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Clinical research courses

  • Cell Design Labs, Inc. announced the issuance of U.S. Patent No. 9,670,281, entitled “Binding-triggered transcriptional switches and the methods of use thereof.” This patent, issued to UC San Francisco (UCSF) and exclusively licensed to Cell Design Labs, broadly covers composition of matter for proprietary synNotch™ constructs, cells genetically modified with synNotch receptors and methods for treating diseases.

  • Tonix Pharmaceuticals Holding Corp. (Nasdaq:TNXP) (Tonix), a company that is developing innovative pharmaceutical products to address public health challenges, presented entitled “Phase 2 Multisite Double-Blind Placebo-Controlled Trial of TNX-102 SL in Military-Related Posttraumatic Stress Disorder: Mediators and Moderators of Treatment Response” (Poster No. 3001130) at the 72nd Annual Scientific Convention of the Society of Biological Psychiatry in San Diego. The poster can be found on the Scientific Presentations page on Tonix’s website. A moderator is a characteristic of study participants that is associated with a treatment response.

  • Abeona Therapeutics Inc.a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. The ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) is a first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for subjects with Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.

  • Tonix Pharmaceuticals Holding Corp. a company that is developing innovative pharmaceutical products to address public health challenges, announced it will present additional analyses of data from its Phase 2 study in military-related posttraumatic stress disorder, or PTSD, at the 72nd Annual Scientific Convention of the Society of Biological Psychiatry (SOBP) on May 20, 2017 in San Diego.

  • Burcon NutraScience Corporation is pleased to announce that it has been granted a U.S. patent, covering technology for the production of its Peazazz® pea protein:  U.S. patent no. 9,635,875, was granted on May 2, 2017.  This patent is the first to be granted for Burcon’s pea technology by the United States Patent and Trademark Office and confirms that the Company’s approach is unique, distinct and defensible.  Burcon anticipates further positive progress, with its patent filings, in due course.

  • ProQR Therapeutics N.V. announced that the Company can start the Phase 1/2 trial for QR-110, named PQ-110-001, in Leber’s congenital amaurosis Type 10 (LCA 10) patients. QR-110 is a novel investigational RNA therapeutic targeting LCA 10 due to the p.Cys998X mutation, a severe genetic rare disease that causes children to lose sight in the first years of life. LCA 10 is one of the most prevalent forms of gene-related blindness in children worldwide and currently there are no therapies commercially available.

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  • Achillion Pharmaceuticals, Inc.announced the presentation of updated results from the ongoing phase 2 ‘604 Study’ being conducted by Alios BioPharma Inc., part of the Janssen Pharmaceutical Companies (Janssen). These results were presented as an oral presentation during the European Association for the Study of the Liver (EASL) 2017 International Liver Congress in Amsterdam. These results demonstrate that the triple combination of simeprevir, odalasvir and AL-335 has the ability to shorten treatment duration, offer high efficacy and be generally well tolerated in those whose disease is caused by hepatitis C virus (HCV) genotype 1 (GT1), one of the most prevalent causes of hepatitis C globally.

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  • Aptose Biosciences Inc  a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, announced that preclinical data for its pan-FLT3/BTK inhibitor CG’806 will be presented in two separate posters at the 2017 American Association for Cancer Research (AACR) Conference Hematologic Malignancies: Translating Discoveries to Novel Therapies, being held May 6-9 in Boston, MA.

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  • Intellia Therapeutics a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, announced that the European Patent Office (EPO) has decided to grant a patent broadly covering the CRISPR/Cas9 genome editing technology. The patent includes claims covering compositions of the widely adopted CRISPR single guide RNA technology for use in any non-cellular and cellular setting, including eukaryotic cells such as human or mammalian cells, as well as for use in human therapeutics.

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