When I first heard of CRISPR gene editing I was excited for my lab to try it out, but it seemed a long way from the field having even one CRISPR therapy available to treat human disease. In the Knoepfler lab, we study genetic and genomic programming in human development and disease. Gene editing seemed …
Updates on CRISPR therapy development and clinical trials Read More »
I wrote recently about how political pressure on FDA Commissioner Stephen Hahn in 2020 may have helped the Texas firm Direct Biologics move their product ExoFlo forward inside the agency. Today I analyze the ExoFlo Phase I clinical trial paper used in that push on Hahn to get the firm the IND for a Phase II …
Review of ExoFlo paper used to push Direct Biologics IND inside FDA finds red flags Read More »
If you’ve never heard of the KRAS G12C mutation, it’s a particularly frightening mutation present in numerous cancers. Like the MYC oncogene, many people view mutations in RAS as undruggable, but new efforts show some glimmers of hope. The drug Sotorasib has been approved by the FDA to target the KRAS G12C mutation. Here’s a …
Weekly reads: KRAS G12C, eLife journal controversy, bird iPSCs Read More »
When I first started The Niche more than a dozen years ago, I hoped we’d see a Parkinson’s disease stem cell therapy by now. A proven one. I was a little too optimistic back then. Now in 2022, I know much better how hard translation to the bedside can be in general. Still, I’m hopeful. …
Parkinson’s disease stem cell therapy research update Read More »
It’s always great to read about stem cell clinical trials that are looking encouraging. Stem cell clinical trials Proving a new therapy is safe and effective is so challenging. Remarkably, there are more than 9,300 stem cell clinical trials listed for a “stem cell” search on Clinicaltrials.gov. Of course, not all of these are actual …
As we age a higher proportion of our cells end up in a senescent state, which has sparked interest in senolytic drugs and a broader area called senolytics research. The goal of today’s post is to give you an overview of this important area. How much data are there on efficacy or safety? Is the …
What are senolytics? Drugs, activator, supplements, risks Read More »
An FDA FOIA dump of text messages of former Commissioner Stephen Hahn suggests that a Texas regenerative firm called Direct Biologics benefited from political pressure on him in mid-2020. Then he was asked again to help in early 2021. This was shortly before he resigned at the start of the Biden Administration. The push came from …
FDA leader Stephen Hahn was pressured to aid Texas firm Direct Biologics Read More »
There was a time when I thought transdifferentiation based approaches might quickly move into clinical trials. Then things kind of cooled off. We didn’t see many papers reporting methods to transdifferentiate cells. I still think that this technology, sometimes called direct reprogramming, has major potential. A new paper on making human brain cells this way got …
Paper sparks renewed interest in transdifferentiation & clinical potential Read More »
Exosome therapy is a still-experimental cell therapy approach aimed at treating specific diseases using secretions from cells. It’s novel since most attention on cell therapies has been given to using the actual cells themselves. In addition, this approach is a newer idea than using cells as the drug to treat diseases. Interestingly, the term “cell …
Fact-checking exosome therapy: costs, risks, & lack of data Read More »
The recent news of Vertex Pharma buying small stem cell biotech ViaCyte and Doug Melton moving to Vertex brought to mind an old stem cell biotech called Advanced Cell Technology (ACT) and its leader, stem cell biologist Dr. Robert Lanza. Years ago, big pharma firm Astellas bought ACT and Lanza moved to be a leader of regenerative …