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Clinical Trials

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Number of results: 4542

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Date Product Disease Phase Company Therapeutic area
2019-06-07 ADV7103 (tripotassium citrate monohydrate and potassium hydrogen carbonate) cystinuria 2-3 Advicenne (France) Rare diseases - Kidney diseases - Renal 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
2019-05-01 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
2019-04-29 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
2019-04-16 AVXS-101 (adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene) spinal muscular atrophy (SMA) Type 1 3 Avexis (USA - IL), now a Novartis company (Switzerland) Rare diseases - Genetic diseases - Neuromuscular diseases
2019-04-15 ARO-AAT (N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene) alpha-1 antitrypsin deficiency 2-3 Arrowhead Pharmaceuticals (USA - CA) Rare diseases - Genetic diseases - Liver diseases - Hepatic diseases
2019-04-04 ATL1102 Duchenne muscular dystrophy (DMD) 2 Antisense Therapeutics (Australia) Rare diseases - Genetic diseases - Neuromuscular diseases
2019-03-24 aducanumab (BIIB037) Alzheimer's disease (AD) 1b Biogen (USA - MA) Neurodegenerative diseases
2019-03-21 aducanumab (BIIB037) Alzheimer's disease 3 Biogen (USA - MA) Neurodegenerative diseases
2019-03-21 aducanumab (BIIB037) Alzheimer's disease 3 Biogen (USA - MA) Neurodegenerative diseases
2019-03-18 lipid nanoparticle formulation for systemic delivery of mRNA Fabry disease preclinical Translate Bio (USA - MA) Rare diseases - Genetic diseases
2019-03-11 QR-421a (2'-O-(2-methoxyethyl)-modified antisense oligonucleotide targeting exon 13 in the USH2A gene) Usher syndrome 2A or non-syndromic retinitis pigmentosa (RP) caused by an exon 13 mutation of the causative USH2A gene 1-2 ProQR Therapeutics (The Netherlands) Rare diseases - Genetic diseases - Ophtalmological diseases
2019-03-11 RTX-134 phenylketonuria 1 Rubius Therapeutics (USA - MA) Rare diseases - Genetic diseases - Metabolic diseases
2019-03-11 ALN-AGT hypertension 1 Alnylam Therapeutics (USA - MA) Cardiovascular diseases
2019-03-11 ARO-APOC3 hypertriglyceridemia, Familial Chylomicronemia Syndrome 1 Arrowhead Pharmaceuticals (USA - CA) Cardiovascular diseases - Metabolic diseases
2019-03-11 AXO-AAV-GM2 advanced infantile Tay-Sachs disease 1-2 Axovant Sciences (USA - NY) Rare diseases - Genetic diseases - Neurodegenerative diseases
2019-03-06 givosiran (ALN-AS1) acute hepatic porphyria 3 Alnylam Therapeutics (USA - MA) Rare diseases - Metabolic diseases
2019-02-27 MYO-101 (self-complementary non-replicating, recombinant adeno-associated virus serotype rhesus 74 (rh 74) expressing the human beta-sarcoglycan gene under the control of the muscle creatine kinase (MHCK7) promoter) beta-sarcoglycanopathy, also known as limb girdle muscular dystrophy (LGMD) type 2E 1-2a Myonexus Therapeutics (USA - MA) Rare diseases - Genetic diseases - Neuromuscular diseases