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April 2019 NUCLEUS 3-25 DED UN 18 O 98 F http://www.nesacs.org N Y O T R E I T H C E N O A E S S S L T A E A C R C I N S M S E E H C C TI N O CA April 2019 Vol. XCVII, No.8 N • AMERI Monthly Meeting 2019 Esselen Award to Carolyn R. Bertozzi at Harvard University 2018 Richards Medal Address By Chad A. Mirkin How to Allocate Resources to Intellectual Property as a Start-Up By Katherine Ann Rubino 2019 NESACS Candidates for Election 2018 Richards Medal Address Rational Vaccinology: In Pursuit of the Perfect Vaccine By Chad A. Mirkin, Northwestern University The field of nanomedicine, broadly defined, uses concepts and tools in nanoscience to develop new ways of tracking and treating disease and making and delivering drugs. In this talk, I focus on one exciting class of nanostructures - spherical nu- cleic acids, or SNAs - that organize nucleic acids into densely packed and highly oriented three-dimensional forms not seen in nature (Figure 1).1 SNAs are highly potent gene regulation agents and components of innovative vaccines and cancer im- munotherapies.2-5 The reason this field is so important and garners such broad scientific interest is because nanostructures have en- abling properties as a result of their size, shape, and compo- sition. In the field of nanotechnology, size is often the focus; but, when compared to the size of small molecules, which have been the primary basis for drugs for decades, nanostruc- tures are actually relatively large. This issue of size is impor- tant for two reasons. First, a general tenet of nanotechnology is that when bulk materials are miniaturized to the nanoscale, they take on new properties even if the “large” and “small” materials are compositionally identical. These emergent prop- erties have driven immense technological development, espe- Fig. 1: Schematic of a SNA with a liposomal core surrounded by a dense cially in molecular diagnostics, imaging, and cellular studies shell of highly oriented nucleic acids and assays. They are also beginning to influence the way attacked in many different ways. With gene regulation or medicines are developed. Many nanostructures are inherently knock-down strategies, such as those based on anti-sense and modular (multi-component) and lend themselves to multifunc- siRNA, one can use nucleic acid medicines to interfere with tionality, something not easily achieved with small molecules. nucleic acid signatures that produce too much of a particular In addition, because they are on the same length scale as many protein associated with a given disease. By regulating protein sub-cellular components that are responsible for trafficking expression, a cell can be transformed from a diseased to a medicines, they can easily interface with them. healthy one or, in the case of cancer, cause a diseased cell to Nanotechnology is rapidly accelerating the development selectively die or be sensitized towards treatment with of nucleic acid therapeutics by enabling key challenges to be chemotherapeutics. However, the opportunities are far greater. overcome that are allowing us to realize the long-standing Through studies of viral and bacterial disease, we now know dream of creating medicines that can target any disease with specific DNA and RNA signatures that can be utilized to stim- a known genetic basis. Let us consider conventional nucleic ulate or suppress the immune system. Therefore, in the lab, acid medicines. Just 15 years ago, small molecule drugs dom- we can synthesize DNA or RNA molecules, that if delivered inated the top 10 list of the most prescribed drugs.6 Today, 7 appropriately, can regulate the immune system to treat disease, of the top 10 most prescribed drugs are biologics — a remark- including many forms of cancer and autoimmune diseases. able shift in a relatively short period of time.7 While it is clear With all of these advantages, one might ask why nucleic that the field of medicine has overwhelmingly transitioned acid medicines, which have been around for 30 years, are not from small molecules to biologics, many people, including widely used already. Initially, researchers were extremely ex- myself, believe that the next wave, and perhaps the most cited, but quickly realized that a number of key requirements promising one, will be the era of nucleic acid medicines. The had to be addressed before such drugs could be successfully reasons are straightforward. First, with nucleic acid medicines, implemented. First, there had to be ways to make nucleic acid disease can be treated at its genetic roots, and we now know medicines at a reasonable cost – it turns out that this is no many of the genetic origins of disease. Second, once one de- longer a problem, and we can now make large quantities of velops one type of drug, another one can be prepared by sim- nucleic acid medicines at pharmaceutical scale. Second, bio- ply changing the sequence of the nucleic acid (the logical pathways through which nucleic acid medicines can identification and synthesis of a whole new molecule is not function must be thoroughly understood – this, also, is no required). This means that the timeline required for drug de- longer a limiting factor. Through the Human Genome Project velopment will be significantly shortened, an exciting and mass sequencing, we now understand and can regulate prospect because much of the cost lies in that process. Another many of the relevant pathways for a wide variety of disease attractive aspect of nucleic acid drugs is that disease can be continued on page 6 2 The Nucleus April 2019 The Northeastern Section of the American- Chemical Society, Inc. Contents Office: Anna Singer, 12 Corcoran Road, Burlington, MA 01803 (Voice or FAX) (781) 272-1966. 