Month: July 2017

31
Jul 2017

Bionest

Human Germline Gene Editing Becomes Reality — at Least at the Research Stage

Back in April of this year, we wrote about the National Academies of Science and Medicine cautiously endorsing the possible use of gene editing at the germline level to correct serious diseases or medical conditions. Their report commented that scientists would likely be in a position to accomplish such activities safely within 5 to 10...
26
Jul 2017

Bionest

CAR-T Nears the Marketplace, with Unanimous Approval Recommendation – but Challenges Remain

On July 12, Novartis’ CAR-T therapy, CTL019 (tisagenlecleucel), received a unanimous recommendation for approval from the reviewing U.S. Food and Drug Administration’s advisory panel for use in the treatment of children and young adults with advanced acute lymphoblastic leukemia (ALL). The FDA is now expected to make its final decision by October 3.   CTL019...
17
Jul 2017

Bionest

Gene Therapy: Coming of Age Perhaps, but Who Will Pay?

New technologies tend to go through cycles of interest and attention. Initial excitement about the potential of a technological advance too often builds to over-blown hype, followed by a quick reversal of interest in the aftermath of a big disappointment. But if continued research later produces truly encouraging results, interest can build again as time...
12
Jul 2017

Bionest

CRISPR Reaches the Clinic

Sometime mid-month, the first clinical trial employing CRISPR gene editing technology within the human body will initiate in China, at Sun-Yat Sen University. Scientists there will seek to use CRISPR and TALEN, another gene editing method, to treat persistent HPV infection and disable the tumor growth mechanism in infected cells. In a non-invasive approach, scientists...
5
Jul 2017

Bionest

FDA: Thinking “Outside the Box”

On May 17, the U.S. Food and Drug Administration granted an expanded approval to Vertex’s Kalydeco (ivacaflor), tripling the number of mutations in the cystic fibrosis (CF)-associated gene CFTR that the drug can treat, from 10 to 33. The agency’s action was unusual in that, for the first time ever, they based their decision on...