Can you introduce Sangamo Therapeutics?

Sangamo Therapeutics specializes in genomic medicine. We have various ongoing projects, such as Hemophilia A in partnership with Pfizer, a wholly-owned project for sickle cell disease, and a CAR-Treg cell therapy for renal transplant, which is currently in clinical trials and progressing well. Additionally, we have a pipeline of projects targeting inflammatory bowel disease, multiple sclerosis, and diseases of the central nervous system (CNS). Our proprietary zinc finger technology allows us to power a complete suite of genomic medicine capabilities, providing a unique approach to gene therapy, cell therapy, and gene editing. We are strategically focused on developing genomic cures for serious conditions with high unmet patient needs and where our technology has the potential to create first- or best-in-class medicines.

What are the recent business highlights?

In our most recent quarterly call, we reported having US$308 million in cash. In the last several years, we have managed to raise approximately US$1.6 billion, about half of which came from financial raises and the other half through partnerships with leading pharmaceutical companies. Partnerships are crucial to us because they help us deliver on our mission to get new medicines to patients as quickly and safely as possible by extending the reach of our technology into new therapeutic areas and providing revenue to fuel our science. For instance, our partnership with Pfizer for Hemophilia A is of immense significance as it will provide a potentially transformative treatment for patients. Additionally, we expect to receive potentially US$240 million in milestones and 14-20% royalties until the drug launch.

Our zinc finger platform is uniquely versatile, allowing us to power a complete suite of genomic medicine capabilities. However, as a small company, there are limits to what we can take forward on our own. Therefore, we regularly evaluate inquiries from other companies interested in partnering with us to utilize our technology.

Could you provide an update on the Hemophilia A program with Pfizer and what steps are planned for a pivotal readout?

The phase 3 trial dosing is almost complete, and the results are expected to be presented in the first half of 2024. We have also been advised that they will be filing the Biologics License Application (BLA) in the second half of the same year. For our company, this program is a transformative moment.

What are the clinical advantages observed in the Phase 1-2 STAAR Study conducted for Fabry disease?

When we speak to patients with Fabry disease, we can sense the importance of having a treatment that frees them from the burden of enzyme replacement therapy infusions. Patients with Fabry disease spend approximately five hours every other weekend for infusion, which can be quite challenging. Thus, the idea of potentially receiving a single infusion of gene therapy that frees them from the disease is a liberating and important factor that eases the burden of their condition.

Can you give an outlook on the future trends of data technologies and gene therapy in the life sciences industry?

Being the first gene editing company to use zinc finger technology, Sangamo’s versatile technology enables us to power a complete suite of genomic medicine capabilities: gene activation or repression, DNA cleavage, base editing, site-specific integration, and epigenetic modification. Sangamo has the whole toolbox, enabling us to find the right tool for each clinical need. While technology is important, it is the application of that technology that matters.

What are the primary trends propelling the demand for genomic medicines in the US in 2023?

Cell and gene therapy is becoming increasingly important. So far, these therapies have been used mainly for rare conditions or in cases where the patient's life is at risk. However, Hemophilia A may be the first large-scale use of genomic medicine, where patients have an alternative treatment option. We believe that the benefit of giving patients a "once and done" treatment that keeps them out of hospitals and reduces their pain is enormous. The benefit to society of treating patients with a one-time gene therapy that extends their lives and reduces their pain must be taken into account when considering cost and benefit.