The Amazon of Gene Therapy
How scientists used genetic engineering and ML to bring a virus back from the dead as a better deliver method for gene therapies
Gene therapy is a powerful tool to treat diseases from cancer to deafness, but it requires safe and effective delivery to the correct cells in the body. This post is about the unique genesis of Anc80, a new viral delivery system.
Gene therapy has two parts: 1) the genetic package, to fix a disease-causing error, and 2) the delivery system, to deliver the fix to its intended location.
Existing delivery systems cannot reach all cells, limiting gene therapy’s utility.
Anc80 has proven 1000X more effective in targeting difficult-to-reach cells in mice and the first human trials start this year. If successful, Anc80 will enable drugs for many currently-untreatable diseases.
Delivering Gene Therapy: A Very Brief History (1985–2015)
Delivering genetic packages is hard, but nature has given us a bespoke cellular infiltration system: the adenovirus.
Adenoviruses normally cause respiratory infections, but scientists in the 1980s began hijacking the infectious machinery to deliver genetic packages into cells…
