V

viewpoints

DOI:10.1145/3292517 Michael A. Cusumano

Technology Strategy
and Management
CRISPR: An Emerging
Platform for Gene Editing
Considering a potential platform candidate in
the evolving realm of gene-editing technologies research.

W
HEN THINKING ABOUT
which areas of research
might form the basis for
new industry platforms,
in the past we have
focused on information technologies
such as computers, Internet software,
smartphones, cloud services, artificial
intelligence and machine learning, and
even quantum computing (see “The
Business of Quantum Computing,”
Communications, Oct. 2018). These
technologies early on had the potential
to generate what we call “multi-sided
markets” with powerful “network ef-
fects.” Network effects are self-rein-
forcing feedback loops where, as the
number of users or complementary
innovations increase, the more widely
used and valuable the platform be-
comes (see “The Evolution of Platform
Thinking,” Communications, Jan. 2010).
Another early-stage technology
suited to platform dynamics is gene
editing. Research began several de-
cades ago, leading to various tools and viruses. What scientists observed years CRISPR-associated enzymes as “mo-
techniques. It is still uncertain which ago is that specialized segments of RNA lecular scissors” to cut, modify, or re-
approach will become the dominant and associated enzymes in one organ- place genetic material. The potential
foundation for further research and ism can modify genes (DNA sequences) applications include diagnostic tools
applications development, but there in another organism. For example, this and treatments for genetic diseases
IMAGE F RO M SH UTT ERSTOCK.CO M

are some platform candidates. happens naturally when the immune as well as genetic reengineering more
One particularly promising technol- system in bacteria fight against an in- broadly.8 An August 2016 article in Na-
ogy is CRISPR, or “Clustered Regularly vading virus. In 2012, several scientists tional Geographic magazine described
Interspaced Short Palindromic Re- discovered they could use CRISPR se- CRISPR’s potential: “CRISPR places an
peats.”12 CRISPR refers to small pieces quences of DNA as well as “guide RNA” entirely new kind of power into human
of DNA that bacteria use to recognize to locate target DNA and then deploy hands. For the first time, scientists can

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viewpoints

quickly and precisely alter, delete, and searchers and applications, which in
rearrange the DNA of nearly any living turn have inspired more research, tool
organism, including us. In the past development, applications, venture
three years, the technology has trans- capital investments, and so on.
formed biology … No scientific dis- At the center of an emerging CRISPR
covery of the past century holds more ecosystem is a non-profit foundation
promise—or raises more troubling called Addgene, founded in 2004 by
ethical questions. Most provocatively, if MIT students. It funds itself by selling
CRISPR were used to edit a human em- plasmids, small strands of DNA used in
bryo’s germ line—cells that contain ge- laboratories to manipulate genes. Since
netic material that can be inherited by 2013, it has been collecting and dis-
the next generation—either to correct tributing CRISPR technologies to help
a genetic flaw or to enhance a desired researchers get started on their experi-
trait, the change would then pass to that ments.14 The Addgene tools library con-
person’s children, and their children, sisted of different enzymes and DNA or
in perpetuity. The full implications of RNA sequences useful to identify, cut,
Advertise with ACM! changes that profound are difficult, if edit, tag, and visualize particular genes.a
not impossible, to foresee.”10 There were also numerous startups,
DNA resembles a programming some of which have already gone pub-
Reach the innovators language and data-storage technology, lic. CRISPR Therapeutics (founded in
and thought leaders useful in different applications. Gene 2013) was trying to develop gene-based
editing provides opportunities for com- medicines to treat cancer and blood-re-
working at the panies to pursue product solutions, lated diseases, and collaborating closely
cutting edge such as to build standalone diagnostic with Vertex and Bayer. Editas Medicine
tools or gene therapies. It also enables (2013) and Exonic Therapeutics (2017)
of computing some institutions and companies to were tackling diseases such as cancer,
and information create products, tools, or components sickle cell anemia, muscular dystrophy,
that other firms can build upon. Like and cystic fibrosis.b Beam Therapeutics
technology through today’s quantum computers, each use (2018) planned to use CRISPR to edit
ACM’s magazines, of CRISPR seemed to require special- genes and correct mutations.1 Mam-
ized domain knowledge (that is, the moth Biosciences (2018) was following
websites genome of a particular organism and more of a platform strategy and devel-
and newsletters. disease) and then tailoring to the appli- oping diagnostic tests that could be the
cation, such as to use CRISPR to design basis for new therapies. It was broadly li-
a diagnostic test or therapeutic product censing its technology and encouraging
◊◆◊◆◊ for a specific disease, or to reengineer a other firms to explore therapies based
plant to fight off insects. But, along with on its testing technology.11 In fact, Mam-
rising numbers of CRISPR research- moth’s goal was to create “a CRISPR-en-
Request a media kit ers, platform-like network effects and abled platform [italics added] capable of
with specifications multisided market dynamics were also detecting any biomarker or disease con-
appearing and helping the industry taining DNA or RNA.” In a recent public
and pricing: evolve. In particular, more research statement, the company summarized
publications have led to improvements its strategy to cultivate an applications
Ilia Rodriguez in tools and reusable component li- ecosystem: “Imagine a world where you
braries, which have attracted more re- could test for the flu right from your liv-
+1 212-626-0686 ing room and determine the exact strain
[email protected] you’ve been infected with, or rapidly
DNA resembles screen for the early warning signs of can-
cer. That’s what we’re aiming to do at
a programming Mammoth—bring affordable testing to
language and data- everyone. But even beyond healthcare,
we’re aiming to build the platform for
storage technology, CRISPR apps [italics added] and offer the
useful in different technology across many industries.”3

