DNA RNA and Cells

31 Oct 2017 Mustang Bio Reports Preclinical Data on Regional Delivery of MB-103 HER2 Chimeric Antigen Receptor Engineered T (CAR T) Cells to Target HER2+ Breast Cancer Metastasis to the Brain
31 Oct 2017 Akcea and Ionis Report Positive Phase 1/2a Data with AKCEA-APOCIII-LRx
31 Oct 2017 Novartis submits application to FDA for KymriahTM (tisagenlecleucel) in adult patients with r/r DLBCL, seeking second indication for first-ever FDA approved CAR-T therapy
28 Oct 2017 Tocagen Presents Updated Durable Complete Response Data from Phase 1 Trial of Toca 511 & Toca FC in Recurrent High-Grade Glioma
26 Oct 2017 Transgene: First Patient Treated in a Phase 1/2a Trial (Oncovirac) of Novel Oncolytic Virus TG6002 in Recurrent Glioblastoma
26 Oct 2017 FDA Grants Breakthrough Therapy Designation for BioMarin's Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A
25 Oct 2017 Atara Biotherapeutics Announces Six of Ten Progressive Multiple Sclerosis (MS) Patients Experienced Clinical Improvements in an Ongoing Phase 1 Study of Autologous ATA190
24 Oct 2017 Arbutus Presents HBV Data at 2017 AASLD Liver Meeting
23 Oct 2017 Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting(R) 2017
21 Oct 2017 uniQure Announces Hemophilia B Gene Therapy Program To Enter Pivotal Study With FIX-Padua Variant in 2018
19 Oct 2017 AOBiome Therapeutics Reports Positive Efficacy Results from Phase 2b Clinical Trial of Ammonia Oxidizing Bacteria (AOB) for the Treatment of Acne Vulgaris
19 Oct 2017 OncoSec Presents Positive Phase 2 Data for ImmunoPulse® IL-12 in Combination with Pembrolizumab Demonstrating a Best Overall Response Rate (BORR) of 50% in Predicted Anti-PD-1 Non-Responder Melanoma Patients
19 Oct 2017 Atara Biotherapeutics Initiates 60 Patient, Global Phase 1 Clinical Study to Evaluate Allogeneic ATA188 in Patients with Progressive or Relapsing-Remitting Multiple Sclerosis (MS)
19 Oct 2017 Kite’s Yescarta™ (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy
18 Oct 2017 MeiraGTx Presents Preclinical Data on its Ocular Gene Therapy Studies for the Treatment of Leber's Congenital Amaurosis, Achromatopsia and X-Linked Retinitis Pigmentosa at ESGCT 2017 Congress in Berlin
17 Oct 2017 Dicerna Files Clinical Trial Application for DCR-PHXC, the Company’s Most Advanced GalXC™ Product Candidate, for Phase 1 Study in Primary Hyperoxaluria (PH)
17 Oct 2017 Incysus Announces FDA Approval of IND Application for a Phase 1 Study of a Gamma-Delta (γδ) T Cell Leukemia and Lymphoma Immunotherapy
16 Oct 2017 ZIOPHARM Oncology Announces First Patient Dosed in New Phase 1 Study of Ad-RTS-hIL-12 plus Veledimex for the Treatment of Pediatric Brain Tumors
16 Oct 2017 BrainStorm Enrolls First Patients in Phase 3 Trial of NurOwn® in ALS
13 Oct 2017 FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease
13 Oct 2017 miRagen Therapeutics Presents New Clinical Data Suggesting Positive Impact From Systemically Administered MRG-106 on Mycosis Fungoides Form of Cutaneous T-Cell Lymphoma at EORTC Global Task Force Meeting
13 Oct 2017 Ionis Pharmaceuticals Initiates Clinical Study of IONIS-MAPT Rx in Patients with Alzheimer's Disease
11 Oct 2017 Mustang Bio Announces $12.8 Million Grant to Fund Phase 1 Trial of CAR T Therapy MB-101 in Malignant Glioma
06 Oct 2017 Capricor Therapeutics Reports Intravenous Administration of CAP-1002 Shown to be Effective in Preclinical Models of Duchenne Muscular Dystrophy
06 Oct 2017 Abeona Announces Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa

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