Archives: 2019-10-13

RPE65 Gene Therapy Effective for Retinal Dystrophy

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

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Elsevier and phactMI to develop new tool for medical data

Analytics firm Elsevier has teamed up with non-profit phactMI to develop a new solution for providing scientifically accurate information to healthcare professionals. The deal will leverage Elsevier’s Entellect life sciences platform to create a semantic search portal. Scheduled to launch in the fourth quarter of this year, the new solution will facilitate rapid access to

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Melanoma variability at the single-cell level predicts treatment responses

Source: H. Lee Cancer Center & Research Institute Patients with advanced melanoma have been able to live longer because of several newly approved targeted treatment options, including BRAF and MEK inhibitors. However, patients will often have different responses to the same treatment due to genetic variability. Melanoma varies from patient to patient, but genetic variability is

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FDA Updates Digital Health Guidances to Align with 21st Century Cures Act

Source https://www.mintz.com/insights-center/viewpoints/2791/2019-10-fda-updates-digital-health-guidances-align-21st-century On September 26, 2019, FDA released a suite of revised digital health guidances, which includes the following: Changes to Existing Medical Software Policies Resulting from Section 3060 of the 21st Century Cures Act (“Section 3060 Changes Guidance”) – Final Policy for Device Software Functions and Mobile Medical Applications (“MMA Guidance”) – Final General Wellness: Policy for

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New articles on the ethics of genome editing published in The CRISPR Journal

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

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Overcoming the barriers to cell and gene therapies in Europe. BIA and ARM position

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

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