DNA RNA and Cells

02 Apr 2020 Vir and Alnylam Expand Collaboration to Advance Investigational RNAi Therapeutics Targeting Host Factors for the Treatment of COVID-19
02 Apr 2020 Axial Biotherapeutics Announces Positive Topline Results from Phase 1b/2a Clinical Trial of AB-2004 for the Treatment of Autism Spectrum Disorder
02 Apr 2020 Biogen Announces First Patient Treated With Higher Dose of SPINRAZA® (nusinersen) in Phase 2/3 DEVOTE Study
01 Apr 2020 ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421a for Usher Syndrome and Provides Business Update
01 Apr 2020 Kleo Pharmaceuticals Enters into Research Collaboration with Green Cross LabCell (GCLC) To Rapidly Develop COVID-19-Targeting Allogeneic NK Cell Combination Therapy
01 Apr 2020 BiomX Announces Positive Topline Data from Phase 1 Study for Lead Candidate BX001 for Acne-Prone Skin
01 Apr 2020 Dicerna Provides Initial Observations From PHYOX™3 Trial of Nedosiran for Treatment of Primary Hyperoxaluria and an Update on Data Presentation Plan
01 Apr 2020 FDA accepts Mesoblast’s Biologics Licence Application for Ryoncil™ and agrees to priority review
01 Apr 2020 Horizon Discovery and Pharmahungary report positive early-stage results for novel micro-RNA therapeutic for ischemic heart diseases
01 Apr 2020 FDA Accepts Investigational New Drug Application for CRISPR/Cas9-Based Sickle Cell Disease Therapeutic Candidate Developed Under Collaboration with Intellia Therapeutics
31 Mar 2020 Bristol Myers Squibb and bluebird bio Announce Submission of Biologics License Application (BLA) for Anti-BCMA CAR T Cell Therapy Idecabtagene Vicleucel (Ide-cel, bb2121) to FDA
31 Mar 2020 Generation Bio and Vir Biotechnology to Collaborate on Research to Leverage Scalable Non-Viral Gene Therapy Platform for Durable Production of Monoclonal Antibodies Against Coronavirus That Causes COVID-19
31 Mar 2020 Spray-On Gene Therapy With RenovaCare SkinGun™ in New Study Underway at King’s College London
30 Mar 2020 Clinical outcomes of Mesoblast’s cell therapy in end-stage ischemic heart failure presented at American College of Cardiology virtual scientific sessions
30 Mar 2020 Daiichi Sankyo Submits Application for CAR T Therapy Axicabtagene Ciloleucel for Treatment of Patients with Certain Relapsed/Refractory B-cell Lymphomas in Japan
29 Mar 2020 AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
27 Mar 2020 uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial of AMT-061 (Etranacogene Dezaparvovec) in Hemophilia B
27 Mar 2020 Arbutus Announces Positive Preliminary Phase 1a/1b Clinical Trial Results for AB-729, a Proprietary GalNAc Delivered RNAi Compound in Development for People Living with Chronic Hepatitis B
26 Mar 2020 VBL Therapeutics Announces Positive Outcome of the Interim Analysis in the OVAL Phase 3 Ovarian Cancer Pivotal Study
24 Mar 2020 MaxCyte and Allogene Therapeutics Sign Clinical and Commercial License Agreement to Enable the Advancement of Allogeneic CAR T (AlloCAR T™) Therapies
24 Mar 2020 Triumvira Immunologics and Lonza Announce Collaboration to Develop Innovative Point-of-Care Manufacturing for TAC T-Cell Therapeutic Targeting Solid Tumors
24 Mar 2020 First-of-its-kind Engineered Macrophage Cell Therapy Platform Shows Ability to Reduce Tumor Burden and Activate Anti-Tumor Immunity, Improving Overall Survival in Pre-Clinical Cancer Models
24 Mar 2020 AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2
24 Mar 2020 Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing
23 Mar 2020 Regulatory authorities approve JTA Phase III study in osteoarthritic knee pain and ALLOB Phase IIb study in difficult fractures

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