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AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
We work at the intersection of biology, engineering, and design, developing technologies that emulate human biology. Our products are enabling a new era of precision medicine and the development of applications for personalized health. Our Human Emulation System, which uses Organs-on-Chips technology, is being used by industry, government, and academia to understand how different diseases, medicines, chemicals and foods affect human health. The system offers researchers a new standard for predicting human response — with greater precision and control than today`s cell culture or animal-based experimental methods. We are also leading a bold, new precision medicine initiative with our clinical partners. Named "Patient-on-a-Chip," the program is developing personalized chips with patients` own cells. This initiative will help transform the way each of us understands our own bodies and manages our own health. Research conducted using our Organ-Chips has been published in high-impact scientific journals, including Science. In addition, Organ-Chips have been acquired by the Museum of Modern Art (MoMA) in New York for their permanent collection, and have also been awarded Product Design of the Year 2015 by London`s Design Museum. Our founding team pioneered the original Organs-on-Chips technology during their residency within the Wyss Institute for Biologically Inspired Engineering at Harvard University.
ARCA was founded on the belief that a precision medicine approach to drug development, tailoring medical treatment to the individual genetic characteristics of patients, can enable more effective therapies, improve patient outcomes and reduce healthcare costs. ARCA`s lead development program is intended to be a direct implementation of those ideas. Gencaro™ (bucindolol hydrochloride) is being developed as a potential treatment for atrial fibrillation (AF). ARCA has identified genetic variations in cardiac receptors that we believe may predict individual patient response to Gencaro™, giving Gencaro™ the potential to be the first genetically-targeted prevention treatment for AF. ARCA is also developing rNAPc2 as potential treatment for RNA-associated diseases, initially focused on COVID-19.
Concentric Analgesics, Inc. is a private, biotechnology company focused on discovering and developing novel, non-opioid therapeutics for the management of acute and chronic pain.
Human Genome Sciences, Inc. is a Rockville, MD-based company in the Healthcare, Pharmaceuticals, & Biotech sector.