January 16, 2021

The Niche

Trusted stem cell blog & resources

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

4 min read

It’s been a great couple days so far here at the World Stem Cell Summit in Atlanta. You can follow it on Twitter using the #WSCS15 hash tag. I first attended it 5 years ago in Pasadena. One of the special aspects of WSCS is it brings together diverse stakeholders in a way that just doesn’t happen elsewhere. For instance, you can have the FDA, patients, physicians, scientists, and funding agency people all in one room together. Some strong impressions so far include the …Read More

2 min read

Stem cell biotech company Bioheart ($BHRT) has had a rough 2015 so far. Could this year be a decisive, negative tipping point for the company? It has a number of clinical trials going, but from my view things seem increasingly uncertain. An oddity amongst stem cell biotechs, Bioheart and its leadership have at times seemed to toy with what some might view as controversial projects. Kristin Comella, CSO of Bioheart, has played a major role in other ventures including in the past with the Ageless Institute, a stem …Read More

7 min read

I invited the Chief Scientific Officer (CSO) of Bioheart, Kristin Comella, to do an interview after hearing some buzz that this could be a critical time for the company and that it might have been recently visited by the FDA. Note that Comella not just Bioheart CSO, but also the primary instructor for physician training in stem cell methods offered by the company US Stem Cell Training. Can you update us on how Bioheart is doing with its clinical trials/INDs? Comella: Our completed clinical trials of …Read More

4 min read

The big stem cell news already this week is the acquisition by Fujifilm of Cellular Dynamics International (CDI) for a whopping $307 million dollars or about $16.50 per CDI share. You can read the press release here. Update: The entity is now known as Fujifilm Cellular Dynamics. It was less than two years ago that CDI did its IPO with an initial offering of about $46 million. Fujifilm reportedly has a multibillion-dollar war chest for buying into the life sciences sector and a growing interest in regenerative …Read More

5 min read

In the interview below I talk with Dr. Masayo Takahashi, who is leading a team conducting the first ever in-human clinical study based on iPS cells. The work began with patient enrollment on Aug. 1, 2013 in Japan. 1. Can you tell us a bit about your background? As an M.D./Ph.D. and ophthalmologist do you also see patients in addition to doing research? How did you first get interested in stem cells? Are your interests primarily in iPS cells? Yes, I have outpatient clinics in …Read More

3 min read

Stem cell technology is poised to wow the world with biomedical advances in the coming decades and Japan wants to lead the way. They want to forge ahead particularly with applications that focus on a relatively new kind of stem cells called induced pluripotent stem cells or iPS cells. Japanese researcher, Shinya Yamanaka, first reported production of iPS cells in 2006 in mice and in 2007 in humans. Japan has raced ahead of other countries and they appear to have the intellectual property rights and …Read More