Lysosomal Therapeutics

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.

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 focused on translational research and molecular diagnostics, headquartered in Seattle, Washington. Established in 2003, the company utilizes a unique digital molecular barcoding technology to create a range of products that facilitate gene expression analysis, microRNA studies, and copy number variation detection. Key offerings include the nCounter Analysis System, which consists of the nCounter MAX and FLEX systems, and the nCounter SPRINT Profiler, all designed for automated sample processing and data collection. The nSolver Analysis Software aids researchers in data normalization and analysis. Additionally, NanoString provides various custom CodeSets and panels tailored for specific applications, including cancer profiling and autoimmune disease research. The company is advancing the field of spatial genomics with its GeoMx Digital Spatial Profiler system and is developing the Hyb & Seq molecular profiling system for gene sequence analysis. NanoString collaborates with several pharmaceutical companies and research institutions to enhance its product offerings and expand its impact in the scientific community.