Cells Weekly – July 5, 2015

by Alexey Bersenev on July 6, 2015 · 0 comments

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Cells Weekly is a digest of the most interesting news and events in stem cell research, cell therapy and regenerative medicine. Cells Weekly is posted every Sunday night!

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1. CRISPR genome editing news
Famous geneticist from MIT Eric Lander wrote an opinion piece in NEJM. I’d highly recommend you to read this article!

The discussions that will begin in the fall may solidify a broad international consensus that germline editing should be banned — with the possible exception of correcting severe monogenic disease genes, in the few cases in which there is no alternative. For my own part, I see much wisdom in such a position, at least for the foreseeable future. A ban could always be reversed if we become technically proficient, scientifically knowledgeable, and morally wise enough and if we can make a compelling case. But authorizing scientists to make permanent changes to the DNA of our species is a decision that should require broad societal understanding and consent.

Nature posted interesting piece on “the biggest bioethical debate of the year” – politics of gene editing:

If the budget passes, this clause would be the first time that lawmakers have used the FDA to limit human embryo research. A 1996 law known as the Dickey–Wicker Amendment bans the use of federal funds to create human embryos for research, but does not pertain to FDA regulation. The National Institutes of Health (NIH) reaffirmed in April that heritable genetic modification falls under the Dickey–Wicker rule, and director Francis Collins said that clinical application of such technology is “viewed almost universally as a line that should not be crossed”.

Nature also has a very nice flipboard feature – Embryo Editing: the Ethics of CRISPR

Yet another piece that I’d like to recommend is about the CRISPR commercialization and competition between startups:

The uncertainty has not stopped venture capitalists and pharmaceutical companies from snapping up deals with the warring genome-editing startups. “It’s a gold rush. It’s a tsunami. This pace is rare,” says Rodolphe Barrangou, one of the first scientists to decipher this bacterial defense mechanism, a co-founder of Intellia and a member of Caribou’s board of advisers. “You see some of the smartest investors in the world putting tens of millions into almost nothing: great names and founders, but no tangible assets, no products on the market, sometimes they don’t even have CEOs. They’re putting millions of dollars on a case, on an argument, on future potential.”

Finally, on research side, a new study offers improved methodology for CRISPR-Cas genome editing:

This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of biotechnological and therapeutic applications of the CRISPR-Cas technology.

2. Pause in the first iPS cell-based trial in Japan
Very unfortunate pause in first famed iPS cell-based trial was recently announced in Japanese media and RIKEN web-site. The reason for it is an unwanted gene mutation found in QC assays while product was prepared for the second patient. Paul Knoepfler wrote about it on his blog.

3. FDA news
A couple of recent letters from FDA. The first is to BS Medical for adulteration of PRP-making device:

A review of our databases disclosed that your firm has not obtained premarket approval or clearance for these Kit in the United States and has not received an investigational device exemption from premarket approval for these Kit either. Nevertheless, the Internet website above offers the sale of the Yes PRP Kit to buyers in the United States.

Very typical for PRP guys.

The second letter is to BioDlogics on misbranding of the series of amniotic tissue products as 361:

These morselized amniotic – membrane based products are human cells, tissues, and cellular and tissue-based products (HCT/Ps) as defined in 21 CFR 1271.3(d). However, the products are HCT/Ps that do not meet all of the criteria in 21 CFR 1271.10(a) and therefore are not regulated solely under section 361 of the Public Health Service Act (PHS Act) and the regulations in 21 CFR Part 1271.

Very typical for amniotic guys.

Finally, FDA recently claimed a jurisdiction over veterinary cell-based products meeting the definition of a new animal drug. You can read the full guidance for industry here.

4. Two biggest deals in cell/ gene therapy
Two deals worth $1B were announced this week. The first $1B deal is between Celgene and Juno Therapeutics on CART cell therapies. You can read about it here, here and here.

The second $1B deal is between Biogen and AGTC on gene therapy of eye diseases. Read more here.

2 Billions in one week – good time for cell/ gene therapy!

5. Lawsuit against cell therapy company
Cellular Biomedicine Group is involved in lawsuit with investor. Here is some interesting details:

The lawsuit was filed by a shareholder who bought 100 shares of CBMG stock the day before an anonymous blogger, who professes to have had a short position in CBMG’s stock, published a false and misleading article about the company.

Here is an article on Seeking Alpha and company’s response.

6. Magnetic levitation for cell sorting
Utkan Demirci group from Stanford published a method for cell sorting by magnetic levitation:

Here, we demonstrate that both eukaryotic and prokaryotic cells can be levitated and that each cell has a unique levitation profile. Furthermore, our levitation platform uniquely enables ultrasensitive density measurements, imaging, and profiling of cells in real-time at single-cell resolution. This method has broad applications, such as the label-free identification and monitoring of heterogeneous biological changes under various physiological conditions, including drug screening in personalized medicine.

Science magazine did a nice coverage of this study. Highly recommended!

7. Gene therapy of cystic fibrosis – results of the clinical trial
Results of Phase 2 gene therapy trial for cystic fibrosis were published this week. The authors reported modest improvement in experimental group over placebo:

Further improvements in efficacy and consistency of response to the current formulation are needed
before gene therapy is suitable for clinical care; however, our findings should also encourage the rapid introduction of more potent gene transfer vectors into early phase trials.

Not very impressive, lots things to learn, but will they continue? Perhaps, with modifications:

Alton said his team is now planning follow-up studies to look at whether higher, more frequent doses might have a greater benefit, and to test ways of combining the gene therapy with drug treatments to potentially boost its effect.

8. New Methods and Protocols:
Acquisition of pluripotency by human fibroblasts through continued OCT4-induced plastic (Stem Cell Res)
Effects of integrating and nonintegrating reprogramming methods on genomic stability of human iPS cells (PLoS ONE)
Automated identification and localization of hematopoietic stem cells by 3D intravital microscopy (Stem Cell Reports)
Engraftment of genetically-selected parthenogenetic stem cell-derived cardiomyocytes (PLoS ONE)
Allogenic iPSC-derived RPE cell transplants induce immune response in pigs (Sci Reports)
Bovine ocular fluid as a replacement for serum in cell cryopreservation (PLoS ONE)

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