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Intrexon: Chimeras, CRSPR and Gene Therapy

02/16/2017 7:00 am EST


John McCamant

Editor, Medical Technology Stock Letter

Intrexon (XON) has made a small yet strategic acquisition in buying GenVec (GNVC); with the purchase, XON gets AdenoVerse, a platform of adenovirus vectors for delivering drugs and vaccines that dovetails nicely with its existing suite of gene therapy technologies, suggests John McCamant, editor of The Medical Technology Stock Letter.

The ability to increase the payloads of viral vectors so they can carry multiple genes and regulatory elements will expand the opportunity for gene therapy to treat a wide range of diseases.

XON continues to build out its broad gene therapy platform with various viral as well as non-viral transfer techniques at their disposal to pursue an array of in-vivo and ex-vivo gene and cell therapy approaches.

The substantial payload capacity and a helper-dependent adenoviral system will allow the company to pursue new drug development targets and will also provide more partnering opportunities.

XON's subsidiary Exemplar Genetics, has been awarded a subcontract to create genetically-engineered mini-swine models of sickle cell disease as part of a national resource that could lead to new treatments for the disorder.

Genetically engineered miniature swine represent a powerful investigational platform with greater similarities to humans. Insufficient animal models create a significant barrier to progress in the discovery of disease mechanisms and in the development of therapeutics.

This may hold particularly true in therapeutic development for rare genetic diseases and orphan indications where there is no clear path for evaluation or an insufficient patient population

To further support XON’s growing efforts in this field, The Salk Institute recently announced that they had successfully grown embryos containing cells from both pigs and humans.

The Salk researchers see a host of potential applications for their technology. In the Cell publication, they point out that CRISPR-edited rodents may offer a more efficient platform for medical research than models that are currently used, which are often cross-bred mice with various gene mutations.

They also suggest that human-pig chimeras could help researchers better understand embryo development and the progression of human diseases. They might also prove to be a good platform for testing human drugs, they write.

The next step for the Salk researchers is to see if they can use CRISPR to create pigs that are lacking key organs, and then use human cells to fill in the gaps—just as they did with the rat-mouse chimera.

XON is uniquely positioned to take gene therapy to the next level and be a leader in this cutting edge field which could dramatically change many areas of drug development and provide a news source for human organs.

In our view, Intrexon is amassing both the tools and the experts necessary to develop second generation gene and cell therapies.

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