Bailey told the US Food and Drug Administration’s independent advisory committee Tuesday that she believes a cutting-edge therapy that is currently under review offers the sickle cell community something many haven’t ever had before: hope.
CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop.
In company studies, the treatment was considered safe, and it had a “highly positive benefit-risk for patients with severe sickle cell disease,” Dr. Stephanie Krogmeier, vice president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel.
Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshapen red blood cells block normal circulation and can cause moderate to severe pain.
Dr. Scot Wolfe, a temporary voting member of the committee and professor at UMass Chan Medical School, said he liked that the company promised to monitor patients for 15 years to see if there were any problems down the road.
Asked by the committee how he would advise patients how to evaluate the risks with this treatment, Bauer said he would be honest that there is some uncertainty, but most of the human genome is non-coding, meaning it doesn’t provide instructions to the cells to act a certain way.
The original article contains 1,216 words, the summary contains 222 words. Saved 82%. I’m a bot and I’m open source!
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Bailey told the US Food and Drug Administration’s independent advisory committee Tuesday that she believes a cutting-edge therapy that is currently under review offers the sickle cell community something many haven’t ever had before: hope.
CRISPR, or clustered regularly interspaced short palindromic repeats, is a technology researchers use to selectively modify DNA, the carrier of genetic information that the body uses to function and develop.
In company studies, the treatment was considered safe, and it had a “highly positive benefit-risk for patients with severe sickle cell disease,” Dr. Stephanie Krogmeier, vice president for global regulatory affairs with Vertex Pharmaceuticals Incorporated, told the panel.
Thirty-nine of the 40 people tested with the treatment did not have a single vaso-occlusive crisis, which means the misshapen red blood cells block normal circulation and can cause moderate to severe pain.
Dr. Scot Wolfe, a temporary voting member of the committee and professor at UMass Chan Medical School, said he liked that the company promised to monitor patients for 15 years to see if there were any problems down the road.
Asked by the committee how he would advise patients how to evaluate the risks with this treatment, Bauer said he would be honest that there is some uncertainty, but most of the human genome is non-coding, meaning it doesn’t provide instructions to the cells to act a certain way.
The original article contains 1,216 words, the summary contains 222 words. Saved 82%. I’m a bot and I’m open source!