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Researchers at the University of Texas at Austin shared results showing that their redesign of Cas9 makes it 4,000 times less likely to target the wrong stretch of DNA while still maintaining its efficiency.
The FDA has not yet issued any hold orders to Cassava, but the company expects that its time is coming after rivals Denali and Cortexyme received theirs.
The FDA said that the shared NDA documents did not show ample proof that bardoxolone methyl effectively slows down kidney function loss in patients with Alport syndrome.
A new project uncovering genetic changes linked to damage following a heart attack could potentially pave the way for gene therapies to prevent long-term cell damage.
Shares of Cassava Sciences are up more than 5% in premarket trading after the FDA denied a Citizen Petition filed last year to suspend the company’s Alzheimer’s clinical trials.
Taysha said the data for TSHA-101 is the “first-ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.”
Some might go as far as to consider the pharmaceutical industry as mankind’s best hope for survival. But now, as the world enters the third year of the pandemic, has that perception changed?
Shares of Mustang Bio have fallen more than 13% in premarket trading after announcing the FDA had placed a hold on the company’s Investigational New Drug application for its bubble boy gene therapy.
Last week was a relatively quiet week for clinical trial news, particularly around COVID-19. Here’s a look.
The Biotechnology Innovation Organization provides a glimpse of economic development in the biosciences ecosystem at the state and regional levels.
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