Researchers at the University of California at San Diego have used RNA-targeted CRISPR to reverse symptoms in an animal model of myotonic dystrophy type 1 (DM1). They reported their findings in the Sept. 14, 2020, issue of Nature Biomedical Engineering.

LONDON – The international commission convened in the aftermath of Chinese scientist He Jiankui’s shock announcement of the birth of gene edited twins has set a possible course to approval of heritable gene editing, but said the technique is far from ready for use.

Mammoth Biosciences Inc., of South San Francisco, and London-based Glaxosmithkline plc (GSK) have joined forces to develop a point-of-care test to detect active COVID-19 infections using Mammoth’s CRISPR-based DETECTR platform. The two companies hope to submit an application to the U.S. FDA for emergency use of the test before the end of the year.

Mammoth Biosciences Inc., of South San Francisco, and London-based Glaxosmithkline plc (GSK) have joined forces to develop a point-of-care test to detect active COVID-19 infections using Mammoth’s CRISPR-based DETECTR platform. The two companies hope to submit an application to the U.S. FDA for emergency use of the test before the end of the year.

CYBERSPACE – At the virtual annual meeting of the American Society of Gene and Cell Therapy, CAR T cells were the subject of both historical overviews and cutting-edge research alike.

Chinese scientists have shown for the first time that the down-regulation of a single RNA-binding protein, polypyrimidine tract-binding protein 1 (Ptbp1), locally converted glial cells to neurons and showed promise for treating the symptoms of neurodegenerative diseases in mice.

An experimental gene editing therapy for an inherited form of blindness has become the first in vivo CRISPR medicine to be administered to patients, according to Editas Medicine Inc. and its partner, Allergan plc, which licensed the candidate in 2018.

BEIJING – Chinese scientist He Jiankui has been sentenced to three years in prison and fined ¥3 million (US$429,421) for illegally carrying out the human embryo gene editing that led to the birth of twin girls and another baby with heritable changes to their genomes.  

For biopharma, 2019 can be described as a terrific year – with a few asterisks. The financial markets were flourishing, with venture capital dollars, in particular, flowing to the sector, while dealmaking reached historic proportions. Meanwhile, scientific breakthroughs led the way as cell and gene therapies gained ground, the first signs of success emerged with new technologies like CRISPR and the long-awaited promise of genomics found its way to the front lines of health care.

ORLANDO, Fla. – “The Wright brothers showed that you could fly a plane, but it wasn’t very far and it wasn’t very safe,” Wendell Lim told his audience at the 61st American Society of Hematology (ASH) annual meeting this weekend. “That’s where cell therapy is now.”