Cambridge, MA – March 15, 2010 – Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, today announced the appointment of Nick Leschly as the President of the company, effective April 1, 2010. Leschly, who joined the company in 2004, will be taking over from Adam West who is retiring after 30 years with the company.
“Filling the shoes of a remarkable, experienced, and thoughtful leader such as Mr West is a formidable task. However, I am really honored to have been given such a wonderful opportunity and excited about the challenges and opportunities that lie ahead,” said Mr. Leschly. This is a huge step forward in my career and I am looking forward to leading the company as we embark on this new journey. The company has a reputable history of leadership and success in the pharmaceutical industry and I hope to
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Its products treat the underlying cause of genetically based diseases by delivering corrective genes to the patient’s own bone marrow, providing the potential for a one-time transformative treatment. Genetix's lead clinical product in development is Lenti-D™ for Adrenoleukodystrophy (ALD), also known as Lorenzo's Oil disease. The results of the ongoing ALD trial, based upon work conducted by the National Institute of Health and Medical Research (INSERM), were named to Science magazine's "Scientific Breakthroughs of 2009." In addition to ALD, Genetix is conducting Phase I/II trials with its LentiGlobin™ product in beta-Thalassemia. To date, all patients receiving the full therapy with both products have shown disease arrest. In addition to Genetix’s lead programs, the company’s proprietary stem cell processing, GMP manufacturing and gene therapy vector technologies are applicable to treating other genetic diseases, opening the door to develop more
Case 1: In this case. As a certified public accountant, Erickson oversaw and initiated an arbitrary adjustment to increase cash and decrease accounts receivable. Also, Erickson signed Form 10-K with full knowledge that the financial statements include therein incorporated entries misstating revenues. As we can see from this case, Erickson’s behavior not only violate the Business and Professions Code, Division 3, Chapter 1, § 5100(g) and (i), but also against the ethical theories.
...Restricted After Gene Therapy Death. The New York Times - Breaking News, World News & Multimedia. Retrieved September 22, 2011, from http://partners.nytimes.com/library/national/science/health/052500hth-gene-therapy.html?scp=9&sq=FDA%20jesse%20gelsinger&st=cse
Background: Merck & Co. is an American pharmaceutical company and one of the largest pharmaceutical companies in the world. In 1971 the United States approved the use of an MMR vaccine made by Merck, containing the Jeryl Lynn strain of mumps vaccine. In 1978 Merck introduced the MMR II, using a different strain of the rubella vaccine. In 1997 the FDA required Merck to conduct effectiveness testing of MMRII. Initially it was over 95%; to continue the license; Merck had to convince the FDA that the effectiveness stayed at a similar rate over the years.
... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today. As scientist discovers more genes and their functions, the potential of this treatment is limitless. Though gene therapy is an auspicious treatment choice for numerous diseases (including inherited disorders, some types of cancer, and certain viral infections), the procedure remains precarious and is still under study to make sure that it will be safe and effective. Thus government regulators and scientist must take a lead role in adopting a practical approach to address these issues and determining the correct procedures for dealing with them.
Being presented with the problems in the implementation of the SAP ERP system, it is evident that Novartis Pharmaceuticals requires a comprehensive action plan that resolves key issues and the underlying problem. Refer to Exhibit A for a graphical representation of the action plan.
An Analysis of GlaxoSmithKline The business that I have done research into is GlaxoSmithKline. This company is a globalised research-based pharmaceutical public limited company. Its ownership structure has changed a great deal since the original company was first established in 1715. Originally a pharmacy, the company has expanded, merged with and taken over other companies over the decades.
Threat of new entrants is relatively high. Companies forming alliances are potential rivals. Even if earlier such company was not considered to be a threat, after merging with some research and development company or forming alliance with another pharmaceutical company it would become a rival to Eli Lilly. The threat is however weakened by significant research and development costs necessary to successfully enter the business. Eli Lilly’s focus on a relatively narrow market of sedatives and antidepressants weakens the threat of new entrants, but other products that form lesser part of company’s sales such as insulin and others are exposed to high threat of new entrants. The need of obtaining certificates and licenses also weakens the threat of new entrants. Discussed above leads to the conclusion that threat of new entrants is medium.
