The budding field of genome editing holds dizzying possibilities for medical research, and could even unlock cures for previously untreatable genetic diseases. One firm at the forefront, Editas Medicine, plans to turn cutting-edge science into medical reality. Founded by scientists from MIT, Massachusetts General Hospital, the University of California, Berkeley, and Harvard University, Editas is developing medical therapies using a new gene editing technology called CRISPR that edits DNA at specific locations in the human genome. Editas is working on a number of different diseases and initial programs target severe ocular diseases and cancer immunotherapies.
Last year the company’s IPO added $109 million to its coffers — this in addition to $163 million in private funding from a blue-chip list of VCs and crossover investors. Katrine Bosley, former CEO of Avila Therapeutics and head of business development at Adnexus Therapeutics, joined Editas three years ago as CEO. We spoke to her about Editas’s CRISPR technology, and its application in severe ocular diseases and cancer immunotherapies.
What is the idea behind CRISPR?
Conceptually it’s an idea that’s been around ever since we understood that a mutation or mistake in DNA could lead to a disease. Decades ago, scientists were able to understand the sequence of DNA; later they sequenced the full human genome. CRISPR is a tool that takes advantage of this scientific foundation because we can use it to manipulate DNA and apply it to making medicines that will work at the level of DNA. Essentially, it has the potential to repair broken genes.
What is the promise of genome editing?
It has the potential to enable us to treat diseases that we could not treat before, working at the level of DNA to really correct genetic mistakes in patients who suffer from genetically-defined diseases. We may also be able to create treatments that have the kind of durable impact that we aren’t able to achieve with other kinds of drug modalities. There are small molecule drugs, antibody drugs, and many other kinds of pharmaceuticals, but with genomic-based medicines you have the opportunity to accomplish previously unreachable clinical goals.
There are about 6,000 different genetically defined diseases and 95% of them don’t have any approved therapeutics. They’re managed clinically but they don’t have approved therapeutics to actually treat the disease itself.
Gene editing is being touted as one of the biggest medical breakthroughs, is it?
There are many reasons for this to be considered an exciting breakthrough. One is absolutely the potential to develop medicines that treat diseases that don’t currently have good therapies. At the same time, the technology is being used in basic research to ask and answer all kinds of other scientific questions that you couldn’t attempt before because you didn’t have the tools. For instance, we can have a better understanding of how cells work or create model systems to mimic a disease that allows you to study the disease in different ways. Part of the reason you see such great excitement in the world of science about CRISPR is because there are so many different applications, not just the therapeutic possibilities but for basic science as well.
What are some of the challenges in determining how to apply CRISPR to humans?
One critical question we are looking at is the technical challenge of how to deliver a CRISPR-based medicine and what part of the body we need get the medicine to reach. A nerve cell is different from a blood cell, and they’re both different from skin cells. Each cell type will require different delivery approaches. A second critical technical question is exactly what kind of repair to the gene needs to be made? As you might imagine with 6,000 different genetic diseases, there is a wide variety of different kinds of mutations that underlie those diseases, some are easier to correct than others.
How does that apply to developing an entirely new medical therapy?
The type of technical challenges we need to overcome and solve are what I’ll call translational science challenges: it’s going from the kinds of basic research that occurs in academic science and then translating that into the applied world of developing a medicine. For instance, while we are figuring out how to deliver to the right cells in the body, we also have to invent technologies to support our progress. No one has ever developed this kind of medicine before, so supporting technologies like analytical methodologies and measuring techniques don’t always exist. When you’re developing a new category of medicines like this, it’s not just about developing the medicine itself, you’re also developing a lot of capabilities so that you can understand your medicine as you develop it.
What are your primary areas of focus?
Our lead program currently is a treatment for a particular form of genetic blindness called Lebor Congenital Amaurosis 10, or LCA 10. Our therapy for LCA10 is currently in pre-clinical development, and earlier this year, we announced that we achieved in vivo proof of editing in the retinas of non-human primates. This achievement is a big step forward in advancing a genome editing therapy. The next step is to apply to the US Food and Drug Administration for an Investigational New Drug application. Then, the next step is clinical trials.
In addition to our ophthalmology work, we have a number of programs in diseases of the blood and bone marrow, including both internal programs and our programs partnered with another biotech company, Juno Therapeutics. We’re combining our technology for gene editing with Juno’s technology for engineering T cells. T cells are an important part of your immune system. Juno engineers them to better fight cancer cells. We can add gene editing on top of that, to further enhance the T cells’ capability. The goal is to be able to take cells from the body’s own immune system, engineer them to better fight cancer, and then give them back to the cancer patient. This is a very exciting emerging area within cancer therapy.
This is a field that moves at breathtaking speed. The science is robust, but even in a world of robust science this has moved really quickly. The basic science in our hands is working well, and we continue to work on further improving and expanding what it can do in addition to working on new programs. We’ve made progress in developing a potential treatment for LCA10 blindness. Through that program, we’re figuring out how to do editing in the eye more generally, which may enable work in other diseases of the eye such as genetic and infectious diseases.
One of the exciting but also challenging parts of genome editing is that you work across the whole genome, so any genetically defined disease is potentially a disease that we can work on. But they’re not all equally technically feasible just yet. As we make progress with the basic technology, it does start to open up more of that territory. What comes next is working on more diseases and hopefully in a wider range of therapeutic areas; solving the challenge of how to deliver to more and different tissues in the body then helps us work on a wider range of diseases.
Ultimately it comes down to this: What if you could repair broken genes? We’re here because we think we can do that.
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