DNA RNA and Cells

18 Jan 2017 Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis
18 Jan 2017 Servier appoints MaSTherCell for the development of its CAR-T cell therapy manufacturing platform
18 Jan 2017 Jazz Pharmaceuticals Announces First Patient Enrolled in Phase 3 Clinical Trial Evaluating Defibrotide for the Potential Prevention of VOD in High Risk Patients
14 Jan 2017 New Data Show SPINRAZA™ (nusinersen) Significantly Reduces Risk of Death or Permanent Ventilation in Infantile-Onset Spinal Muscular Atrophy
13 Jan 2017 Pluristem Completes Patient Enrollment in a Large Multinational Phase II Trial in Intermittent Claudication
12 Jan 2017 CureVac Provides Clinical Update at 35th Annual J.P. Morgan Healthcare Conference
09 Jan 2017 Adaptimmune confirms GSK Nomination of Second Adaptimmune Target under Strategic Multi-Target Collaboration
09 Jan 2017 Adaptimmune Announces FDA Acceptance of IND Application for Affinity Enhanced T-Cell Therapy Targeting MAGE-A4 in Multiple Solid Tumors
09 Jan 2017 The Medicines Company Announces Positive Top-Line Results from Day 180 Interim Analysis in Ongoing ORION-1 Phase 2 Study of Inclisiran (formerly, PCSK9si)
07 Jan 2017 Genprex Announces Positive Interim Data from Phase II Clinical Trial of Oncoprex™ for Late Stage Non-Small Cell Lung Cancer
07 Jan 2017 Allele to Develop iPSC-Derived Cell Therapies with Focus on Diabetes and Spinal Cord Injury
05 Jan 2017 Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For SB-525 Gene Therapy Program For Hemophilia A
04 Jan 2017 Rebiotix Treats First Patient in Phase 1 Study of RBX7455, an Orally Delivered Broad-Spectrum Non-Frozen Microbiota Capsule for Recurrent Clostridium difficile Infection
04 Jan 2017 Ionis Pharmaceuticals Reports Positive Data from Phase 2 Study of IONIS-GCGR Rx in Patients with Type 2 Diabetes
04 Jan 2017 Cellectis Submits IND Application for UCART123, an Allogeneic Gene Edited CAR T-Cell Product Candidate, in AML and BPDCN
03 Jan 2017 Kite Pharma Submits Investigational New Drug (IND) Application for KITE-718, an Investigational Next Generation T-Cell Receptor (TCR) Therapy Targeting Cancer Antigens MAGE A3 and MAGE A6
29 Dec 2016 CardiAMP Pivotal Heart Failure Trial Initiated at Two US Centers
29 Dec 2016 City of Hope Researchers Achieve Remission Using CAR-T Cell Therapy for Aggressive Brain Tumors
24 Dec 2016 SPINRAZA™ (nusinersen) Approved in U.S. to Treat Broad Range of Patients with Spinal Muscular Atrophy
21 Dec 2016 Lion Biotechnologies Announces New Sponsored Research and Clinical Grant Agreements with H. Lee Moffitt Cancer Center and Research Institute
21 Dec 2016 MaxCyte Announces Strategic Immuno-Oncology Collaboration to Advance New Generation of CAR-based Cell Therapies
21 Dec 2016 GenSight Biologics Reports Sustained Visual Acuity Gain at 78 Weeks in Its Phase I/II Study with GS010 for the Treatment of Leber’s Hereditary Optic Neuropathy (LHON)
21 Dec 2016 Juno Therapeutics’ and Celgene Corporation’s Investigational Drug JCAR017 Granted Breakthrough Therapy Designation from FDA and Priority Medicines Eligibility from EMA for Relapsed/Refractory Diffuse Large B-cell Lymphoma
21 Dec 2016 Ionis and AstraZeneca Advance First Generation 2.5 LICA Drug into Preclinical Development to Treat Cardiovascular Disease
19 Dec 2016 Akcea and Ionis Announce Positive Results from Compass Phase 3 Study of Volanesorsen

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