The big gene edit: the view from the front line

Tim Farries, PhD

Reflections on a lively panel debate at the 2022 Cell & Gene Meeting on the Mesa

by Tim Farries, Ph.D, Principal Consultant and Senior Director

In October I had privilege of chairing an expert panel discussion at the above event, on the accelerating progress in genome (gene) editing.

We explored the technology’s promise (and how it differs from gene therapy: it modifies the DNA permanently), and the remaining challenges as science becomes reality. Unsurprisingly, the session was very well attended and we covered a lot of important ground.

Mark Shearman from Editas Medicine talked about gene editing’s potential for longer term efficacy, because of its scope to ‘fix’ a problem at its source (longer term outcomes can be limited by cell division in gene therapy, which sees healthy genes introduced while mutated or damaged genes remain in place.)

Bringing regulators on the journey

At least as importantly as the science itself is the ability of regulators to adapt safety requirements to allow breakthrough treatments to progress when they don’t fit the usual criteria for approval. The panel highlighted the important work being done here by the Alliance for Regenerative Medicine (ARM), the global advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies.

Devyn Smith of Arbor Biotechnologies talked about the value of presenting a consolidated view from across the sector to agencies such as the FDA – rather than individually take 15 different approaches to the regulator. This will help to reduce complexity where possible, and streamline the development of new, fit-for-purpose regulatory pathways.

The risk/benefit calculations are critical, too. In rare disease areas where there isn’t much of a precedence, companies are running into situations where the outcome measures are being transposed from known indications to new indications. This isn’t ideal, so there needs to be more dialogue with agencies such as the FDA around how to interpret information, as well as an openness to accept data from multiple endpoints, the panel agreed.

Everyone accepted that it is still very early days for gene editing, even for gene therapy, so it’s probably too soon to differentiate between likely regulatory pathways for ex vivo vs in vivo approaches and techniques (that is, where gene modification happens external to or inside the body), though again some exciting developments are happening at a research level.

The key in the meantime is to continue to partner with the FDA and other agencies to help them understand the good, the bad and where the opportunities are, and how we can collectively ensure that any applications are safe.

Pooling insights

LogicBio’s Fred Chereau hoped the industry would continue to educate the various authorities and share learnings with them, to help drive the best possible guidance – to ensure not only the safety of the patient, but also the feasibility of drug development. When it comes to such innovative therapies, partnerships between the industry and its regulators are vital, he said.

ARM plays an invaluable role here too, as a unifying force, Albert Seymour of Homology Medicines noted. It enables companies to share early experiences, brainstorm ideas and best approaches, and generally learn from each other – again as a catalyst for positive industry advancement.

Tremendous potential

The panel’s enthusiasm for the entire field of gene technology, and gene editing in particular, was palpable.

As Arbor’s Devyn Smith put it, “To think that you can actually edit and completely fix the problem, and that we can do that today, is amazing. It brings an ability not just to treat disease but to actually cure it, and do that in the actual endogenous DNA rather than having an ectopic DNA – that’s the promise. Are we going to get there tomorrow? No – it’s going to it’s a journey. But we’re seeing a lot of great progress.”

As a final but important note, the panel considered the evolving attitude of investors backing this cutting-edge science. Crucially, they understand the promise. But it’s incumbent on all of us to continue to generate the data required to support that backing and deliver the returns.

Mark Shearman described the multi-layered expectations: “The focus is on clinical data, proving safety and efficacy. They want to see a projection of where the program’s going, and the timeline over which you’ll be able to submit applications. They want to be confident that you can actually manufacture the product in a controlled fashion. And they want to see examples of where your analytics have been reviewed by regulatory authority.”

Pricing and reimbursement scheme are still to be defined. And the potential will obviously grow as the range of indications expands, and companies can target wider populations – conditions such as sickle cell anemia, for example.

The debate then took us deeper into the science, and was followed by extensive audience interaction.

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