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11 Mar 2019 |
ProQR Doses First Patient in Phase 1/2 STELLAR Trial of QR-421a for Usher Syndrome Type 2
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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
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11 Mar 2019 |
Alnylam Submits CTA Application for ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension in High Unmet Need Settings
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11 Mar 2019 |
Arrowhead Pharmaceuticals Begins Dosing in Phase 1 Study of ARO-APOC3 for Treatment of Hypertriglyceridemia
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07 Mar 2019 |
AOBiome Doses First Patient in Phase 1b Clinical Trial of B244 for the Treatment of Pediatric Atopic Dermatitis
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07 Mar 2019 |
Alnylam Reports Positive Topline Results from ENVISION Phase 3 Study of Givosiran in Acute Hepatic Porphyria
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06 Mar 2019 |
NKMax America Announces FDA Approval of IND for Phase 1 Trial of SNK01 in Patients with Refractory Cancer
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02 Mar 2019 |
CARsgen Therapeutics Receives IND Clearance for BCMA-CAR-T Cells from the NMPA
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02 Mar 2019 |
Akcea and Ionis Receive Positive EU CHMP Opinion for WAYLIVRATM (volanesorsen)
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28 Feb 2019 |
Translate Bio Provides Updates on Cystic Fibrosis (CF) and Ornithine Transcarbamylase (OTC) Deficiency Programs
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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
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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
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25 Feb 2019 |
CRISPR Therapeutics and Vertex Announce Progress in Clinical Development Programs for the Investigational CRISPR/Cas9 Gene-Editing Therapy CTX001
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21 Feb 2019 |
Torque Announces Clinical Trial Collaboration with Merck
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21 Feb 2019 |
ReNeuron Group plc Announces Positive Preliminary Data in U.S. Retinitis Pigmentosa Trial
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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
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21 Feb 2019 |
Inovio announces first subject dosed with its DNA-encoded monoclonal antibody (dMAb) in first-ever human trial
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20 Feb 2019 |
Phase 1 Data Published in Nature Communications Show Potential of mRNA Encoding VEGF-A as a Regenerative Therapeutic
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20 Feb 2019 |
Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
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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
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20 Feb 2019 |
Modis Therapeutics Announces that MT1621 Receives Breakthrough Therapy Designation from FDA for the Treatment of TK2 Deficiency
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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)
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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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14 Feb 2019 |
Sarepta Announces FDA Acceptance of Golodirsen (SRP-4053) New Drug Application for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 53
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12 Feb 2019 |
Precigen Announces Clearance of IND to Initiate Phase 1 Study for First-in-Class PRGN-3005 UltraCAR-T to Treat Advanced Stage Platinum Resistant Ovarian Cancer Patients
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