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In Case You Missed It: Three Things We Learned In Our Gene Editing Webinar

Missed our gene editing webinar? Fear not! Here are a few of the highlights from our panel of experts which included Verve Therapeutics’ CEO, Sekar Kathiresan as well as our home-grown specialists. And if that’s not enough, the webinar is available on demand.

Gene editing is a hot topic right now. With the first Pdufa date for a Crispr-edited therapy (Crispr Therapeutics/Vertex’s ex vivo project exa-cel) now set for late this year, has the moment finally come for gene editing to hit the mainstream? Obviously, it’s too early to know, but our panel had plenty to discuss in the session. Here are three of my key takeaways.

  1. Determinism and optimism rule
    At the recent ASGCT conference, the mood was upbeat. This year’s ASGCT was the biggest in the event’s 10-year history and there are high hopes for what might be possible in terms of meeting the huge unmet need across a range of diseases. One note of caution, though, was that investors are being very careful. Companies looking to secure funding need more than “just” good science now, and passing the baton from series A to series B is taking longer. There’s excitement, but it’s tempered by the current financial climate.
  2. More trial results are on their way
    With scores of trials for a wide range of diseases taking place around the world, there will be plenty for bio-watchers to look out for. Verve Therapeutics is currently running a trial in the UK and New Zealand for its gene editing contender VERVE-101 in heterozygous FH (Familial hypercholesterolaemia). And data due later this year are set to be the first results of base editing trials in humans. It’s worth adding that VERVE-101 remains on clinical hold in the US and it’s unclear when this will be lifted. Verve’s CEO Sekar Kathiresan noted that they are having open discussions with the FDA and that all regulators recognise the unmet need in FH and the potential of a “one and done approach that has the potential to be a definitive genetic treatment”.
  3. Deal making is driven by good data AND good science
    There are always deals to be made and Amanda Micklus took us through some of the key deals and the trends from recent years. One this is clear, there’s a big appetite for gene editing from big players. In vivo in particular, is attracting investment from the likes of Novartis and Pfizer with several deals worth over $1bn now in place. Since 2020, we’ve seen a decline in the average deal spend but a rise in the upfront payments. This front-loading of the deals may be an indication of higher confidence from the big guys.

These are just a few highlights from the webinar so do check out the full recording which is available here if you have time. We also have our report, Gene Editing: Overhyped or Unstoppable Tide. That report features interviews with a number of the leading gene editing companies including many of those mentioned during the webinar.


Carolyn Hall

Director, Content & Thought Leadership Marketing


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