Taking CAR T-Cell Therapy from the Lab to the Patient Means Commercializing Where Few Have Succeeded
If you want to get a sense of the excitement level around immunotherapy cancer treatments, look at the frenzy of acquisitions and other large investments pharmaceutical companies are engaged in as they keep up with new developments. You’ll go dizzy trying to stay current. One of the promising therapies involves equipping T cells with chimeric antigen receptors (CARs), which enable them to identify, target and destroy cancer cells. CAR T-cell therapy shows incredible potential, but the companies looking to manufacture these super-soldier cells have some big questions ahead of them.
I watched the beginning of CAR T cell research some twenty years ago, and we seem to be nearing the crescendo from its first breakthrough into applications for patient care. Results from trials testing the efficacy of CAR T cells against blood cancers like leukemia have been positive. Many pharmaceutical companies are in Phase III of their research, almost ready to step into commercialization. However, taking their cells from the lab to the marketplace means scaling up production capability, which isn’t easy.
As companies choose from different devices and processes for possible commercialization, they’re building the bridges between research and the patients they’ll ultimately serve. The tricky part is that there hasn’t been enough time to determine the best ways to build these bridges.
While the healthcare community is still inventing the wheel for commercializing these treatments, there are some pointers that can help guide companies involved in CAR T-cell therapies as they consider their options.
Thinking about flexibility in your CAR T-cell commercialization is thinking about the future of your business. For example, one of the questions facing us is whether cell enrichment or cell purity will prove to be more important when separating cells for this therapy. Some devices offer remarkable purity, while others offer enrichment, and still others offer some of both.
Flexibility is valuable because CAR T-cells may have many more surprises in store for us. There’s work underway to see how well these cancer killers perform against solid tumors, and we may find that using different types of white blood cells can work better to attack different forms of cancer.
Keeping pace with the evolution of cancer immunotherapy is a tall order for companies with rigid, single-purpose manufacturing devices. Of course, anyone working in a lab knows processes need to be consistent so results stay the same. However, it’s good to have devices that are able to adapt to new processes you may start in the future.
Closed, Automated Systems
If you have a closed system for your CAR T-cell manufacturing, you’re ahead of the curve. The difficulty in moving toward process automation stems from the comfort people often have with manual steps, as well as the limited availability of devices that enable closed manufacturing. Especially in this field, where many companies are still in the research phase, moving to a closed system is a dramatic but necessary change to scale up efficiently.
The shift from performing the work by manual means to using automated devices is a fundamental step for the commercialization challenge. Once you’ve finally completed Phase III of your research, it may feel strange to suddenly start looking for closed, automated systems to do much of the work for you.
The manual processes developed during research are often difficult to scale up. It’s a large capital investment, and it would create more demand for people and time than a closed, automated system. Think of it this way: The higher a cleanroom’s classification, the more it will cost, and closed systems may not require the same cleanroom demands as the processes typically used in research phases.
End-to-end solutions can help for optimum cell therapy practices like cell collection, cell separation, and more. Cell therapy manufacturing poses unique obstacles because key devices and processes don’t always come from the same supplier. There are many device manufacturers in cell therapy, but not many offer a portfolio broad enough to carry CAR T-cell manufacturing processes from beginning to end, especially such processes that include third-party locations like hospitals and vector manufacturing facilities. To work around this dilemma, some companies are mixing and matching devices from different suppliers or using devices in new ways for certain steps in their processes.
With the pace of CAR T-cell developments and the substantial investments needed, it is understandable how tempting it can be to create a manufacturing process with devices from different suppliers, but those devices may not work together as well as if they all came from the same supplier. For example, devices from the same supplier could have data and workflow management enabled with software interconnectivity, which is critical to maintain chain of custody for cell products.
Whatever you choose, end-to-end solutions composed of automated devices at every step are probably going to be used by nearly every CAR T-cell manufacturer in the future. They provide efficiency and productivity in manufacturing for what may become a vital therapy for many patients.
