Swiss firm licenses gene therapies from UMass Medical School

Photo/Grant Welker UMass Medical School in Worcester.

Miguel Sena-Esteves

Heather Gray-Edwards

Terence Flotte

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By Grant Welker

A Swiss company developing gene therapies for neurological diseases has licensed programs researched at UMass Medical School in Worcester.

The therapies are meant to treat Tay-Sachs and Sandhoff diseases, two rare and similar inherited conditions. Research on the therapies was developed by a team of researchers at UMass Medical School, including Miguel Sena-Esteves, Heather Gray-Edwards and Terence Flotte, the dean of the School of Medicine.

The announcement Thursday the Swiss company Axocant Sciences has licensed the therapy programs from UMass comes just over a month after another highlight for the medical school's research on Tay-Sachs. In November, the Blu Genes Foundation, a Toronto foundation dedicated to the development of gene therapy treatments for rare disease, donated $1.4 million to UMass Medical School for the advancement of an early-stage clinical trial for the Tay-Sachs.

Tay-Sachs and similar diseases are attractive for researchers because they've already been able to identify the underlying genetic cause, Sena-Esteves said. Researchers now have well-understood methods for delivering corrective genes, he said.

The therapies are designed to introduce functional copies of genes to improve survival and enable children with the diseases to reach key developmental milestones.

Tay-Sachs and Sandhoff are fast-developing diseases destroying nerve cells and leading to loss of motor skills. They have an incidence of approximately one out of 180,000 live births worldwide, according to the licensing announcement. Children with the diseases often die within a few years.

UMass researchers have already reached milestones in treating Tay-Sachs and Sandhoff, including development of a gene-therapy vector used to deliver functional copies of defective genes that cause the diseases.

Axovant's exclusive worldwide licensing of the therapies from UMass Medical School includes additional payments tied to development, regulatory and commercial milestones, the school said. Financial details of the deal were not disclosed.

Axovant is working on therapies for other neurological diseases including Parkinson's and ALS.