May 31, 2020

The Niche

Knoepfler lab stem cell blog

Search Results for: 21st century cures

1 min read

The 21st Century Cures Act specifies some new FDA rules about investigational stem cell-based regenerative medicine therapies and perhaps most importantly defined a new class of product called a “Regenerative Advanced Therapy” or RAT, which is entitled to an expedited review. The FDA already has a new page up on RATs, which specifies the specifics of RAT designation and how an entity may submit an application for RAT designation. This was remarkably fast of the FDA. Some things on the RATs page stand out including …Read More

4 min read

The 21st Century Cures Act has some important provisions that would impact the stem cell and regenerative medicine field.  What is driving these efforts is a widespread sense that the status quo of regenerative medicine oversight isn’t working very well. I’m talking with key stakeholders and still trying to sort out my thoughts on the stem cell provisions in Cures. What would it mean? Cures’ language on stem cells is nuanced and its provisions also are not so black and white as REGROW was in …Read More

3 min read

One of the hottest topics in the world of stem cells this year has been the REGROW ACT and at a larger level the movement for changes in FDA oversight of investigational regenerative medicine therapies (see my past posts on it here), so the related provisions in the 21st Century Cures Act are stirring much discussion today. The bill is called Cures for short and you can read the latest draft here. Cures may get a vote in Congress as early as tomorrow so this is …Read More

3 min read

The Japan stem cell and regenerative medicine oversight system has been designed to move investigational therapies forward fast, much faster than the systems in other countries such as the U.S. But how well is it working so far? It’s an important question both for that country and more globally as the Japanese system was touted by some as a model and a reason why the U.S., to remain more competitive in this industry. Some of the same folks felt that the U.S. should also …Read More

3 min read

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

2 min read

I can see many reasons to be jolly on the regenerative medicine front as we get deeper into the holiday season as there is quite a lot of stem cell good news even if there is also some not so good news. In today’s post the focus is on the good news of late. Semma Therapeutics, a stem cell diabetes biotech, raised more than $110 million. I’m eager to see how the next few years play out with the healthy competition between them, ViaCyte, & …Read More