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12 Dec 2021 |
New Data from SLN124 Healthy Volunteer Study Reinforce Broad Therapeutic Potential in Hematological Diseases
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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
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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
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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
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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
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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
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07 Dec 2021 |
Adicet Bio Announces Positive Interim Clinical Data From First-Ever Allogeneic, Off-The-Shelf, Gamma Delta CAR T Investigational Cell Therapy
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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
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02 Dec 2021 |
Astellas and Dyno Therapeutics Announce Research Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle
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30 Nov 2021 |
Krystal Biotech Announces Positive Topline Results from GEM-3 Pivotal Trial of VYJUVEK™ in Patients with Dystrophic Epidermolysis Bullosa
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30 Nov 2021 |
Talaris Therapeutics Announces Initiation of Phase 2 Clinical Trial of FCR001 in Individuals with a Severe Form of Scleroderma
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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
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25 Nov 2021 |
Selecta Biosciences Provides Update on Phase 1/2 Clinical Trial of SEL-302 for the Treatment of Methylmalonic Acidemia
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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
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25 Nov 2021 |
Ionis announces that Pfizer reports topline results from Phase 2b clinical study of vupanorsen
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24 Nov 2021 |
Synlogic Presents Data Demonstrating Reductions in Plasma Phenylalanine Levels in Patients with Phenylketonuria Treated with SYNB1618
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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
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19 Nov 2021 |
Ionis initiates Phase 3 clinical program of donidalorsen in patients with hereditary angioedema
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18 Nov 2021 |
Novome Biotechnologies Reports Positive Results from a Phase 1 Study of NOV-001
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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
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18 Nov 2021 |
Arrowhead Announces JNJ-75220795 in Development for NASH
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16 Nov 2021 |
RIBOMIC Announces RBM-007 Phase 1 Clinical Trial Results for Achondroplasia
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16 Nov 2021 |
Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease
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16 Nov 2021 |
NKGen Biotech Announces Positive SNK01 Clinical Trial Results Presented at the Society for Immunotherapy of Cancer (SITC) 2021 Annual Meeting
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16 Nov 2021 |
AZD8601 EPICCURE Phase II trial demonstrated safety and tolerability in patients with heart failure
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