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
High-throughput synthetic biology approach reveals hidden AAV features and could help fast-track future gene therapies Adeno-associated viruses (AAVs) have become the go-to vehicle for delivering therapeutic gene cargo to target tissues for the wave of gene therapies that are in development in academic and biotechnology laboratories. However, natural AAVs do not specifically target diseased cells
New data released shows cell and gene therapy manufacturing facilities are rapidly becoming operational reflecting job expansion in the sector. Additional 6,000m2 of cell and gene therapy manufacturing space expected to become available within the next 12 months. The Cell and Gene Therapy Catapult (CGT Catapult) published the sixth annual UK cell and gene therapy
European regulations might be responsible for lower numbers of advanced therapy clinical trials running in the region when compared to the US and Asia. Europe has historically been a pioneer in advanced therapies, such as cell and gene therapy. Europe was the first region to approve a gene therapy, and it boasts the highest number
