DNA RNA and Cells

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
31 Oct 2017 Mustang Bio Reports Preclinical Data on Regional Delivery of MB-103 HER2 Chimeric Antigen Receptor Engineered T (CAR T) Cells to Target HER2+ Breast Cancer Metastasis to the Brain
31 Oct 2017 Akcea and Ionis Report Positive Phase 1/2a Data with AKCEA-APOCIII-LRx
31 Oct 2017 Novartis submits application to FDA for KymriahTM (tisagenlecleucel) in adult patients with r/r DLBCL, seeking second indication for first-ever FDA approved CAR-T therapy
28 Oct 2017 Tocagen Presents Updated Durable Complete Response Data from Phase 1 Trial of Toca 511 & Toca FC in Recurrent High-Grade Glioma
26 Oct 2017 Transgene: First Patient Treated in a Phase 1/2a Trial (Oncovirac) of Novel Oncolytic Virus TG6002 in Recurrent Glioblastoma
26 Oct 2017 FDA Grants Breakthrough Therapy Designation for BioMarin's Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A
25 Oct 2017 Atara Biotherapeutics Announces Six of Ten Progressive Multiple Sclerosis (MS) Patients Experienced Clinical Improvements in an Ongoing Phase 1 Study of Autologous ATA190
24 Oct 2017 Arbutus Presents HBV Data at 2017 AASLD Liver Meeting
23 Oct 2017 Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting(R) 2017
21 Oct 2017 uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study With FIX-Padua Variant in 2018
19 Oct 2017 AOBiome Therapeutics Reports Positive Efficacy Results from Phase 2b Clinical Trial of Ammonia Oxidizing Bacteria (AOB) for the Treatment of Acne Vulgaris
19 Oct 2017 OncoSec Presents Positive Phase 2 Data for ImmunoPulse® IL-12 in Combination with Pembrolizumab Demonstrating a Best Overall Response Rate (BORR) of 50% in Predicted Anti-PD-1 Non-Responder Melanoma Patients
19 Oct 2017 Atara Biotherapeutics Initiates 60 Patient, Global Phase 1 Clinical Study to Evaluate Allogeneic ATA188 in Patients with Progressive or Relapsing-Remitting Multiple Sclerosis (MS)
19 Oct 2017 Kite’s Yescarta™ (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy
18 Oct 2017 MeiraGTx Presents Preclinical Data on its Ocular Gene Therapy Studies for the Treatment of Leber's Congenital Amaurosis, Achromatopsia and X-Linked Retinitis Pigmentosa at ESGCT 2017 Congress in Berlin
17 Oct 2017 Dicerna Files Clinical Trial Application for DCR-PHXC, the Company’s Most Advanced GalXC™ Product Candidate, for Phase 1 Study in Primary Hyperoxaluria (PH)

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