Is gene therapy ready for a development renaissance?

Genetix is betting $35 million in fresh financing that gene therapy is ready for a renaissance. Profiled by the Boston Globe, Genetix is using a new viral vector approach to deliver healthy replacement genes needed to check rare diseases. Once the dummy virus--which is first made harmless--infects stem cells in the blood, new genes are copied into the system.

"If you are successful in getting enough genes into enough of those cells, you can have the presence of your therapeutic genes for the remainder of the life of the organism,'' Mitchell Finer, Genetix's chief scientific officer, tells the Globe. Genetix has targeted two rare and fatal diseases: beta-thalassemia major and Adrenoleukodystrophy, or ALD. And because Genetix researchers work with the patient's cells, they can avoid the issue of an immune system rejection.

Genzyme Ventures is one of the investors backing Genetix's gene therapy venture. Alan Walts, managing director of the fund, says that Genzyme would be interested in either an acquisition or a licensing deal if the work looks promising.

Gene therapy had been a hot field in the drug development world back in the ‘90s. But the death of one patient in a high-profile trial set the field back by years as researchers were forced to correct some serious safety issues.

- here's the story from the Boston Globe

Suggested Articles

Removing the IRE1-alpha gene from beta cells in mouse models of Type 1 diabetes restored normal insulin production, scientists found.

Selectively targeting TGF-beta1 with Scholar Rock's SRK-181 overcame primary resistance to checkpoint inhibitor therapy in mice.

Enhertu produced a 55.6% objective response rate in HER2-positive non-small cell lung cancer patients in a phase 1 trial.