Takeda broadens gene therapy scope, turns to Poseida for non-viral approach


Takeda Pharmaceutical already has gene therapy prospects, but it wants a piece of this market in a way that avoids the safety risks from the engineered viruses that deliver these treatments to their destinations in the body. Poseida Therapeutics has several non-viral gene therapy technologies, and Takeda is putting up some money now with the promise of more later to begin an R&D alliance with the biotech company.

Takeda is interested in Poseida’s technology for in-vivo gene therapies, which work inside the patient. The deal announced Tuesday calls for the companies to work together to develop up to six such therapies that use three Poseida non-viral gene therapy technologies.

The deal gives Takeda the option to add two more programs to the collaboration, bringing the total to eight partnered programs. Takeda is paying Poseida $45 million up front to begin the partnership. Milestone payments tied to the progress of all eight programs could bring San Diego-based Poseida up to $3.6 billion more.

The initial six programs for the partnership come from Poseida’s pipeline and include the biotech’s most advanced gene therapy program, P-FVIII-101, a preclinical candidate for the inherited blood clotting disorder hemophilia A. Competitors are further ahead. Pfizer and partner Sangamo Therapeutics have reached Phase 3 testing with their gene therapy candidate. BioMarin Pharmaceutical is conducting another Phase 3 study after the FDA rejected its hemophilia A gene therapy last year. Both of these gene therapies are delivered by adeno-associated viruses (AAV).

Poseida’s P-FVIII-101 uses the company’s piggyBac gene insertion technology and nanoparticle delivery to introduce a therapeutic gene to address the deficiency of Factor VIII. In mouse studies, Poseida has reported its gene therapy corrected the deficiency of the blood clotting protein to near normal levels.

The other five programs covered by the Takeda/Poseida partnership are in the discovery stage and address undisclosed diseases. According to the terms of the agreement, Poseida is responsible for funding the research until candidate selection. If Takeda opts to continue development of one or more of those candidates, the pharma giant will be responsible for funding their development and, if approved, commercialization.

Takeda already has hemophilia gene therapy prospects via another alliance. A partnership with Asklepios Biopharmaceuticals (now a part of Bayer) is developing gene therapies for hemophilia A and B. AskBio’s approach uses AAV. Earlier this year, Takeda invested in and formed a partnership with gene therapy startup Ensoma. Instead of using AAV, the Boston-based biotech uses adenoviruses that have been stripped of their genetic material. Takeda’s partnership with Ensoma covers the development of up to five gene therapies for rare diseases.

In a prepared statement, Madhu Natarajan, Takeda’s rare diseases drug discovery unit head, said that Poseida’s different approach shows promise for developing non-viral in vivo gene therapies that complement the Japanese company’s existing alliances.

“This partnership reinforces Takeda’s commitment to investing in next-generation gene therapy approaches that have the potential to deliver functional cures to patients with rare genetic and hematologic diseases,” he said.

If a safety problem emerges in the Poseida pact, the agreement offers Takeda a quick way out. While the agreement offers a way for either party end the deal if there’s a breach by the other party – standard contract language – it also specifies that Takeda may terminate the pact “for convenience upon prior written notice or in the event of a safety concern immediately upon written notice,” according to a Poseida regulatory filing.

Photo: Scott Eisen/Bloomberg, via Getty Images

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