DNA RNA and Cells

14 Nov 2017 Akcea Initiates AKCEA-ANGPTL3-LRx Phase 2 Program in Patients with Rare Hyperlipidemias
13 Nov 2017 DNAtrix Virus Expressing T-Cell Co-stimulator OX40L (CD252) Presented at the 2017 Society for Immunotherapy of Cancer Meeting
13 Nov 2017 International Stem Cell Corporation Announces Positive Interim Clinical Results for Parkinson's Disease Clinical Trial
13 Nov 2017 Mesoblast Phase 2 Trial Results Show Early and Durable Effects of Single Mesenchymal Precursor Cell Infusion in Biologic Refractory Rheumatoid Arthritis Patients
12 Nov 2017 Three-year Follow-up Phase 3 Data Provide Additional Information on Efficacy, Durability and Safety of Investigational LUXTURNA™ (voretigene neparvovec) in Patients with Biallelic RPE65-mediated Inherited Retinal Disease
12 Nov 2017 CRISPR Therapeutics Highlights New Additions to Portfolio of Allogeneic CRISPR-based CAR-T Therapies at SITC Annual Meeting
09 Nov 2017 Abeona Enrolls First Subject in Spain in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
09 Nov 2017 NY-ESO Data Presented at the Connective Tissue Oncology Society (CTOS) Annual Meeting Confirm Potential of Adaptimmune’s SPEAR T-Cell Therapy
08 Nov 2017 Synlogic Reports Positive Top-Line Phase 1 Data Demonstrating Safety and Tolerability and Proof of Mechanism in Healthy Volunteers for SYNB1020, a Synthetic BioticTM Medicine for the Treatment of Hyperammonemia
08 Nov 2017 Fourth Gene Therapy Program Targets the Most Common Inherited Juvenile Macular Dystrophy
08 Nov 2017 AMAG Announces Autologous Cord Blood Cells Improve Motor Function in Some Children with Cerebral Palsy
08 Nov 2017 Alnylam Initiates ENVISION Phase 3 Clinical Study with Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias (AHPs)
08 Nov 2017 Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051
07 Nov 2017 Cancer-Fighting ‘Super T-cells’ to Enter Clinical Evaluation
07 Nov 2017 FDA Lifts Clinical Hold on Cellectis Phase 1 Clinical Trials with UCART123 in AML and BPDCN
07 Nov 2017 Ionis Announces Submission of New Drug Application (NDA) for Inotersen to the U.S. FDA
06 Nov 2017 The Medicines Company and Alnylam Pharmaceuticals Announce Initiation of Phase III Clinical Trials of Inclisiran
06 Nov 2017 ORYX: Positive data from Phase I/IIa clinical trial in glioblastoma with oncolytic virus ParvOryx published in peer-reviewed journal, Molecular Therapy
06 Nov 2017 Novartis reaches another regulatory milestone for CTL019 (tisagenlecleucel) with submission of its MAA* to EMA for children, young adults with r/r B-cell ALL and adult patients with r/r DLBCL
04 Nov 2017 Quark Pharmaceuticals, Inc. Presents Results from a Positive Phase 2 Study of QPI-1002 for Prevention of Acute Kidney Injury Following Cardiac Surgery at the American Society of Nephrology Meeting
03 Nov 2017 Vedanta Biosciences Awarded Up To $5.4 Million from CARB-X to Accelerate Development of VE303, a Novel Human Microbiome-Derived Treatment for Serious Bacterial Infection
03 Nov 2017 Alnylam Reports Positive Preliminary Results from Ongoing Phase 1/2 Study of Lumasiran (ALN-GO1) in Patients with Primary Hyperoxaluria Type 1 (PH1)
03 Nov 2017 Sarepta Therapeutics and Nationwide Children’s Hospital Announce U.S. Food and Drug Administration (FDA) Clearance of the IND Application for the GALGT2 Gene Therapy Program
03 Nov 2017 Ionis Announces Submission of Marketing Authorization Application for Inotersen to the European Medicines Agency
02 Nov 2017 Sanofi and Alnylam present positive complete results from APOLLO Phase 3 study of investigational patisiran in hereditary ATTR (hATTR) amyloidosis patients with polyneuropathy

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