Man In First Muscular Dystrophy CRISPR Treatment Trial Dies
The Boston Globe, reporting on the news, said 27-year-old Terry Horgan was due to be the first person to get a custom CRISPR therapy for Duchenne muscular dystrophy, but it's unclear when or if he got the treatment. AP notes he was the lone volunteer in the study.
The Boston Globe:
Man Who Was First In Line For A Custom CRISPR Therapy Dies
A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The creation of the first-of-its-kind therapy for Terry Horgan, 27, was helmed by the Boston and Connecticut based nonprofit Cure Rare Disease — founded and led by Terry’s older brother, Richard Horgan. (Cross, 11/4)
AP:
Death In US Gene Therapy Study Sparks Search For Answers
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him. Terry Horgan, a 27-year-old who had Duchenne muscular dystrophy, died last month, according to Cure Rare Disease, a Connecticut-based nonprofit founded by his brother, Rich, to try and save him from the fatal condition. Although little is known about how he died, his death occurred during one of the first studies to test a gene editing treatment built for one person. (Ungar, 11/4)
More on gene therapy —
The Boston Globe:
Duke, MIT, And Stanford Scientists Create RNA Technology That Could Improve Genetic Therapies
Scientists from Duke, MIT, and Stanford have independently devised a molecular trick that could help make genetic therapies safer and more effective. The technology, which was disclosed in three separate papers published recently, could help ensure that treatments based on DNA or RNA are only activated once they reach the right part of the body. (Cross, 11/6)
In other pharmaceutical news —
NPR:
Statins 'Vastly Superior' To Supplements To Cut Heart Attack Risk, Study Finds
"What we found was that rosuvastatin lowered LDL cholesterol by almost 38% and that was vastly superior to placebo and any of the six supplements studied in the trial," study author Luke Laffin, M.D. of the Cleveland Clinic's Heart, Vascular & Thoracic Institute told NPR. He says this level of reduction is enough to lower the risk of heart attacks and strokes. The findings are published in the Journal of the American College of Cardiology. (Aubrey, 11/6)
The Wall Street Journal:
Walgreens Unit Close To Roughly $9 Billion Deal With Summit Health
A unit of Walgreens Boots Alliance Inc. WBA 3.72%increase; green up pointing triangle is nearing a deal to combine with a big owner of medical practices and urgent-care centers in a transaction worth roughly $9 billion including debt, according to people familiar with the matter, the latest in a string of acquisitions by big consumer-focused companies aiming to delve deeper into medical care. (Cooper, 11/6)
Stat:
Supreme Court Agrees To Review A Closely Watched Patent Case
In a boost for Amgen, the U.S. Supreme Court agreed to hear a case in which the company argued a federal appeals court incorrectly determined its patent claims on a cholesterol medication are invalid. (Silverman, 11/4)
Stat:
A 'Blank Check': Bill To Boost Antibiotic Development Blasted As A 'Flawed' Giveaway To Pharma
Amid mounting concern over antibiotic resistance, a coalition of academics and advocacy groups is urging Congress not to pass legislation to reward drug companies for developing new treatments, because they argue the bill fails to require acceptable clinical trial standards and is tantamount to a giveaway to the pharmaceutical industry. (Silverman, 11/4)
