, /PRNewswire/ -- Astellas Pharma US, Inc. ("Astellas") announced today 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 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 disease 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 to two years. 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. 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. "We are pleased to partner with UMass Chan, a leading medical academic center in , and .
