DNA RNA and Cells

02 Aug 2016 Abeona Therapeutics Provides Update on Initial Subjects in Sanfilippo Type A Gene Therapy Trial, Demonstrating Encouraging Early Biopotency Signals
02 Aug 2016 Takeda and TiGenix Announce Publication in The Lancet of 24-Week Results of the Phase 3 ADMIRE-CD Trial Investigating Cx601 in the Treatment of Complex Perianal Fistulas in Patients with Crohn's Disease
01 Aug 2016 Biogen and Ionis Pharmaceuticals Report Nusinersen Meets Primary Endpoint at Interim Analysis of Phase 3 ENDEAR Study in Infantile-Onset Spinal Muscular Atrophy
30 Jul 2016 Seres Therapeutics Announces Interim Results from SER-109 Phase 2 ECOSPOR Study in Multiply Recurrent Clostridium difficile Infection
28 Jul 2016 uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B Demonstrating up to 9 Months of Sustained Levels of Factor IX Activity and Therapeutic Effect
28 Jul 2016 BioMarin Provides Positive Proof-of-Concept Data for BMN 270 Gene Therapy in Hemophilia A in Late Breaking Oral Presentation at the World Federation of Hemophilia (WFH) 2016 World Congress
28 Jul 2016 Fibrocell Announces First Subjects Enrolled in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa
27 Jul 2016 Stemedica Begins First Clinical Trials In The U.S. Using Adult Allogeneic Stem Cells To Treat Alzheimer's Disease
27 Jul 2016 Sangamo BioSciences Announces New Gene Therapy Clinical Development Program for Treatment Of Hemophilia A
26 Jul 2016 Alnylam Announces New Positive Interim Phase 1 Study Results for Fitusiran, a Once-Monthly, Subcutaneous, Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia and Rare Bleeding Disorders
26 Jul 2016 AstraZeneca and Moderna Announce Filing of First Clinical Trial Application in Messenger RNA Therapeutics™ Collaboration
25 Jul 2016 FDA Lifts Clinical Hold On CGF166
21 Jul 2016 Targazyme Enrolls First Patient in Phase 1/2 Study of TZ101 Fucosylated Regulatory T Cells to Treat and Prevent Graft vs Host Disease in the Transplant Setting
20 Jul 2016 AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
20 Jul 2016 Arbutus Reports Topline Results from TKM-PLK1 HCC Clinical Trial
19 Jul 2016 ZIOPHARM Provides Update Regarding Phase I Study of Gene Therapy Candidate Ad-RTS-hIL-12 in Brain Cancer
18 Jul 2016 BrainStorm Announces Positive Top Line Results from the U.S. Phase 2 Study of NurOwn® in Patients with Amyotrophic Lateral Sclerosis (ALS)
15 Jul 2016 ZIOPHARM Issues Statement Regarding Phase I Study of Gene Therapy Candidate Ad-RTS-hIL-12 in Brain Cancer
14 Jul 2016 Atara Bio Doses First Patient in Multi-Center Expanded Access Protocol for Study of Allogeneic Epstein-Barr Virus Specific Cytotoxic T-lymphocytes (EBV-CTLs) in EBV-Associated Lymphomas and Lymphoproliferative Disorders
13 Jul 2016 Juno Therapeutics to Resume JCAR015 Phase II ROCKET Trial
12 Jul 2016 ZIOPHARM Announces Plans for Phase I Clinical Trial with CD33 CAR-T Cell Therapy Targeting Acute Myeloid Leukemia
12 Jul 2016 MyoKardia Announces Topline Clinical Data Supporting Advancement of MYK-461 to Phase 2 PIONEER-HCM Study in Symptomatic, Obstructive Hypertrophic Cardiomyopathy Patients
11 Jul 2016 First Patient is Randomized in STEMTRA Trial For Traumatic Brain Injury
08 Jul 2016 Alnylam Initiates Phase 1/2 Clinical Trial for ALN-HBV, an Investigational RNAi Therapeutic for the Treatment of Chronic Hepatitis B Virus Infection
08 Jul 2016 Kite Pharma Completes Enrollment of All Patients with Diffuse Large B-Cell Lymphoma (DLBCL) in Pivotal Multi-Center Phase 2 Study of KTE-C19 (ZUMA-1)

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