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10 Oct 2018 |
Newly Published Data in Nature Medicine Show Potential of mRNA to Produce Therapeutic and Protective Protein Levels in Pre-Clinical Models of Acute Intermittent Porphyria
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09 Oct 2018 |
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate
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09 Oct 2018 |
Inovio Successfully Develops First DNA-Encoded Monoclonal Antibody (dMAb™) Checkpoint Inhibitor and Demonstrates Tumor Shrinkage in Preclinical Studies
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06 Oct 2018 |
Audentes Therapeutics Presents New Positive Interim Data from ASPIRO, the Phase 1/2 Clinical Trial of AT132 in Patients with X-linked Myotubular Myopathy, at 23rd International Annual Congress of the World Muscle Society
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06 Oct 2018 |
Akcea and Ionis Receive FDA Approval of TEGSEDI™ (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
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05 Oct 2018 |
Vedanta Biosciences Announces Successful Phase 1a/1b Data Demonstrating Safety, Tolerability, and Proof of Mechanism for Lead, Rationally Defined Bacterial Consortium Product Candidate, VE303
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05 Oct 2018 |
bluebird bio Announces European Medicines Agency’s Acceptance of Marketing Authorization Application for LentiGlobin™ Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia
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03 Oct 2018 |
Alnylam Submits Clinical Trial Authorization (CTA) Application for ALN-AAT02, an Investigational RNAi Therapeutic for the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease (Alpha-1 Liver Disease)
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02 Oct 2018 |
CytoSen Therapeutics and KBI Biopharma Enter into Strategic Partnership to Manufacture NK Cells and Nanoparticles
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02 Oct 2018 |
Hitachi Chemical Advanced Therapeutics Solutions Signs Three-Year Agreement with GSK to Provide Clinical Manufacturing for T Cell Therapy
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27 Sep 2018 |
Alnylam Announces Positive Topline Results from Interim Analysis of ENVISION Phase 3 Study of Givosiran in Patients with Acute Hepatic Porphyria
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27 Sep 2018 |
Ultragenyx Announces Positive Topline Cohort 2 Results from Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency and Progression to Higher Dose
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27 Sep 2018 |
Targovax Announces Interim Results From Phase I Trial of ONCOS-102 in Checkpoint Inhibitor Refractory Melanoma
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27 Sep 2018 |
BriaCell Announces Positive Phase IIa Proof of Concept Data in Advanced Breast Cancer; Initiates Combination Study
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24 Sep 2018 |
Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
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24 Sep 2018 |
Akcea and Ionis Report Positive Data from Phase 2 Study of AKCEA-APO(a)-LRx
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23 Sep 2018 |
Nightstar Reports Positive Proof of Concept Data from Dose Escalation Study in XIRIUS Trial for NSR-RPGR in XLRP Patients
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21 Sep 2018 |
Exicure Announces Positive Phase 1 Results for its Lead Immuno-Oncology Asset
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21 Sep 2018 |
AVITA Medical Announces FDA Approval of the RECELL® System for the Treatment of Severe Burns in the U.S.
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21 Sep 2018 |
Novartis announces positive CHMP opinion for one-time gene therapy Luxturna® to treat children and adults with rare inherited retinal disease
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21 Sep 2018 |
First neuroblastoma patient successfully dosed with innovative CAR therapy utilizing natural killer T cells (CAR-NKT)
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17 Sep 2018 |
Oncolytics Biotech® Announces Investigator Sponsored Multiple Myeloma Study Combining REOLYSIN® and Nivolumab (Opdivo®) with Standard of Care
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14 Sep 2018 |
Bone Therapeutics SA announces final results from Phase I/IIA ALLOB delayed-union fracture study
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13 Sep 2018 |
Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in Spain
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12 Sep 2018 |
Vital Therapies Announces That Topline Results of VTL-308 Fail to Achieve Primary and Secondary Endpoints of Improvement in Survival
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