The Power of Partnerships to Unlock the Promise of Gene Therapy

“We can’t do it alone.”?

That was the thought that came to my mind after joining Poseida a few years ago.?As I began to fully understand the breadth and depth of the genetic engineering technologies that were being developed by the company, it quickly became apparent that to do justice to the multitude of opportunities that exist within our walls we would need significant resources and support from industry leading strategic partners. At Poseida, we are focused on developing the next wave of cell and gene therapies for patients with cancer and rare genetic diseases. ?By partnering with world-class strategic collaborators, we can accelerate our progress and expand our ability to bring new and better treatment options to more patients in need.

Our platform technologies in gene insertion, gene editing, gene delivery and beyond are unique tools that have broad applicability across both cell therapy and gene therapy. Used alone or in combination, they allow us to pursue some of the most promising opportunities in drug development today. ?Our Super piggyBac? DNA Delivery System, Cas-CLOVER? Site-Specific Gene Editing System and non-viral, biodegradable nanoparticle delivery technology all represent potentially disruptive leaps forward over other technologies.?PiggyBac and Cas-CLOVER in particular have broad utility and the potential to be used both ex vivo, or outside the body, and in vivo, or directly in the body, in many cell types and multiple applications.

?In order to fully exploit such broad technologies, we had to develop a strategy that would allow us to make demonstrable technical progress to validate our platforms and attract the right strategic partners at the right time.?To accomplish this, we focused our attention on two primary genetic engineering applications: CAR-T therapy for oncology with a focus on allogeneic approaches and in vivo liver-directed gene therapy for rare genetic diseases.?While some may have questioned our approach in waiting to partner, we know that industry acceptance of new technology is often slow to start, and patience and perseverance are needed.?

Unlocking the True Promise of Gene Therapy

In gene therapy, the industry continues to pay the cost of many, many companies using decades-old technology.?The FDA recently held an advisory panel meeting on the topic of the numerous safety issues associated with adeno-associated virus, or AAV, the most common gene therapy approach, which have resulted in clinical holds of many trials in recent times.

To be fair, AAV does work to a degree – but the risks can and, in some cases, certainly do outweigh the benefits, especially when considering that AAV therapies are temporary by design.?In other words, AAV does not enable permanent gene correction and so the risks and benefits need to be seriously considered in light of the transient treatment effect.?While many companies are trying to develop a “better AAV,” we think it’s time to move on to a better way with a better technology.

Charting a New Course

At Poseida, our gene therapy approach has two distinct characteristics that set us apart from others in the space.?First, we are pursuing permanent gene correction with our piggyBac technology – in other words, we are seeking single treatment cures. Those pursing AAV or RNA approaches are unlikely to ever achieve cures because those technologies are not integrating – meaning they do not modify and correct the DNA. ?Second, while piggyBac has the potential to be used with AAV to achieve permanent correction at lower doses, we are ultimately moving to non-viral delivery of piggyBac and Cas-CLOVER using biodegradable nanoparticles to avoid the serious safety concerns and other limitations of AAV.

While we have been working on our gene therapy platform for several years, we waited until now to partner as we can now demonstrate the true long-term value of our novel technologies and unique approach to attract a world class partner who could bring complementary and synergistic skills and resources to the relationship.

Strategic Collaboration with Takeda

To that end, we are pleased that we recently announced a strategic partnership with Takeda Pharmaceuticals to pursue non-viral approaches to both gene insertion and gene editing using our piggyBac and Cas-CLOVER technologies delivered by biodegradable nanoparticles.?Our in vivo gene therapy work with Takeda will initially focus on both liver-directed and hematopoietic stem cell (HSC)-directed indications, including our Factor VIII program for Hemophilia A and five other targets that we have not yet disclosed.

We are extremely excited to be working with Takeda, which is not only one of the world’s leading pharmaceutical companies, but a leader in rare diseases and gene therapy as well as Hemophilia A.?The partnership with Takeda represents the beginning of a new chapter for Poseida as we expand the reach of our platform technologies in gene therapy.

Takeda and Poseida are passionate about bringing innovative treatments to patients with rare genetic diseases. By working together and leveraging the power of our platform technologies, we have the opportunity to change the future of medicine and unlock the true promise of genetic engineering in gene therapy.