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Juventas Therapeutics is a private clinical-stage company developing novel therapies for ischemic cardiovascular disease. The company`s lead product` JVS-100` is a non-viral plasmid that encodes for stromal cell-derived factor-1 (SDF-1). SDF-1 has been shown to significantly increase end-organ function following tissue injury by promoting cell survival` recruiting endogenous stem cells to the damaged region` and promoting new blood vessel growth. The SDF-1 repair pathway is well conserved throughout end-organ systems providing the opportunity to impact a broad range of diseases. Target cardiovascular clinical indications address large markets with high unmet medical need and significant market potential. Juventas is currently enrolling multiple clinical trials to test therapy efficacy in heart failure and critical limb ischemia patients.
Precision NanoSystems Inc. (PNI) creates innovative solutions that empower researchers to harness powerful nanotechnology to transform our understanding and treatment of diseases.
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
Diagenics is a Woburn, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.