No one knows for certain what new discoveries will shape the future of CAR T-cell therapy. Blood cancers seem to be the most promising, but don’t be surprised if new buzz is created around other applications. Professionals at pharmaceutical companies, medical device manufacturers, cancer centers, and other organizations are pondering the many unanswered questions around cancer immunotherapy. For now, building a flexible, automated, closed, end-to-end manufacturing process with devices that can adapt to new uses will position CAR T-cell manufacturers for present and future success in supplying cancer-fighting cells.
at IMVACS in Boston August 24th -28th.
10:30 Engineering T Cells for One and All
Harjeet Singh, Ph.D., Research Investigator, Pediatrics, MD Anderson Cancer Center
The administration of genetically modified T cells is championed by academia and industry alike. T cells are precision tools and one challenge now is to render them suitable for broad appeal as immunology is translated into immunotherapy. To help democratize T-cell therapy, I will reveal strategies how immune cells can be engineered ex vivo using a transposon/transposase system for in vivo applications. I will discuss how this non-viral approach to gene therapy can be combined immunotherapy to redirect specificity and improve the effector functions of T cells manufactured for clinical trials. For example, T cells can be genetically modified to express chimeric antigen receptors (CARs) to redirect specificity for cell-surface tumor-associated protein antigens and carbohydrates on fungi. I will reveal how the Sleeping Beauty (SB) system can be adapted and used to stably express CARs and TCRs to improve the therapeutic potential of clinical grade T cells. These clinical data serve as a foundation for additional genetic engineering to co-express transgenes to improve persistence as well as provide the opportunity for genome editing to eliminate undesired endogenous genes to improve T-cell potency and broaden their distribution and application.
Published: July 30, 2015 7:26 p.m. ET
GLOBAL & USA CANCER IMMUNOTHERAPY MARKET ANALYSIS TO 2020: Antibody Drug Conjugates (ADCs), Bispecific Monoclonal Antibodies, Cancer Vaccines, Cytokines, Interferons, Chimeric Antigen Receptor (CAR) T-Cell Therapy, PD-1/PD-L1 inhibitors, Dendritic Cells, Checkpoint Inhibitors, Adopted Cell Therapy (ACT) & IDO Inhibitors
LONDON, July 30, 2015 /PRNewswire/ -- This report provides a comprehensive overview of the size of cancer immunotherapy market, the segmentation of the market, key players and the vast potential of therapies that are in clinical trials. Oncologic therapeutics cannot cure cancer and yet in 2014, the overall market for cancer therapeutics stood at about $84.3 billion. Any drug that can provide a reasonable survival of more than five years for the cancer patients can achieve a blockbuster status. Within the cancer therapeutics, the immunotherapeutic drugs have gained worldwide acceptance, because they are targeted drugs targeting only the cancer cells. Today, cancer immunotherapy drugs have captured nearly 50% of the overall oncology drugs market, generating about $41.0 billion in 2014 alone.
I expect they will do the same again next week.
should be the same day as earnings, scheduled for next week. I believe it's the 5th
DONOR-DERIVED, CD19-DIRECTED, CAR-MODIFIED T CELLS INFUSED AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION AS PRE-EMPTIVE DONOR LYMPHOCYTE INFUSION IN PATIENTS WITH CD19+ MALIGNANCIES
Author(s): Partow Kebriaei ,Helen Huls ,Stefan Ciurea , Tingting Liu , Harjeet Singh , Shihuang Su ,
Chitra Hosing , Simon Olivares , Matthew Figliola , Pappanaicken Kumar , Bipulendu Jena ,
Sourindra Maiti , Ian McNiece , Dean Lee , Priti Tewari , Uday Popat , Amin Alousi , Betul Oran ,
Nina Shah , Katayoun Rezvani , Marie Forget , Sonny Ang , Rineka Jackson , Gabriela Rondon ,
Hagop Kantarjian , Perry Hackett , Elizabeth Shpall , Richard Champlin , Laurence Cooper
EHA Learning Center. Kebriaei P. Jun 14, 2015; 103162
Label: EHA Latest recommended materials
Disclosure(s): MD Anderson Cancer Center
Type: Oral Presentation
Presentation during EHA20: From 14.06.2015 08:30 to 14.06.2015 08:45
Location: Room Lehar 1 + 2
Allogeneic hematopoietic stem cell transplantation (HSCT) can be curative in a subset of patients with advanced B-lineage acute lymphoblastic leukemia (ALL), but relapse remains the main reason for treatment failure. Donor-derived, non-specific lymphocyte infusions (DLI) have been ineffectively infused and are associated with significant graft-versus-host-disease (GVHD). Chimeric antigen receptor (CAR)-modified T cells directed toward CD19 have demonstrated dramatic efficacy in patients with refractory ALL. However, responses are often associated with life-threatening cytokine release.
