Before gene therapy can be used to treat renal diseases, delivery of therapeutic genes to the kidney must become much more efficient. A novel approach in which three different gene delivery vectors were injected intravenously and directly into the kidneys of mice was reported in an article published in Human Gene Therapy, a peer-reviewed journal from
Early results suggest that prime editors are cleaner than CRISPR–Cas9 and more versatile than base editors, but many questions remain. A paper recently published in Nature from David Liu and co-workers discloses a ‘prime’ gene-editing system many years in the making. In principle, the system, comprising a catalytically impaired Cas9 enzyme and an engineered reverse transcriptase, may
Drugs that manipulate the code of life are finally changing lives Source Scientific American The idea for gene therapy—a type of DNA-based medicine that inserts a healthy gene into cells to replace a mutated, disease-causing variant—was first published in 1972. After decades of disputed results, treatment failures and some deaths in experimental trials, the first
The strategy has long been seen as having enormous potential to cure diseases Paving the way for solving a major safety issue associated with gene therapies, scientists have developed a special molecular switch that could be embedded into such therapies to allow doctors to control dosing. The feat, published in the journal Nature Biotechnology, offers
Potential 1st Gene Therapy in Europe Directed at Any Type of Hemophilia Application to be Reviewed Under Accelerated Assessment BioMarin Pharmaceutical announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January
Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.
The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug. Sarepta announced Thursday evening that the agency had approved its second drug, called Vyondys 53. After the drug was originally rejected by the FDA in August due to concerns over the
The first two patients treated with the 3e13 vg/kg dose achieved stable Factor VIII (FVIII) levels demonstrating durability in the normal range through 44 and 37 weeks, respectively– All five patients in the 3e13 vg/kg dose cohort achieved normal range FVIII levels within 5-7 weeks following treatment, with no bleeding events with up to 44 weeks
Polymer-based nanocontainers could improve the delivery of gene therapies, a Johns Hopkins team reported. One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune
Source REUTERS Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the world’s priciest medicines. The full scope of Novartis’ $500 million plan, revealed to Reuters in an interview
