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Regado Biosciences, Inc., is a biopharmaceutical company focused on the discovery and development of novel, oligonucleotide-based actively controllable therapeutics. Our initial focus is on applications in the acute and sub-acute cardiovascular therapeutic area. The company's lead product candidate, REG1, is a two-component system consisting of pegnivacogin, an anticoagulant aptamer specifically targeting coagulation Factor IXa, and its complementary oligonucleotide active control agent, anivamersen. REG1 is currently being evaluated in the REGULATE-PCI trial, a worldwide Phase 3 trial enrolling 13,200 patients with acute coronary syndromes (ACS) undergoing percutaneous coronary intervention (PCI), a hospital-based procedure used to mechanically open or widen obstructed coronary arteries. Regado's actively controllable product candidates have the potential to improve patient outcomes and enhance the patient experience, provide direct therapeutic control to physicians and reduce overall treatment costs.
Faze Medicines is a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates.
The mission of 4D Molecular Therapeutics is to design and develop transformative gene therapy products using our proprietary technology to create novel adeno-associated virus variants to be utilized as treatments for serious unmet medical conditions. Our products are unlocking the full potential of gene therapy to treat, and potentially cure, genetic diseases. A large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4D products. 4D has one of the deepest and most diverse product pipelines in the gene therapy field. Adeno-associated virus (AAV) vectors have emerged as a favored delivery vehicle for gene therapy in the human body. They can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. Several AAV gene therapy products are in late-stage clinical development, and one product is approved in the EU (Glybera, Uniqure). However, these first-generation AAV vectors have limited utility for the majority of diseases. Many of these common AAV were discovered as laboratory contaminants and monkey infections, for example; they have not been customized as targeted medicines. In contrast, 4D customized AAV vectors, and the products made from them, are designed to unlock the full potential of gene therapy. Through 4D`s Therapeutic Vector Evolution, we are able to create customized vectors solutions to overcome the hurdles identified with first-generation AAV vectors. 4D is creating the ultimate gene therapy products to cure genetic diseases using new customized delivery vehicles (vectors) to shuttle genes into the cells in any organ in the body. This 4D “library” of vectors will soon include several for the liver, several for the brain, several for the heart, the eye, muscle, and so on. Doctors will be able to hand pick a vector for any patient based on the organ that is diseased.
Potenza Therapeutics is a biotechnology company focused on building a diverse portfolio of oncology programs that utilize the body`s own immune system to seek out, recognize and destroy tumors through a diversity of mechanisms. Our programs are built upon novel biological insights into the tumor microenvironment and mechanisms by which cancer cells evade detection and destruction by the immune system. Potenza Therapeutics was founded in 2014 and is located in Cambridge, MA.
Biohaven is a privately-held biopharmaceutical company engaged in the identification and development of clinical stage compounds targeting the glutamatergic system and other neurological pathways. Biohaven has licensed intellectual property from Yale University, Catalent, ALS Biopharma LLC and Massachusetts General Hospital. Biohaven is owned by a group of investors including Portage Biotech Inc.