DNA RNA and Cells

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
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
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
12 Feb 2019 Arcturus Therapeutics Reassumes Full Worldwide Rights to ARCT-810, a Clinical Development Candidate for Ornithine Transcarbamylase (OTC) Deficiency
12 Feb 2019 Adverum Biotechnologies Announces Presentation of Preclinical Long-Term Safety Data of ADVM-022 at 2019 Angiogenesis, Exudation, and Degeneration Conference
11 Feb 2019 Immunicum AB (publ) Announces First Patient Treated in Phase Ib/II ILIAD Combination Trial
09 Feb 2019 Finch Therapeutics’ Investigational Drug CP101 Granted Breakthrough Therapy Designation from FDA for Recurrent C. Difficile Infection
09 Feb 2019 uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B
07 Feb 2019 Sangamo Announces Interim Results Of Phase 1/2 CHAMPIONS Study Showing Preliminary Evidence Of In Vivo Genome Editing In Patients With MPS II Treated With SB-913
07 Feb 2019 Sangamo Announces Interim Results Of Phase 1/2 EMPOWERS Study Evaluating SB-318 Zinc Finger Nuclease (ZFN) In Vivo Genome Editing Demonstrating Increased Leukocyte IDUA Activity In Patients With MPS I
07 Feb 2019 Neurogene Announces Presentation of Preclinical Data for Gene Therapies to Treat AGU and CMT4J, Two Rare and Devastating Neurodegenerative Disorders
07 Feb 2019 Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy

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