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 Inc. is a biotechnology company focused on developing therapies for autoimmune and inflammatory diseases. Incorporated in 2013 and headquartered in Cambridge, Massachusetts, the company specializes in small-molecule drugs that modulate immune responses. Its lead compound, AX-024, selectively inhibits the interaction between T-cell receptors and Nck, which plays a crucial role in T-cell activation. This mechanism allows for the suppression of harmful immune reactions against self-tissues while maintaining the body's ability to combat infections. Artax Biopharma’s research portfolio includes therapies targeting conditions such as asthma, rheumatoid arthritis, psoriasis, graft-versus-host disease, and multiple sclerosis, aiming to provide innovative treatments with reduced side effects for patients suffering from these complex health issues.

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, Inc. is a life science company that specializes in providing innovative tools for translational research and molecular diagnostics globally. Founded in 2003 and headquartered in Seattle, Washington, the company developed a unique digital molecular barcoding technology for various applications in basic research and clinical diagnostics. Its flagship product, the nCounter Analysis System, consists of automated systems for sample preparation and data collection, allowing for the simultaneous profiling of numerous genes and proteins from single tissue samples. Additionally, NanoString offers the GeoMx Digital Spatial Profiler, which enhances spatial genomics research. The company also provides a range of services, including custom assay design, data analysis software, and molecular diagnostic test kits, such as Prosigna for breast cancer prognosis. Collaborations with major pharmaceutical companies and research institutions further bolster its position in advancing genomic and proteomic research.