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 is a clinical-stage biotechnology company founded in 2007 and based in Cambridge, Massachusetts. It develops cancer therapies using tumor-targeted pseudovirions, employing viral nanoparticles conjugated to infrared-activated molecules to selectively destroy cancer cells. The lead program AU-011 targets ocular melanoma, including primary choroidal melanoma, with the aim of treating tumors locally while reducing damage to surrounding tissue. The company pursues precision therapies for solid tumors and has ongoing development in other ocular oncology indications and bladder cancer, focusing operations in the United States.

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.