetically optimized microbes that can treat patients suffering from pharmacoresistant and underserved diseases. Bloom Science seeks to translate the therapeutic advantages of the ketogenic diet into innovative biopharmaceuticals, providing effective treatment options for individuals facing serious and life-altering neurological conditions.

Prilenia Therapeutics B.V., a clinical stage biotech company, researches and develops products for movement disorders and neurodegenerative diseases affecting adults and children. It offers Pridopidine, an orally bioavailable small molecule investigational drug for huntington disease, amyotrophic lateral sclerosis, Parkinson’s disease levodopa induced dyskinesia, neurodegenerative eye disease, Parkinson’s disease, rett syndrome, fragile X, and Alzheimer’s disease. The company was founded in 2018 and is based in the Netherlands.

Apic Bio, Inc. is a biotechnology company focused on developing gene therapies for rare and complex neurological and liver diseases. Founded in 2017 and based in Cambridge, Massachusetts, the company specializes in treating conditions such as alpha-1 antitrypsin deficiency. Apic Bio utilizes a gene therapy platform that employs a Dual Function Vector (df-AAV) to simultaneously silence the Z-AAT protein and enhance the M-AAT protein in the liver. This innovative approach aims to address the challenges of genetic diseases through targeted therapies, providing hope for patients with undertreated conditions.

Verge Genomics focuses on accelerating drug discovery for neurodegenerative diseases through the use of artificial intelligence and genomic data. The company has developed a platform that leverages machine learning to analyze large datasets, identifying genetic targets and predicting effective therapeutics. By mapping genes associated with various neurodegenerative conditions, Verge aims to discover drugs that can simultaneously target multiple genetic factors. Founded by experts in machine learning and neuroscience, Verge Genomics seeks to improve patient outcomes and reduce the costs associated with pharmaceutical development. The company is based in Mountain View, California, and has formed a strategic alliance with Datavant to enhance its data-driven approach.

Emulate, Inc. specializes in developing organs-on-chips technology that emulates human biology to better understand the effects of diseases, medications, and environmental factors on human health. The company offers organ-chips that replicate the functions of various organs, including the lung, intestine, liver, and skin, and can simulate both normal and disease states. Emulate’s platform aims to provide more accurate predictions of human responses compared to traditional experimental methods, such as cell cultures or animal testing. The company serves a diverse clientele that includes biotechnology and pharmaceutical firms, consumer health and cosmetics companies, government agencies, and academic researchers. Founded in 2013 and based in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. It has shifted its focus towards advancing product innovation and safety across multiple sectors, including drug development and personalized medicine.

Orphazyme A/S is a biopharmaceutical company based in Copenhagen, Denmark, that specializes in developing therapies for neurodegenerative orphan diseases, particularly those linked to protein misfolding such as lysosomal storage diseases. Founded in 2009, the company is focused on its lead candidate, Arimoclomol, which is undergoing clinical trials for multiple orphan diseases, including Niemann-Pick disease type C, Gaucher disease, sporadic inclusion body myositis, and amyotrophic lateral sclerosis. Orphazyme’s research is grounded in academic discoveries from its founders at the Danish Cancer Society, and the company has established collaborations with leading academic institutions in Europe and the United States. In addition, Orphazyme has entered a strategic partnership with CombiGene AB to enhance its research and development efforts in the biopharmaceutical sector.

Amylyx Pharmaceuticals, Inc. develops novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. It offers AMX0035, an investigational therapy and a fixed-dose co-formulation of two active compounds, such as sodium phenylbutyrate and Taurursodiol optimized to treat both the energy crisis in the mitochondria and the toxic, unfolded proteins in the endoplasmic reticulum, disrupting the neurological chain of events that leads to patient suffering. The company’s pipeline includes amyotrophic lateral sclerosis, Wolfram syndrome, and Alzheimer’s diseases. Amylyx Pharmaceuticals, Inc. was founded in 2013 and is based in Cambridge, Massachusetts.