We hypothesized that infusing CAR-modified, CD19-specific T cells after HSCT as a directed DLI would be associated with less GVHD while providing cellular-based disease control, and may be associated with less cytokine released when administered in a minimal disease state.
We employed a non-viral gene transfer using the Sleeping Beauty (SB) transposon/transposase system to stably express a 2nd generation CD19-specific CAR (designated CD19RCD28 that activates via CD3z/CD28) in donor-derived T cells for patients with advanced CD19+ lymphoid malignancies. T cells were electroporated using a Nucleofector device to synchronously introduce two DNA plasmids coding for SB transposon (CD19RCD28) and hyperactive SB transposase (SB11). T cells stably expressing the CAR were retrieved over 28 days of co-culture by recursive additions of g-irradiated activating and propagating cells (AaPC) in presence of soluble recombinant interleukin (IL)-2 and IL-21. The AaPC were derived from K562 cells and genetically modified to co-express CD19 as well as the co-stimulatory molecules CD86, CD137L, and a membrane-bound version of IL-15.
To date, we have successfully treated 16 patients with advanced CD19+ ALL (n=13) or NHL (n=3); 7 patients had active disease at time of HSCT. Donor-derived CAR+ T cells (HLA-matched sibling n=9; haplo-family n=5; double cord blood n=2) were infused at a median 64 days (range 42-91 days) following HSCT to prevent disease progression. Transplant preparative regimens were myeloablative, busulfan-based (n=8) or reduced intensity, fludarabine-based (n=8). All patients were maintained on GVHD prophylaxis at time of CAR infusion with tacrolimus, plus mycophenolate mofeteil for cord, plus post-HSCT cyclophosphamide for haplo donors. The starting CAR+ T-cell dose was 106 (n=7), escalated to 107 (n=6), and currently at 5x107 (n=3) modified T cells/m2 (based on recipient body surface area). Patients have not demonstrated any acute or late toxicity to CAR+ T cell infusions. Three patients developed acute grades 2-4 GVHD (liver n=1, upper GI n=1, skin=1) which was within the expected range after allogeneic HSCT. Eight patients have relapsed at a median of 90 days following HCT (range 68-185 days). Fifty percent of patients (n=8) remain alive and in complete remission (CR) at median 7.2 months (range 2.1-21.3 months) following HSCT.
We report the first human application of the SB and AaPC platforms to genetically modify clinical-grade cells. We demonstrate that infusing donor-derived CD19-specific CAR+ T cells in the adjuvant HSCT setting as pre-emptive DLI may provide an effective and safe approach for maintaining remission in patients at high risk for relapse. Modification of the CAR construct is underway in efforts to improve CAR T-cell in vivo proliferation and persistence.
Jul 29, 2015
NEW YORK (GenomeWeb) – The National Institutes of Health this month awarded MD Anderson Cancer Center researchers a four-year grant to bioinformatically and functionally investigate genomic alterations as novel therapeutic targets for head and neck squamous cell carcinoma (HNSCC).
In recent years, genomic studies have identified numerous genetic alterations in HNSCC, but such alterations "are dominated by tumor suppressor genes and untargetable oncogenes," MD Anderson's Jeffrey Myers, who is leading the research, wrote in the grant's abstract. "Nevertheless, we hypothesize that novel molecular therapeutic targets are present in HNSCC and that these targets exist in parts of the data that have not been effectively analyzed."
With the support of the NIH grant, administered by the National Institute of Dental & Craniofacial Research and worth $971,667 in its first year, Myers and his colleagues plan to examine existing genomic data using a combination of computational and functional approaches to identify candidate drug targets.
