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Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Continues to Show Safety and Efficacy in Phase 1/2 Trial
05 Feb 2025 01:05 GMT
… virus (AAV) vector-based gene therapy, has continued to show safety … to treatment with the gene therapy, and that GCase expression … range following treatment with the gene therapy. Although, they noted … trial of FLT201 AAV gene therapy in adult patients with …
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Ocugen, Inc. receives European Medicines Agency ATMP designation for new gene therapy
05 Feb 2025 03:46 GMT
… review timeline of the new gene therapy. The classification will also allow … potentially provide this novel modifier gene therapy candidate to all RP patients … Medicinal Product Classification for Modifier Gene Therapy Candidate OCU400 for Retinitis Pigmentosa …
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Senator Brandon Shippy Introduces S1036, Human Gene Therapy Moratorium
05 Feb 2025 01:19 GMT
… moratorium on the administration of gene therapy products for infectious disease indications … pause the use of experimental gene therapy shots while we further assess … . This bill only applies to gene therapy for infectious disease indications (immunizations …
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Italian charity seeks EMA approval for rescued gene therapy
04 Feb 2025 23:25 GMT
… in the European Union.
The gene therapy, named etuvetidigene autotemcel, was developed … (EMA) for evaluating the gene therapy. Etuvetidigene autotemcel is designed as … combined immunodeficiency (ADA-SCiD). That gene therapy also originated in the laboratories …
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USC receives grant for gene therapy targeting glioblastoma
04 Feb 2025 23:25 GMT
… .
The treatment is the first gene therapy for this aggressive brain cancer … , a surgical procedure for administering gene therapy.
USC investigators are developing a … laboratory model outcomes.
Cell & Gene Therapy coverage on Pharmaceutical Technology is …
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NJBioFutures Launches to Develop Cell and Gene Therapy Workforce
04 Feb 2025 22:05 GMT
… certification programs on cell and gene therapy biomanufacturing, biotechnology and biopharmaceuticals. These …
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Gene Therapy Shows Promise for Rare Bone Disease in Mice
04 Feb 2025 20:50 GMT
… treating HPP will be a gene therapy in which a single … safety and effectiveness of a gene therapy for HPP.
The new … including different dosages of the gene therapy, tests in male and female … rather than infants, the gene therapy was more effective on female …
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New gene therapy shows promise for lifelong treatment of hypophosphatasia
04 Feb 2025 20:15 GMT
… and effectiveness of a gene therapy for HPP.
The new … including different dosages of the gene therapy, tests in male and … rather than infants, the gene therapy was more effective on female … enzymatic activity spurred by the gene therapy. In adult female mice …
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Spur Therapeutics Announces Positive Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Gene Therapy Candidate for Gaucher Disease, at WORLDSymposium™
04 Feb 2025 13:00 GMT
… , an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type … an adeno-associated virus (AAV) gene therapy candidate in clinical development as … a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well …
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New grant funds first-of-its-kind gene therapy to treat aggressive brain cancer
03 Feb 2025 22:07 GMT
… treatment would be the first gene therapy for glioblastoma to use a … the tumor to deliver the gene therapy and cellular treatment. This clinical …