In September 14, 1990, an operation, which is called gene therapy, was performed successfully at the National Institutes of Health in the United States. The operation was only a temporary success because many problems have emerged since then. Gene therapy is a remedy that introduces genes to target cells and replaces defective genes in order to cure the diseases which cannot be cured by traditional medicines. Although gene therapy gives someone who is born with a genetic disease or who suffers cancer a permanent chance of being cured, it is high-risk and sometimes unethical because the failure rate is extremely high and issues like how “good” and “bad” uses of gene therapy can be distinguished still haven’t been answered satisfactorily.
Despite the significant portion of Americans that do not support embryonic stem cell research, it should be federally funded because of the potential health benefits, the definition of human, and the opportunity to clearly define regulations for ethical research. The wide range of prospective uses for stem cells could greatly improve the health and wellbeing of many people. In stem cell treatments, undifferentiated cells are programmed to form specific cells, which can then be transplanted to the afflicted area. Stem cells can possibly treat afflictions including “Alzheimer’s disease, spinal cord injury, stroke, burns, heart disease, diabetes, osteoarthritis, and rheumatoid arthritis” (“Stem Cell Basics”). Another important use is drug testing.
Merck & Co. has to be aware of the economy as with any industry. Within the recession, more and more were looking towards generic substitutes. This can at times not be a problem with patents. However, once a patent is up, a competitor who develops generic versions of Merck’s products becomes a low-cost competitor. However, during the recession from 2008 – 2009, Merck didn’t see any drop in sales. Actually, they were able to keep a continual increase in sales and net income.
Gene therapy is the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat diseases such as Severe Combined Immunodeficiency (SCID) and Adenosine Deaminase Deficiency (ADA) and possibly cancer as well. It was first researched in 1985
The first gene therapy trial was started by Dr.William French Anderson. And the patient was Ashanthi,a four year old girl who had SCID. Ashanthi had the disease because of the missing enzyme adenosine deaminase (ADA). This missing ADA stopped her body from constantly producing white blood cells that are needed to fight infections. Ashanthi was in a very vulnerable state with infections that are seen as mild. Ashanthi was able to get two choices of treatment, Antimicrobial drugs that can be used to treat SCID but only with short term benefits or a bone marrow transplant from a matching donor. The bone marrow is a soft tissue located in the centre of our bones and its purpose is to make red blood cells, platelets and white blood cells. For Ashanthi, a transplanted bone marrow would increase the production of white blood cells and would give her a strong immune system to fight the infections. Sadly however, this option was not successful because of the lack of compatible bone marrow donors. Then scientists decided to start a new gene therapy to treat Ashanthi. The scientists would get Ashanthi’s white blood cells from her blood and working copies of the adenosine deaminase gene were put into the cells by a vector. A vector is a “vehicle” used by scientists to add new genes into the DNA, But for Ashanthi, the
Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy. Although initially considered as a promising approach for treating the genetic of disease, the field has attracted disappointment for failing to fulfil its potential. With the resolution of many of the barriers that restricted the progress of gene therapy and increasing reports of clinical success, it is now generally recognised that earlier expectations may have been premature.
The case under analysis, Eli Lilly & Company, will be covering the positives and negatives with regards to the business situation and strategy of Eli Lilly. One of the major pharmaceutical and health care companies in its industry, Lilly focused its efforts on the areas of "drug research, development, and marketed to the following areas: neuroscience, endocrinology, oncology, cardiovascular disease, and women's health." Having made a strong comeback in the 1990's due to its remarkably successful antidepressant Prozac, was now facing a potential loss in profits with its patent soon to expire. The problem was not only the soon to expire patent on Prozac, but the fact that Prozac accounted for as much as 30% of total revenue was the reality Eli Lilly now faced. (Pearce & Robinson, 34-1)
The United States has long been a leader in scientific research, but it will take industry, academia, and government working together for our country to stay there. Since the implementation of the Bayh-Dole Act of 1980, which gave universities greater control over intellectual property, research universities have teamed up with partners during early-stage development to further their resources. The new task of universities was not to conduct research with the intent to make money, but to present their findings to the public domain for the sake of knowledge and the public good. In 2004, David Sinclair and Christopher Westphal, two innovative scientists following their intuition, founded Sirtris Pharmaceuticals. The founding idea arose from Sinclair’s