LogicBio team to present data on preclinical effectiveness of GeneRide platform

May 14 2018

LogicBio Therapeutics, Inc., a genetic medicine company founded to develop safe medicines with lasting therapeutic benefit for children with life-threatening diseases, today announced it will be presenting data on the company's technology and robust preclinical pipeline at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) at the Hilton Chicago, from May 16-19, 2018.

The LogicBio team will present abstracts demonstrating the preclinical effectiveness of the company's platform technology, GeneRide™, in hemophilia B and methylmalonic acidemia (MMA)—a rare, life-threatening metabolic disease. GeneRide™ enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LogicBio recently announced a development program targeting MMA.

In addition, LogicBio collaborators from the National Human Genome Research Institute (NHGRI) at the National Institutes of Health (NIH), Oregon Health and Science University (OHSU) and Stanford University will also highlight the preclinical effectiveness of the GeneRide™ platform in presentations during Saturday's oral abstract session. NIH scientist Randy Chandler will present data demonstrating the preclinical efficacy of GeneRide™ in mouse models of MMA; OHSU researcher Sean Nygaard will describe a novel approach for selecting gene edited hepatocytes; and Stanford researcher Gustavo De Alencastro will present data on a strategy to increase the efficiency of GeneRide™.

Details of LogicBio presentations:

Promoterless Targeting without Nucleases of Hyperactive Factor IX Corrects the Bleeding Diathesis in Hemophilia B Mice
Session: Gene Targeting & Gene Correction I
Abstract Number: 188
Time: Wednesday, May 16, 5:30 pm - 7:30 pm
Place: Stevens Salon C & D

Generide™, a Novel AAV Strategy to Treat Pediatric Patients with Methylmalonic Acidemia
Session: Gene Targeting & Gene Correction III
Abstract Number: 797
Time: Friday, May 18, 5:45 pm - 7:45 pm
Place: Stevens Salon C & D