CAR-T Executive Summary

Improvements in health care and life sciences are an important source of gains in health and longevity globally. The development of innovative pharmaceutical products plays a critical role in ensuring these continued gains. To encourage the continued development of new drugs, economic incentives are essential. These incentives are principally provided through direct and indirect government funding, intellectual property laws, and other policies that favor innovation. Without such incentives, private corporations, which bring to market the vast majority of new drugs, would be less able to assume the risks and costs necessary to continue their research and development (R&D). In the United States, government action has focused on creating the environment that would best encourage further innovation and yield a constant flow of new and innovative medicines to the market. The goal has been to ensure that consumers would benefit both from technological breakthroughs and the competition that further innovation generates. The United States also relies on a strong generic pharmaceutical industry to create added competitive pressure to lower drug prices. Recent action by the Administration and Congress has accelerated the flow of generic medicines to the market for precisely that reason. By contrast, in the Organization for Economic Cooperation and

The GDC standardizes data from current data storage projects and will collect data which researchers choose to share. The NCI is also working with the DOD and the VA on the Applied Proteogenomics Organizational Learning and Outcomes (APOLLO) consortium to move forward with cancer detection and prevention techniques. Collaborating with pharmaceutical and biotech companies, the NCI is also creating a list of pre-approved shared compounds for researchers to use more easily. The Departments of Defense and the Veterans Affairs are increasing their investments in cancer research by funding Centers of Excellence focused on specific cancers, and by conducting large longitudinal studies to help determine risk factors and enhance treatment. The FDA is developing an Oncology Center of Excellence to accelerate development and coordination of new technologies. It has also set up the “Navigator” program in order to make new treatments more rapidly

Some of traditional drugs may be effective in patients whose cancers have a specific molecular target, and not for other patients. To solve this problem of patient-specificity, pharmaceutical research have seen the expansion of individually tailored cancer treatment, which is an application of targeted therapy, and this is where biopharmaceuticals are. As an increasing part of the population is diagnosed with cancer and as these patients live longer, increasing care will be given to patients who have received these drugs. Moreover, in the case of cancer therapy, those drugs and especially with mABs are a promise of less side effects : recombinant DNA technology makes it possible to genetically engineer an antibody to reduce the risk of host immune response.

Many doctors, physicians, researchers and biotech companies--including the revolutionary Seattle Genetics research facility--are now turning to antibody-assisted cancer treatments and precisely targeted cures instead of treating cancer with a cocktail of chemicals and radiation that generate risky side effects and damage the healthy tissue that patients need to recover. Cancers are among the most frightening and difficult-to-treat illnesses. Ranked as the leading cause of death and disability, cancer is actually an umbrella term that covers many different diseases. Each person faces a unique disease because cancers interact with the body's existing cells, so each case has a

Once the T-cells have been isolated, synthetic antibodies are produced and integrated into the surface of the T-cells that recognize specific antigens on the affected tumor and cancer cells. The timeline for manufacturing Kymriah is approximately three to four weeks. The period in which the gene immunotherapy is being prepared, patient will undergo immunosuppression chemotherapy to prepare the body to accept the genetically modified T-cells.

“It is critical to the nation’s future health that innovative practices and biomedical and clinical research be promoted and incentivized” (Torrens & Williams, 2009). It is the medical research team that has to develop the drug, device or technology and make it accepted and available for patients and clinicians to use. “This phase involves private companies and private capital almost entirely and calls for a mixture of scientific promotion to the technical expert community and broader general marketing to the health care system that must ultimately approve it for purchase and clinical use” (Torrens & Williams, 2009). It is through clinical trials that will eventually gain the FDA approval, if a drug or medical device is deemed safe for the community to use. “Once the appropriate regulatory approvals are obtained for a new device or drug, the development process moves into an active marketing phase designed to encourage the use of the product by individual clinicians and

Nucleon is a small biotechnology start-up company focused on developing biotechnological pharmaceutical products based on a class of proteins known as cell regulating factors. The company has been in the market for five years, and currently, they are in the process of human trials for their first potential product, “cell regulating protein-1” (CRP-1). Overcoming these phases, Nucleon has to decide among several alternatives on producing CRP-1. Knowing that the process involved a tremendous amount of time and money, Nucleon has to choose the right decision for their long-term survival in the intensively competitive and high-stakes drug industry.

