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Clinical Trials

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Number of results: 4557

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Date Product Disease Phase Company Therapeutic area
2019-08-01 scAVV9.CB.CLN6 CLN6 Batten disease 1-2 Amicus Therapeutics (USA - NJ) Paragon Gene Therapy, a unit of Catalent (USA - NJ) Rare diseases - Genetic diseases - Neurological diseases - Neurodegenerative diseases
2019-07-15 AVR-RD-01 Fabry disease 2 Avrobio (USA - MA) Rare diseases - Genetic diseases
2019-07-11 GS010 Leber hereditary optic neuropathy (LHON) 3 Gensight Biologics (France) Rare diseases - Genetic diseases - Ophtalmological diseases
2019-07-01 Oncoprex™ non-small cell lung cancer (NSCLC) Genprex (USA - TX) Cancer - Oncology
2019-06-18 BMN 111 (vosoritide) achondroplasia 2 Biomarin Pharmaceutical (USA - CA) Rare diseases - Genetic diseases
2019-06-18 BMN 111 (vosoritide) achondroplasia 3 Biomarin Pharmaceutical (USA - CA) Rare diseases - Genetic diseases
2019-06-17 lumasiran Primary Hyperoxaluria Type 1 (PH1) 3 Alnylam Therapeutics (USA - MA) Rare diseases - Genetic diseases
2019-06-07 ADV7103 (tripotassium citrate monohydrate and potassium hydrogen carbonate) cystinuria 2-3 Advicenne (France) Rare diseases - Kidney diseases - Renal diseases
2019-06-04 leriglitazone (MIN-102), Friedreich’s Ataxia 2 Minoryx Therapeutics (Spain) Rare diseases - Neurodegenerative diseases - Neurological diseases
2019-06-02 isatuximab relapsed/refractory multiple myeloma 3 Sanofi (France) Cancer - Oncology
2019-05-31 ALN-AGT hypertension 1 Alnylam Therapeutics (USA - MA) Cardiovascular diseases
2019-05-28 BMN 270 - valoctocogene roxaparvovec- adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene hemophilia A 1-2 Biomarin Pharmaceutical (USA - CA) Rare diseases - Genetic diseases - Hematological diseases
2019-05-15 GS010- adeno-associated viral vector containing the human NADH dehydrogenase 4 gene Leber hereditary optic neuropathy (LHON) 3 Gensight Biologics (France) Ophtalmological diseases - Genetic diseases - Rare diseases
2019-05-08 IDE196 metastatic uveal melanoma and other solid tumors harboring GNAQ or GNA11 (GNAQ/11) mutations and PKC fusions 1-2 Ideaya Biosciences (USA - CA) Cancer - Oncology
2019-05-07 GS030 retinitis pigmentosa 1-2 Gensight Biologics (France) Rare diseases - Genetic diseases - Ophtalmological diseases
2019-05-01 bb2121 anti-BCMA CAR T cells multiple myeloma 1 Bluebird bio (USA - MA) Celgene (USA - NJ) Cancer - Oncology
2019-04-30 AXO-Lenti-PD (also known as OXB-102) Parkinson's disease 1-2 Axovant Sciences (USA - NY) Oxford Biomedica (UK) Neurological diseases - CNS diseases
2019-04-30 AAV-vector containing engineered hGAA Pompe disease preclinical Amicus Therapeutics (USA - NJ) Rare diseases - Genetic diseases
2019-04-29 AVR-RD-01  Fabry disease 2 Avrobio (USA - MA) Rare diseases - Genetic diseases
2019-04-24 JNJ-3989 (formerly ARO-HBV) chronic hepatitis B virus (HBV) infection 1-2 Arrowhead Pharmaceuticals (USA - CA) Infectious diseases