Lysosomal Therapeutics

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.

Artax Biopharma Inc. is a biotechnology company focused on developing therapies for autoimmune and inflammatory diseases. Founded in 2013 and based in Cambridge, Massachusetts, the company specializes in small-molecule drugs that act as immunomodulators. Its lead compound, AX-024, specifically targets Nck, disrupting the interaction that activates T-cells, thereby preventing harmful responses against antigens while maintaining the immune system's ability to combat infections. Artax Biopharma's research encompasses a range of conditions, including asthma, rheumatoid arthritis, psoriasis, graft versus host disease, and multiple sclerosis. By aiming to treat both symptoms and underlying disease mechanisms, the company's therapies are designed to minimize side effects associated with traditional treatments.

Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing therapies to target and destroy cancer cells using viral nanoparticle conjugates. The company’s lead product, AU-011, utilizes viral nanoparticles modeled on the human papillomavirus, conjugated with infrared-activated small molecules, specifically for the treatment of ocular melanoma, a primary eye cancer. By employing nanotechnology, Aura aims to safely eliminate cancer locally, particularly in early-stage cases, to prevent progression to more severe forms of the disease, such as metastasis to the liver. Additionally, the company is advancing its bel-sar candidate for treating primary choroidal melanoma and exploring applications in bladder cancer. Founded in 2007, Aura Biosciences is committed to developing precision immunotherapies that preserve the function of organs affected by cancer.

NanoString Technologies, Inc. is a provider of life science tools for translational research and molecular diagnostics on a global scale. The company specializes in the nCounter Analysis System, an automated platform for digital detection and quantification of nucleic acids. Key products include the nCounter MAX and FLEX systems, which feature an automated liquid handling component and a digital analyzer for data collection. Additionally, NanoString offers the nCounter SPRINT Profiler, nSolver Analysis Software, and custom CodeSets for tailored research needs. The company has developed various gene expression panels for cancer, autoimmune diseases, and other conditions. Furthermore, NanoString is advancing spatial genomics through its GeoMx Digital Spatial Profiler and Hyb & Seq molecular profiling systems. Collaborations with prominent organizations, including Celgene and Merck, enhance its research capabilities, particularly in developing novel protein assays for breast cancer. Founded in 2003 and headquartered in Seattle, Washington, NanoString continues to innovate in the field of molecular diagnostics and research tools.