Archives 2019

NIAID scientists develop “mini-brain” model of human prion disease

National Institutes of Health scientists have used human skin cells to create what they believe is the first cerebral organoid system, or “mini-brain,” for studying sporadic Creutzfeldt-Jakob disease (CJD). CJD is a fatal neurodegenerative brain disease of humans believed to be caused by infectious prion protein. It affects about 1 in 1 million people. The

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How will we pay for the coming generation of potentially curative gene therapies? Bill Cassidy US Senator proposals

We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these expensive treatments are entering a market structure that was not built to price them. Congress will likely need to play a part in developing a new paradigm for financing such

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Fecal Microbiota for Transplantation: Safety Communication- Risk of Serious Adverse Reactions Due to Transmission of Multi-Drug Resistant Organisms

The FDA is now aware of bacterial infections caused by multi-drug resistant organisms (MDROs) that have occurred due to transmission of a MDRO from use of investigational fecal microbiota for transplantation (FMT): Two immunocompromised adults who received investigational FMT developed invasive infections caused by extended-spectrum beta-lactamase (ESBL)-producing Escherichia coli (E.coli). One of the individuals died. FMT

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RESTORE Community produced a new video for ATMP’s diffusion

Large Scale Research Initiative RESTORE The visionary unifying goal of Large Scale Research Initiative RESTORE is to make the transforming promise associated with Advanced Therapies a reality for the benefit of patients and society. RESTORE sees itself as a “place” where medicine, basic research, technology development and engineering meet, communicate and work together. Through this

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Replicating Fetal Bone Growth Process Could Help Heal Large Bone Defects

Animal study finds large bone defects could be regenerated through stem cell implantation and allowing movement To treat large gaps in long bones, like the femur, which result from bone tumor removal or a shattering trauma, researchers at Penn Medicine and the University of Illinois at Chicago developed a process that partially recreates the bone

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Gene Therapies clinical trials see 17% jump year-over-year, with two approvals near and pipelines expanding

Data released May 9 by the Alliance for Regenerative Medicine (ARM) illustrates the growth of clinical activity where gene therapy is concerned. ARM’s Quarterly Regenerative Medicine Global Data Report for the first quarter of 2019 showed 372 gene therapy clinical trials were in progress as of the end of Q1. Interestingly, a majority (217 or 58%) were

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ARM Releases Regenerative Medicine & Oncology Report: Metrics and Trends Related to Companies Developing Cell and Gene Therapies for Cancer

This report includes an overview of regenerative medicine approaches to treating a variety of cancer types, along with a list of ARM member companies active in developing these therapies, global financings, an overview of the oncology-focused clinical pipeline, as well as commentary from select therapeutic developers active in this area. Key findings include: There are 416 companies worldwide active in

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