In a recent press announcement, The U.S. National Institutes of Health (NIH) has agreed to invest in the development of gene therapies in an attempt to cure HIV and sickle cell diseases. The program, for which NIH will partner with The Bill & Melinda Gates Foundation, will involve funding of $100 million each over the next four
ReNeuron, the UK based, clinical-stage stem cell therapeutics company, announced that new data relating to its CTX stem cell platform will be presented today at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Dr. Steve Pells, Principal Investigator at ReNeuron, will present new data showing the phenotypic stability and scalability
Applied Genetic Technologies Corporation (AGTC), a clinical-stage biotechnology company, developing transformational genetic therapies for patients suffering from rare and debilitating diseases, announced new data from non-clinical studies evaluating the effect of pre-existing anti-AAV antibodies on the transduction and expression efficiency of AAV vectors. According to the company’s CSO, Preexisting immunity to AAV remains a challenge for
Cell and gene therapies are the new entrants in the pharma Superstar Club. They are selling tickets faster than a Queen’s concert in the 1980s. In fact, as per the Alliance of Regenerative Medicine’s recent findings, there has been more than 75% year on year increment in funding to support the development of various cell and gene therapies. With
Over 500 cell and gene therapy candidates are being investigated in different stages of clinical development across various geographies. In fact, in the past five years alone, more than 1,000 clinical trials, that are currently active, were initiated to evaluate the potential of these therapies across multiple therapeutic indications. Due to several development challenges, such
Over the last few decades, various viral and non-viral vectors have been developed, optimized and standardized for introduction of therapeutic DNA / gene of interest into a patient’s body / cells. Currently, the most popular viral vectors, on the basis of their use in active clinical trials, are those based on AAV, adenovirus, lentivirus and retrovirus.
The first gene therapy, Gendicine®, was only approved in 2003 in China; since then, the domain has evolved significantly. The ‘Gene Therapy Market (2nd Edition), 2018-2030’ report provides an extensive study on the current market landscape of gene therapies, with a prime focus on gene augmentation based therapies and oncolytic viral therapies, featuring an elaborate discussion on the
Scientists around the word are debating the scientific and ethical possibilities of using powerful gene editing tools, such as the CRISPR/Cas9 system, to treat heritable diseases in human embryos. In 1987, the discovery of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas) genes in single celled prokaryotic systems revolutionized genetic engineering. Initially thought to be