DNA RNA and Cells

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
09 Oct 2018 Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate
09 Oct 2018 Inovio Successfully Develops First DNA-Encoded Monoclonal Antibody (dMAb™) Checkpoint Inhibitor and Demonstrates Tumor Shrinkage in Preclinical Studies
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
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
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
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
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)
02 Oct 2018 CytoSen Therapeutics and KBI Biopharma Enter into Strategic Partnership to Manufacture NK Cells and Nanoparticles
02 Oct 2018 Hitachi Chemical Advanced Therapeutics Solutions Signs Three-Year Agreement with GSK to Provide Clinical Manufacturing for T Cell Therapy
27 Sep 2018 Alnylam Announces Positive Topline Results from Interim Analysis of ENVISION Phase 3 Study of Givosiran in Patients with Acute Hepatic Porphyria
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
27 Sep 2018 Targovax Announces Interim Results From Phase I Trial of ONCOS-102 in Checkpoint Inhibitor Refractory Melanoma
27 Sep 2018 BriaCell Announces Positive Phase IIa Proof of Concept Data in Advanced Breast Cancer; Initiates Combination Study
24 Sep 2018 Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
24 Sep 2018 Akcea and Ionis Report Positive Data from Phase 2 Study of AKCEA-APO(a)-LRx
23 Sep 2018 Nightstar Reports Positive Proof of Concept Data from Dose Escalation Study in XIRIUS Trial for NSR-RPGR in XLRP Patients
21 Sep 2018 Exicure Announces Positive Phase 1 Results for its Lead Immuno-Oncology Asset
21 Sep 2018 AVITA Medical Announces FDA Approval of the RECELL® System for the Treatment of Severe Burns in the U.S.
21 Sep 2018 Novartis announces positive CHMP opinion for one-time gene therapy Luxturna® to treat children and adults with rare inherited retinal disease
21 Sep 2018 First neuroblastoma patient successfully dosed with innovative CAR therapy utilizing natural killer T cells (CAR-NKT)
17 Sep 2018 Oncolytics Biotech® Announces Investigator Sponsored Multiple Myeloma Study Combining REOLYSIN® and Nivolumab (Opdivo®) with Standard of Care
14 Sep 2018 Bone Therapeutics SA announces final results from Phase I/IIA ALLOB delayed-union fracture study
13 Sep 2018 Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in Spain
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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