Posted on 12/08/2023 11:39:13 AM PST by bitt
The Food and Drug Administration has approved the world's first medicine employing CRISPR gene-editing technology to treat sickle cell disease, giving thousands new hope to treat the painful disease.
"Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today," said Nicole Verdun, director of the Office of Therapeutic Products within the FDA's Center for Biologics Evaluation and Research.
Two new treatments, Casgevy and Lyfgenia, were approved on Friday in a long-anticipated move that tackles genetic disorders by turning off or replacing specific genes.
Approximately 100,000 people in the United States are affected by sickle cell disease, which is the result of an inherited genetic mutation that produces a dysfunctional form of the protein hemoglobin, which carries oxygen in the bloodstream. Hemoglobin proteins in those affected with the disease are shaped in the form of a sickle, giving the disorder its name.
Casgevy targets the specific gene that causes the dysfunction, switching it off and allowing for the production of fetal hemoglobin proteins. Lyfgenia works by producing a gene-therapy-derived hemoglobin that functions similarly to the adult form of the protein.
Both products come from a patient's blood stem cells, which are delivered in a one-time, single-dose infusion once they have been genetically modified. The treatments are approved for sickle cell disease patients over 12 years old with recurrent vaso-occlusive events.
"Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited," Verdun said.
Sickle cell disease occurs most frequently in black populations, affecting about 1 in every 365 black births in the United States.
(Excerpt) Read more at washingtonexaminer.com ...
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It may be good, but the FDA squandered their credibility with the COVID vax. History says hoax.
CRISPR? no thanks
FDA....You're credibility was pissed away years ago. We all are in a worse place for that. Your legacy. Our problem. We will solve it. Soon.
Sickle cell anemia is a disease that does not discriminate.
So we need to watch VERY closely and see how the different demographic group experience side effects.
CRISPR?
Where I keep my lettuce.
(Yeah I know what it is)
https://freerepublic.com/focus/chat/4202231/posts?page=6#6
They have competition from an mRNA/nanolipidparticle (NLP) injection.
Good if that’s really what it is. If.
“ Both products come from a patient’s blood stem cells, which are delivered in a one-time, single-dose infusion once they have been genetically modified.”
I need to know more about how this works. It sounds like they have to kill off almost all the original stem cells before they inject the modified ones. That would leave the recipient very immune-compromised for many months.
First thought was cremation.
That follows the treatment...
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