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UniQure's Stock Is Trading Higher As FDA Lifts Clinical Hold On Its Hemophilia B Gene Therapy Program
Monday, April 26, 2021 - 8:34am | 333Read More...The FDA has removed the clinical hold on uniQure NV's (NASDAQ: QURE) hemophilia B gene therapy program after determining the company had satisfactorily addressed all identified issues related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE...
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Vertex Pharma Inks Research, Licensing Agreement With Obsidian Therapeutics For Controllable Genetic Therapies
Thursday, April 22, 2021 - 9:06am | 207Read More...Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and Privately-held Obsidian Therapeutics have entered into a research collaboration and licensing agreement to discover novel therapies that regulate gene editing. The collaboration leverages Obsidian's cytoDRiVE platform...
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Taysha Gene Therapies Stock Is Trading Higher After Buying Rights To New Gene Therapy Candidate For Neurodegenerative Disease
Monday, April 12, 2021 - 11:47am | 317Read More...Taysha Gene Therapies Inc (NASDAQ: TSHA) has unveiled a deal for global rights to the AAV9 gene therapy program, now known as TSHA-120, to treat giant axonal neuropathy (GAN) Under the terms of the agreement, the GAN patient advocacy group will receive an upfront payment of $5....
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Dicerna's RNAi Technology Shows Encouraging Action Against CNS Targets In Animal Studies
Tuesday, March 30, 2021 - 1:59pm | 253Read More...Dicerna Pharmaceuticals Inc (NASDAQ: DRNA) has announced new data from preclinical studies of its GalXC-Plus RNAi technology demonstrating its potential to deliver messenger RNA (mRNA) knockdown against prespecified gene targets across the central nervous system (CNS) and to...
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Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient
Monday, March 29, 2021 - 9:26am | 464Read More...Uniqure NV (NASDAQ: QURE) has reported results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec, its hemophilia B gene therapy candidate. What Happened: The...
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Solid Bio's DMD Gene Therapy Shows Promising Early Data, No New Safety Concerns, But Fails To Cheer Investors
Tuesday, March 16, 2021 - 7:57am | 591Read More...Solid Biosciences Inc (NASDAQ: SLDB) reported interim functional, biomarker data, and patient-reported outcome measures from its ongoing IGNITE Phase 1/2 trial of its lead gene therapy candidate, SGT-001 in Duchenne muscular dystrophy (DMD). While IGNITE-DMD was designed...
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BioMarin's Hemophilia Gene Therapy Scores RMAT Designation In US
Monday, March 8, 2021 - 1:05pm | 272Read More...The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) valoctocogene roxaparvovec investigational gene therapy for the treatment of adults with severe hemophilia A. RMAT is an expedited...
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Benitec Biopharma's Gene Therapy For Rare Muscle Disorder Shows Encouraging Action In Animal Studies
Wednesday, February 24, 2021 - 10:26am | 354Read More...Benitec Biopharma Inc (NASDAQ: BNTC) reports an interim analysis of the BB-301 Pilot Dosing study conducted in large animal subjects. BB-301 a genetic medicine directly injected into the pharyngeal muscles. It employs the proprietary platform, which allows for a "Silence...
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Selecta Biosciences/Asklepios Start Early-Stage Study To Assess ImmTOR In Gene Therapy
Wednesday, February 17, 2021 - 10:55am | 195Read More...Selecta Biosciences Inc (NASDAQ: SELB) and Asklepios BioPharmaceutical, a subsidiary of Bayer AG (OTCMKTS: BAYRY), have initiated a Phase 1 dose-escalation trial evaluating SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) without...
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AbbVie, Caribou Biosciences Ink Licensing Agreement For (CAR)-T Cell Therapeutics
Wednesday, February 10, 2021 - 11:04am | 245Read More...AbbVie Inc (NYSE: ABBV) and Caribou Biosciences have entered into a multi-year collaboration and license agreement to research and develop chimeric antigen receptor (CAR)-T cell therapeutics. AbbVie will utilize Caribou's Cas12a CRISPR hybrid RNA-DNA genome editing and cell...
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Avrobio Shares Rally On Fabry Disease Gene Therapy Encouraging Benefit In Early-Stage Study
Monday, February 8, 2021 - 9:07am | 350Read More...Avrobio Inc (NASDAQ: AVRO) has reported positive data from the ongoing Phase 2 FAB-GTi Phase 1/2 trial evaluating AVR-RD-01, an ex vivo lentiviral gene therapy for Fabry disease. 100% reduction, or complete clearance, of the toxic substrate in the kidney biopsy, was...
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Dosing Underway In Sio Gene's Early-Stage Trial For GM2 Gangliosidosis Gene Therapy
Wednesday, February 3, 2021 - 12:49pm | 222Read More...Sio Gene Therapies Inc (NASDAQ: SIOX) has dosed the first patient in Phase 1/2 trial evaluating AXO-AAV-GM2 investigational gene therapy to treat GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease, a rare genetic disorder that progressively destroys nerve...
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Bayer Acquires AskBio For $4B To Strengthen Its Gene Therapy Platform
Monday, October 26, 2020 - 6:12am | 426Read More...German pharma and life sciences company Bayer AG (OTC: BAYRY) has acquired U.S.-based gene therapy biotech company Asklepios Biopharmaceutical Inc (AskBio) in a deal valued up to $4 billion. The purchase price of $2 billion will be paid upfront, and the remaining half...
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BioMarin Strikes Gene Therapy Partnership Targeting Rare Genetic Cardiomyopathies
Monday, May 4, 2020 - 10:43am | 415Read More...BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is beefing up its gene therapy portfolio. The large-cap biopharma BioMarin said it has entered into a pre-clinical collaboration and licensing agreement with DiNAQOR, a gene therapy platform company headquartered in Switzerland, to develop...
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Regulators Give Roche Green Light For $4.3B Spark Therapeutics Acquisition
Tuesday, December 17, 2019 - 10:21am | 297Read More...Roche Holding (OTCQX: RHHBY) has received approval from the Federal Trade Commission and Britain’s Competition and Markets Authority to acquire Spark Therapeutics, Inc. (NASDAQ: ONCE), the companies announced Tuesday. The regulatory authorities concluded the deal would not hurt...
