Gene therapy is ready for prime time

by Alexey Bersenev on December 28, 2011 · 2 comments

in clinical trials and cases

I was not very optimistic about the future of gene therapy 3-4 years ago. Well, I was wrong about it. I was obsessed by cell therapy so much that underestimate the progress in gene therapy. Both fields have matured and so did I. Last two years brought us tremendous development in both fields. I realized how much gene and cell therapy could be bind together. Today I’d like to share some links and opinions on why gene therapy is ready for prime time.

First of all, I’d like to highlight the recent notable advances in gene therapy. Last two years there were many of them. I’d mention a few:

But not everything went so smoothly this year. There were some setbacks. I’d mention two:

Now, I’d like to share an opinion of two important figures in the field.

Ted Friedmann:

We now passed the stage which we’re trying to prove something to somebody and trying to overcome our history of maybe promising too much and being criticized for that. And now we’re at the point when we can begin to deliver real treatment for real people.

James Wilson:

Gene therapy is ready for prime time and will revolutionize practice of many aspects of medicine and soon to follow will be stem cells.

So, what does gene therapy readiness mean?
In the recent poll of Genetic Engineering News magazine 61.4% of professionals were positive about first FDA or EMA approval of gene product on the market in the next 2 years. There are more than one and half thousand of currently ongoing gene therapy clinical trials. 60 of them on a Phase 3. There are 3 commercially available gene therapy drugs on a market.

Based on all clinical data that gene therapy has accumulated in the last decade, we can say that it works! It could cure a disease or provide better quality of life at some indications. We are ready for expansion and advance of clinical trials. We are ready for marketing approval of few more gene products in the next one-two years. We are ready for post-marketing analysis and commercial manufacturing of the products. So, let field to explode!

{ 2 comments… read them below or add one }

richard December 28, 2011 at 9:25 pm

We hope StarGen the gene program of Oxford biomedica have positive results to cure Stargardt


SammyJo Wilkinson March 1, 2014 at 11:27 pm

JAMA: Less Oversight Needed for Gene Transfer Studies

An additional layer of review by the National Institutes of Health (NIH) for gene transfer studies is no longer needed because the field has become established and many of the early concerns about safety have been resolved, according to an Institute of Medicine (IOM) report.


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