The most promising targets will be tested in a high-throughput in vivo screening system in HNSCC lines with known genotypes, with validated targets further tested for genotype co-dependencies. Known drug targets will be studied in preclinical xenograft models.
For targets that are currently undruggable, the researchers will computationally and experimentally analyze their pathways for additional targets that can be functionally tested.
Through the work, the MD Anderson investigators aim to generate a broad list of functionally validated novel targets for HNSCC as candidates for drug development.
Biotech Report: Will Gene Therapy Go Mainstream?
The products are safe. The science is rock-solid. Patients are champing at the bit. But as much as the industry may be lining up behind gene therapies, marketing and operational questions abound. Larry Dobrow reports on the current state of the gene therapy union
Within the healthcare marketing community, gene therapy is an object of intense fascination. In understanding the potential curative promise of such drugs, marketers have fallen over themselves to tout their virtues and rip off list after list of “best practices” for promoting them—even though it's hard to codify best practices when so few of the products have actually reached the market. Nonetheless, you'd be hard-pressed to find even a C-list pharma marketer who hasn't long since awakened to the eventual virtues of gene therapy. Enthusiasm within the scientific and investment communities couldn't be higher.
And yet within patient populations and many provider circles, gene therapy remains an object of interest for different reasons. The moniker gene therapy is one of them, given how in certain uneducated circles it conjures images of mad scientists attempting to, say, create a superhuman being or performance-enhance their livestock. So with gene therapy targeting any number of conditions on its way, the question needs to be asked: Is the US market ready for gene therapy and everything that comes with it?
The short answer is yes, of course it is. The products are marvels. So far there hasn't been a major safety hiccup. Are you joking? Seriously, dude.
The longer answer is more complicated, marketing and pharma execs say. To a person they marvel at the transformative science … and fret about manufacturing and operational challenges and the ever-thorny question of asking payers to pick up six-figure tabs. They're also a little wary about making predictions when the landscape seemingly reinvents itself monthly—and that's before a huge dump of clinical-trial data arrives sometime in the second half of 2015.
“I wish I could say we have an extensive roster of clients and we're starting to take messages to various audiences,” says Amy Graham, general manager of Ogilvy CommonHealth Specialty Marketing. “But to be honest, there is an extremely limited number of clients that have gotten anywhere near needing an ad agency yet.” Adds FreshBlood CEO Bob Finkel, “We all know that [gene therapy] is the future. The science behind it is solid. But there's always going to be a bit of a wait-and-see attitude with anything that's novel. That means you need education, awareness and so much else to lower the barriers to resistance.”
Thus any potential look at the challenges of marketing gene therapies must necessarily begin with a cursory look at the current environment—which, frankly, will likely evolve between the moment MM&M hits “publish” on this piece and the one in which you read it. 23andMe, known for most of its life as a provider of $99 DNA tests, launched a drug discovery unit it dubbed 23andMe Therapeutics. While it claimed it wasn't becoming a quote-unquote drug company, 23andMe hired well-regarded former Genentech EVP of research and early development Richard Scheller to serve as the new unit's chief scientific officer. It's also partnered with a host of pharma giants, among them Pfizer and Genentech, during the last 18 months or so.
Along those lines, formal collaborations between big pharma and smaller gene-therapy specialists are thriving: between Bristol-Myers Squibb and uniQure (for cardio therapies), between Bayer and Dimension Therapeutics (Hemophilia A), between Celgene and bluebird bio (cancer). Such partnerships are born largely out of pragmatism. Big Pharma has the financial resources and the will, while gene therapy specialists have the production know-how.
“It's a rapidly evolving field,” notes Hans Duerr (photo, left), head of Bayer's global hematology business. “Many companies need a partner that's looking at this holistically and learning from other indications. Dimension understands how to package the gene the right way. They have the focus to produce [gene therapies] on a commercial scale.”
Small company/big company
Consider the example of Dutch gene therapy firm uniQure, which markets lipoprotein lipase gene therapy Glybera (photo, below left) in Europe. The company's secret sauce, such as it is, is its ability to handle the technical demands associated with the production of gene therapies. “We're driven by our abilities to design and manufacture,” says CEO Jörn Aldag (photo, right). “We have a fully integrated value chain; we can manufacture to industrial scale. We go well beyond proof of concept.” Left unsaid? Many pharma giants and would-be gene therapy companies don't.
