DNA RNA and Cells

24 Nov 2020 Alnylam Announces U.S. Food and Drug Administration (FDA) Approval of OXLUMO™ (lumasiran), the First and Only Treatment Approved for Primary Hyperoxaluria Type 1 to Lower Urinary Oxalate Levels in Pediatric and Adult Patients
24 Nov 2020 Ionis announces third investigational antisense medicine to treat nonalcoholic steatohepatitis (NASH) enters development
23 Nov 2020 Catamaran Bio Launches with $42 Million Financing to Develop Off the Shelf CAR-NK Cell Therapies to Treat Solid Tumors
22 Nov 2020 DNAtrix Announces Positive Data from Phase 2 CAPTIVE (KEYNOTE-192) Study with DNX-2401 in Patients with Recurrent Glioblastoma Highlighted in an Oral Late-Breaking Presentation During Society for Neuro-oncology (SNO) Annual Meeting
20 Nov 2020 INOVIO Presents Clinical Results of its DNA Medicines INO-5401 + INO-9012 in Novel Combination with PD-1 Inhibitor Libtayo® (cemiplimab) in the Treatment of Newly Diagnosed Glioblastoma Multiforme at Society for Neuro-Oncology 2020 Annual Meeting
20 Nov 2020 Glycostem initiates phase I/IIa (pivotal) trial to evaluate the safety and efficacy of oNKord® in patients with Acute Myeloid Leukemia (AML)
20 Nov 2020 uniQure Announces Positive Top-Line Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
20 Nov 2020 Novartis secures exclusive rights for potential acute respiratory distress syndrome cell therapy
19 Nov 2020 Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD)
19 Nov 2020 Bio-Path Holdings Announces First Patient Dosed in Phase 1 Clinical Trial of BP1002
19 Nov 2020 Durable Responses with ADP-A2M4 in Synovial Sarcoma with Confirmed Responses in 44% of Patients and Disease Control Rate of 94% Presented at CTOS
19 Nov 2020 Alnylam Receives Approval for OXLUMO™ (lumasiran) in the European Union for the Treatment of Primary Hyperoxaluria Type 1 in All Age Groups
19 Nov 2020 Bristol secures £45M to advance gene therapy treatment of chronic kidney diseases
19 Nov 2020 Genoscience Pharma completes successful phase 1b trial evaluating GNS561, PPT-1 inhibitor, in patients with primary and secondary liver cancer
18 Nov 2020 FDA Lifts Clinical Hold on MELANI-01 Study Evaluating Cellectis’ Product Candidate UCARTCS1 in Multiple Myeloma
18 Nov 2020 Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet’s Investigational Gene Therapy for Wilson Disease
17 Nov 2020 Silence Therapeutics Presents Positive Pre-Clinical Data for SLN360 for the Treatment of Elevated Lipoprotein(a) at American Heart Association 2020
16 Nov 2020 Dicerna Announces FDA Acceptance of Lilly’s Investigational New Drug (IND) Application for First GalXC™ RNAi Candidate Under Companies’ Global Research Collaboration and Licensing Agreement
16 Nov 2020 Arbutus Announces Presentation of Phase 1a/1b Clinical Trial Results for AB-729 in Chronic Hepatitis B Subjects at The Liver Meeting Digital Experience™, The American Association for the Study of Liver Diseases Meeting
15 Nov 2020 Adverum Biotechnologies Announces Positive Interim Data from Cohorts 1-4 from OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for Wet AMD
13 Nov 2020 Alnylam Reports Positive Interim Results from Ongoing Phase 1 Study of ALN-AGT, an Investigational RNAi Therapeutic for the Treatment of Hypertension
13 Nov 2020 Arrowhead Interim Clinical Data Demonstrate ARO-AAT Treatment Improved Multiple Biomarkers of Alpha-1 Liver Disease
13 Nov 2020 Nkarta Announces Treatment of First Patient in First-in-Human Clinical Trial of Engineered NKG2D-Based NK Cell Cancer Immunotherapy NKX101
13 Nov 2020 MaaT Pharma Treats First Acute Myeloid Leukemia Patient in Phase 1 Clinical Trial to Evaluate Capsule Formulation of Microbiome Restoration Biotherapeutic
13 Nov 2020 Atara Biotherapeutics Presents New Preclinical Data on ATA3271, a Next-Generation Allogeneic Mesothelin-Targeted CAR T to Treat Solid Tumors, at the 35th Society for Immunotherapy of Cancer Annual Meeting (SITC 2020)

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