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GenSight Biologics reports positive data in Phase III clinical trial of GS010 gene therapy for Leber Hereditary Optic Neuropathy

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...

GenSight Biologics announces positive DSM Board review and continuation of PIONEER Phase I/II clinical trial of GS030 gene therapy for Retinitis Pigmentosa

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...

Neurocrine Biosciences and Voyager Therapeutics announce positive phase 1 results of genetic therapy for Parkinsons disease.

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 ...

Promising Treatment for Periodontitis Gum Regeneration tested at Tohoku University

Researchers have designed a safer, faster and cheaper cell-based regenerative therapy approach for the treatment of one of the most common human dental diseases, namely periodontitis. A destructive and painful condition that is marked by inflammation and subsequent loss of the gums and the supporting structures of the teeth, periodontitis is caused by bacteria that trigger inflammation of the gums that surround teeth....

Three-antibiotic cocktail clears ‘persister’ Lyme bacteria in mouse study

A new study from researchers at the Johns Hopkins Bloomberg School of Public Health found that a slow-growing variant form of Lyme bacteria caused severe symptoms in a mouse model. The slow-growing variant form of Lyme bacteria, according to the researchers, may account for the persistent symptoms seen in ten to twenty percent of Lyme patients that are not...

Researchers putting the brakes on lethal childhood cancer

Malignant rhabdoid tumor (MRT) is one of the most aggressive and lethal childhood cancers. Although rare — about 20 to 25 new cases are diagnosed annually in the United States — there is no standard effective treatment for the disease, which is driven by loss of an anti-cancer protein called SNF5. The chances are very small that a child will...

Rocket Pharmaceuticals Presents Preclinical Data of RP-A501 for Danon Disease at the American Society of Gene and Cell Therapy 2019 Annual Meeting

- 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...

Anti-CRISPR molecules discovered that block the gene editing technology

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...