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Results seen across multiple trials bolster Sarepta’s “confidence in the potential disease-modifying benefits of this therapy,” said the company’s CEO, Doug Ingram. Read More
BioMarin plans to meet with the FDA to discuss these latest findings and expects to file a biologics license application in the second quarter of this year. Read More
Bayer and Mammoth Biosciences have announced a deal in which Bayer will shell out $40 million now and potentially up to $1 billion over time for the use of Mammoth’s CRISPR systems to develop in vivo gene-editing therapies. Read More
After flopping a phase 3 trial in hospitalized COVID-19 patients, Molecular Partners’ antiviral ensovibep has scored in a new phase 2 study, reducing viral load and cutting the risk of hospitalization or death by 78 percent in patients with mild-to-moderate disease. Read More
Tirzepatide, Eli Lilly’s potential dual-molecule blockbuster for type 2 diabetes (T2D), should be priced at $5,500 to $7,500 per year to be a cost-effective therapeutic option, according to the Institute for Clinical and Economic Review (ICER). Read More
Single-dose protection was 63 percent; by 13 days after boosting, it had risen to 84 percent, where it hovered for up to two months after the booster. Read More
The study did return positive results in overall quality of life and some hints of survival benefit at 12 months, so BridgeBio isn’t throwing the molecule in the dustbin yet. Read More
CoronaVac, Sinovac’s COVID-19 vaccine, appears to be completely ineffective against the Omicron variant unless boosted with an mRNA vaccine, new data suggest. Read More
The approach taken by drugmakers during the pandemic turned out COVID-19 vaccines in record time, but it has some shortcomings for use in future drug development and manufacturing, according to a survey of more than 500 industry leaders. Read More
BridgeBio’s investigational cardiomyopathy molecule acoramidis (AG10) fell short of the phase 3 finish line this week, when it not only failed its primary endpoint, but was associated with decreases in a functional measure of cardiac fitness relative to placebo. Read More