applications.
a See https://www.addgene.org/crispr/
b See A. Regalado, “Startup Aims to Treat Mus-
cular Dystrophy with CRISPR,” MIT Technology
Review (Feb. 27, 2017) and http://www.editas-
medicine.com/pipeline

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Commercialization of CRISPR sys-

tems was still years away, and the tech-
Ethics and social
Calendar
nology had limitations. It was better at
screening, cutting, and rewriting rather
than inserting DNA.4 And only recently
issues might of Events
have medical centers and companies hinder widespread January 13–19
applied to start CRISPR-related clinical use of gene editing. POPL ‘19: The 46th Annual
ACM SIGPLAN Symposium
trials. There were also alternative tech-
on Principles of
nologies with different strengths and Programming Languages,
weaknesses. In particular, TALEN (Tran- Lisbon, Portugal,
scription Activator-Like Effector Nucle- Sponsored: ACM/SIG,
Contact: Fritz Henglein,
ases), another gene-cutting enzyme tool, brown eyes, or a higher IQ?5 Email: [email protected]
was more precise than CRISPR and more In sum, platform dynamics were in-
scalable for some non-laboratory appli- fluencing areas outside of information January 14–18
cations, though it was more difficult to technology. It was not so clear, though, AFIRM ‘19: ACM SIGIR/SIGKDD
African Workshop on
use.6 In general, CRISPR was in the lead, how to use the power of the platform Machine Learning for
with several universities and research wisely and safely, and what types of Data Mining and Search,
centers, startup companies, and estab- government monitoring and self-reg- Cape Town, South Africa,
lished firms actively publishing papers, ulation were most appropriate. These Co-Sponsored: ACM/SIG,
Contact: Hussein Suleman,
applying for patents, and sharing their issues were likely to become fierce Email: [email protected]
tools and depositories of genetic compo- topics of debate as CRISPR and other
nents. Most researchers were also focus- gene-editing technologies evolved into January 29–31
ing on CRISPR-Cas9, a specific protein widely used platforms for medical, FAT* ‘19: Conference
on Fairness, Accountability,
that used RNA to edit DNA sequences. food, and other applications. and Transparency,
One concern is that the business Atlanta, GA,
References Sponsored: ACM/SIG,
models of biotech startups and pharma- 1. Al Idrus, A. Feng Zhang and David Liu’s base-editing
Contact: danah boyd,
ceutical companies depended on pat- CRISPR startup officially launches with $87 million.
Email: [email protected]
FierceBiotech.com, (May 14, 2018).
ent monopolies, making the industry 2. Boettcher, M. and McManus, M.T. Choosing the right tool
ultra-competitive and locking applied for the job: RNAi, TALEN, or CRISPR. Molecular Cell 58,
4 (May 21, 2015), 575–585; https://bit.ly/2DOHZB5.
research into protected silos. The result 3. CRISPR company cofounded by Jennifer Doudna February
was potentially a “zero-sum game” men- comes out of stealth mode. Genome Web (Apr. 26,
2018); https://bit.ly/2QYHkjo February 11–15
tality. This contrasted to the more co- 4. Cyranoski, D. CRISPR alternative doubted. Nature WSDM 2019: The 12th ACM
operative (but still highly competitive) (Aug. 11, 2016), 136–137.
International Conference on
