Late last week, the FDA held its two day Advisory Committee meeting to examine the toxicity risks of AAV vectors for gene therapy. During that meeting, the FDA advisory panel heard of several cases of AAV gene therapies leading to vector-induced liver cancers in mice.
And now, already this week, the FDA has put a hold on a Phase 1/2 study of Biomarin’s BMN 307 gene therapy in phenylketonuria following the discovery of tumours in mice one year after their dosing in pre-clinical studies. Of seven mice that received the highest dose of BMN 307, six developed tumours, with evidence of integration of the AAV vector into the genome.
In the opinion of Evaluate Vantage
“This raises the spectre of insertional mutagenesis, wherein a vector-delivered gene tampers with a target cell’s chromosomal arrangement, triggering cancer.”
https://www.evaluate.com/vantage/articles/news/trial-results/another-gene-therapy-safety-scare-hits
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$1.15 |
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Open | High | Low | Value | Volume |
$1.15 | $1.18 | $1.11 | $172.3K | 151.3K |
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1 | 592 | $1.15 |
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$1.16 | 4211 | 1 |
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No. | Vol. | Price($) |
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1 | 2000 | 1.120 |
3 | 9261 | 1.100 |
1 | 2000 | 1.050 |
2 | 33803 | 1.000 |
1 | 1020 | 0.980 |
Price($) | Vol. | No. |
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1.160 | 4211 | 1 |
1.175 | 10000 | 1 |
1.180 | 33000 | 2 |
1.200 | 25749 | 4 |
1.205 | 2270 | 1 |
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