DNA RNA and Cells

10 Aug 2017 REGENXBIO Announces IND Active for Phase I Trial of RGX-111 to Treat Mucopolysaccharidosis Type I
08 Aug 2017 RXi Pharmaceuticals Advances Immuno-Oncology Pipeline by Selecting Two sd-rxRNA Compounds for Preclinical Development and Sourcing cGMP Manufacturing
08 Aug 2017 Kite Submits Investigational New Drug (IND) Application for KITE-585, Anti-BCMA CAR-T Therapy Candidate for Multiple Myeloma
07 Aug 2017 Rebiotix Announces First Patient Enrolled in Phase 3 Clinical Trial of RBX2660 for the Prevention of Recurrent Clostridium difficile Infection
07 Aug 2017 Kite Announces Initiation of Axicabtagene Ciloleucel CAR-T Clinical Program in the European Union
05 Aug 2017 MiMedx Files With The FDA To Initiate Its Investigational New Drug Achilles Tendonitis Phase 3 Clinical Trial
03 Aug 2017 VM BioPharma Announces First Patient Dosed in Phase 3 Study of Gene Therapy Candidate VM202 for Non-Healing Diabetic Foot Ulcers
03 Aug 2017 BioMarin Announces Plans to Progress Both the 6e13vg/kg and 4e13 vg/kg Doses of BMN 270, its Investigational Gene Therapy for Hemophilia A, into Phase 3 Studies
03 Aug 2017 MiMedx Reports Exceptional Results in Phase 2B Clinical Trial on the Use of AmnioFix® Injectable in the Treatment of Plantar Fasciitis
02 Aug 2017 Kite Doses First Patient in the Phase 2 Trial of Axicabtagene Ciloleucel in Indolent B-Cell Non-Hodgkin Lymphoma (ZUMA-5)
02 Aug 2017 Orbsen Therapeutics Receives Approval from European Regulators for Clinical Trial of Breakthrough Cell Therapy for Diabetic Kidney Disease
02 Aug 2017 Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA™ (voretigene neparvovec)
01 Aug 2017 uniQure Reacquires Development and Commercialization Rights for its Gene Therapy Candidate in Hemophilia B
01 Aug 2017 Homology Medicines Announces $83.5 Million Series B Financing to Advance Lead Development Candidate and AMEnDR™ In Vivo Gene Editing Technology
01 Aug 2017 NantKwest Announces Durable Complete Responses in Phase I Trial of aNK Cell Therapy in Patients with Relapsed Hematological Malignancies
01 Aug 2017 Transgene: First Patient Dosed in a Phase 1/2 Trial of Pexa-Vec + Opdivo® for the First-Line Treatment of Advanced Liver Cancer
01 Aug 2017 GenSight Biologics Completes Enrollment of RESCUE Phase III Study of GS010 in the Treatment of Leber’s Hereditary Optic Neuropathy
31 Jul 2017 Kite Files the Industry’s First CAR-T Marketing Authorization Application in Europe for Axicabtagene Ciloleucel
27 Jul 2017 Quark Pharmaceuticals Reports Positive Results from a Phase 2 Study to Evaluate the Efficacy and Safety of QPI-1002 for the Prevention of Acute Kidney Injury (AKI) in Subjects at High Risk of AKI Following Cardiac Surgery
27 Jul 2017 Moderna Provides Update on mRNA Rare Disease R&D Strategy
27 Jul 2017 Akcea and Ionis Announce Submission of Marketing Authorization Application for Volanesorsen to the European Medicines Agency
25 Jul 2017 NantKwest Announces Publication of Preclinical Data of haNK Cell Therapy in Combination with Checkpoint Inhibitor Avelumab Providing Strong Rationale for Advancement to Human Clinical Trials
24 Jul 2017 Sernova-HemAcure Consortium Announce Significant Progress in Development of ‘First in World’ Regenerative Medicine Therapy for Treatment of Hemophilia A Patients
24 Jul 2017 Cytori Announces Top-Line 24- and 48-Week Results from the STAR Trial of Habeo™ Cell Therapy in Patients with Scleroderma
24 Jul 2017 Transgene Announces Cancer Research Publication Confirming the Potential of its Next Generation Armed Engineered Oncolytic Virus

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