Astellas invests $50 million in Taysha Gene Therapies

Astellas has announced it will acquire 15 percent of Taysha Gene Therapies, Inc through a $50 million investment, gain licensing to two of its adeno-associated virus (AAV) gene therapy programmes for Rett syndrome and giant axonal neuropathy (GAN). 

Taysha develops and commercialises drug therapies for rare and large-market indications such as monogenic central nervous system (CNS) diseases. Its first intrathecally-delivered therapy candidate, TSHA-102, is the first-and-only gigaxonin gene therapy for Rett syndrome, which is currently in clinical development. The second, TSHA-120, is designed for GAN. It is in Phase I/II clinical development and awaiting regulatory feedback. These innovative drugs offer potential for better, more effective treatments for Rett syndrome and GAN.

TSHA-120 has received Orphan Drug Designation (ODD) and Rare Paediatric Disease designation from the US Food and Drug Administration (FDA) and ODD from the European Commission (EC).

TSHA-102 uses the miRNA-Responsive AutoRegulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. The technology inhibits toxicity associated to transgene overexpression and there is potential to use the therapy for other indications. 

“…our goal is to bring new transformative treatment options to patients living with serious genetic diseases and limited treatment options,” explained Naoki Okamura, Chief Strategy Officer at Astellas. She shared that the partnership fits strategically with Astellas’ long-term vision to expand the company’s gene therapy capabilities.

Under the agreement, Astellas will have rights to any potential change of control of Taysha. Astellas will also receive one Board observer seat on Taysha’s Board of Directors.

Astellas Gene Therapies works on research for rare diseases of the eye, CNS and neuromuscular system. Their products are designed using a gene therapy drug discovery engine built around innovative science, a validated AAV platform and manufacturing capability.