DNA RNA and Cells

15 Apr 2019 ProQR Doses First Patient in Phase 2/3 ILLUMINATE Trial of Sepofarsen for LCA10
15 Apr 2019 Alnylam Initiates ILLUMINATE-B Phase 3 Pediatric Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 and Presents New Positive Results from Phase 2 Open-Label Extension Study
15 Apr 2019 Promethera Biosciences Presents Updated Phase 2a Results for World’s First Stem Cell Clinical Trial in ACLF at The International Liver Congress™/ EASL 2019
15 Apr 2019 Arrowhead Pharmaceuticals Receives FDA Clearance to Begin Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
15 Apr 2019 Adverum Biotechnologies Provides Clinical Program Update on ADVM-022 Gene Therapy for Wet AMD
13 Apr 2019 Alnylam Presents Positive Complete Results from ENVISION Phase 3 Study of Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria
13 Apr 2019 Arrowhead Preclinical Data Demonstrates RNAi Therapy Prevents and Reverses Alpha-1 Liver Disease at The International Liver Congress™
13 Apr 2019 Arrowhead Presents Clinical Data on JNJ-3989 (ARO-HBV) at The International Liver Congress™
09 Apr 2019 Immatics Initiates Third Phase I Clinical Trial of its Unique ACTengine® Platform in Patients with Advanced Solid Cancers
09 Apr 2019 Translate Bio Presents Preclinical Data Supporting MRT5201 for the Treatment of Ornithine Transcarbamylase (OTC) Deficiency at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
08 Apr 2019 SanBio Announces SB623 Regenerative Cell Therapy for Traumatic Brain Injury Has Received Ministry of Health, Labour and Welfare (MHLW) Sakigake Designation
05 Apr 2019 Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington's Disease
05 Apr 2019 AnGes Obtains Conditional Approval in Japan for HGF Gene Therapy to Treat Critical Limb Ischemia
05 Apr 2019 Homology Medicines Announces FDA Clearance of Investigational New Drug Application for HMI-102 Gene Therapy Candidate for PKU
04 Apr 2019 Janssen Announces Investigational CAR-T Therapy JNJ-68284528 Granted PRIME Designation by the European Medicines Agency
03 Apr 2019 Fate Therapeutics Announces First Patient Treated with iPSC-derived NK Cell Cancer Immunotherapy FT500 Successfully Completes Initial Safety Assessment
03 Apr 2019 FDA Clears the IND for UCARTCS1, the First Allogeneic CAR-T to Treat Multiple Myeloma Patients
03 Apr 2019 Idera Pharmaceuticals Presents ILLUMINATE-101 Data Demonstrating Tilsotolimod Activates Innate and Adaptive Immunity as Monotherapy in Patients with Refractory Solid Tumors at the American Association for Cancer Research (AACR) 2019 Annual Meeting
03 Apr 2019 Inovio Presents Cancer Killing Data of Its Transformative DNA-Encoded Bi-specific T Cell Engagers (dBiTEs) at AACR
03 Apr 2019 Sangamo Provides Clinical Development Update Including Early Phase 1/2 Beta Thalassemia Gene-edited Cell Therapy Data
03 Apr 2019 Sangamo and Pfizer Announce Phase 1/2 Interim Data For Investigational Hemophilia A Gene Therapy
02 Apr 2019 Sensei Biotherapeutics Presents First Preclinical Data on SNS-723 CAR-T and Long-Term Results on SNS-301 Data at the 2019 American Association for Cancer Research (AACR) Annual Meeting
02 Apr 2019 MiNA Therapeutics Presents Pre-Clinical Data Supporting Combination of MTL-CEBPA with Sorafenib and Other Cancer Treatments at AACR
02 Apr 2019 Adaptimmune Presents Preclinical Data for its Next Generation SPEAR T-cell Targeting MAGE-A4 at the American Association for Cancer Research (AACR) Annual Meeting
02 Apr 2019 Allogene Therapeutics Presents Preclinical Data Demonstrating the Potential of AlloCAR T™ Therapy in Renal Cell Carcinoma (RCC) at the 2019 AACR Annual Meeting

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