RARE Daily

Astellas Enters Sponsored Research with UMass for Alexander Disease Gene Therapy

June 24, 2024

Rare Daily Staff

Astellas Pharma US said it has entered into a sponsored research agreement with the UMass Chan Medical School to proceed with research for an adeno-associated virus vector mediated gene therapy for the treatment of Alexander disease, a fatal, ultra-rare disease for which the only current treatment option is supportive care.

This collaboration is part of a focused initiative to deliver value to patients with rare diseases through innovative R&D models including investigator-led research.

Under the terms of the agreement, UMass Chan will lead research activities to accelerate the program toward clinical study while Astellas will support UMass Chan activities by providing drug discovery research expertise. The current agreement lasts for one year with an opportunity for Astellas to extend it to two years.

Alexander disease is an ultra-rare and progressive central nervous system disorder caused by mutations in the glial fibrillary acidic protein gene that usually has a fatal outcome. Most affected patients do not live beyond their first few years. Alexander disease affects astrocytes, a type of cell that makes up the majority of cells in the CNS and leads to the destruction of white matter in the brain, causing progressively worsening severe intellectual and physical disabilities; and eventually affected individuals will lose life-sustaining abilities.

The sponsored research agreement supports investigations being done by Jun Xie, associate professor of microbiology and physiological systems, and Guangping Gao, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research at UMass Chan, who have developed an adeno-associated virus vector that can express a small RNA silencer that suppresses the mutant proteins that cause AxD in mice.

“Silencing GFAP, the mutant protein that causes AxD, with our artificial miRNA could be a promising therapeutic approach to treatment,” said Xie. “Collaborating with Astellas will bring us one step closer to our goal of helping patients with this disease.”

Photo: Jun Xie, associate professor of microbiology and physiological systems at UMass

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