In a new report, we spoke to executives from a number of gene editing companies to learn more about their view on the progress of the science, the challenges and opportunities ahead and the regulatory hurdles that face gene editing technologies. It’s been an illuminating process and highlights a number of big questions that remain.
- Will in vivo eclipse ex vivo?
The first wave of gene editing has been all about ex vivo – extracting patient cells, editing them and then re-implanting them. It’s an expensive, cumbersome and pretty grim process for the patient. The alternative is to edit the genes in vivo instead, but there are concerns about unleashing such new technology inside patients. Within the group we interviewed for the report, there’s a pretty bullish sense that in vivo will be the approach of choice eventually but many felt that there will always be a place for ex vivo too.
- Are next-generation technologies like base and prime going to overtake Crispr?
While Crispr might feel like the new kid on the block, there are already newer platforms being investigated. Base editing and prime editing, for example, do not cause the double-stranded breaks that are a concern with Crispr. Both approaches are still at very early stages so only time will tell if Crispr will eventually look clunky in comparison.
- How important will new delivery technologies be?
In case there’s not already enough development and uncertainty in this area, there are also new delivery technologies being tested. One of the current most popular delivery vehicles, lipid nanoparticles, tend to head to the liver, which limits the potential scope of treatable indications. Other delivery technologies in development – such as protein nanoparticles (PNPs) and virus-like particles (VLPs) – might widen this scope dramatically. The importance of technologies like these will depend to some degree on the success that Cas9/Crispr-led approaches have, but there are biotechs investing heavily in this area.
- Are the regulators being extra-harsh on gene editing?
Any new drug faces regulatory hurdles but there has been a great deal of focus on gene editing, particularly with in vivo approaches. Given that in vivo editing permanently changes the patient’s genome, this is hardly surprising. Many of the earlier-stage companies we spoke to felt that being a little later to the party would work in their favour as it would enable them to learn what the regulator is looking for from the frontrunners. John Evans, CEO at Beam pointed out “the difficulty is when there’s something new, the FDA doesn’t know exactly what to ask you for, because they don’t know the science”. The consensus across the group we spoke to was that in vivo was being treated with an appropriate level of caution by the FDA.
- Is gene editing really going to be as big as everyone hopes?
This is the big one and there are certainly plenty of investors who are banking on gene editing becoming huge. In a rough market, there have been some significant funding rounds over the past couple of years. As well as potentially offering life-changing therapies to patients with few options, there are potentially some hefty rewards for the companies who get successful gene editing products to market.
To summarise the optimism in the gene editing world, we can look to a quote in our report from David Hallal, CEO of ElevateBio who told us “It’s not a matter of if, it’s when this becomes a dominant modality that changes the world”. Let’s wait and see…
Find out more in our new report, Gene Editing: Overhyped or Unstoppable Tide? or register for our webinar featuring Sekar Kathiresan of Verve Therapeutics.