DNA RNA and Cells

10 Jan 2018 GenSight Biologics Receives MHRA Approval to Initiate Phase I/II PIONEER Clinical Trial of GS030 Gene Therapy in Retinitis Pigmentosa
09 Jan 2018 Incysus Submits Second Investigational New Drug Application for Gamma-Delta T Cell Immunotherapy Candidate
09 Jan 2018 Moderna Announces an Array of Clinical Advances and Outlines 2018 Priorities; 19 Development Candidates, including 10 Clinical Programs, Highlight Productivity of mRNA Platform
09 Jan 2018 Ionis' Inotersen NDA Accepted for Priority Review by the FDA
08 Jan 2018 Merck’s KEYTRUDA® (pembrolizumab) Significantly Improved Recurrence-Free Survival Compared to Placebo as Adjuvant Therapy in Patients with Stage 3 Resected High-Risk Melanoma (EORTC1325/KEYNOTE-054)
08 Jan 2018 Adaptimmune Announces Two Manufacturing Achievements on Its way to Become the First Fully Integrated TCR T-cell Therapy Company
08 Jan 2018 Adaptimmune Announces Positive Safety Data from Pilot Studies with MAGE-A10 SPEAR T-cells and First Patient to Receive 1 billion Target Cell Dose
08 Jan 2018 Ultragenyx Announces Positive Interim Topline Results From First Cohort of Phase 1/2 Clinical Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
07 Jan 2018 Akcea Initiates Phase 2b Study of AKCEA-APOCIII-LRx in Patients with Hypertriglyceridemia and Established Cardiovascular Disease
04 Jan 2018 Audentes Announces Positive Interim Data from First Dose Cohort of ASPIRO, a Phase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy
04 Jan 2018 Celsion Announces FDA Clearance of the OVATION II Study for the Evaluation of GEN-1 Immunotherapy to Treat Newly Diagnosed Stage III/IV Ovarian Cancer
29 Dec 2017 Atara Biotherapeutics Announces FDA Clearance to Initiate Two Phase 3 Clinical Studies to Evaluate Tabelecleucel in Patients with Rituximab-Refractory Epstein-Barr Virus Associated Post-Transplant Lymphoproliferative Disorder (EBV+PTLD)
28 Dec 2017 Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT Deficiency
27 Dec 2017 VBL Therapeutics Announces First Patient in Pivotal Phase 3 Study of VB-111 in Platinum Resistant Ovarian Cancer in Collaboration with the GOG Foundation, Inc.
22 Dec 2017 Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1/2 Study of ARO-HBV
22 Dec 2017 Ionis Pharmaceuticals Announces The Initiation Of A Clinical Study Of Its First Orally Delivered Antisense Drug For The Treatment Of Gastrointestinal Disorders
21 Dec 2017 Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial for MPS IIIB
21 Dec 2017 Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-AAT
20 Dec 2017 Moderna Publishes Preclinical Data in Cell Reports on mRNA Therapeutic to Treat Rare Liver Disease Methylmalonic Acidemia (MMA), A Disorder of Organic Acid Metabolism
20 Dec 2017 BioMarin Doses First Patient in Global GENEr8-1 Phase 3 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
20 Dec 2017 FDA Approves Spark Therapeutics’ LUXTURNA™ (voretigene neparvovec-rzyl), a One-time Gene Therapy for Patients with Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
19 Dec 2017 Regulus Announces First-in-Human Dosing for Phase I Study of RGLS4326 for the Treatment of Autosomal Dominant Polycystic Kidney Disease
18 Dec 2017 RXi Pharmaceuticals Announces Positive Results from Phase 2 Trial with RXI-109 for Dermal Scarring
18 Dec 2017 Poseida Therapeutics Announces First Patient Treated in Phase 1 Study of P-BCMA-101 CAR-T Stem Cell Memory Therapy in Patients with Multiple Myeloma
18 Dec 2017 Sanofi and Alnylam submit Marketing Authorization Application to the European Medicines Agency for patisiran for the treatment of hereditary ATTR amyloidosis

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