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.
Emendo Biotherapeutics Inc. CEO David Baram told BioWorld his firm’s allele-specific gene-editing approach offers such an advantage over previous methods that “we decided to take the challenge of curing diseases that require the highest precision possible,” and the New York-based firm bears an impressive list of partners. “Doors opened immediately and collaborations formed very fast,” sometimes “even faster than we could digest,” he said.
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.”
DUBLIN – Crispr Therapeutics AG has delivered what appears, so far at least, to be a safe, functional cure for the first patient enrolled in each of its phase I/II trials of lead CRISPR/Cas9 gene editing therapy CTX-001, in beta-thalassemia and in sickle cell disease.
HONG KONG – U.S.-based biotech firm Egenesis Inc., headquartered in Cambridge, Mass., has received $100 million through a series B funding round for the development of safe and effective human-compatible organs through gene editing.
LONDON – A paper that raised concerns for the future health of Lulu and Nana, the world's first gene edited babies, has been fully retracted at the request of the authors, after they failed to identify a problem in data from the U.K. Biobank on which their analysis was based.
