Vertex heads to the clinic with a next-gen stem cell candidate for … – Endpoints News


Vertex will start human testing of a new-and-improved version of its stem cell therapy for type 1 diabetes.

Regulators cleared Vertexs IND for its stem cell-derived pancreatic islet cell therapy VX-264 in type 1 diabetes, the company announced Thursday.

While the new candidate uses the same islet cells as VX-880, Vertexs original stem cell program for diabetes, it also makes use of an immunoprotective device that potentially eliminates the need for immunosuppression. Patients who take immunosuppressants may face a range of side effects, including an increased risk of infection. The device used in this program was invented by Semma Therapeutics, which was acquired by Vertex in 2019, and further developed at Vertex, according to a spokesperson.

The news comes several months after Vertex struck a $320 million deal to acquire ViaCyte and its other stem cell program for diabetes. The deal was intended to accelerate Vertexs overall diabetes pipeline, but did not play into the development of either VX-880 or VX-264, according to the company.

Both companies had been attempting to rejuvenate patients abilities to produce their own insulin using stem cell transplants. ViaCyte was going for an off-the-shelf gene-edited approach encapsulated in immune-evading devices that researchers thought could prevent rejection.

VX-880 has successfully demonstrated clinical proof of concept in T1D, and the acquisition of ViaCyte will accelerate our goal of transforming, if not curing T1D by expanding our capabilities and bringing additional tools, CEO Reshma Kewalramani said in a statement at the time.

Vertex plans to launch a Phase I/II trial in the first half of this year and is already conducting a Phase I/II in Canada, it said on Thursday.

Editors Note: This story has been updated throughout to reflect that ViaCytes technology was not used in the development of VX-880 or VX-264. A previous version of this story incorrectly stated that VX-264 was developed using technology from the ViaCyte acquisition. The story has been corrected to reflect that the device used in this program was invented by Semma Therapeutics, which was acquired by Vertex in 2019, and further developed at Vertex.

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