Pfizer announced in February 2025 that it was taking Beqvez (fidanacogene elaparvovec), its gene therapy for hemophilia B, ...
Pfizer and BioMarin have pulled their hemophilia gene therapies, citing low demand. To prepare for his presentation at the ...
The FDA’s recently unveiled plausible mechanism framework may have been forged with i | The FDA’s newly launched plausible ...
Cancer cells excel at evading detection, but subtle chemical differences set them apart from healthy cells. Now, a team of ...
Pharmaceutical Technology on MSN
FDA shares guide on genome editing best practices
This guidance emphasises the potential of NGS methods in mitigating safety risks associated with therapies incorporating genome editing.
Cancer drugs are designed to shut tumors down. But sometimes, in the very act of attacking a tumor, treatment can also help a ...
A lab in Hawaiʻi is developing nanobodies, tiny proteins that help the immune system recognize and destroy cancer cells, ...
Following favorable phase 1 data showing significant reductions in HBV DNA and RNA, the FDA has fast tracked pevifoscorvir sodium for chronic HBV infection.
Researchers from Children's Hospital of Philadelphia (CHOP) developed a new RNA sequencing strategy that can reveal how ...
Morning Overview on MSN
Compact CRISPR tool boosts in-body gene editing to 90% in lab tests
For the millions of people living with genetic diseases like muscular dystrophy and inherited liver disorders, one of the ...
"I want more time, more memories, and the chance for a better life than my father’s," writes Rachel Reising, who has Huntington's disease.
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