A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge. Source EPR According to a new report, ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies
The International Society for Cell and Gene Therapy has created a group to tackle the rising number of commercial cell bank services that are misleading patients. The International Society for Cell and Gene Therapy (ISCT) has announced the formation of a global consortium to combat the growing number of unproven commercial cell banking services. The group will
Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects. This new technique is called the “prime editing” and is being developed
Source NIH Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates
A more efficient approach to gene therapy that could lower costs and improve patient outcomes has recently been developed by a team from Scripps Research. This work, published on October 17 in the journal Blood, offers a potential alternative to the standard process of delivering gene therapy, which is expensive, time-consuming, and requires many steps to administer healthy
NIH Director Francis Collins describes evolution of existing collaborations and how the two agencies might facilitate development of gene therapy, such as by having a common template for regulatory review. Source Pink Sheet The National Institutes of Health is looking to build on its partnerships with the US Food and Drug Administration to tackle new
A California “human biohacking” bill calls for warnings on do-it-yourself genetic-engineering kits. Source Technology Review It’s going to be illegal in California to sell “gene-therapy kits” unless they carry a warning that says not to use them on yourself. Just one wrinkle: we’re not sure any such kit exists. Not yet, anyway. The consumer protection
Neurologic scores stabilized, seizures diminished with tailored antisense oligonucleotide therapy The researchers who developed a personalized antisense oligonucleotide (ASO) for a little girl with a form of Batten disease — all in record time — have detailed their case in the New England Journal of Medicine. Timothy Yu, MD, PhD, of Boston Children’s Hospital, and colleagues explained how
Subretinal administration of the gene therapy AAV2/5-OPTIRPE65 showed improvements in vision-guided mobility, retinal sensitivity, and foveal-driven visual function for patients with RPE65-associated retinal dystrophy, according to findings from a phase 1/2 study presented at the 2019 American Academy of Ophthalmology Annual Meeting. In the early-stage study, there was a significant change in the ability to complete a
The number of cell and gene therapy clinical trials in the UK continues to increase with 85 ongoing trials at present, representing a 37% increase over last year. For the first time the majority of trials have commercial sponsors, many of whom are non-UK companies. There has also been an increase in the proportion of
