Lysosomal Therapeutics

Amylyx Pharmaceuticals, Inc. is a biotechnology company focused on developing innovative therapies for neurodegenerative diseases, particularly Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome. Founded in 2013 and based in Cambridge, Massachusetts, the company is best known for its investigational therapy AMX0035, a fixed-dose co-formulation of sodium phenylbutyrate and Taurursodiol. This treatment aims to address the energy crisis within mitochondria and the accumulation of toxic, unfolded proteins in the endoplasmic reticulum, both of which contribute to the progression of neurodegenerative conditions. Through its research and development efforts, Amylyx seeks to provide effective solutions to mitigate the suffering caused by these debilitating diseases.

Artax BioPharma is a biopharmaceutical company developing therapies for autoimmune and inflammatory diseases. Its flagship product, AX-024, is an oral small-molecule that selectively targets T-cell activation to treat conditions like asthma, rheumatoid arthritis, psoriasis, and multiple sclerosis. Incorporated in 2013, the company is headquartered in Cambridge, Massachusetts.

Aura Biosciences, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapies aimed at targeting and destroying cancer cells through 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 cancer of the eye. Aura Biosciences aims to address the challenges of treating small ocular melanomas early in the disease progression to prevent metastasis, particularly to the liver, which is often fatal. In addition to ocular oncology, the company is exploring applications of its platform in other indications, including bladder cancer. Founded in 2007, Aura Biosciences is committed to advancing precision immunotherapies designed to preserve organ function while effectively managing a range of solid tumors.

NanoString Technologies provides life science tools for translational research and molecular diagnostics. It offers the nCounter Analysis System, featuring automated liquid handling and digital detection, along with customizable assays and software for data analysis. The company also develops spatial genomics systems like GeoMx DSP and Hyb & Seq. Its products enable various applications in basic research, translational medicine, and diagnostics.