Takeda enlists Poseida in $3.6B+ nonviral gene therapy deal, though data remain years away from human studies
Takeda has been behind other major players in terms of cell and gene therapy development, but after announcing a collaboration with Selecta Biosciences last week, it’s now on a bit of a spending spree.
The Big Pharma has recruited Poseida Therapeutics to develop in vivo gene therapies in six different indications, the companies announced Tuesday, with Takeda retaining an option to ask for two more. Poseida will get a $45 million upfront payment in the deal, $125 million in short-term preclinical milestones and up to $435 million in clinical milestones per program.
If all eight candidates hit all their checkpoints, Takeda will be on the hook for more than $3.6 billion. Each of the programs will be directed at the liver and hematopoietic stem cells, with hemophilia A being the first target.
Though last week’s Selecta deal was much narrower in scope, focusing on tamping down the immune response seen in AAV-based gene therapies, Takeda’s Tuesday agreement is a bet on the future of gene therapy, Poseida CEO Eric Ostertag told Endpoints News. The biotech’s gene therapy platform is centered around a nonviral approach, which Ostertag says can provide a true cure for genetic diseases.
Poseida argues it is different from earlier gene therapy attempts because that nonviral approach is better suited at integrating into a patient’s genome. Ostertag said the programs in Tuesday’s deal will use its proprietary nanoparticle technology to edit targeted genes in vivo, resulting in higher expression levels, and give Poseida the opportunity to go after pediatric patients.
It’s an approach unlike those being investigated by other companies that he says are primarily treating adults, though Ostertag did not name names.
“I think there are some approaches that aspire to be single treatment cures. We’ve actually proven it with numerous animal models,” Ostertag told Endpoints. “That is unique.”
As Ostertag points out, however, Poseida has yet to put any of its gene therapies in humans and likely won’t do so for several years. The earliest gene therapy slated for IND-enabling studies is an OTC deficiency program not involved in Tuesday’s deal shooting for 2023, and even that isn’t entirely certain — Poseida is still choosing between two different technologies.
The lack of integration seen in AAV-based gene therapy and some nanoparticles-based approaches are “particularly a problem when you have a rapidly dividing tissue,” Ostertag said. “Transient approaches are not going to work at all in that [pediatric] patient population.”
He later added, “To date, the other gene therapy companies are really only addressing those few patients that had mild disease that made it into adulthood. We can address the pediatric population.”
Poseida has largely made noise for its CAR-T programs, providing proof of efficacy for a next-gen solid tumor CAR-T in a data readout at the end of August. The results came about 10 months after the FDA removed a clinical hold, spurred by a patient death on its lead cancer drug.