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 country’s health department is now sharing study results on new drugs and treatments online. Transparency advocates want the U.S. to do the same. Health Canada has posted safety and efficacy data online for four newly approved drugs; it plans to release reports for another 13 drugs and three medical devices approved or rejected since March. Source
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
By Annie Hubert, Senior Director, European Public Policy at Alliance for Regenerative Medicine ATMPs are varied and complex, ranging from potential one-off curative treatments to treatments tailored to a specific individual. As such, they differ significantly from ‘traditional’ medicinal treatments, something which has been recognised by regulators in Europe: the European Parliament introduced ATMPs as a
Source Wake Forest Baptist Medical Center The Wake Forest Institute for Regenerative Medicine (WFIRM) is the recipient of a major research funding award from the U.S. Department of Health and Human Services (HHS) to support its lung-on-a-chip technology as a model to develop chemical injury treatments. The five-year $24 million program has been approved with an
Source Universitat de Barcelona The League of European Research Universities (LERU) has published a new report on how to improve patients’ access to Advanced Therapy Medicinal Products (ATMPs) in medical centers and university hospitals. In this report, LERU presents recommendations to improve ATMP within the academic context in order to ensure that EU institutions, national
Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial, published in Nature Medicine. Researchers of MRC Centre for Regenerative Medicine, University of Edinburgh, Edinburgh, UK who tested the potential treatment in patients with liver cirrhosis – where long term damage produces scarring – found
Source CancerNetwork CAR T cells are a more effective therapy if manufactured for patients with multiple myeloma prior to the onset of relapsed or refractory disease, according to study results1 published in Blood Advances. “CAR T cells directed against the B-cell maturation antigen (BCMA) have demonstrated impressive initial results, but available data suggest that most patients with initial responses
SMART has developed a new way to study cells, paving the way for a better understanding of how cancers spread and become deadly. Source Singapore-MIT Alliance for Research and Technology Researchers at Singapore-MIT Alliance for Research and Technology (SMART) and MIT’s Laser Biomedical Research Center (LBRC) have developed a new way to study cells, paving
