Nearly all patients treated so far have been relieved of the blood-clogging crises of the disease.
One after another, the men and women testified how a new gene-editing treatment had relieved their suffering from sickle-cell disease. They addressed a Tuesday hearing in which the Food and Drug Administration heard patients and scientists weigh in on what’s likely to be the country’s first-approved genetic treatment for the disorder.
All but one of the dozens of patients treated with exa-cel in clinical trials have gone for more than a year without the blood-clogging crises of sickle cell, and have been spared the frequent hospital stays that those crises provoked. The one exception was a patient whose condition was aggravated by an infection, and his symptoms were relatively mild.
FDA documents posted online in preparation for the meeting don’t reveal great concerns at the agency over exa-cel. After reading them last week, William Blair analyst Sami Corwin wrote that they set up “a near-best-case scenario” for the meeting—and for exa-cel’s approval as a treatment for sickle cell.
At the meeting, agency reviewers were mainly concerned with the exa-cel developers’ tests for off-target DNA edits that might cause side effects. That crowded field is one reason why shares of Vertex and Crispr Therapeutics haven’t gotten much lift from exa-cel’s approaching approval. Vertex has roughly tracked the Nasdaq Composite Index, while Crispr has lagged behind them both.
United States Latest News, United States Headlines
Similar News:You can also read news stories similar to this one that we have collected from other news sources.
FDA Advisors Consider Crispr Gene-Editing Treatment for Sickle Cell AnemiaNearly all patients treated so far have been relieved of the blood-clogging crises of the disease.
Read more »
CRISPR/Cas9 unlocks TuMV resistance in Chinese cabbage: A leap forward in genome-edited plant breedingGenome editing, especially the CRISPR/Cas9 technology, holds immense promise in enhancing plant traits, primarily disease resistance, offering a more efficient alternative to traditional breeding.
Read more »
Unlocking the genetic potential of poplars: A new era of precision genome editing with CRISPRThe Populus genus, commonly known as poplars, cottonwoods, and aspens, consists of approximately 30 tree species native to the northern hemisphere. Because of their diverse usages in landscape, agriculture, bioenergy, and industry, Populus species have been the focus of many tree breeding and genetic improvement programs.
Read more »
VinFast Enters Into Standby Equity Subscription Agreement of Up to $1 Billion With Yorkville AdvisorsVietnamese electric vehicle maker VinFast and global investment fund manager Yorkville Advisors have entered into a standby equity subscription agreement of up to $1 billion.
Read more »
Left-wing Arabella Advisors dark money groups cut ties with Palestinian terror-tied charityGabe Kaminsky is an investigative reporter for the Washington Examiner. You can follow him on X: gekaminsky.
Read more »
A ‘new turning point’: Patients could soon have a new shot to protect them from deadly meningitisThe CDC's independent vaccine advisors recommended a new vaccine that could potentially prevent meningitis B.
Read more »