January 25, 2021

The Niche

Trusted stem cell blog & resources

Search Results for: rmat

2 min read

This post includes my updated list of firms/products that have received FDA regenerative medicine advanced therapy designation or RMAT, which are in the public domain. The numbers really jumped in the last month. I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm names are to relevant press releases or other reference material. A special thanks to some folks at ARM for helping me keep the list up to date. You can …Read More

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Our California stem cell agency, CIRM, started as a baby even before I came to California in 2006 to start my lab here at UC Davis School of Medicine, but now CIRM or to use its full name the California Institute for Regenerative Medicine is I’d say a full-grown, mature adult at about age 13 in humans years. Maybe CIRM years don’t go for the 7-year equivalent of dog years to human years, but perhaps 2 or 3 CIRM years to one human year. …Read More

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The year of 2018 has already been a wild one for stem cell news. There are many developments on a variety of fronts. Here are some of the most notable news bites of the last month or so. RMAT Wave. The FDA continues its warp-speed issuance of Regenerative Medicine Advanced Therapy (RMAT) designations with 20 such designations now in the public domain with Cellerant Therapeutics’ announcement and possibly a few more not yet out there. I wrote about the “carrot” of RMATs as relative …Read More

Here is a list of firms/products that have self-reported that they have received FDA regenerative medicine advanced therapy designation or RMAT. I’m assuming for the purposes of this resource page that all self-reports are accurate. Note that the links embedded into the firm names are to relevant press releases. A special thanks to some folks at ARM for helping me keep the list up to date. It was updated as of December 28, 2020 with 49 RMAT total. Note that the FDA has recently …Read More

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Australian stem cell biotech Mesoblast announced that it has received regenerative medicine advanced therapy (RMAT) designation from the U.S. FDA. This is very good news for the company and an encouraging development for the field. Interestingly, last month the FDA clarified that there is expanded RMAT designation that can include gene therapies too. At the Meeting on the Mesa in October, an FDA official reportedly presented data indicating that about 1/3 applications for RMATs were granted (see image below from Twitter). This recently announced RMAT …Read More

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In the same way perhaps that some excited relatives or parents-to-be both gush and worry about a baby before it is even born, our field has been transfixed for a week by the Mitalipov paper on CRISPR’ing human embryos even though the paper just now came out. Now that the paper is out, we can take a closer look at this “baby” and for us scientists that involves giving it a critical review. In science, “critical” often means a thorough once over with a …Read More

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Can a stem cell clinic business get FDA RMAT designation? At least one announced it was trying, but now seems to have given up. Stem cell clinic business US Stem Cell, Inc. has reportedly announced that it is at least temporarily abandoning its efforts at getting Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The company’s penny stock $USRM has been gyrating for months and I had earlier wondered if could be some fake news about it. The USRM news now seems real and not …Read More

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Many stem cell clinics are in a bind in 2017 in the new RMAT approval reality. Stem cell clinics say that they only want to help people by providing stem cell therapies to patients in a speedier way that they claim getting FDA approval for a trial just doesn’t allow, but that doesn’t ring true. It never has and now even less so. The new RMAT designation out there based on the 21st Century Cures Act whereby the FDA can rapidly accelerate truly promising …Read More

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The 21st Century Cures Act has some important regenerative medicine language in it. One part of that was attempting to accelerate FDA review of promising investigation regenerative medicine therapies. The mechanism for this was a new designation called Regenerative Advanced Therapy, now renamed Regenerative Medicine Advanced Therapy (RMAT) Designation. I don’t think anyone was sure how this would play out in terms of how quickly the FDA would give RMAT designations and to how many investigational products. Already it’s clear the FDA seems to …Read More

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What kind of scientist are you? I suspect that all of us scientists like to think that most of the work that we do is transformative and that we are a transformative scientist. In science we hear quite a lot about the importance of transformative, innovative science. But can everyone be doing transformative science all the time? I don’t think that’s realistically possible. There are of course probably degrees of transformativeness for various specific projects. Today the research world often defines success not using “transformative” as the …Read More