Gene therapy for sickle cell moves closer as last hurdle cleared : statnews

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Gene therapy for sickle cell moves closer as last hurdle cleared

Gene therapy for sickle cell moves closer as last hurdle cleared

Researchers have cleared the last scientific hurdle to a clinical trial of gene therapy to cure sickle cell disease, they reported on Tuesday, fueling hopes that they will begin enrolling patients early next year.But they dodged a bullet.The new study, conducted in mice, addressed a sometimes calamitous risk in gene therapy: the difficulty of changing only one thing when tweaking the DNA of a cell.


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David Epstein: Industry should 'remain optimistic' on cell and gene therapy after Novartis cull

David Epstein: Industry should 'remain optimistic' on cell and gene therapy after Novartis cull

David Epstein says that despite Novartis ($NVS) scaling back on its gene and cell therapy work, the industry should still be positive about the medical impact it can have, while he tells FierceBiotech that he is now "starting to consider biopharma options" for his next role.Epstein and former head of Novartis Oncology Hervé Hoppenot were pivotal in championing and nurturing the gene and cell therapy unit for the Swiss major.But after both men left the company (Hoppenot became the CEO of Incyte in 2014 while Epstein unexpectedly left a few months back), Novartis announced that it was to make major cuts to its internal unit dedicated to cell and gene therapy.


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Gene therapy for sickle cell disease passes key preclinical test

Gene therapy for sickle cell disease passes key preclinical test

A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center report today in the Journal of Clinical Investigation.The work sets the stage for bringing a decades-old discovery about sickle cell disease to the bedside.A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in the coming year.