DUBLIN – Cellectis SA is picking up $15 million worth of equity in Cytovia Therapeutics Inc. and could earn as much as $760 million in development, regulatory and sales milestones from a deal involving up to five gene-edited allogeneic natural killer (NK) cell or chimeric antigen receptor (CAR-NK) cell therapies employing its Talen (transcription activator-like effector nuclease) gene editing technology.
Sekar Kathiresan is building Verve Therapeutics Inc. around the concept of a one-and-done treatment for cardiovascular disease because only half of patients are disciplined enough to take a statin every day. “That’s a huge issue for durable cholesterol lowering after a heart attack,” Verve’s CEO and co-founder told BioWorld.
Tessera Therapeutics Inc., a Cambridge, Mass.-based company working to "write therapeutic instructions into the genome," has raised $230 million in series B financing to back its development of potential cures and treatments for cardiovascular, oncological, neurodegenerative and infectious diseases.
Metagenomi Inc. has raised a $65 million series A financing to expand its gene editing abilities, advance its research and validate its pipeline in preclinical studies. The company’s CRISPR-based systems use algorithms for screening thousands of genomes from microorganisms to advance therapies for use in oncology, genetic diseases and possibly much more.
DUBLIN – Versant Ventures, the founding investor of Crispr Therapeutics AG, is teaming up with one of the academic founders of that company, Stanford University’s Matt Porteus, to start a new gene editing firm, Graphite Bio Inc., which is focused on targeted integration of DNA to achieve a therapeutic benefit.
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.”
