Question: In 1995, Edward Lanphier founded Sangamo
In 1995, Edward Lanphier founded Sangamo Biosciences for the purpose of developing zinc-finger nucleases (ZFNs), a new technology that offered potential for “editing†the genetic code of a living individual to correct genetically based diseases (e.g., hemophilia, sickle cell anemia, Huntington’s disease, and many others) or to confer genetic resistance to non-genetically based diseases.
ZFNs work by cutting the DNA in a chosen spot. The cell then typically attempts to repair the cut by either by polishing the two ends of DNA and sealing them back together or by copying the corresponding section of DNA in the other half of the chromosome pair. Since many diseases occur because of a gene on a single half of the chromosome pair, this “homologous substitution†from the other chromosome corrects the faulty gene. Alternatively, scientists can even provide a template gene sequence that they want to use to substitute for the cleaved portion of the DNA (see Figure 8.1).
Gene editing offered a radical new way to cure or prevent diseases, but it required a significant amount of R&D work both to develop ZFNs that were precise and reliable enough to safely edit human genes and to develop a delivery mechanism that would ensure the ZFNs penetrated enough of an individual’s cells to make a difference. Clinical trials to establish the treatment’s safety and efficacy to get FDA approval would also be a huge hurdle to overcome.
Since none of Sangamo’s products were commercially available yet, the company was entirely reliant upon grants and funding from partners for its survival. Though Sangamo had signed big collaboration agreements with Shire AG and Biogen IDEC for several of its treatment areas, it had ambitions to develop its revolutionary treatment for HIV on its own, establishing itself as a fully integrated biopharmaceutical company.
Correcting Monogenic Diseases
Monogenic diseases are diseases that are caused by a defect in a single gene. One example is hemophilia. People with hemophilia lack sufficient clotting factors in their blood, resulting in them bleeding longer after an injury. Internal bleeding, in particular, can cause significant damage and be life threatening. Individuals with hemophilia need regular infusions to replace the clotting factor in their blood. Sangamo’s ZFN treatment offered the hope of a cure, rather than lifelong treatment.a Sangamo had already demonstrated that its ZFN method for treating hemophilia worked in mice and was preparing to file an application to begin clinical trials. Sangamo also had developed treatments for sickle cell anemia and beta-thalassemia, also monogenic diseases. Normally patients with sickle cell anemia or beta-thalassemia require lifelong care or bone marrow transplants…………..
Required:
1. What were the pros and cons of Sangamo pursuing its gene editing programs alone versus working with a partner?
2. Does the HIV program offer any special opportunities or challenges?
Figure 8.1:
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Transcribed Image Text:
FIGURE 8.1 Gene editing with nucleases Nuclease MÁVV Chromosome Cut Non homologous end joining (NHEJ) Loss of few DNA repair matrix DNA repair matrix base pairs homologous | Paste recombination Paste Gene insertion Gene repair Gene inactivation knock in knock in
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> Why do you think so many innovation projects fail to generate an economic return?
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> Gavriel Iddan was an electro-optical engineer at Israel’s Rafael Armament Development Authority, the Israeli authority for development of weapons and military technology. One of Iddan’s projects was to develop the “eye” of a guided missile, which leads t
> In the global video game industry, the introduction of each generation of console has ushered in a new battle for market dominance. New entrants have made startling entrances and toppled seemingly invincible incumbents. Game developers, distributors, and
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> Can you identify an example of a development project and what type of team you believed they used? Do you think this was the appropriate type of team given the nature of the project?
> What are some of the ways that managers can ensure that a team reaps the advantages of diversity while not being thwarted by some of the challenges team diversity raises?
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> What kinds of people make good project champions? How can a firm ensure that it gets the benefits of championing while minimizing the risks?
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> Consider a group project you have worked on at work or school. Did your group use mostly sequential or parallel processes?
> What are some of the advantages and disadvantages of a parallel development process? What obstacles might a firm face in attempting to adopt a parallel process?
> Is the Stage-Gate process consistent with suggestions that firms adopt parallel processes? What impact do you think using Stage-Gate processes would have on development cycle time and development costs?
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> What are some of the advantages and disadvantages of the transnational approach advocated by Bartlett and Ghoshal?
> Why is the tension between centralization and decentralization of R&D activities likely to be even greater for multinational firms than firms that compete in one national market?
> What factors should a firm take into account when deciding how centralized its R&D activities should be? Should firms employ both centralized and decentralized R&D activities?
> What are some of the advantages and disadvantages of having formalized procedures for improving the effectiveness or efficiency of innovation?
> Are there particular types of innovation activities for which large firms are likely to outperform small firms? Are there types for which small firms are likely to outperform large firms?
> Describe a technological innovation not discussed in the chapter, and identify where you think it lies on the control continuum between wholly proprietary and wholly open.
> Can you identify a situation in which none of the legal protection mechanisms discussed (patents, copyrights, trademarks, trade secrets) will prove useful?
> When will trade secrets be more useful than patents, copyrights or trademarks?
> Consider a firm that is considering marketing its innovation in multiple countries. What factors should this firm consider in formulating its protection strategy?
> What marketing strategies are used by the producers of the product you identified for question 3? What are the advantages and disadvantages of these marketing strategies?
> What are the differences between patents, copyrights, and trademarks?
> Are their some industries in which you would expect to see particularly short technology cycles? Are their some industries in which you would expect to see particularly long technology cycles? What might be some of the factors that influence the length o
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> What are some of the reasons that a firm might use both qualitative and quantitative assessments of a project?
> How does the mode of collaborating (e.g., strategic alliance, joint venture, licensing, outsourcing, collective research organization) influence the success of a collaboration?
> What are some of the advantages and disadvantages of collaborating on a development project?
> Identify a particular development project you are familiar with. What kinds of methods do you believe were used to assess the project? What kinds of methods do you believe should have been used to assess the project?
> For what kind of development projects might a real options approach be appropriate? For what kind of projects would it be inappropriate?
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> What determines whether an industry is likely to have one or a few dominant designs?
> Why is it necessary to perform an external and internal analysis before the firm can identify its true core competencies?
> What makes an ability (or set of abilities) a core competency?
> What is the difference between a strength, a competitive advantage, and a sustainable competitive advantage?
> Are dominant designs good for consumers? Competitors? Complementors? Suppliers?
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> What are some of the advantages of entering a market early? Are there any advantages to entering a market late?
> What are some of the ways a firm can try to increase the overall value of its technology, and its likelihood of becoming the dominant design?
> What factors will (or should) influence a firm’s pricing strategy?
> What are some examples of industries not mentioned in the chapter that demonstrate increasing returns to adoption?
> What are some of the sources of increasing returns to adoption?
> Why do technologies often improve faster than customer requirements? What are the advantages and disadvantages to a firm of developing a technology beyond the current state of market needs?
> What are some of the reasons that both technology improvement and technology diffusion exhibit s-shaped curves?
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