5. Hayden, E.C. Should you edit your children’s genes?
spirit of “growing the pie” together that Nature (Feb. 23, 2016). Web Search and Data Mining,
we generally see with basic science and 6. Labiotech Editorial Team. The most important battle Melbourne, VIC Australia,
in gene editing: CRISPR versus TALEN (Mar. 13, Co-Sponsored: ACM/SIG,
which we saw in the early days of the per- 2018); https://bit.ly/2TwHLmC. Contact: Alistair M. Moffat,
sonal computer, Internet applications, 7. Lander, E. The heroes of CRISPR. Cell (Jan. 14, 2016).
8. McKinsey & Company. Realizing the potential of Email: ammoffat@unimelb.
and even smartphone platforms such CRISPR. (Jan. 2017); https://mck.co/2Bl2MK0 edu.au
9. Molteni, M. A new startup wants to use CRISPR to
as Google’s Android. Of course, CRISPR diagnose disease. Wired (Apr. 26, 2018). February 24–26
scientists openly shared and published 10. Specter, M. How the DNA revolution is changing us.
FPGA ‘19: The 2019 ACM/SIGDA
National Geographic (Aug. 2016).
their basic research.7 And though the 11. Vayas, K. New CRISPR-based platform could soon International Symposium
U.S. Patent Office already has granted diagnose diseases from the comfort of your home. on Field-Programmable
hundreds of patents related to CRISPR, Science (Apr. 29, 2018). Gate Arrays,
12. Zimmer, C. Breakthrough DNA editor born of bacteria. Seaside, CA,
patent holders usually offered free li- Quanta Magazine (Feb. 6, 2015.
Sponsored: ACM/SIG,
13. Zimmer, C. What is a genetically modified crop? A
censes to academic researchers, even European ruling sows confusion. The New York Times, Contact: Kia Bazargan,
those still under litigation. (July 27, 2018) Email: [email protected]
14. Zyontz, S. Running with (CRISPR) scissors:
Ethical and social issues might hin- Specialized knowledge and tool adoption.
February 25–26
der widespread use of gene editing. Technological Innovation, Entrepreneurship, and
HotMobile ‘19: The 20th
Strategic Management Research Seminar, MIT Sloan
The controversies centered on how School of Management (Oct. 22, 2018). International Workshop on
much genetic engineering should we, Mobile Computing Systems
Michael A. Cusumano ([email protected]) is a professor and Applications,
as a society, allow? Experts already dis- Santa Cruz, CA,
at the MIT Sloan School of Management and founding
agreed about the safety of genetically director of the Tokyo Entrepreneurship and Innovation Sponsored: ACM/SIG,
altered plants and animals that con- Center at Tokyo University of Science. Contact: Alec Wolman,
tributed to the human food supply.13 Email: [email protected]
The author thanks Samantha Zyontz as well as David
Scientists can deploy similar technol- Fritsche, Gigi Hirsch, and Pierre Azoulay for their
comments. This column is derived from a forthcoming
ogy to change human embryos and book by Michael A. Cusumano, Annabelle Gawer, and
cells, such as to treat genetic diseases David B. Yoffie, The Business of Platforms: Strategy in
the Age of Digital Competition, Innovation, and Power,
or potential disabilities. But should we Harper Business, June 2019.
allow parents to edit their children’s
genes, such as to select for blue versus Copyright held by author.

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