That tech platform was among the primary factors that made uniQure an ideal partner for Bristol-Myers Squibb. Announced in early April, the collaboration gives BMS access to uniQure technology and a pre-clinical gene therapy program in the cardiovascular space designed to restore the heart's ability to synthesize S100A1, a regulator of heart function. UniQure will handle manufacturing of clinical and commercial supplies while BMS will head up regulatory activities and commercialization and pay for all R&D–related expenses. Ultimately, the two companies may end up collaborating on as many as 10 programs. “It's very validating,” Aldag says.
On the other side of the coin are large organizations like Bayer. Long among the leaders in hemophilia, the company recognized the potential of gene therapies to revolutionize the space some time ago. “One of the key challenges that's always existed in hemophilia is, ‘How do I make it easier for patients to stay adherent and get regular prophylaxis?' ” Duerr explains. Indeed, while Bayer's Kogenate has ranked among the leading Hemophilia A treatments for two decades, it requires two- or three-time-weekly injections. Bayer's gene-therapy push aims to reduce this burden on patients—to a single injection per week, perhaps, or even oral treatments at some point in the future. “The development that has been accomplished over the last couple of years has been spectacular,” he says.
But even as Duerr describes himself as an “inherent optimist,” he still sees a need to temper expectations. “With [Hemophilia A] it's not as simple as, ‘Here's a cure—one injection and you're done for life,' ” he explains. “That's difficult to prove. You'd have to test for extremely long periods of time. It's hard to make predictions.”
Which is why Bayer—and, presumably, any number of pharma companies that could soon find themselves in a similar situation—isn't making any assumptions about patient uptake of gene therapies. Kogenate may require regular shots, but it has stood the test of time; patients have come to rely on it. That's part of the reason why Duerr doesn't expect any gene therapy that treats Hemophilia A to render Kogenate obsolete anytime soon.
“Hemophilia is characterized by very strong brand loyalty and the strong experience customers have with existing products,” he says. “What we anticipate, assuming successful development and licensure of gene therapy products, is that there will be a wave of patients who will jump on [new products] quickly, but that will be a relatively small segment. Others will take a more measured approach.”
Why? Because of any number of factors. Let's say Bayer develops a therapy that, with a single shot, greatly moderates the effect of Hemophilia A, making thrice-weekly shots a thing of the past. What happens if the new therapy has a fuller expression in some patients than in others? What if those patients with the less-full expression need surgery or a tooth extraction?
“It's going to be critical for companies to offer a full suite of products,” Duerr says.
Patients, physicians and payers
While there's no set timetable for the arrival of gene therapies, marketers are already getting ready to sell them—or, rather, their efficacy and safety—to any number of potential publics, some more skeptical than others. Experts agree that the top concern, whether valid or not, is affirming that gene therapy products are safe. This shouldn't be much of a challenge at all: Aldag notes that over the course of more than 100 clinical trials of adenovirus-based gene therapy, there hasn't been a single material adverse event reported.
“Obviously we can't guarantee no long-term safety problems, but there have been no adverse events that are drug-related. None,” he says. “From a general and from a regulatory perspective, AD-based gene therapy is considered safe by the authorities, the industry and increasingly by doctors.”
Even less of a challenge, and one that makes believers shake their heads bemusedly, is the notion that gene therapy is the equivalent of “playing God” and thus crosses an ethical line drawn somewhere in the far-off sand. “Gene therapy is a radical departure from all forms of medicine that have been in existence until now,” says Margaret Cianci, executive director of the Alliance for Cancer Gene Therapy (see sidebar, "The DNA of ACGT," below). “But let's be clear: Nothing is being done that influences future generations or anything like that. It's not the type of research we're funding, nor anyone else in the US.”
