Crispr/Cas9 is working well…but we’re still learning how to move this cargo into the correct cells. (1/3)
Hello friends,
I’m Kevin Curran, a molecular biologist and life science educator. My newsletter explores advanced medicine and the future of biotech.
In my next three newsletters, I review the current state of gene editing medicine, specifically focusing on the challenge of delivering gene editing tools into the correct tissue in a patient’s body. Solving for gene editing delivery is one of the most critical challenges in biotech today.
In the video below, I provide an introduction to gene editing delivery and describe how viral vectors and ‘naked cargo’ could be used to deliver this medicine. In subsequent newsletters, I’ll review the use of LNPs and VLPs.
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The image below illustrates different locations in the human body where scientists are currently focused on the delivery of gene editing tools. To put it simply, most diseases are caused by a malfunction in one particular tissue in the human body. To address these diseases, we need to master the skill of delivering precise gene edits into these specific tissues.
Much of the delivery work summarized above is still in the early stage of clinical trials or else still in pre-clinical (animal work). However, we do have one FDA approved gene editing drug (Casgevy, from Vertex) that is delivered to bone marrow stem cells once the cells are removed from the body.
Another company, named Intellia, has just begun a late-stage trial for NTLA-2001 for a liver disease, called ATTR. If approved, NTLA-2001 would be the first gene editing drug that is administered directly to the human body. NTLA-2001 relies on a LNP (lipid nanoparticle) to deliver CRISPR/Cas9 into liver cells via administration into the bloodstream.
In the You Tube video below, I summarize this field of research and articulate the key themes and objectives that are central to gene editing delivery.
My intention was to make a 10 minute video…. but apparently I like to talk because I now see the final video is 37 minutes. Yikes, well hopefully long form content is coming back in fashion.
If these videos are helpful, then let me know and I’ll keep making them.
Take care,
Kevin
