Preclinical
Amicus Therapeutics, headquartered in Cranbury, New Jersey, held a Type C meeting with the U.S. Food and Drug Administration (FDA) regarding its regulatory path for AT-GAA for Pompe Disease. The company released its plans for the drug.
There are plenty of great scientific research stories out this week. Here’s a look at just a few of them.
Researchers at the University of Texas Southwestern Medical Center used CRISPR gene editing to treat Duchenne muscular dystrophy (DMD) in dogs. Their work was published in the journal Science.
Cystic fibrosis drugmaker Vertex Pharmaceuticals forged a three-year collaborative deal with U.K.-based machine learning specialist Genomics plc to improve the discovery of targets for precision medicines.
Maryland-based REGENXBIO is expanding its gene therapy pipeline to include a new treatment for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease.
Citing safety concerns, a watchdog organization is calling on the National institutes of Health to halt enrollment in a clinical trial evaluating a treatment for sepsis that the organization said is risky and flawed.
Researchers at the University of Texas Health Science Center at San Antonio have found a type of cellular stress in Alzheimer’s disease for the first time that is involved in cancer and aging. The stress is called cellular senescence, which is linked to the tau protein tangles associated with end-stage Alzheimer’s disease. The research was published in the journal Aging Cell.
The U.S. Food and Drug Administration has posted new draft guidance that will guide drug and medical device manufacturers that aim to develop treatments for “the underlying pathophysiology and structural progression” of osteoarthritis (OA).
In the biotech world, only 7 to 9 percent of companies have women as their chief executives. Antiva BioSciences is a good example of how this problem is perpetuated.
Biohaven Pharma, headquartered in New Haven, Conn., announced positive data from a proof-of-concept trial for its BHV-0223 in social anxiety and public speaking anxiety disorders.
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