Finding a way to restore the function of the Histocompatibility Complex genome so that it can activate antibodies such as β2-microglobulin (β2-m) to kill the tumour should be an area focused on because it can then be introduced into one infected Devil that can bite others and so the genome is passed on. (Siddle H et.al 2012)

In an exclusive interview posted on Inspirery, Dr. Clay Siegall, the CEO of Seattle Genetics, explains why he has focused on targeted therapies for cancer. Siegall, who holds a Ph.D in genetics from George Washington University, co-founded the biotech company to update cancer treatments so that chemotherapy is not so traumatic for cancer patients. Since Seattle Genetics's first ADC, which targets cancer cells as opposed to both cancer and healthy cells, became FDA-approved, Siegall has generated substantial revenue for company by arranging licensing agreements. They took a risk, said Siegall, since only one out of every ten drugs submitted to the FDA receives their approval. Under Siegall's leadership, Seattle Genetics is developing a diverse

There is a strong pipeline in Fate therapeutic. Their pipeline can be divided into two areas: immuno-oncology, including 3 products named FATE-NK100, engineered hnCD16 iNK and engineered CAR iTcell, and immuno-regulation, including 2 products named ProTmune and ToleraCyte. It shows that Fate has a great interest in nature killer cells, T cells and CD34+ cells. This report will mainly introduce the technique in FATE-NK100 and ProTmune which are the only two products in clinical stage. In addition, the entire pipeline is built on the induced pluripotent stem cell technique. [7]

The strategic implications for Vertex attempting to fund and develop four drugs are as following:

Market Analysis – The combination of lengthy approval process of the FDA, intellectual property laws, and R&D capital requirements, poses a high entry barrier in the blood substitute markets. The human market is expected to

Achieve a median composite eight-year product development cycle by 2010. Deliver two new molecular entity (NME) launches on average per year from 2010. In order to achieve the above objective, ensure that we have 10 or more NMEs in Phase III development by 2010. Development cycle times and quality for small molecules and biologics. Number of NME launches per year. Attrition rates. Number of development projects by phase. Number of in-licensing deals, alliances and acquisitions. R&D investment levels. Improving R&D quality and speed through leading-edge science, effective risk management and decision-making and overall business efficiency. Maximising the value of our biologics business and continuing to build a major presence in this fast-growing sector. Investing in external opportunities to enhance our internal innovation through in-licensing, alliances and acquisitions. 2008 target exceeded for small molecule development cycle times. NME and life-cycle management progressions

Unlike Gilead that has only one product in its Oncology line, Bristol-Myers Squibb presently have four different drugs namely: Erbitux –an epidermal growth factor receptor (EGFR) antagonist for the treatment of Head & Neck cancer and Colorectal cancer,(2) Opdivo (nivolumab) for the treatment of unresectable or metastic melanoma and lungs cancer, (3) Syrcel( dasatinb) for the treatment of newly diagnosed adult with Philadelphia chromosome –positive (ph+) chronic myeloid leukemia and (4) Yervoy (ipilimumob) for the treatment of melanoma a type of skin cancer that spread and as such cannot be remove by surgery. Like Seattle Genetics, the company products use either Antibody-drug Conjugate (ADC) though in a different version by linking potent cytotoxic to monoclonal antibodies targeted to specific tumor cells or immune-oncology, an innovative technology that unlocks the body own immune system to fight against cancerous cells. It also expanded its focus to Nolch inhibitor (used in blocking powerful pathway that promotes tumor cell survival for certain other types of cancer. (Bristol-Myer Squibb, 2014) Because the technology is similar but used differently, BMS would be considered a close competitor who currently has the advantage of having four targeted specific drugs and 12 other oncology and immune-oncology in various trial phases.

The research and development of the pharmaceutical industry is very important as the industry relies on it to develop new products to maintain and sustain the growth of the industry (ALRC 2014). According to the Australian Government Law Reform Commission, every year, the total spending in research and development in pharmaceutical industry, which includes drug discovery, pre-clinical testing and clinical trials on drugs is around $300 million (ALRC 2014). Mergers and acquisitions are intensifying in the global pharmaceutical industry, especially over the last 10 years. With factors like exorbitant research and development costs, the relatively shorter product life cycles, and the rarity of discovering a new life-changing drug acting as catalysts, leading pharmaceutical companies now have more cause to step out and look for external collaboration. This results in an increasing number of smaller biotechnology companies merging with bigger pharmaceutical companies (The