Takeda and Poseida Therapeutics Announce $3.6 Billion Collaboration to Advance Non-Viral In Vivo Gene Therapies

Compiled by | Tom Li

Recently, Takeda's investment in gene therapy reached a new peak this Tuesday with a research collaboration, as the gene therapy partnership agreement with Poseida Therapeutics is valued at up to $3.6 billion.

Takeda and Poseida Therapeutics have entered into a research collaboration and exclusive license agreement, under which the two companies will leverage Poseida’s piggyBac, Cas-CLOVER, biodegradable DNA and RNA nanoparticle delivery technologies, and other proprietary genetic engineering platforms to develop up to eight gene therapies.

This collaboration will focus on the development of non-viral in vivo gene therapy programs, including Poseida’s hemophilia A program. Takeda will fund the R&D expenses for all collaborative programs. Poseida will receive up to $45 million in upfront and preclinical milestone payments, with milestone payments tied to the progress of all eight programs potentially bringing up to $3.6 billion to the San Diego-based company.

Poseida will lead research activities until Takeda completes the selection of drug candidates, after which Takeda will assume responsibility for further development and commercialization. The timeline and specific details of this collaboration remain confidential, even in Securities and Exchange Commission (SEC) filings, presumably because most programs have not yet advanced to human clinical trials.

While this marks Poseida Therapeutics' first major collaboration, Takeda has long sought to secure a foothold in the gene therapy sector. Just last week, Takeda announced it would provide up to $1.12 billion in funding to support Selecta Biosciences' technology platform for developing gene therapies for lysosomal storage diseases. Prior to this, in August, the company also entered into a $303 million collaboration agreement with Genevanti focused on gene therapies for liver diseases.

Poseida Therapeutics’ P-FVIII-101 leverages the company’s piggyBac gene insertion technology and nanoparticle delivery system to introduce a therapeutic gene, aiming to address Factor VIII deficiency in hemophilia patients. In mouse studies, Poseida reported that its gene therapy can restore clotting protein levels to near-normal. Previously, Takeda partnered with Asklepios BioMedical (AskBio), a Bayer subsidiary, to develop gene therapies for hemophilia A and hemophilia B. AskBio’s approach utilizes adeno-associated virus (AAV) vectors. Earlier this year, Takeda also formed a partnership with gene therapy startup Ensoma, which employs adenovirus vectors with their genetic material removed. The Takeda-Ensoma collaboration encompasses the development of up to five gene therapies for rare diseases.

Non-viral gene therapy is increasingly attracting interest within the pharmaceutical R&D sector, as this technology circumvents the safety concerns associated with adeno-associated virus (AAV)-based therapies. Following an advisory committee meeting to evaluate the safety of Novartis’s Zolgensma, the FDA has also heightened its focus on the safety of similar products. Madhu Natarajan, Head of Rare Disease Drug Discovery at Takeda, stated that Poseida’s distinct technological approach demonstrates promising potential for developing non-viral in vivo gene therapies, which can complement and expand Takeda’s existing product portfolio.

However, Takeda’s competitors appear to be ahead. Pfizer and its partner Sangamo Therapeutics have advanced their gene therapy candidate to Phase 3 clinical trials. Following the FDA’s rejection of its hemophilia A gene therapy last year, BioMarin Pharmaceutical is conducting another Phase 3 study; both gene therapies utilize adeno-associated virus (AAV) vectors.

References:

1.Billions club: Takeda taps Poseida for 2nd $1B-plus gene therapy pact in 2 weeks

2.Poseida Therapeutics Announces Research Collaboration with Takeda for Novel Non-Viral In Vivo Gene Therapies

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