Ann: Successful GLP Tox Studies in Second Blinding Eye Disease, page-10

When results start coming through, there will be a belated realization of the potential.

There are currently around 7,000 known monogenic diseases caused by mutations in a single gene. However, a recent study identified 72 monogenic disorders that have a prevalence of at least 1 in 20,000 individuals in the human population. Of these, 34 are caused exclusively by mutations in a single gene. The number of known monogenic diseases continues to grow as gene discovery efforts advance, aided by technologies like whole exome/genome sequencing.
https://pubmed.ncbi.nlm.nih.gov/34515378/

Stoke therapeutics have identified approximately 1,200 monogenic diseases and approximately 6,500 additional genes with RNA target signatures which we believe to be amenable to TANGO.