What Happened to Broadly Accessible Gene Therapy?

As the year 2000 crept in, the Y2K bug imperiled life as we once knew it, threatening to wreak havoc with our computers and the world’s networks. Seven months later, in July, even bigger news, which was actually true, had parts of the world trembling with joy. In Letters to Nature, published in Nature (magazine), a team of scientists led by Dr. Michel Sadelain informed the world that they expressed the human β-globin gene in mice. The suspected result was that the two largest genetic blood diseases in the world, Sickle Cell Disease (SCD) and β-thalassemia (Thal), would shortly be cured.
Unfortunately, this did not go quite as the scientists or patients had hoped. Finally, over 23 years later on December 7, 2023, both Bluebird Bio’s Lyfgenia gene replacement and Casgevy, CRISPR’s gene editing, were approved, for $3.1M and $2.2M per patient.