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BioWorld - Tuesday, December 23, 2025
Home » Topics » Gene therapy, BioWorld Science

Gene therapy, BioWorld Science
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Liver
Hematologic

Poseida Therapeutics advances nonviral liver-directed gene therapies

April 18, 2024
Poseida Therapeutics Inc. has announced progress with its fully nonviral liver-directed gene therapies.
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mRNA on digital background
Drug Design, Drug Delivery & Technologies

Circio establishes in vivo proof of concept for Circvec circular RNA platform

April 18, 2024
Circio Holding ASA has established technical in vivo proof of concept for its proprietary Circvec circular RNA platform by demonstrating statistically significant improvement in durability over mRNA-based expression.
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Medical vector illustration showing cross section of an eyeball with close up of the macula
Ocular

Saliogen nominates development candidate for Stargardt disease

April 18, 2024
Saliogen Therapeutics Inc. has nominated a development candidate, SGT-1001, for the treatment of Stargardt disease. SGT-1001 is being developed as a one-time, nonviral therapy to slow or stop the progressive loss of central vision in people with Stargardt disease, regardless of the type of mutation.
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Red blood cells, DNA
Hematologic

NHLBI grant supports Geneventiv’s development of universal gene therapy for hemophilia

April 17, 2024
Geneventiv Therapeutics Inc. has been awarded a Direct to Phase II Small Business Innovation Research (SBIR) grant for approximately $2.5 million from the National Heart Lung Blood Institute (NHLBI) at the National Institutes of Health (NIH) to support development of a universal gene therapy for hemophilia A or B with or without inhibitors.
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DNA, drug development concept art.
Neurology/Psychiatric

Voyager and Neurocrine nominate development candidate in GBA1 gene therapy program for Parkinson’s disease

April 16, 2024
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
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Musculoskeletal

Precision Biosciences presents promising results for PBGENE-PMM gene therapeutic

April 2, 2024
Mitochondria contain circular DNA encoding 37 genes that are crucial for oxidative phosphorylation.
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DNA in drug capsules, digital background
Neurology/Psychiatric

Atamyo’s gene therapy for LGMD2C/R5 cleared to enter clinic in Europe

March 27, 2024
Atamyo Therapeutics SAS has received clinical trial application (CTA) authorizations in Italy and France for ATA-200, its gene therapy for the treatment of γ-sarcoglycan related limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
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Scientist, microscope and dropper
Drug Design, Drug Delivery & Technologies

Samsung Ventures makes investment in Brickbio

March 19, 2024
Samsung Life Science Fund, created jointly between Samsung Biologics Co. Ltd., Samsung Bioepis Co. Ltd. and Samsung C&T, and managed by Samsung Venture Investment Corp., and Brickbio Inc. have announced Samsung’s investment in preclinical-stage biopharmaceutical company Brickbio.
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DNA illustration
Immuno-oncology

Asgard Therapeutics financing to advance in vivo cell reprogramming

March 14, 2024
Asgard Therapeutics AB has announced a €30 million (US$32.7 million) series A financing to advance its first-in-class in vivo cell reprogramming platform for immuno-oncology.
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DNA illustration
Endocrine/Metabolic

Fractyl Health reports preclinical data supporting RJVA-001 and Rejuva pancreatic gene therapy platform

March 13, 2024
Fractyl Health Inc. has reported promising new preclinical findings supporting RJVA-001, the first clinical candidate in its Rejuva pancreatic gene therapy platform. The company’s GLP-1 gene therapy candidate has potential to treat metabolic diseases, including obesity and type 2 diabetes.
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