https://theprogressnetwork.org/gene-editing-therapies/
"It was 2024, and a baby named KJ had just been born. He had his mother’s
bright blue eyes, just like his three older siblings. Within days, however, it
was clear something was seriously wrong. Little KJ was diagnosed with CPS1
deficiency, a rare liver condition in which ammonia builds up in the blood and
eventually causes organ damage.
CPS1 is often treated with a liver transplant, but KJ was too small and
medically unstable to receive one. Instead, doctors came to his parents with an
idea. What if a gene editing therapy—which, by altering a small section of his
DNA, would correct a malfunctioning enzyme in KJ’s liver—could be built just
for him?
Six months later, KJ received his first dose of the therapy. Four months after
that, in June 2025, he was released from the hospital where he’d spent his
entire life. The treatment had stabilized his condition, with no serious side
effects. It was the first time a CRISPR-based therapy had been custom-built for
one patient.
CRISPR has made gene editing therapies for common genetic conditions, such as
sickle cell disease, a reality. Theoretically, similar therapies are also
possible for the more than 7,000 rarer ones, like KJ’s. But most people with
rare diseases are out of luck; it’s not worth it for biotechnology companies to
try to develop a cure given the small number of future customers. KJ’s story
raised the exciting possibility that bespoke gene editing therapies could
become viable workarounds.
Until last week, one barrier to that future was the Food and Drug
Administration (FDA). Though KJ’s therapy was built just for him, it could be
tweaked to treat others with related liver disorders. But traditional FDA
regulations required that each tweak be treated as a totally new drug,
subjected to its own lengthy review and large-scale clinical trials. Those, of
course, are impossible to conduct for most rare diseases.
Now, however, regulations have been updated so that researchers can “bundle”
data from distinct but related rare-disease treatments. This new clearance
pathway was developed specifically to fast-track gene editing solutions,
especially those that could help children. But it may eventually apply to a
broader range of conditions and therapy types—good news for both patients and
innovation."
Cheers,
*** Xanni ***
--
mailto:xanni@xanadu.net Andrew Pam
http://xanadu.com.au/ Chief Scientist, Xanadu
https://glasswings.com.au/ Partner, Glass Wings
https://sericyb.com.au/ Manager, Serious Cybernetics
