October 27, 2020

The Niche

Knoepfler lab stem cell blog

induced pluripotent stem cells

3 min read

It’s notable how the FDA now considers gene-editing a kind of regenerative medicine. This means that various gene therapy products in development technically qualify as regenerative therapies. Ever since the agency began its rapidly growing regenerative medicine advanced therapy (RMAT) designation program, we’ve seen an increasing number of gene therapy biotechs and products qualify as RMATs. As a result, the RMAT-qualified gene therapies get to go into a speedier lane of FDA oversight. Today’s post is a mix of recommended reads in the regenerative …Read More

2 min read

Where do things stand with IPS cell translational research? The newest development is that regulators in Japan just have given conditional approval to an Osaka University team to an induced pluripotent stem (IPS) cell-based study for ischemic heart disease. For years IPS cell-based products have been on-again off-again in active clinical study for macular degeneration, work led by Dr. Masayo Takahashi. The new heart disease study is led by Yoshiki Sawa. This appears to be the third conditionally approved IPS cell study in Japan. I’m not …Read More

3 min read

There’s much more to CRISPR-Cas9 than just gene editing and a new paper from the lab of Rudy Jaenisch in Cell highlights that in an exciting way. It reports epigenetic reversal of a Fragile X Syndrome phenotype in induced pluripotent stem cell (IPSC) neurons. Fragile X Syndrome is a neurological disorder in boys resulting from CGG repeat expansions in the regulatory region of the FMR1 gene and associated epigenetic alterations including DNA methylation that tend to shut off gene expression. The new paper, Liu, et al., …Read More

3 min read

A team of researchers at RIKEN and Kobe City Medical Center reported at a press conference today a serious adverse event in a clinical trial participant receiving an induced pluripotent stem cell (IPS cell)-based therapy for vision loss. According to Japan Times and Yahoo! Japan (in Japanese), the patient in question in the IPS cell trial for macular degeneration developed a problem with his eye following the cell transplant. From Yahoo! Japan: “The patient had an operation on Monday to remove pre-retinal membrane, deemed to …Read More