Q&A with Matthew Kane, Co-founder & CEO, Precision BioSciences – Speaker at PMWC 2018 Silicon Valley

Q: What are the benefits of the Precision BioSciences’ ARCUS genome editing platform and what makes it unique?

A: ARCUS is a next-generation genome editing platform derived from a class of naturally occurring enzymes called homing endonucleases. In fact, you can think of homing endonucleases as nature’s genome editing system. The key advantages of ARCUS include exquisite control over off-target modifications, ease of delivery to the intended tissue or cell type, and greater efficiency for gene repair or insertions. Put simply – safety and efficacy. This novel nuclease architecture required over nine years and $20 million to develop but today gives us what, I believe, is the first therapeutic-grade genome editing platform and the only that has both clear freedom to operate and the ability to fully control its use.

Q: How Precision BioSciences applies the ARCUS genome editing technology to cancer immunotherapy?

A: To develop cancer immunotherapies, our team uses ARCUS to re-program human immune cells to become highly targeted, cancer-eliminating, cellular machines. This is part of a class of therapies broadly known as Chimeric Antigen Receptor T cells (CAR Ts). Precision’s first CAR T program is, in many ways, a next generation version of the recently approved CD19 targeting CAR Ts, Kymriah and Yescarta, now being used to treat certain types of acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). While representing significant steps towards an effective solution for ALL and NHL, these first generation treatments are limited by the scope of patients they can address and the product’s ability to efficiently scale in a manner that will reach the patients in greatest need. Precision’s 2nd generation approach uses ARCUS genome editing to enhance and arm qualified, healthy donor immune cells – rather than cells taken from cancer patients – to create a highly scalable, off-the-shelf product that could greatly improve and expand upon the effectiveness and safety of the first generation class of products. We anticipate seeking approval for our first human clinical trial to test this method in the second half of 2018.

Q: What are some of the clinical applications that your approach for gene therapy has the potential to address the unmet needs of patients and families dealing with devastating and, as yet, untreatable diseases?

A: Today, our gene therapy pipeline focuses on diseases that may be mediated by editing in either the liver or the eye. This includes potential cures for diseases such as Hepatitis B, Hemophilia A, Familial Hypercholesterolemia (think extremely high levels of bad cholesterol), and a form of Retinitis Pigmentosa, a disorder that leads to blindness. I am incredibly excited about the promise of genome editing for patients and families dealing with devastating genetic diseases; this is something I can say that every member of the team at Precision cares deeply about. However, we also understand the challenges that in vivo genome editing presents beyond that of ex vivo cell therapies. We can’t simply treat cells in the lab and then analyze and sort them before administering to the patient. We have to get it right the first time, every time. But, as a company dedicated to improving life, these are the challenges that we both embrace and are uniquely qualified to succeed in.