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
Source http://www.liebertpub.com/ The CRISPR Journal announces the publication of its October 2019 Special Issue on The Ethics of Human Genome Editing. The Journal is led by Editor-in-Chief Rodolphe Barrangou, PhD (North Carolina State University) and Executive Editor Dr. Kevin Davies. For full-text copies of articles or to arrange interviews with Dr. Barrangou, Dr. Davies, authors, or
Source MedCity News Interim Phase I/II data for Zolgensma showed improvements in children aged 2 to less than 5 with spinal muscular atrophy. The therapy received FDA approval in May for infants younger than 2. A gene therapy approved for treating spinal muscular atrophy in infants has also shown improved efficacy in older children. Basel,
Penn study shows delivering a “distant cousin” of a key protein prevented muscle damage without triggering an immune response in large animal models A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal
Of an estimated 6,500 to 7,000 known rare diseases, only a fraction – maybe 5% – have U.S. Food and Drug Administration-approved treatments. To increase that percentage, the National Institutes of Health has awarded approximately $31 million in grants in fiscal year 2019 to 20 teams – including five new groups — of scientists, clinicians,