Don't underplay the patient–customer service angle. Myriad Genetics, a maker of diagnostics like myRisk Hereditary Cancer, which evaluates 25 genes associated with eight cancer sites, believes that supporting individuals (physicians included) with questions about products or conditions is crucial. According to EVP, corporate communications Ron Rogers, Myriad has more than 80 genetic counselors on staff and more than 200 in its customer-service department. “There has to be an ease of use with this information,” he says.
There's reason to believe such approaches will resonate among patient and caregiver audiences. “They get it,” Finkel says. “They know it's not Botox or some other elective procedure. For some people, [gene therapies] could be the difference between life and death, not between looking beautiful or looking ugly.” And that doesn't even get into the high level of interest among today's empowered patients. “Patients know their options,” Duerr says. “They're informed about clinical trials. There is lots of awareness in disease communities.”
Some experts joke that there's more awareness in these communities than there is among physicians themselves. At the same time, most of their questions are likely to be about the logistics of the process rather than about the validity of the science. “I was chatting with a physician who's published a lot of work in hemophilia. Some of the things that came up were like, ‘Is it even ethical to have patients in gene therapy trials?” Graham reports. “We're going to have to address their concerns and questions. It's a tremendous learning curve for many of them.”
Of all potential audiences for marketing in and around gene therapies, payers will likely prove hardest to crack. It's unlikely that the next wave of gene therapies, which mostly target small patient populations, will threaten the economic underpinnings of the healthcare system the way that Gilead hep.-C cures Sovaldi and Harvoni supposedly did (it's worth noting for the 275th time: A single $80,000 drug regimen is a lot cheaper than a lifetime's worth of chronic care). “The impact of gene therapies on the system, at least for now, isn't as meaningful. It's digestible for the system,” Aldag says.
But if or when gene therapies are approved to treat conditions with large populations—like the cardiovascular ones BMS and uniQure are targeting or Parkinson's disease—all bets are off. Aldag is hopeful that pharma companies and insurers will have adopted new financial models by the time gene therapies of that kind hit the market, but he's far from certain that they will.
“The key message regarding reimbursement is that it's a one-time treatment and doesn't require delivery of product to a hospital, and that you can provide higher value to patients who are already being treated or help patients where there's no treatment at all,” Aldag explains. “If you look at this modality versus the lifetime cost of a therapeutic, you can price a gene therapy very profitably at a cost way inferior to the current lifetime cost to the healthcare system.”
Aldag then brings up the dreaded “A” word: annuity. “I think payers will increasingly accept annuity payments over the lifetime of a patient,” he says. “There won't be enormous peaks of upfront price—it would be spread over time. There's no risk of dosing a patient for $1 million and then three days later he gets run over by a car.”
Will payers buy it, literally and figuratively? Several declined requests to discuss the eventualities associated with the economics in or around gene therapy. Graham, for one, is skeptical. “Some commentators say it might require Congressional action to make this happen—and, well, we've been really successful getting consensus around health legislation,” she cracks.
“You have to get the third-party payers convinced that gene therapy is not experimental but rather an appropriate treatment path for specific individuals,” Finkel adds. “In the long run they'll save money—never been an easy argument to make with payers.”
The DNA of ACGT
It's not inaccurate to say that, in 2001, few healthcare entities devoted a lot of thought to gene therapy. On the product front, pharma companies were focusing energy and resources on prospecting for the next blockbuster. On the marketing front, they were still giddy in the wake of the opening of the DTC door a few years earlier. If you were deep into gene therapy in 2001, you likely spent many hours inside a lab or within the walls of academia.
In retrospect, then, the foresight and ambition of the Alliance for Cancer Gene Therapy's founding fathers and mothers seem all the more impressive. Back in 2001 the organization's co-founder and first president, successful business exec Edward Netter, was helping tend to a daughter-in-law stricken with breast cancer. To hear current ACGT executive director Margaret Cianci tell it, his entire outlook was changed after he attended a lecture at the Mt. Sinai School of Medicine by Dr. Savio Woo, the founding director of the Baylor College of Medicine's Center for Gene Therapy and a former president of the American Society for Gene Therapy.
