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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
ADVENTRX Pharmaceuticals is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Pathogen Detection Systems, Inc. is a Thunder Bay, ON-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Rubius Therapeutics is a biopharmaceutical company pioneering a new era of cellular medicines. Our proprietary RED PLATFORM™ was designed to genetically engineer and culture Red Cell Therapeutics™ that are selective, potent and ready to-use cellular therapies for the potential treatment of several diseases across multiple therapeutic areas. Our initial focus is to advance RCT™ product candidates for the treatment cancer and autoimmune diseases by leveraging two distinct therapeutic modalities — potent cell-cell interaction and tolerance induction. We are currently enrolling patients in a Phase 1/2 clinical trial of RTX-240; the trial has two Phase 1 arms enrolling adult patients with advanced solid tumors: an ongoing monotherapy dose escalation arm in adults with relapsed/refractory or locally advanced solid tumors and a combination therapy dose escalation arm with pembrolizumab in adults with relapsed/refractory or locally advanced solid tumors. In April 2022, the combination arm with pembrolizumab was expanded to focus on non-small cell lung cancer and renal cell carcinoma patients. We are also enrolling patients in a Phase 1/2 clinical trial of RTX-224 for the treatment of patients with certain advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer.
Sage Therapeutics is a clinical-stage biopharmaceutical company committed to developing novel medicines to transform the lives of patients with life-altering central nervous system (CNS) disorders. Sage`s lead product candidate, ZULRESSO(TM) (brexanolone) injection, has completed Phase 3 clinical development for postpartum depression and a New Drug Application is currently under review with the U.S. Food and Drug Administration. Sage is developing a portfolio of novel product candidates targeting critical CNS receptor systems, including SAGE-217, which is in Phase 3 development in major depressive disorder and postpartum depression.