The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds. According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign
Source https://www.nature.com/articles/s41467-019-12281-8#Sec14 Highly specific Cas9 nucleases derived from SpCas9 are valuable tools for genome editing, but their wide applications are hampered by a lack of knowledge governing guide RNA (gRNA) activity. Here, we perform a genome-scale screen to measure gRNA activity for two highly specific SpCas9 variants (eSpCas9(1.1) and SpCas9-HF1) and wild-type SpCas9 (WT-SpCas9) in
Wake Forest Institute for Regenerative Medicine scientists have fine-tuned their delivery system to deliver a DNA editing tool to alter DNA sequences and modify gene function. The improved “hit and run” system works faster and is more efficient. “With this new method we are able to package together in one lentiviral capsid both essential components
At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously
Long-Term Follow-up Data Show That the 88% of Patients Treated in the Starbeam Study (ALD-102) Were Free of Major Functional Disabilities (MFDs) at Two Years, and Continued to Remain MFD-Free at up to Five Years of Follow-up bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D™ gene
New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who
Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus
Mark Zylka, PhD, in The UNC Neuroscience Center was awarded NIH funding for two 5-year projects, one jointly with Ben Philpot, PhD, to develop new knowledge with the potential to advance treatments for neurodevelopmental disorders. The National Institutes of Health have awarded two separate grants totaling $6.1 million to Mark Zylka, PhD, director of the
CRISPR Research Might Lead to Cure for Duchenne Muscular Dystrophy At this time, there is no cure for Duchenne muscular dystrophy (DMD), although there is one treatment for a subgroup of the disease. That is Sarepta Therapeutics’ Exondys 51 for DMD patients with a confirmed mutation amenable to exon 51 skipping. Recently the U.S. Food and
Applications and the limitations of real-world data. Source Contract Pharma There are approximately 7,000 distinct rare diseases that exist affecting 350 million people worldwide, and approximately 80% of those rare diseases are caused by faulty genes. Scientific advances such as the CRISPR/Cas9 genome-engineering system1 have simplified the pharmaceutical and biotech industry’s ability to develop gene therapies,
