… -stage study of its investigational gene therapy in patients aged two to …

On 30 December 2024, Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached its primary endpoint in a Phase III study in paediatric patients aged two to 17 with type II spinal muscular atrophy (SMA). The …

… partnership to develop an investigational gene therapy product to treat hemophilia A … was discussing data from the gene therapy with regulatory authorities.  It also … to patients,” said Macrae.  While gene therapy trials have shown positive results …

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… the first time researchers studied gene therapy to treat this disease, which … for the first time whether gene therapy could offer a potentially transformative … cells. They found that this gene therapy approach significantly boosted hemoglobin and …

… terminated its hemophilia A gene therapy partnership with Sangamo Therapeutics, … last year, linking the gene therapy to a significant reduction … for a hemophilia A gene therapy in June 2023, … options” for bringing the gene therapy to market, including looking …

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… important target cell types for gene therapy, and while there is a … is difficult to target with gene therapy because it is composed of … in both basic research and gene therapy. It shows that the best …

… to giroctocogene fitelparvovec, an investigational gene therapy product candidate for the treatment … pipeline and progressing our Fabry gene therapy programme towards a potential BLA … development discussions for its Fabry gene therapy programme, will allow it …