On June 20, 2024 Astellas Pharma US, Inc. ("Astellas") reported the company has entered into a sponsored research agreement with the UMass Chan Medical School to proceed with research for an adeno-associated virus (AAV) 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 (Press release, Astellas, JUN 20, 2024, View Source [SID1234644449]). This collaboration is part of a focused initiative to deliver value to patients with rare disease through innovative R&D models including investigator-led research.

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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 to two years.

Alexander disease is an ultra-rare and progressive central nervous system (CNS) disorder caused by mutations in the glial fibrillary acidic protein (GFAP) 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.1

Yoshitsugu Shitaka, Ph.D., Chief Scientific Officer at Astellas

"We are pleased to partner with UMass Chan, a leading medical academic center in Massachusetts, and help foster innovation in this important ecosystem. At Astellas, we strive to find innovative ways to treat patients with the highest unmet medical needs, who have traditionally been left behind, including rare and ultra-rare diseases.

Gene therapy is a complex breakthrough area where traditional approaches might not be sufficient or optimal, and ultra-rare diseases like Alexander disease cannot be appropriately addressed with the usual pharma models. Only by working alongside all the key players such as patients, physicians, academic centers, can we advance pioneering work to challenge the status quo, by turning innovative science into meaningful treatments for those patients who are waiting."

The sponsored research agreement supports investigations being done by Jun Xie, PhD, associate professor of microbiology & physiological systems, and Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, 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.

Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems, director of the Horae Gene Therapy Center and co-director of the Li Weibo Institute for Rare Diseases Research, UMass Chan

"We are enthused to be working with Astellas. Their drug discovery research expertise is a critical component in translating this work from bench to bedside, and bringing gene therapy to patients in the clinic who are struggling with this deadly disease."

Jun Xie, Ph.D., associate professor of microbiology & physiological system, UMass Chan

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