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California Life Sciences Association (CLSA) advances Californias world-leading life sciences innovation ecosystem
Amyloid diseases are a diverse group of disorders, characterized by the deposition of amyloid protein fibrils in vital organs and tissues. There are approximately 30 different types of amyloidosis, each resulting from the misfolding and aggregation of a specific protein, and they are all severely debilitating, progressive, and often fatal.
Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare and devastating metabolic diseases with limited treatment options. Aeglea`s lead product candidate, pegzilarginase, is in a pivotal Phase 3 trial for the treatment of Arginase 1 Deficiency and has received both Rare Pediatric Disease and Breakthrough Therapy Designation. In the second quarter of 2020, the Company initiated a Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has also been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have big impact on the lives of patients and their families.
PDS Biotech is a clinical-stage biopharmaceutical company focused on the development and commercialization of therapies based on the Versamune® platform, a T-cell activating technology, designed to harness the power of the body`s immune system in the fight against cancer and infectious disease.
Sienna Biopharmaceuticals is focused on the discovery, clinical development and commercialization of products in aesthetics and dermatology based on scientific breakthroughs that dramatically improve patient outcomes, and physicians’ practice of medicine.