DNA RNA and Cells

20 Nov 2017 U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy
17 Nov 2017 Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
17 Nov 2017 bluebird bio Announces First Patient Treated in Northstar-3 (HGB-212), Phase 3 Study of LentiGlobin™ in Patients with Transfusion-Dependent β-Thalassemia (TDT) and β0/β0 Genotype
17 Nov 2017 Celgene Corporation and bluebird bio Announce bb2121 Anti-BCMA CAR-T Cell Therapy Has Been Granted Breakthrough Therapy Designation from FDA and Prime Eligibility from EMA for Relapsed and Refractory Multiple Myeloma
17 Nov 2017 Seattle Children's Opens First CAR T-Cell Immunotherapy Trial in the U.S. for Children and Young Adults With Leukemia that Targets CD22 and CD19 Proteins Simultaneously
16 Nov 2017 BioCanCell Announces Updated Results from the Phase 2 Clinical Trial of BC-819 and BCG in Patients with Bladder Cancer
15 Nov 2017 Significant Improvements Reported in Duchenne Muscular Dystrophy Patients Treated with Capricor's Investigational Cell Therapy
15 Nov 2017 Replimune Announces First Patients Dosed in a Phase 1/2 Clinical Trial of its RP1 Oncolytic Immunotherapy in Patients With Advanced Solid Tumors
15 Nov 2017 Poseida Therapeutics Presents on the Development of Allogeneic and Autologous BCMA-Specific CAR-T Therapies at Precision: Blood Cancer Summit
15 Nov 2017 Sangamo Announces Treatment of First Patient in Landmark Phase 1/2 Clinical Trial Evaluating In Vivo Genome Editing for MPS II
14 Nov 2017 Akcea Initiates AKCEA-ANGPTL3-LRx Phase 2 Program in Patients with Rare Hyperlipidemias
13 Nov 2017 DNAtrix Virus Expressing T-Cell Co-stimulator OX40L (CD252) Presented at the 2017 Society for Immunotherapy of Cancer Meeting
13 Nov 2017 International Stem Cell Corporation Announces Positive Interim Clinical Results for Parkinson's Disease Clinical Trial
13 Nov 2017 Mesoblast Phase 2 Trial Results Show Early and Durable Effects of Single Mesenchymal Precursor Cell Infusion in Biologic Refractory Rheumatoid Arthritis Patients
12 Nov 2017 Three-year Follow-up Phase 3 Data Provide Additional Information on Efficacy, Durability and Safety of Investigational LUXTURNA™ (voretigene neparvovec) in Patients with Biallelic RPE65-mediated Inherited Retinal Disease
12 Nov 2017 CRISPR Therapeutics Highlights New Additions to Portfolio of Allogeneic CRISPR-based CAR-T Therapies at SITC Annual Meeting
09 Nov 2017 Abeona Enrolls First Subject in Spain in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
09 Nov 2017 NY-ESO Data Presented at the Connective Tissue Oncology Society (CTOS) Annual Meeting Confirm Potential of Adaptimmune’s SPEAR T-Cell Therapy
08 Nov 2017 Synlogic Reports Positive Top-Line Phase 1 Data Demonstrating Safety and Tolerability and Proof of Mechanism in Healthy Volunteers for SYNB1020, a Synthetic BioticTM Medicine for the Treatment of Hyperammonemia
08 Nov 2017 Fourth Gene Therapy Program Targets the Most Common Inherited Juvenile Macular Dystrophy
08 Nov 2017 AMAG Announces Autologous Cord Blood Cells Improve Motor Function in Some Children with Cerebral Palsy
08 Nov 2017 Alnylam Initiates ENVISION Phase 3 Clinical Study with Givosiran, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyrias (AHPs)
08 Nov 2017 Sarepta Therapeutics Announces FDA Clearance of IND for the Company’s PPMO Exon 51 Candidate, SRP-5051
07 Nov 2017 FDA Lifts Clinical Hold on Cellectis Phase 1 Clinical Trials with UCART123 in AML and BPDCN
07 Nov 2017 Ionis Announces Submission of New Drug Application (NDA) for Inotersen to the U.S. FDA

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