Can you provide an overview of Sangamo and its unique contribution to the life sciences sector?

Sangamo is a genomic medicine pioneer, the first to edit human cells in vivo. Our mission is to translate groundbreaking science into medicines that improve patients' lives and assist those suffering from severe diseases for which current medicine can only provide symptom relief at best. 

We are based on Zinc Fingers (ZF), an editing technology that allows us to find a unique part of the DNA and make changes to it to address the disease. ZF technologies are extremely adaptable and customizable. We can append a variety of functional domains, allowing us to adapt and optimize functionality based on the disease of interest. We currently have patients being treated for Hemophilia A, Fabry disease, and Sickle Cell disease, and in each of those cases, patients have improved and are living more normal lives as a result of a single, one-time treatment.

What vision did you bring to the company when you joined as CEO in 2016?

The fact that Sangamo was at the forefront of a new technology drew me in. With my pharma experience, I insisted on only doing science that could be translated into benefit for patients; it is the connection between understanding patients and unmet medical needs that truly distinguishes a biotechnology company. Many labs and universities are conducting excellent research, but it is our responsibility to translate that research into medicines.

Can you elaborate on Sangamo's pipeline?

We are advancing a robust pipeline of wholly owned and partnered programs in rare disease, neurology, oncology, and autoimmune indications. Currently, in pre-clinical development, we have programs targeted at addressing neurological disorders, multiple sclerosis, inflammatory bowel disease, and renal transplant. We have, for instance, dosed the first patient in a Phase 1 clinical study of an investigational CAR-Treg for renal transplant, and the patient is generally well tolerating the treatment.

In 2018, Sangamo acquired TxCell based in Valbonne, France where we now have over 100 people developing these Treg therapies. Moving forward, we feel that our ZF editing technology has unique advantages that will allow it to control genes associated with brain diseases, and in this space, we are working with our partners on Alzheimer's disease, Parkinson's disease and autism spectrum disorder as well as advancing our own wholly owned programs in neurology.

What are your preferred methods of funding your R&D efforts?

Since joining Sangamo, I have raised USD 1.5 billion, with partnerships accounting for USD 800 million. Our company costs between USD 280 million and USD 300 million to run each year. We will continue to raise funds by forming partnerships and telling our story to investors and shareholders until we have a revenue stream from direct sales of our medicines or royalties from our partners.

Obtaining funds from partners is extremely beneficial in funding research; however, what is more important is that partners bring clinical expertise to a small company like ours, which cannot be an expert in everything. A collaborative ecosystem enables us to get medicines to patients more quickly.

What are the biggest challenges Sangamo is currently facing, especially after COVID?

Challenges in finding and retaining talent, legislation and policy, and finding patients for clinical trials are a reality in the entire biotechnology sector. The current market downturn has caused share prices to go down which makes it harder to raise money. Sangamo is fortunate to have over USD 300 million in the bank, which will take us into 2024. 

The biotech industry is rapidly expanding, particularly in areas such as San Francisco and Boston, with a large number of emerging companies competing for talent. In terms of regulations, every new therapy or technology is met with great excitement before it enters the real world, where it must be approved by the FDA and European regulatory authorities and meet certain standards. These standards evolve as new areas of medicine emerge, which is normal, but this also requires additional efforts when submitting a new therapy for approval.

What impact has COVID had on the perception and focus of innovation?

People have been working on mRNA vaccines for a long time, so Covid vaccines did not appear overnight.

The onset of the pandemic, on the other hand, provided a platform for urgency, focus, and funds to be directed toward the rapid development of mRNA vaccines for human use. It also provided a positive risk-benefit equation in that there was an enormous benefit to taking on the risks of potential side effects, which have been shown to be exceptionally well tolerated for a new technology.