Astellas will sink $50 million into Taysha Gene Therapies in exchange for a 15% stake in the biotech and the option to license clinical-stage candidates for two rare neurological disorders.
The two adeno-associated virus (AAV) gene therapies in question are TSHA-102, the only gene therapy in the clinic for Rett syndrome, and TSHA-120, which is in phase 1/2 development for axonal neuropathy.
Astellas touted the deal as the continuation of a gene therapy strategy that saw the Japanese Big Pharma acquire Audentes for $3 billion in January 2020 and open a gene therapy manufacturing facility in North Carolina four months ago.
Both of the candidates covered by the deal are AAV9 gene replacement therapies. TSHA-102 utilizes Taysha’s miRNA-responsive auto-regulatory element (miRARE) platform to regulate transgene expression on a cell-by-cell basis, while TSHA-120 delivers the gene gigaxonin.
“Taysha is an industry leader in CNS gene therapies and this partnership fits strategically with our long-term vision of expanding Astellas’ gene therapy capabilities, allowing the company to impact the lives of a broader range of patients with urgent unmet medical needs,” Astellas Chief Strategy Officer Naoki Okamura said in a postmarket release Monday.
The agreement also gives Astellas certain rights when it comes to signing off on any potential change of control of Taysha. In the meantime, Astellas will take up an observer seat on Taysha’s board of directors, which the companies said would enable the biotech to leverage Astellas’ clinical and commercial expertise.
Taysha has one other candidate in the clinic that isn't covered by the Astellas deal—a therapy called TSHA-118 in phase 1/2 development for an inherited nervous system disorder called CLN1 disease.
For Astellas, the route into gene therapy offered by the Audentes acquisition hasn’t always gone smoothly. Plans to file AT132 for approval in a rare neuromuscular disease were knocked off course by patient deaths, while the Big Pharma decided to terminate work on a clinical-phase therapy called AT702 for Duchenne muscular dystrophy as well as two earlier-stage candidates for the condition after taking a look at a preclinical study.