This isn't the exazt deal but lots of good info from IV board
They have a great track record/reputation and have been on fire the past four yrs....this is a major positive and should be followed by more news for sure.
New Recommendation: ZIOPHARM Oncology (ZIOP) is a second-generation gene therapy company focused on the development of new cancer treatments with a laser focus on the red-hot immunooncology (I/O) sector. ZIOP licensed its gene expression control technology from synthetic-biology innovator Intrexon for use in the development of DNA-based oncology treatments. Under an exclusive channel partner agreement, the two companies have collaborated to develop a tightly controlled system that injects a gene-expression package into tumors to express the anti-tumor cytokine interleukin-12 (IL-12), but only when patients take a pill that contains a ligand to activate the IL-12 gene. When the patient stops taking the pill, IL-12 expression stops, allowing for specific dosing for a set time. Expression is controlled by Intrexon’s proprietary biologic switch known as the RheoSwitch. The ability to administer or withdraw the pill to control gene expression is a key safety benefit.
Game Changing News for ZIOP: Last week, ZIOP and Intrexon announced they had licensed a unique MD Anderson immunotherapy technology, creating an alliance to hone a new way of customizing immune system attack cells to find and kill cancer cells. The two companies each agreed to pay MD Anderson $50 million in company stock — a total of $100 million — to license technology developed in the lab of Laurence Cooper, M.D., Ph.D., professor of Pediatrics, including a new method for genetically engineering immune T cells to target cancer.
Importantly, the companies and MD Anderson expect the collaboration to yield next-generation therapies based on designer cytokines and CAR-T cells under the control of RheoSwitch to target both blood and solid tumor cancers. First generation CAR-T companies are limited to blood cancers at this point due to severe toxicity. They project five patient derived CAR-T cells to enter the clinic in 2015 with off-the-shelf programs starting in 2016.
MD Anderson’s Sleeping Beauty Delivery System Key to Deal: The technology licensed from MD Anderson, known as Sleeping Beauty, is, in our view, much better than recombinant viral DNA delivery methods used to create first generation CAR-T cells. Sleeping Beauty is a faster, simpler and more nimble process for customizing T cells, while also being far less expensive. The system is named Sleeping Beauty because it is able to “awaken” an extinct transposon – DNA that can replicate itself and insert the copy back into the genome.
“It is a shared vision to maximize the speed and breadth of multigenic innovation for patients through the use of nimble, nonviral DNA cell manufacturing strategies that can further overcome viral packaging constraints and economic limitations,” stated Gregory Frost, Ph.D., senior vice president and head of Intrexon’s Health Sector. “Collectively, this will assemble the most advanced set of technologies to empower the strongest adoptive cell therapy pipeline that can drive innovation.”
Why CAR-T? It is amazing the huge buzz that CART has created given that only an estimated 300 patients have been treated. The CAR-T technology has been established broadly in the elite academic centers with the University of Pennsylvania, Baylor College of Medicine, City of Hope, Memorial Sloan Kettering Cancer Center (MSKCC), Fred Hutchinson Cancer Research Center (FHCRC)/Seattle Children’s, MD Anderson and the National Cancer Institute leading the way in the U.S. In the last two years, agreements have been formed tocommercialize CAR-T technologies, with KITE and Juno examples of newly established pure-play T cell therapeutic companies. In addition several companies have licensed CAR-T technology including NVS, JNJ, PFE, and CELG.
1st Generation CAR-T More Box-CART than Race-CART: First-generation CAR-T therapies have been notorious for their severe toxicity including cytokine release syndrome/tumor lysis syndrome, which in some cases has been fatal, in fact the NCI terminated a HER2 CAR-T trial after the first patient enrolled died. In our view, the first-generation CART treatments are akin to pushing a box-cart down a hill and hoping for the best with your tennis shoes as breaks. In stark contrast, ZIOP has the ability to turn on/off their treatment while also controlling gene expression. In our view, Box-CARTs will be very expensive personalized medicines that will only treat a niche sub-set of very sick blood cancer patients due to their toxicity and expense. The real opportunity in CAR-T therapy is to create off-the shelf blood and solid tumor treatments that can address the underlying cause of the disease without major toxicity. The addition of non-viral delivery and the RheoSwitch for precise gene control makes ZIOP’s second-generation technology a Race-CART with the brakes and steering necessary to addressthese huge cancer markets all at a reasonable cost.
Investment Conclusion: In our view, the MD Anderson partnership provides significant validation for ZIOP’s second-generation gene therapy platform as MD Anderson had many deep-pocketed suitors to choose from, yet chose a small-cap biotech (backed by synthetic-biology leader Intrexon). While CAR-T success in solid tumors may be more difficult to achieve, ZIOP's orally activated RheoSwitch combined with the non-viral Sleeping Beauty delivery system could be a game changer.
Development could be very rapid in CAR-T space as we have recently witnessed the rapid transition from Phase I trials to registration studies in the space. In our view, ZIOP has the potential to catch up rapidly and become a leader in second-generation CAR-T treatments.
ZIOP has all the tools to be a second generation gene therapy leader in the exciting field of I/O. The ability to package multiple genes combined with a non-viral delivery system all precisely controlled by RheoSwitch has the potential to create off-the-shelf therapies that will treat solid tumors in addition to hem/onc. We believe the Sleeping Beauty delivery system is a critical key to unleashing ZIOP’s technology. With all the critical IP now locked down with MD Anderson, the next announcement from ZIOP should be a partnership with a majorpharma/bio company looking to enter the red hot I/O space. We expect the company to execute multiple partnerships as their technology could address the myriad commercial opportunities within the spectrum of oncology. ZIOP is a BUY under $9 with a TARGET PRICE of 14
Market Data 
Markets 
AI
