Genetic Modification

The health authorities canceled the license for Kolon Life Sciences' osteoarthritis gene therapy drug Invossa, Tuesday, over the mislabeling of a key ingredient. In addition, they have decided to pursue criminal charges against the pharmaceutical firm.Invossa was the first regenerative gene therapy drug for patients with the degenerative joint disease. The Ministry of Food and Drug Safety approved it use in Korea in July 2017 on the basis that it used cartilage-originated cells.After the approvals process,...

A new study challenges the presumption that people born with developmental brain disorders such as severe autism will benefit from medical interventions only if treated during a narrow window in infancy or early childhood. Writing in the journal eLife, an open-access scientific journal, the Rumbaugh lab at Scripps Research in Florida reports improvement in measures of seizure and memory in adult mouse models of a genetic cause of autism, called SYNGAP1 disorder.

Durable improvement in best-corrected visual acuity (BCVA), as GS010-treated eyes sustain +15.4 ETDRS letters equivalent improvement versus baseline and +28.1 ETDRS letters equivalent versus nadirBilateral improvement continues in key visual functions BCVA and contrast sensitivityResponder analyses suggest improved clinical outcomes for GS010-treated eyes relative to sham-treated eyesGS010 safety and tolerability established over the course of the trialBriefing packages incorporating findings under preparation for meetings with regulatory authorities GenSight Biologics today reported a first set...

GenSight Biologics announced that the independent Data Safety Monitoring Board (DSMB) completed its first safety review of the ongoing PIONEER Phase I/II clinical trial of GS030 combining gene therapy and optogenetics for the treatment of Retinitis Pigmentosa. The DSMB confirmed the absence of any safety issues for the first cohort of three subjects who received a single intravitreal injection of 5e10 vg combined with a wearable optronic visual stimulation device. The...

Results Confirm the Posterior Trajectory as an Additional Surgical Delivery Route for VY-AADC in Patients with Parkinson’s DiseaseTreatment with VY-AADC Improved Good ON Time (ON Time Without Troublesome Dyskinesia) by 1.7 Hours and Reduced OFF Time by 2.2 Hours at 12 Months in Patients with Parkinson’s Disease SAN DIEGO, CA and CAMBRIDGE, MA, USA I May 05, 2019 I Neurocrine Biosciences, Inc. (NASDAQ: NBIX) and Voyager Therapeutics, Inc. (NASDAQ: VYGR) today announced...

- Biodistribution of RP-A501 Demonstrates High Concentration in Heart, the End-Organ Target in Danon Disease - - Phase 1 Clinical Trial to Begin in Second Quarter of 2019 - NEW YORK, NY, USA I May 02, 2019 I Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today presents preclinical data of RP-A501 at the American Society of Gene and Cell Therapy 2019 Annual Meeting in...

Scientists have identified the first chemical compounds able to inhibit and regulate CRISPR systems, which could ultimately make CRISPR gene-editing technologies more precise, efficient, and safe. To identify these compounds, the researchers developed a new platform for rapidly finding small molecules that suppress CRISPR enzymes. Sometimes referred to as “anti-CRISPRs,” such molecules allow researchers to fine-tune CRISPR gene editing. The compounds could prevent CRISPR enzymes from unintentionally affecting other...

Researchers at Jefferson University have been experimenting with ways to weaken the defenses of antibiotic resistant bacteria, by targeting a common feature of the resistant bacteria - a pair of membranes which are able to expel antibiotics by pumping them out of the cell. The researchers found a way to interfere with transfer RNA that is unique to the bacteria, crippling it's ability to produce the membrane proteins, using a method which appears to,...