{"id":2527,"date":"2015-12-07T19:10:43","date_gmt":"2015-12-08T00:10:43","guid":{"rendered":"https:\/\/digital.hbs.edu\/platform-rctom\/submission\/reprogramming-cells-to-fight-disease-rewriting-the-rules-of-biopharma-rd\/"},"modified":"2015-12-07T19:13:29","modified_gmt":"2015-12-08T00:13:29","slug":"reprogramming-cells-to-fight-disease-rewriting-the-rules-of-biopharma-rd","status":"publish","type":"hck-submission","link":"https:\/\/d3.harvard.edu\/platform-rctom\/submission\/reprogramming-cells-to-fight-disease-rewriting-the-rules-of-biopharma-rd\/","title":{"rendered":"Reprogramming cells to fight disease: Rewriting the rules of biopharma R&D"},"content":{"rendered":"

Intro<\/strong><\/p>\n

Earlier this year, CNBC recognized Moderna Therapeutics (Moderna) as the country\u2019s most ambitious and innovative private company changing the economy and overall business landscape.[1]<\/a> The accolade speaks not only to the potential of Moderna\u2019s new technology, which allows the human body to help heal itself, but also to Moderna\u2019s unique business and operating models that are revolutionizing drug R&D.<\/p>\n

The Technology: <\/strong>Moderna is a pioneer in the revolutionary new field of RNA Therapeutics. Moderna is specifically developing messenger RNA (mRNA) therapies which reprogram cells to produce proteins and antibodies.[2]<\/a> In the future, instead of being injected with a drug, your body\u2019s cells could produce the medicine for you.<\/p>\n

The Business and Operating Models:<\/strong> Wyeth Pharmaceuticals took 4-7 years to discover a new drug target and begin preclinical testing.[3]<\/a> Moderna does it in a matter of weeks.[4]<\/a> Rather than developing their own therapies, Moderna has created a network of partners who will leverage the Moderna mRNA platform for development of new products. This business model is combined with a highly automated, standardized manufacturing capability that allows them to create and test new therapies rapidly.<\/p>\n

 <\/p><\/blockquote>\n

A Quick Primer on mRNA<\/strong><\/p>\n

DNA contains genes that provide cells with the instructions for making specific proteins. To make a protein, a gene is transcribed from the DNA into messenger RNA (mRNA) which carriers the instructions to ribosomes that translate the mRNA into a protein. MRNA therapies go directly to ribosomes for translation and do not interact with DNA, so the effects are not permanent and don\u2019t alter a patient\u2019s genes.[5]<\/a><\/p>\n

These new therapeutics are particularly promising from two reasons:<\/p>\n

    \n
  1. They can require much less frequent dosing (in clinical trials some drugs that are injected every two weeks can be replaced with one injection every 6 months)[6]<\/a><\/li>\n
  2. They can produce proteins within cells (i.e., intracellular proteins). Current technologies only allow for transmembrane and secreted protein-based therapies which act on the exterior of cells.<\/li>\n<\/ol>\n

    \u00a0\"1\"<\/a><\/strong>https:\/\/youtu.be\/q8qqAkaPGqo [7]<\/a><\/p>\n

    Business Model<\/strong><\/p>\n

    Moderna\u2019s business model is designed to rapidly bring mRNA therapies to patients. Traditional pharmaceutical companies (like Wyeth) have business and operating models designed to identify drug targets for a specific disease. Discovery research alone can take 4-7 years,[8]<\/a> with another several years before the drugs are approved for use. Moderna, rather than developing individual drugs, \u201cis creating a technology \u2018platform,\u2019 akin to an operating system, that can help multiple business partners develop medicines,\u201d according to CEO Stephane Bancel.[9]<\/a> A platform-based business model is key to achieving Moderna\u2019s stated mission and vision:<\/p>\n

    Our Mission:<\/em><\/strong>\u00a0<\/em>To deliver on the promise of transformative messenger RNA (mRNA) science to bring new medicines to patients.<\/em><\/p>\n

    Our Vision:<\/em><\/strong>\u00a0<\/em>To unlock the potential of mRNA Therapeutics\u2122 by establishing an ecosystem of teams and partners that will work together to develop the broadest possible array of drugs, across diverse therapeutic areas and routes of administration, for serious diseases that are not treatable today.[10]<\/a><\/em><\/p><\/blockquote>\n

    A universal platform allows partners to work on drug targets they already know a lot about and replace protein-based therapies they\u2019ve already had approved by the FDA with an mRNA version. Moderna sources initial discovery through partnerships with research institutes and traditional pharma companies. In fact, Merck, AstraZeneca and Alexion have each paid tens of millions of dollars to have Moderna develop therapies for specific drug targets (DTs).[11]<\/a> Since their founding in 2011, they\u2019ve already got more than 60 DTs in preclinical testing and one drug in clinical trials.[12]<\/a><\/p>\n

    This business model also allows them to build their platform using partner money. While they\u2019re very well financed (~$1Bn in financing), they continue to have $900Mn in cash on hand,[13]<\/a> preferring instead to use money from partnership contracts to fund development of their platform and run tests on particular targets.<\/p>\n

    \"2\"<\/a><\/p>\n

    [14]<\/a><\/p>\n

     <\/p>\n

    Operating Model<\/strong><\/p>\n

    Moderna\u2019s operating model seeks to \u201cflatten the mRNA learning curve across the full breadth of competencies needed to drive the platform forward.\u201d In particular, Moderna is building highly automated production and manufacturing capabilities through advanced robotics technology and first-in-class drug discovery software which enables scientists working with Moderna to design, order, and optimize a drug in a matter of minutes, for delivery in weeks, from a cloud-based portal.[15]<\/a><\/p>\n

