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Imara Inc. is dedicated to developing novel therapeutics for people living with sickle cell disease and other hemoglobinopathies. Sickle cell disease is a rare, genetic blood disease that causes red blood cells to sickle and become damaged, activating immune cells and blocking blood flow in capillaries, injuring many organs and causing daily pain. Imara is developing IMR-687, a highly selective, potent small molecule inhibitor of phosphodiesterase-9 (PDE9i), to treat patients with sickle cell disease. Imara was launched following an 18-month scientific collaboration between orphan drug accelerator Cydan Development and H. Lundbeck A/S.
Graphite Bio is a next-generation gene editing company focused on the development of first-in-class therapies for patients suffering from life-threatening genetic diseases. The company`s platform harnesses the natural cellular process of homology directed repair (HDR) and targeted DNA integration to precisely repair genetic defects at their source. Graphite Bio is leveraging its differentiated platform to advance a portfolio of transformative genetic treatments with potential for saving and dramatically improving patients` lives
Vital Therapies, Inc. is a biotherapeutic company focused on developing a cell-based system for the treatment of acute liver failure. Our product candidate, the ELAD® System, is a human cell-based, bio-artificial liver support system that operates outside the body, or extracorporeally, and is designed with the proposed intent to allow the patient’s own liver to regenerate to a healthy state, or to stabilize the patient until liver transplant. The ELAD System incorporates our human liver-derived cells, or VTL C3A cells, contained in four hollow fiber cartridges, that are combined with single use customized disposable sets and an ancillary delivery system. Data from ELAD clinical studies has shown trends that may indicate a potential to increase survival rates in patients with acute liver failure. ELAD has received orphan designation in the United States and Europe for the treatment of acute liver failure. Prior to the initiation of our ongoing Phase III clinical trial program, over 145 subjects have received therapy with the ELAD System in seven clinical trials and through a compassionate use program, which we believe collectively suggests a promising therapeutic profile. In March 2013, we initiated VTI-208, a Phase III randomized, controlled clinical trial in 200 subjects with alcohol-induced liver decompensation. We reached the midpoint in enrollment of this trial in April 2014, and anticipate the release of preliminary data in the first half of 2015. In addition, we are conducting a second Phase III randomized, controlled clinical trial, VTI-210, in 150 subjects with severe acute alcoholic hepatitis, or AAH, which is a subset of AILD, and expect to initiate enrollment of subjects later in 2014. In the second quarter of 2014, we began enrollment of a Phase II clinical trial of the ELAD System in subjects with either fulminant hepatic failure, or FHF, or surgery-induced acute liver failure, or SILF. We anticipate the release of data from VTI-210 in 2016 and the Phase II component of VTI-212 in 2015 or 2016.
Canadian Light Source is a Saskatoon, SK-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Sorrento is an oncology company developing new treatments for cancer and associated pain. Sorrento recently announced that NantPharma acquired the rights to Cynviloq™, which completed a successful TRIBECA study. Sorrento is also developing resiniferatoxin (RTX), a non-opiate TRPV1 agonist currently in a Phase 1/2 study at the NIH to treat terminal cancer patients suffering from intractable pain. In December 2014, Sorrento and NantWorks formed a global joint venture, now called Nantibody, to focus on immunotherapies for cancer. Also in December 2014, Sorrento and Conkwest, Inc., a privately-held immuno-oncology company developing proprietary Neukoplast,® a Natural Killer (NK) cell-line based therapy, entered into an agreement to jointly develop CAR.TNK™ (Chimeric Antigen Receptor Tumor-attacking Neukoplast) immunotherapies for the treatment of cancer and infectious diseases. In March 2015, Sorrento entered into a global collaboration with NantCell, a NantWorks company, to discover and develop immunotherapies against tumor neo-epitopes.