2018 Richards Medal Adress 2 e-mail: [email protected] _____________________________ NESACS Homepage: http://www.NESACS.org Rational Vaccinology: In Pursuit of the Perfect Vaccine Officers 2019 By Chad A. Mirkin, Northwestern University Chair Andrew Scholte How to Allocate Resources to Intellectual Property as a Start-Up 4 Sanofi-Genzyme 153 2nd Ave By Katherine Ann Rubino Waltham, MA 02451-1122 [email protected] Chair-Elect Monthly Meeting 5 Anna W. Sromek _______________________________________ 115 Mill Street, 2019 Esselen Award Meeting at Harvard Faculty Club, Carolyn R. Belmont, MA 02478 Bertozzi to receive award and speak on Chemical Approaches to Problems [email protected] Immediate Past Chair in Global Health Mindy Levine 35 Cottage St Gustavus John Esselen Award 8 Sharon, MA 02067-2130 _____________________________ (516)697-9688 List of Prior Recipients [email protected] Secretary Michael Singer 2019 NESACS Candidates for Election 9 MilliporeSigma _____________________ 400 Summit Drive, Burlington, MA 01803 (781)-229-7037, Business Directory 10 [email protected] ____________________________________ Treasurer Ashis Saha Seminar Calendar 12 67 Bow St _____________________________________ Arlington, MA 02474-2744 (978)212-5462 Cover: [email protected] 2019 Esselen Award recipient, Professor Carolyn R. Bertozzi, Stanford Archivist University (Photo Courtesy of Prof. Bertozzi). Ken Mattes Trustees Editorial Deadlines: Dorothy Phillips, Ruth Tanner, Peter C. Meltzer May 2019 Issue: March 22, 2019 Directors-at-Large Summer-September 2019 Issue: July 22, 2019 David Harris, June Lum, Michael P. Filosa, John M. Burke, James U. Piper, Ralph Scannell Councilors/Alternate Councilors Term Ends 12/31/2019 Thomas R. Gilbert Mary A. Mahaney Mary Jane Shultz Jerry P. Jasinski Michael Singer Matthew M. Jacobsen Lisa Marcaurelle Ajay Purohit Leland L. Johnson, Jr. Hicham Fenniri Term Ends 12/31/2020 Michael P. Filosa Sonja Strah-Pleynet Carol Mulrooney Patrick M. Gordon Patricia A. Mabrouk Patrick Cappillino The Nucleus is published monthly, except June and August, by the Northeastern Section of the American Anna Sromek Raj (SB) Rajur Chemical Society, Inc. Forms close for advertising on the 1st of the month of the preceding issue. Text Sofia A. Santos Ashis Saha must be received by the editor six weeks before the date of issue. Term Ends 12/31/2021 Editor: Michael P. Filosa, Ph.D., 18 Tamarack Road, Medfield, MA 02052 Email: Catherine E. Costello Kenneth Mattes Ruth Tanner Joshua Sacher [email protected]; Tel: 508-843-9070 Andrew Scholte Mariam Ismail Associate Editors: Myron S. Simon, 60 Seminary Ave. apt 272, Auburndale, MA 02466 June Lum Malika Jeffries-EL Morton Z. Hoffman, 23 Williams Rd., Norton, MA 02766 Morton Z. Hoffman Dajit Matharu Board of Publications: Ajay Purohit (Chair), Mary Mahaney, Ken Drew, Katherine Lee, Katherine Rubino All Chairs of standing Business Manager: Vacant: contact Michael Filosa at [email protected] Committees, the editor of THE NUCLEUS, and Advertising Manager: Vacant: contact Michael Filosa at [email protected] the Trustees of Section Calendar Coordinator: Samurdhi Wijesundera, Email: [email protected] Funds are members of the Photographers: Brian D’Amico, Morton Z. Hoffman Board of Directors. Any Coun cilor of the American Chemical Society Proofreaders: Donald O. Rickter, Morton Z. Hoffman residing within the section area is an ex officio Webmaster: Roy Hagen, Email: [email protected] member of the Board of Directors. Copyright 2019, Northeastern Section of the American Chemical Society, Inc. The Nucleus April 2019 3 How to Allocate NESACS Sponsors 2017 Platinum $5000+ Resources to Intellectual Boston Foundation Esselen Award SK Life Science Property as a Start-Up Amgen, Inc Johnson Matthey By Katherine Ann Rubino, NESACS Board of Publications, Patent Attorney, Cald- Vertex Pharmaceuticals well Intellectual Property Law Davos Pharma When a venture is first getting off the the to-do list as bigger issues need to be Biogen ground, there are a lot of items to be accomplished first, such as making sure PCI Synthesis considered. Delegating and protecting payroll will be met each week. In some Navin Fluorine International Ltd resources can be of utmost importance situations, a problem may arise when a Gold $3000 up to $5000 to ensure the future profitability and pro- friend, employee, or family member Merck Research Corp tection of a start-up.
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