National Institute for Health and Medical Research. The French scientists who developed that technology have generated headlines around the world for its use to cure ALD in two high-profile cases.
“We’re at that same point that we were with monoclonal antibodies 12 to 15 years ago,” Finer, Genetix’s new chief scientific officer, says. “You look and see the resurgence of gene therapy data. We are poised to capitalize on those advancements.”
Genetix, which has now closed $75 million in funding since its inception, adds its own twist to gene therapy. Rather than injecting its therapies directly into patients, the company treats patients’ own bone marrow after it has been extracted from them. Once the firm’s treatments have corrected the genes in patients’ bone marrow stem cells, the cells are infused back into the patients to cure their illness. To deliver healthy genes to cells, the company uses viruses derived from deactivated HIV that are designed to die off after their job is done.
Patients with ALD typically die within 10 years after symptoms start to appear, according to the National Institutes of Health. Genetix aims to enter pivotal trials with its ALD therapy in the U.S. and France, with the exact timing partially depending its talks with the FDA and other regulators, according to Leschly. He didn’t say what questions the pivotal trials will have to answer in order for Genetix to win FDA approval for its gene therapy.
The company is also in early-stage development of a gene therapy for the genetic blood disorder thalassemia, which causes the formation of abnormal hemoglobin in blood cells. Leschly says the firm’s technology could lead to treatments for many other diseases, including many genetic disorders that currently require frequent enzyme replacement therapies—the sort of drugs that Genzyme