“When Edward heard Dr. Woo speak about treating cancer at the molecular level, and the promise in the research, he said, ‘We have to fund this kind of work,' ” Cianci recalls. Netter and his team (including his wife, Barbara, ACGT's current president) set about finding a catchy name that would line up with the initials A, C, G and T, representing the four proteins in the DNA strand: adenine, cytosine, guanine and thymine. After assembling an A-list board of directors and scientific advisory council, the group went to work.
It speaks volumes about the organization's serious-mindedness and sense of purpose that it immediately found itself working alongside huge talents, among them groundbreaking cancer researcher Dr. Judah Folkman. Early grant recipients included Juno Therapeutics scientific founder Dr. Michel Sadelain and leukemia research giant Dr. Carl June; the group's first two classes of “young investigators” included Dr. Bob Vonderheide and current Ziopharm Oncology CEO Laurence Cooper.
What distinguished ACGT then—and continues to distinguish it now—was its specific focus. “We knew we wanted to fund a scientific platform—gene therapy—as opposed to cancer in a particular location—a tissue, an organ, whatever,” Cianci says. Nearly 15 years later the group remains stalwart in its mission. “Dr. June put it nicely: What we're trying to solve is no longer a scientific problem. It's an engineering one,” she continues. “How can we
ramp up the pace? How can we keep the cost of drugs down? How can we give access to as many patients as possible? We need to overcome those obstacles as an organization, but we also need to overcome them as a society.”
All the ACGT's work and investment hasn't yet resulted in a gene therapy for cancer making it to market—yet. Cianci says that should change within the next two years, likely with leukemia drugs from Novartis or Juno. Such potential successes would focus additional attention on the alliance and its researchers, which Cianci says will be more than welcome.
“The more people are aware of gene therapy's track record of success, the more they might choose to contribute to us,” she notes. “We're averaging three to five grants a year. We want to increase that.”
That said, ACGT has enjoyed more than its share of validation, whether from external voices acknowledging the role it's played in furthering awareness of gene therapy or from early supporters like Dr. Woo. “When he retired, we had a gala for him,” Cianci says. “For him to stand up and say that working on this organization with the Netters was the greatest thing he's done, that was beyond anything we could have imagined. It was wonderful and exciting. But there's still so much left to do.”
From the August 2015 Issue of MMM
According to a new report by Allied Market Research, titled "Global Synthetic Biology Market (Products, Technologies, Applications and Geography) - Global Opportunity Analysis and Forecast - 2013 - 2020", the global synthetic biology market is forecast to reach $38.7 billion by 2020, at a CAGR of 44.2% during the forecast period (2014 - 2020). Europe occupies largest share in the global market and would hold-on to its position throughout 2020. However, Asia Pacific is the fastest growing market with a CAGR of 46.4% from 2014 - 2020.
Synthetic biology is at a nascent stage and has recently entered the commercial market. Many technologies that utilize synthetic biology are yet to be commercialized, and are waiting for approvals from the respective regional regulatory bodies. However, this market is expected to witness adoption in varied domains, with chemicals, pharmaceuticals, energy and agriculture, as some major application markets. Key factors fueling the growth of this market include assistance from government and private organizations, rising number of entities conducting research and declining cost of DNA sequencing and synthesizing. Bio-safety & bio-security and ethical issues are key restraining factors of the market. The fact that synthetic biology can be misused has raised concerns all around the world. However, as far as the market dynamics are considered, the bottom line is that the overall impact of these factors would be highly positive.
Global synthetic biology market is segmented based on product, technology, application, and geography. Synthetic biology product market is further segmented into enabling products, enabled products and core products. Enabling product is the fastest growing segment in the product market due to ongoing researches that may bring-innovative ideas for application of synthetic biology in new fields. Thus, the need for enabling products, during R&D activities and in the development of enabled products, would rise.
DNA synthesis is the largest segment within enabling products segment, whereas oligonucleotide synthesis is expected to be fastest growing market at 57.8% CAGR during 2014 and 2020. Chassis organism would be the fastest growing core product during the forecast period with synthetic DNA occupying largest market share. Other core products included in the study are synthetic genes, synthetic sells, and XNA. Biofuels, within enabled product segment, is expected to exhibit tremendous growth; registering a CAGR of 110.1% during forecast period. However, synthetic biology-based pharmaceuticals and diagnostics products will generate largest amount of revenue within enabled product segment followed by agriculture and chemicals sub-segments.