    \"3\"<\/a><\/p>\n

    https:\/\/youtu.be\/vR3nauEkiuI [16]<\/a><\/p>\n

    Their operating model gives them opportunities to work on many DTs simultaneously, scale production quickly and rapidly innovate. Combined with their partnership-based business model this allows them to deliver on their mission and vision of quickly bringing the promise of new mRNA therapeutics to patients with a variety of medical needs from infections, to cancer and rare disease.<\/p>\n

     <\/p>\n

    Citations:<\/strong><\/p>\n

    [1]<\/a> \u201cCNBC Disruptor 50 List.\u201d CNBC. <http:\/\/www.cnbc.com\/2015\/05\/12\/cnbc-disruptor-50.html><\/p>\n

    [2]<\/a> Moderna Therapeutics Company Website, \u201cmRNA Expression Platform.\u201d Accessed on December 5, 2015. <http:\/\/www.modernatx.com\/mrna-expression-platform><\/p>\n

    [3]<\/a> Huckman et al. \u201cWyeth Pharmaceuticals: Spurring Scientific Creativity with Metrics.\u201d 性视界 Business School. April 6, 2010. Page 5.<\/p>\n

    [4]<\/a> Moderna Therapeutics Company Website, \u201cmRNA Expression Platform.\u201d Accessed on December 5, 2015. <http:\/\/www.modernatx.com\/mrna-expression-platform><\/p>\n

    [5]<\/a> Alnylam Company Website. \u201cAbout RNAi.\u201d Accessed on December 6, 2015. <http:\/\/www.alnylam.com\/our-approach\/about-rnai\/><\/p>\n

    [6]<\/a> Reuters, \u201cAlnylam, Medicines Co Cholesterol Drug Effective for Six Months: Study.\u201d November 11, 2015. < http:\/\/reut.rs\/1OEnCWn><\/p>\n

    [7]<\/a> YouTube, \u201cA Quick Primer on mRNA.\u201d <https:\/\/youtu.be\/q8qqAkaPGqo><\/p>\n

    [8]<\/a> Huckman et al. \u201cWyeth Pharmaceuticals: Spurring Scientific Creativity with Metrics.\u201d 性视界 Business School. April 6, 2010. Page 5.<\/p>\n

    [9]<\/a> Weisman, Robert. \u201cBoston Area is Leading RNA Renaissance.\u201d Boston Globe. February 5, 2015. <https:\/\/www.bostonglobe.com\/business\/2014\/02\/05\/companies-developing-rna-therapeutics-are-suddenly-upswing-biomedical-world\/CmN09T59A6qu7nteLhzIjP\/story.html><\/p>\n

    [10]<\/a> Moderna Therapeutics Company Website, \u201cMission and Vision.\u201d Accessed on 12\/3\/2015. \u00a0<http:\/\/www.modernatx.com\/about-us\/about-us><\/p>\n

    [11]<\/a> Moderna Company Presenation at HBS. October 20, 2015.<\/p>\n

    [12]<\/a> Ibid.<\/p>\n

    [13]<\/a> Moderna Therapeutics Company Website, \u201cCorporate Facts.\u201d Accessed on 12\/3\/2015. \u00a0<http:\/\/www.modernatx.com\/about-us\/corporate-facts><\/p>\n

    [14]<\/a> Moderna Therapeutics Company Website, \u201cOur Business Model.\u201d Accessed on 12\/3\/2015. \u00a0<http:\/\/www.modernatx.com\/our-business-model><\/p>\n

    [15]<\/a> Moderna Therapeutics Company Website, \u201cManufacturing and Automation.\u201d Accessed on 12\/3\/2015. \u00a0<http:\/\/www.modernatx.com\/our-mrna-platform\/manufacturing-and-automation><\/p>\n

    [16]<\/a> YouTube, \u201cModerna Therapeutics: Drug Design Studio.\u201d <https:\/\/youtu.be\/vR3nauEkiuI><\/p>\n","protected":false},"excerpt":{"rendered":"

    Moderna is a pioneer in the revolutionary new field of RNA therapetutics – a technology which allows the human body to help heal itself. No only are they delivering a new technology, but they're doing it in a novel way. Remember Wyeth Pharmaceuticals? They took 4-7 years to discover a new drug target and begin preclinical testing. Moderna does it in a matter of weeks.<\/p>\n","protected":false},"author":534,"featured_media":2529,"comment_status":"open","ping_status":"closed","template":"","categories":[471,221,222,117,470,336,7,127],"class_list":["post-2527","hck-submission","type-hck-submission","status-publish","has-post-thumbnail","hentry","category-automation","category-biotech","category-pharmaceuticals","category-rd","category-rna","category-tom-winners","category-winner","category-winners"],"connected_submission_link":"https:\/\/d3.harvard.edu\/platform-rctom\/assignment\/the-tom-challenge-tom-winners-and-losers-assignment\/","yoast_head":"\nReprogramming cells to fight disease: Rewriting the rules of biopharma R&D - Technology and Operations Management<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/d3.harvard.edu\/platform-rctom\/submission\/reprogramming-cells-to-fight-disease-rewriting-the-rules-of-biopharma-rd\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Reprogramming cells to fight disease: Rewriting the rules of biopharma R&D - Technology and Operations Management\" \/>\n<meta property=\"og:description\" content=\"Moderna is a pioneer in the revolutionary new field of RNA therapetutics - a technology which allows the human body to help heal itself. 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