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AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC`s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC`s product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products
At LAVA, we are focused on applying our expertise in gamma-delta T-cell engagers (TCEs) to transform cancer therapy. We refer to our compounds as gamma-delta bispecific T-cell engagers, or gamma-delta bsTCEs. Our platform generates therapeutics that specifically activate a unique and relatively abundant effector gamma-delta T-cell subset called Vγ9Vδ2 (Vgamma9-Vdelta2) T-cells that have the ability to trigger activity of immune cells of both the innate and adaptive immune system. Our gamma-delta bsTCEs have demonstrated superior efficacy and safety profiles compared to other bsTCE approaches in preclinical studies and show preferential activity against tumor cells thereby potentially limiting toxicity in healthy tissue. To date, we are the only company developing bispecific gamma-delta T-cell engaging antibodies for the treatment of cancer. We are building a pipeline of gamma-delta bsTCEs that have applications in both solid and hematologic malignancies. Our lead program, LAVA-051, targets the tumor specific antigen CD1d, which can be overexpressed in multiple myeloma, chronic lymphocytic leukemia (CLL), and acute myeloid leukemia (AML), among other tumor types. LAVA-051 will enter a Phase I/IIa study in 1Q21.
R I Analytical Laboratories Inc is a Warwick, RI-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
HomeDNA, Inc. is a Keller, TX-based company in the Healthcare, Pharmaceuticals, & 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.