Synthetic biology technology market is segmented into enabling technology and enabled technology. Enabling technologies segment is growing speedily, with a CAGR of 48.6% during the forecast period. The market by application includes research & development, chemicals, agriculture, pharmaceuticals & diagnostics, biofuels and others. Biofuels is the fastest growing segment during the forecast period. In terms of geography, Europe is the largest revenue-generating segment, whereas Asia Pacific would experience the highest growth rate during the forecast period.
ZIOPHARM Oncology Inc (NASDAQ:ZIOP), A rise of 1,804,439 shares or 5.5% was seen in the short interest of ZIOPHARM Oncology Inc. Even as the interest increased from 33,040,462 shares on June 30,2015 to 34,844,901 shares on July 15,2015, the days to cover came in at 11. The updated interest stood at 37.2% of the stocks floats. The shares have an average daily volume of 3,152,416 shares. The information was released by Financial Industry Regulatory Authority, Inc (FINRA) on July 24th .
ZIOPHARM Oncology Inc (NASDAQ:ZIOP) witnessed a decline in the market cap on Monday as its shares dropped 3.61% or 0.45 points. After the session commenced at $12.27, the stock reached the higher end at $12.38 while it hit a low of $11.62. With the volume soaring to 2,492,357 shares, the last trade was called at $12.03. The company has a 52-week high of $14.4. The company has a market cap of $1,543 million and there are 128,233,000 shares in outstanding. The 52-week low of the share price is $2.31.
ZIOPHARM Oncology Inc (NASDAQ:ZIOP): The mean short term price target for ZIOPHARM Oncology Inc (NASDAQ:ZIOP) has been established at $13.67 per share. The higher price target estimate is at $21 and the lower price target estimate is expected at $10 according to 3 Analyst. The stock price is expected to vary based on the estimate which is suggested by the standard deviation value of $6.35
M.D. Anderson conducts more cancer clinical trials than any institution in the world, receives the most National Cancer Institute funding, and is responsible for more than one-third of all the new FDA approved cancer drugs. The Center’s Moon Shots Program is dramatically accelerating the pace of converting scientific discoveries into clinical advances that reduce cancer deaths.
Investment analysts at Wunderlich upped their price objective on shares of Intrexon Corp (NYSE:XON) from $55.00 to $70.00 in a note issued to investors on Monday, Market Beat Ratings reports. The firm currently has a “buy” rating on the stock. Wunderlich’s price objective suggests a potential upside of 23.11% from the stock’s previous close.
ZIOPHARM Oncology (NASDAQ:ZIOP) was upgraded by investment analysts at Vetr from a “buy” rating to a “strong-buy” rating in a note issued to investors on Monday, Market Beat Ratings reports. The firm currently has a $14.00 price target on the biotechnology company’s stock. Vetr‘s price target would indicate a potential upside of 12.18% from the company’s current price.
ZIOPHARM Oncology (NASDAQ:ZIOP) opened at 12.48 on Monday. ZIOPHARM Oncology has a 1-year low of $2.31 and a 1-year high of $14.40. The stock’s 50-day moving averageis $11.57 and its 200-day moving average is $10.42. The company’s market cap is $1.60 billion.
ZIOPHARM Oncology (NASDAQ:ZIOP) last released its earnings data on Thursday, May 7th. The biotechnology company reported ($0.10) earnings per share (EPS) for the quarter, beating the consensus estimate of ($0.13) by $0.03. The company had revenue of $0.30 million for the quarter, compared to the consensus estimate of $0.20 million. During the same quarter in the previous year, the company posted ($0.10) earnings per share. The company’s revenue for the quarter was up 50.0% on a year-over-year basis. Analysts expect that ZIOPHARM Oncology will post $-0.70 EPS for the current fiscal year.
Shorts will have to cover at much higher prices if there are no sellers.
If Merck is going to be using T Cells transposed through "sleeping Beauty" tech for this study, that's a big paycheck for ZIOP. I expect we will hear an update regarding Merck on the conference call in two weeks.