DNA RNA and Cells

12 Dec 2021 New Data from SLN124 Healthy Volunteer Study Reinforce Broad Therapeutic Potential in Hematological Diseases
12 Dec 2021 Sangamo Therapeutics Announces Updated Preliminary SAR445136 Phase 1/2 Proof-of-Concept Data Showing Tolerability and Sustained Effects in Sickle Cell Disease
10 Dec 2021 Atara Biotherapeutics Announces Preliminary Results for ATA2271, a Next-Generation Autologous Mesothelin-targeted CAR T-cell Therapy for Solid Tumors, at ESMO Immuno-Oncology Congress 2021
09 Dec 2021 uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B
07 Dec 2021 Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2I/R9
07 Dec 2021 Taysha Gene Therapies Announces Late-Breaking Abstract and Poster Presentation on Positive Preclinical Data For TSHA-105 Demonstrating Therapeutic Potential for the Treatment of Epilepsy Caused by SLCA13A5 Deficiency
07 Dec 2021 Adicet Bio Announces Positive Interim Clinical Data From First-Ever Allogeneic, Off-The-Shelf, Gamma Delta CAR T Investigational Cell Therapy
02 Dec 2021 Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III
02 Dec 2021 Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle
30 Nov 2021 Krystal Biotech Announces Positive Topline Results from GEM-3 Pivotal Trial of VYJUVEK™ in Patients with Dystrophic Epidermolysis Bullosa
30 Nov 2021 Talaris Therapeutics Announces Initiation of Phase 2 Clinical Trial of FCR001 in Individuals with a Severe Form of Scleroderma
30 Nov 2021 European Medicines Agency (EMA) Validates Atara Biotherapeutics' Marketing Authorization Application for Tabelecleucel for the Treatment of Epstein-Barr Virus-Positive Post-Transplant Lymphoproliferative Disease
25 Nov 2021 Selecta Biosciences Provides Update on Phase 1/2 Clinical Trial of SEL-302 for the Treatment of Methylmalonic Acidemia
25 Nov 2021 AVROBIO Presents Clinician Experience with FAB-GT Clinical Trial and Updated Safety Data for Investigational Gene Therapies in Fabry Disease and Gaucher Disease Type 1
25 Nov 2021 Ionis announces that Pfizer reports topline results from Phase 2b clinical study of vupanorsen
24 Nov 2021 Synlogic Presents Data Demonstrating Reductions in Plasma Phenylalanine Levels in Patients with Phenylketonuria Treated with SYNB1618
21 Nov 2021 Voyager Therapeutics Announces Positive Preclinical Data from New Vectorized anti-HER2 Antibody Program at the 26th Society for Neuro-Oncology Annual Meeting
19 Nov 2021 Ionis initiates Phase 3 clinical program of donidalorsen in patients with hereditary angioedema
18 Nov 2021 Novome Biotechnologies Reports Positive Results from a Phase 1 Study of NOV-001
18 Nov 2021 Eureka Therapeutics Announces Initiation of Phase I/II ARYA-2 Clinical Trial of ET140203 ARTEMIS® T Cells to Treat Pediatric Liver Cancer
18 Nov 2021 Arrowhead Announces JNJ-75220795 in Development for NASH
16 Nov 2021 RIBOMIC Announces RBM-007 Phase 1 Clinical Trial Results for Achondroplasia
16 Nov 2021 Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease
16 Nov 2021 NKGen Biotech Announces Positive SNK01 Clinical Trial Results Presented at the Society for Immunotherapy of Cancer (SITC) 2021 Annual Meeting
16 Nov 2021 AZD8601 EPICCURE Phase II trial demonstrated safety and tolerability in patients with heart failure

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