Nasdaq

- Read more about Fibrocell Completes Dosing of First Cohort in Phase 1/2 Clinical Trial of FCX-007 Gene Therapy for Treatment of Recessive Dystrophic Epidermolysis Bullosa
Fibrocell Science, Inc. a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, announced that the remaining two patients in the NC1+ cohort have been dosed in the Phase 1 portion of the Phase 1/2 clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), a devastating genetic skin disease with a high mortality rate. The cohort consists of three patients.
- Read more about Cell Design Labs announces Issuance of Broad Patent for Chimeric Notch Receptor
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.
- Read more about Tonix Pharmaceuticals presented analyses of Potential Moderators of Treatment response to USFDA
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.
- Read more about Abeona Therapeutics announces Top-Line Data for ABO-102 Phase 1/2 MPS IIIA Gene Therapy Trial at ASGCT
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.
- Read more about Tonix Pharmaceuticals to Present PTSD Clinical Data
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.
- Read more about Burcon updates on Peazazz® and Multiple Patent Grants
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.
- Read more about ProQR Announces Clearance of IND Application to Start Clinical Trial for QR-110 in Leber’s Congenital Amaurosis Type 10 Patients
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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- Read more about Alder BioPharmaceuticals announced Phase 2b clinical trial evaluating eptinezumab
Alder BioPharmaceuticals, Inc. a clinical-stage biopharmaceutical company developing monoclonal antibody therapeutics, today announced the presentation of positive data from its Phase 2b clinical trial evaluating eptinezumab (formerly ALD403) for the prevention of migraine.
- Read more about Achillion Pharmaceuticals announced results from the ongoing phase 2 ‘604 Study’
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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- Read more about Aptose Biosciences to Submit CG’806 data at AACR Hematologic Malignancies Meeting
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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