DNA RNA and Cells

19 Mar 2019 Arrowhead Pharmaceuticals Files IND for Pivotal Phase 2/3 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
14 Mar 2019 BrainStorm Announces First Patient Enrolled in Phase 2 Clinical Trial of Progressive MS
11 Mar 2019 ProQR Doses First Patient in Phase 1/2 STELLAR Trial of QR-421a for Usher Syndrome Type 2
11 Mar 2019 Rubius Therapeutics Announces FDA Clearance of Investigational New Drug Application for First-Ever Red Cell Therapeutic, RTX-134, for Treatment of Phenylketonuria
11 Mar 2019 Alnylam Submits CTA Application for ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension in High Unmet Need Settings
11 Mar 2019 Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia
07 Mar 2019 AOBiome Doses First Patient in Phase 1b Clinical Trial of B244 for the Treatment of Pediatric Atopic Dermatitis
07 Mar 2019 Alnylam Reports Positive Topline Results from ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria
06 Mar 2019 NKMax America Announces FDA Approval of IND for Phase 1 Trial of SNK01 in Patients with Refractory Cancer
02 Mar 2019 CARsgen Therapeutics Receives IND Clearance for BCMA-CAR-T Cells from the NMPA
02 Mar 2019 Akcea and Ionis Receive Positive EU CHMP Opinion for WAYLIVRATM (volanesorsen)
28 Feb 2019 Translate Bio Provides Updates on Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency Programs
27 Feb 2019 Esperite N.V. (ESP) with The Cell Factory has confirmed a long-term effect of extracellular vesicle drug candidate CF-MEV-132 for the treatment of Bronchopulmonary Dysplasia
27 Feb 2019 Idera Pharmaceuticals Completes Enrollment Into the ILLUMINATE-204 Trial of the Combination of Tilsotolimod and Ipilimumab for Unresectable or Metastatic Melanoma Following Failure of PD-1 Inhibitor Treatment
25 Feb 2019 CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001
21 Feb 2019 Torque Announces Clinical Trial Collaboration with Merck
21 Feb 2019 ReNeuron Group plc Announces Positive Preliminary Data in U.S. Retinitis Pigmentosa Trial
21 Feb 2019 Torque Initiates Phase 1 Clinical Trial of TRQ-1501 Deep IL-15 Primed T Cells in Patients with Advanced Solid Tumors or Lymphomas
21 Feb 2019 Inovio announces first subject dosed with its DNA-encoded monoclonal antibody (dMAb) in first-ever human trial
20 Feb 2019 Phase 1 Data Published in Nature Communications Show Potential of mRNA Encoding VEGF-A as a Regenerative Therapeutic
20 Feb 2019 Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
20 Feb 2019 Autolus Therapeutics Announces Updated Results from Ongoing CARPALL Trial of Pediatric Acute Lymphoblastic Leukemia Presented at the EHA 1st European CAR T Cell Meeting in Paris
20 Feb 2019 Modis Therapeutics Announces that MT1621 Receives Breakthrough Therapy Designation from FDA for the Treatment of TK2 Deficiency
15 Feb 2019 Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
14 Feb 2019 Caladrius Biosciences Reports Top-Line Data for the Phase 2a Sanford Project: T-Rex Trial of CLBS03 for Recent Onset Type 1 Diabetes

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