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Arkuda Therapeutics is a biotechnology company applying insights into lysosomal biology to drive the development of medicines to change the trajectory of neurodegenerative disease. Arkuda`s lead program ARKD-104 aims to correct progranulin deficiency and lysosomal dysfunction in patients with GRN-related frontotemporal dementia (FTD-GRN), a genetically-defined subtype of frontotemporal dementia caused by an autosomal dominant mutation in the progranulin (GRN) gene. The company is further exploring the therapeutic potential of its molecules in other neurodegenerative diseases where genetic links to dysfunction in progranulin biology have been established, including Alzheimer`s Disease and Parkinson`s Disease. Arkuda is backed by leading investors including Atlas Venture, Cormorant Asset Management, Pfizer Ventures, funds managed by Tekla Capital Management LLC, Mission BioCapital, and Eli Lilly and Company.
MonoSol Rx is a specialty pharmaceutical company leveraging its proprietary PharmFilm® drug delivery technology to develop products that improve patient outcomes and address unmet needs. These pharmaceutical and over-the-counter products are developed independently and with partners. PharmFilm can provide a benefit to patients by improving the efficacy, safety, and convenience of currently marketed pharmaceutical products. PharmFilm enhances new pharmaceutical products with the unique, multi-faceted value proposition afforded by film delivery. MonoSol Rx`s leadership in film drug delivery is supported by strong IP protection, a robust pipeline of prescription drug formulations, and two FDA-approved products — Suboxone® (buprenorphine and naloxone) sublingual film and Zuplenz® (ondansetron) oral soluble film.
Accelerated Technologies Partners is a Hackensack, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Omicia is unlocking the potential of individualized medicine. Our mission is to help researchers and clinicians understand and apply the most relevant information from personal genome sequences, to improve disease management and medical outcomes. Researchers and clinical diagnostic organizations use our solutions to analyze and identify the genetic basis of a variety of conditions, including childhood disease, cancer and cardiovascular disease. Opal™, the leading platform for fast, accurate and flexible genome analysis, enables clinicians, researchers and bioinformaticians alike to derive clinically relevant insights from genomic data. VAAST, our robust novel disease gene finder and variant scoring algorithm, is in use at more than 300 academic and clinical institutions including the NIH, the University of Oxford, the University of Cambridge, Seattle Children’s Research Institute, Institut Pasteur, University of Maryland Institute for Genome Sciences, and UCSF.
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