Sangamo Treats First Patient in Hurler Syndrome Study, Buys TxCell

Sangamo Therapeutics is continuing its push to bring its zinc finger nuclease genome-editing technology to market. The company announced this week that it treated the first patient in a clinical trial analyzing an in vivo gene-editing therapy for patients with a rare disease called Hurler syndrome, or mucopolysaccharidosis type I (MPS I).

What’s more, Sangamo has also announced the purchase of TxCell, a French biotech company focused on T-cell immunotherapies, for roughly $84.2 million, further filling its war chest of proprietary cutting-edge technologies.

>> READ: Lost in the CRISPR Hype, a Gene-Editing Giant Is Fighting Back

Both moves strengthen a company that is taking on rare diseases through a different kind of gene editing and other high-tech therapies.

Part of the Empowers Study, the treatment, SB-318, has already received orphan drug, fast track and rare pediatric disease designations from the Food and Drug Administration and similar nods of confidence elsewhere in the world. Sangamo is investigating the genome-editing therapy’s “safety, tolerability and preliminary efficacy” in as many as nine adults with Hurler syndrome at healthcare facilities in California, Florida, Georgia, New York and Ohio, according to the company.

“With this first patient in the Empowers Study of SB-318 for MPS I, and with five patients now treated in the Champions Study of SB-913 for MPS II, we are making strong progress in the evaluation of our in vivo genome-editing approach for these two rare inherited metabolic diseases,” Edward Conner, M.D., Sangamo’s chief medical officer, said in a statement.

The Champions Study evaluates the effects of Sangamo’s treatment for patients with a form of Hurler syndrome. The company expects to release preliminary data from those clinical trials later this summer.

As noted in a comprehensive Healthcare Analytics News™ feature story early this year, Sangamo’s technologies hold great promise for the future of precision medicine.

Its brand of gene editing does not rely on the more popular CRISPR or TALEN technologies, making the organization something of a standout in the space. Instead, Sangamo uses zinc finger nucleases to edit genes inside the body, hopefully correcting problems that cause debilitating diseases.

In the case of Hurler syndrome, Sangamo’s gene editing targeted liver cells with one intravenous infusion. The technology allows the cells to “insert the corrective gene at the precise location,” enabling the patient to then produce a stable supply of enzyme going forward, according to the announcement.

Sangamo’s acquisition of TxCell, meanwhile, places it at the forefront of regulatory T-cell (Treg) therapies. The company plans to investigate Tregs that are genetically modified with chimeric antigen recptor (CAR-Treg), which could boost organ transplant and the treatment of diseases such as Chron’s disease and multiple sclerosis.

“We believe CAR-Treg therapies will prove to be as exciting for immunology as CAR-T has been for oncology,” Sangamo CEO Sandy Macrae said.

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