Takeda Stopping Preclinical Work in AAV Gene Therapy, Rare Hematology

Rare Daily Staff

Takeda informed employees earlier this week that it would discontinue discovery and pre-clinical efforts in AAV gene therapy, as well as research and pre-clinical efforts in rare hematology.

In an emailed statement to Rare Daily, the company said it was taking steps to focus resources in our core therapeutic areas and late stage clinical programs.

“There is no impact to our clinical development pipeline or commercial products,” the company said. “Takeda remains firmly committed to patients with rare diseases and we will maintain our rare disease unit, which will focus on late-stage development programs moving forward.”

The news, first reported by Endpoints, follows a similar move from Pfizer earlier this year as it announced it would externalize rare disease early-stage research in neurology, cardiology, and its pre-clinical gene therapy programs.

It is not clear how many employees will be effected by the changes. Takeda said is working to support its people engaged in AAV gene therapy activities as it explores externalization strategies for those programs.

The company has already entered into a number of strategic collaborations to drive its future gene therapy pipeline. In October 2021, Takeda entered into a collaboration with Poseida for non-viral in vivo gene therapies that involved a $45 million upfront payment and up to $3.6 billion in milestones for a total of eight programs.

That was followed in February 2022 with a collaboration with Code Bio worth up to $2 billion across four programs for a liver-directed rare disease program and for central nervous system-directed rare disease programs.