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Is CRISPR safe? Genome editing gets its first FDA scrutiny
https://www.nature.com/articles/d41586-023-03317-7NEWS EXPLAINER
28 October 2023
Is CRISPR safe? Genome editing gets its first FDA scrutiny
Advisors to the US regulatory agency will examine the safety profile of a CRISPR-based treatment for sickle-cell disease.
Heidi Ledford
A therapy based on the CRISPR-Cas9 genome editing system could become the first of its kind to gain approval from the US Food and Drug Administration. But the treatment — designed to alleviate a painful blood condition — must first face intense scrutiny by the agency and its advisors.
On 31 October, external advisors to the US Food and Drug Administration (FDA) will meet to discuss a DNA-altering therapy for sickle-cell disease, a genetic condition that can cause misshapen blood cells and sometimes debilitating pain. The advisors’ discussions are likely to be laser-focused on safety data submitted by the treatment’s developers, Vertex Pharmaceuticals in Boston, Massachusetts, and CRISPR Therapeutics in Zug, Switzerland.
“The key to this is safety,” says Mark Walters, a paediatrician at the University of California, San Francisco, who has served on a steering committee advising the two companies on the clinical development of the treatment, called exagamglogene autotemcel (exa-cel), “That’s the question that could really affect decision making, and the safety information is still quite limited.”
[...]
28 October 2023
Is CRISPR safe? Genome editing gets its first FDA scrutiny
Advisors to the US regulatory agency will examine the safety profile of a CRISPR-based treatment for sickle-cell disease.
Heidi Ledford
A therapy based on the CRISPR-Cas9 genome editing system could become the first of its kind to gain approval from the US Food and Drug Administration. But the treatment — designed to alleviate a painful blood condition — must first face intense scrutiny by the agency and its advisors.
On 31 October, external advisors to the US Food and Drug Administration (FDA) will meet to discuss a DNA-altering therapy for sickle-cell disease, a genetic condition that can cause misshapen blood cells and sometimes debilitating pain. The advisors’ discussions are likely to be laser-focused on safety data submitted by the treatment’s developers, Vertex Pharmaceuticals in Boston, Massachusetts, and CRISPR Therapeutics in Zug, Switzerland.
“The key to this is safety,” says Mark Walters, a paediatrician at the University of California, San Francisco, who has served on a steering committee advising the two companies on the clinical development of the treatment, called exagamglogene autotemcel (exa-cel), “That’s the question that could really affect decision making, and the safety information is still quite limited.”
[...]