DNA RNA and Cells

10 Dec 2017 Juno Therapeutics Highlights Key Translational Insights with JCAR017 in Patients with DLBCL
09 Dec 2017 BioMarin Provides 1.5 years of Clinical Data for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at 59th American Society of Hematology (ASH) Annual Meeting Concurrent with NEJM Publication
07 Dec 2017 Dicerna Announces First Human Dosed in Phase 1 Clinical Trial of DCR-PHXC for Treatment of All Forms of Primary Hyperoxaluria
07 Dec 2017 CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-thalassemia
07 Dec 2017 Vedanta Biosciences Announces Initiation of Phase 1a/1b Trial for New Drug Class of Rationally-Defined Bacterial Consortia Derived from the Human Microbiome
07 Dec 2017 Evolve BioSystems' Activated B. infantis EVC001 Demonstrates Substantial and Persistent Remodeling of the Infant Gut Microbiome
07 Dec 2017 Spark Therapeutics and Pfizer Announce Publication in The New England Journal of Medicine of Interim Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Hemophilia B
07 Dec 2017 GenSight Biologics reports positive long-term visual acuity gains and safety results from Phase I/II Study of GS010 for the treatment of Leber Hereditary Optic Neuropathy (LHON)
06 Dec 2017 Arrowhead Presents New Clinical Data Demonstrating a Sustained Host Response in Hepatitis B Patients Following RNAi Therapy
06 Dec 2017 DNAtrix Announces First Pediatric Patient Treated with Oncolytic Virus DNX-2401
06 Dec 2017 First patient enrolled in Phase 3 trial for ATIR101™ in adult patients with blood cancer
05 Dec 2017 Poseida Therapeutics Presents Preclinical Solid Tumor Data for P-PSMA-101 at the American Association for Cancer Research Prostate Cancer Meeting
05 Dec 2017 Adverum Biotechnologies Begins Patient Enrollment in the ADVANCE Phase 1/2 Clinical Trial for A1AT Deficiency
30 Nov 2017 Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
29 Nov 2017 Capricor Therapeutics Announces FDA Clearance of Investigational New Drug (IND) Application for CAP-1002
29 Nov 2017 TWOCELLS and Chugai Announce Performing Surgery of the First Patient in Phase III Trial for “gMSC®1,” a Regenerative Cellular Medicine for Chondrogenesis in the Knee
27 Nov 2017 Eureka Announces FDA Allowance of Investigational New Drug Application for ET190L1-ARTEMIS™ T cell Therapy in Relapsed and Refractory CD19+ Non-Hodgkin Lymphoma
20 Nov 2017 U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy
17 Nov 2017 Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
17 Nov 2017 bluebird bio Announces First Patient Treated in Northstar-3 (HGB-212), Phase 3 Study of LentiGlobin™ in Patients with Transfusion-Dependent β-Thalassemia (TDT) and β0/β0 Genotype
17 Nov 2017 Celgene Corporation and bluebird bio Announce bb2121 Anti-BCMA CAR-T Cell Therapy Has Been Granted Breakthrough Therapy Designation from FDA and Prime Eligibility from EMA for Relapsed and Refractory Multiple Myeloma
17 Nov 2017 Seattle Children's Opens First CAR T-Cell Immunotherapy Trial in the U.S. for Children and Young Adults With Leukemia that Targets CD22 and CD19 Proteins Simultaneously
16 Nov 2017 BioCanCell Announces Updated Results from the Phase 2 Clinical Trial of BC-819 and BCG in Patients with Bladder Cancer
15 Nov 2017 Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor's Investigational Cell Therapy
15 Nov 2017 Replimune Announces First Patients Dosed in a Phase 1/2 Clinical Trial of its RP1 Oncolytic Immunotherapy in Patients With Advanced Solid Tumors

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