Bypassing the gene editing roadblock: “vast majority of the 400 million people in this world with genetic diseases suffer from diseases too rare for companies to be able to recoup the costs of drug development”

https://archive.ph/20251119164735/https://www.nytimes.com/2025/11/19/health/gene-editing-rare-diseases.html#selection-857.337-857.507

“he receives many heart-rending emails from anguished patients with genetic diseases or their families, and he doesn’t want them to expect widespread solutions anytime soon.”

Because of redundant systems, disorders from single gene mutations are relatively rare but still require custom edits. One day we custom edits will cost far less than today, but most with disease will be dead by then. Bypassing stop codons is a fairly desperate measure but may become viable sooner.