DNA RNA and Cells

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
16 Dec 2017 Takeda and TiGenix announce that Cx601 (darvadstrocel) has received a positive CHMP opinion to treat complex perianal fistulas in Crohn's disease
16 Dec 2017 FDA lifts clinical hold on fitusiran
14 Dec 2017 OncoSec Announces Dosing of First Patient in Registration-Directed Phase 2b Clinical Trial, PISCES/KEYNOTE-695, of ImmunoPulse® IL-12 in Combination with Pembrolizumab
14 Dec 2017 Akcea Initiates Phase 2 Study of AKCEA-ANGPTL3-LRx in Patients with Hypertriglyceridemia, Type 2 Diabetes Mellitus and Nonalcoholic Fatty Liver Disease (NAFLD)
14 Dec 2017 ImmunoCellular Therapeutics Achieves Key Milestone in Stem-to-T-Cell Research Immuno-Oncology Program
14 Dec 2017 Iovance Biotherapeutics Expands Pipeline of TIL Therapies into Lung Cancer
13 Dec 2017 Alnylam Completes Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
13 Dec 2017 Preliminary Data from Servier and Pfizer’s UCART19 Product Candidate Shows High Complete Remission Rate Across Two Phase I Adult and Pediatric Acute Lymphoblastic Leukemia Trials

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