DNA RNA and Cells

23 May 2018 FDA Grants Breakthrough Therapy Designation to Lenti-D™ for the Treatment of Cerebral Adrenoleukodystrophy
23 May 2018 TCR Engineered T-Cells are coming out of the shadow to the light
23 May 2018 Rocket Pharmaceuticals Presents Updated Data from Phase 1/2 Gene Therapy Trial of RP-L102 in Patients with Fanconi Anemia at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
23 May 2018 Cellectis: Approval of UCART123 Amendment in AML to Accelerate Clinical Development
23 May 2018 MaxCyte Reports Preclinical Results at ASGCT Annual Meeting Showing Efficient Correction of Sickle Cell Mutation in Hematopoietic Stem Cells (HSC)
23 May 2018 Precigen Announces First Patient Dosed in Phase 1 Clinical Study of INXN-4001
23 May 2018 Lupagen & Humanigen to explore point-of-care CAR-T therapy
23 May 2018 uniQure Presents New Data Demonstrating Clinical Benefit in Hemophilia B Patients with Pre-Existing Anti-AAV5 Neutralizing Antibodies
22 May 2018 BioMarin Provides 2 Years of Clinical Data in 6e13 vg/kg Dose from Ongoing Phase 1/2 Study in Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A at World Federation of Hemophilia 2018 World Congress
22 May 2018 Spark Therapeutics and Pfizer Announce Data from 15 Participants with Hemophilia B Showing Persistent and Sustained Factor IX Levels with No Serious Adverse Events
22 May 2018 Arrowhead Pharmaceuticals Presents New Preclinical Data on ARO-ENaC for Treatment of Cystic Fibrosis
22 May 2018 Editas Medicine Reports Data Demonstrating Subretinal Injection of EDIT-101 Well-tolerated in Non-human Primates
18 May 2018 Homology Medicines Presents Data from PKU Gene Therapy Program Demonstrating Long-Term Durability of Treatment in IND-Enabling Studies
18 May 2018 ToolGen, Inc., Demonstrates CRISPR/cas9 Gene Editing Improves Anti-Tumor Activity of Human CAR-T Cells
18 May 2018 Abeona Therapeutics Provides Clinical Update on MPS IIIA Gene Therapy Trial at the 21st Annual ASGCT Meeting
17 May 2018 Oncolytics Biotech® Announces Collaboration with Merck and Northwestern University Combining Keytruda® and REOLYSIN® in a Phase 2 Second Line Pancreatic Cancer Study
16 May 2018 Novo Nordisk increases commitment to stem cell-based therapies
16 May 2018 Turnstone Biologics Announces U.S. FDA Acceptance of IND for Immunotherapy Targeting HPV Positive Cancers
16 May 2018 Bioverativ and Sangamo Announce FDA Acceptance of IND Application for Gene-Edited Cell Therapy BIVV003 to Treat Sickle Cell Disease
15 May 2018 BioMarin Announces First Patient Dosed in Phase 1/2 Study Evaluating Valoctocogene Roxaparvovec Gene Therapy in Severe Hemophilia A Patients with Pre-existing AAV5 Antibodies
11 May 2018 Arrowhead Pharmaceuticals Presents Preclinical Data on Expanding Cardiometabolic Pipeline
11 May 2018 FDA Advisory Committee Votes in Favor of WAYLIVRA for Treatment of Familial Chylomicronemia Syndrome
10 May 2018 Krystal Biotech Announces Dosing of First Patient in the Phase 1/2 trial of KB103, a First-in-Class Topical Gene Therapy for the Treatment of Dystrophic Epidermolysis Bullosa
09 May 2018 miRagen Therapeutics Announces New Interim Data From Phase 1 Clinical Trial of Cobomarsen in Mycosis Fungoides
07 May 2018 The Medicines Company Announces Inclisiran Data Showing Significant Reductions in Potentially Harmful Subtypes of Bad Cholesterol (Atherogenic Lipoproteins) Linked to Heart Attack and Stroke

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