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
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
Bluebird bio Inc on Friday set a price for its gene therapy, Zynteglo, at 1.58 million euros ($1.78 million) over five years, after winning conditional approval in Europe this month to treat a rare genetic blood disorder. The company proposed an installment plan, with 315,000 euros paid up front and four additional annual payments due
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
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
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
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
A new phase of the IGNITE Network will conduct clinical trials of genomic medicine interventions. The National Institutes of Health will fund clinical trials to assess the benefits, applicability and efficacy of applying genomic medicine interventions to improve management of diseases such as high blood pressure, depression and chronic pain. The trials are part of
An international stem-cell body says the country’s proposed law could put patients at risk. An international group of stem-cell researchers is urging China to cancel draft regulations that would permit some hospitals to sell therapies developed from patients’ own cells, without approval from the nation’s drug regulator. The International Society for Stem Cell Research (ISSCR) sent a
For people whose spinal cords are injured in traffic accidents, sports mishaps, or other traumatic events, cell-based treatments have emerged as a potential avenue for encouraging healing. Now, taking advantage of advances in 3D printing technology, researchers have created customized implants that may boost the power of cell-based therapies for repairing injured spinal cords. Made of
