Xconomyawardsprogram.Pdf
Total Page:16
File Type:pdf, Size:1020Kb
Load more
Recommended publications
-
Boston University Journal of Science & Technology
4 B.U. J. SCI. & TECH. L. 1 January 23, 1997 Boston University Journal of Science & Technology Law Symposium Financing the Biotech Industry: Can the Risks Be Reduced? Ronald Cass, Joshua Lerner, Farah H. Champsi, Stanley C. Erck, Jonathan R. Beckwith, Leslie E. Davis, Henri A. Termeer Table of Contents Speeches..........................................................................................................................[1] Dean Ronald Cass.............................................................................................[1] Joshua Lerner....................................................................................................[2] Farah Champsi..................................................................................................[8] Stanley Erck.....................................................................................................[18] Jonathan Beckwith.........................................................................................[24] Leslie Davis......................................................................................................[37] Henri Termeer.................................................................................................[47] Question and Answer Session..................................................................................[60] Financing the Biotech Industry: Can the Risks Be Reduced?† Jonathan R. Beckwith, Farah H. Champsi, Leslie E. Davis,* Stanley C. Erck, Joshua Lerner, Henri A. Termeer Dean Ronald Cass: 1. The biotechnology -
Schedule 14A
QuickLinks -- Click here to rapidly navigate through this document UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 SCHEDULE 14A Proxy Statement Pursuant to Section 14(a) of the Securities Exchange Act of 1934 (Amendment No. ) Filed by the Registrant ý Filed by a Party other than the Registrant o Check the appropriate box: o Preliminary Proxy Statement o Confidential, for Use of the Commission Only (as permitted by Rule 14a-6(e)(2)) ý Definitive Proxy Statement o Definitive Additional Materials o Soliciting Material under §240.14a-12 Verastem, Inc. (Name of Registrant as Specified In Its Charter) (Name of Person(s) Filing Proxy Statement, if other than the Registrant) Payment of Filing Fee (Check the appropriate box): ý No fee required. o Fee computed on table below per Exchange Act Rules 14a-6(i)(1) and 0-11. (1) Title of each class of securities to which transaction applies: (2) Aggregate number of securities to which transaction applies: (3) Per unit price or other underlying value of transaction computed pursuant to Exchange Act Rule 0-11 (set forth the amount on which the filing fee is calculated and state how it was determined): (4) Proposed maximum aggregate value of transaction: (5) Total fee paid: o Fee paid previously with preliminary materials. o Check box if any part of the fee is offset as provided by Exchange Act Rule 0-11(a)(2) and identify the filing for which the offsetting fee was paid previously. Identify the previous filing by registration statement number, or the Form or Schedule and the date of its filing. -
AVEO PHARMACEUTICALS, INC. (Name of Registrant As Specified in Its Charter)
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 SCHEDULE 14A (Rule 14a-101) INFORMATION REQUIRED IN PROXY STATEMENT SCHEDULE 14A INFORMATION Proxy Statement Pursuant to Section 14(a) of the Securities Exchange Act of 1934 Filed by the Registrant x Filed by a party other than the Registrant ¨ Check the appropriate box: ¨ Preliminary Proxy Statement ¨ Confidential, for Use of the Commission Only (as permitted by Rule 14a-6(e)(2)) x Definitive Proxy Statement ¨ Definitive Additional Materials ¨ Soliciting Material Pursuant to §240.14a-12 AVEO PHARMACEUTICALS, INC. (Name of Registrant as Specified In Its Charter) (Name of Person(s) Filing Proxy Statement, if Other Than The Registrant) Payment of Filing Fee (Check the appropriate box): x No fee required. ¨ Fee computed on table below per Exchange Act Rules 14a-6(i)(1) and 0-11. (1) Title of each class of securities to which transaction applies: (2) Aggregate number of securities to which transaction applies: (3) Per unit price or other underlying value of transaction computed pursuant to Exchange Act Rule 0-11 (set forth the amount on which the filing fee is calculated and state how it was determined): (4) Proposed maximum aggregate value of transaction: (5) Total fee paid: ¨ Fee paid previously with preliminary materials: ¨ Check box if any part of the fee is offset as provided by Exchange Act Rule 0-11(a)(2) and identify the filing for which the offsetting fee was paid previously. Identify the previous filing by registration statement number, or the Form or Schedule and the date of its filing. -
Proqr Therapeutics N.V
ANNUAL REPORT 2017 Meaningful progress - Excited about the future PAGE I PAGE II ANNUAL MAGAZINE 2017 ANNUAL MAGAZINE 2017 our courage to do things differently University of Colorado and during his ACCOMPLISHMENTS – and challenge the status quo. career has earned a global reputation A QUICK GLANCE We can and will make a meaningful for translating cutting-edge science impact – changing the lives of into transformational new therapies Eluforsen (formely known as QR-010) for CF patients. Be it in a small way – for rare diseases including cystic fibro- • Completed second trial for eluforsen by improving one’s quality of life. sis, pulmonary fibrosis, pulmonary • Announced positive data from Or in a big way – by stopping artery hypertension, severe immuno- Phase 1b safety and tolerability study a disease or by curing patients. logic, and inflammatory diseases. • Received FDA Fast Track designation and Orphan Drug Designation (ODD) CREATING MEANINGFUL IMPACT At ProQR, we do it all in the interest Dave is excited to be part of ProQR’s in US and Europe of patients. We’re driven, deter- management team: “ProQR has • Granted two key patents mined, excited to make this differ- a patient-centric culture committed ence. Today, tomorrow and more to transforming lives. Applying our QR-110 for LCA 10 days to come, until we get it right. RNA editing platform to precision • IND clearance from FDA and IN THE INTEREST medicine in hereditary forms of commenced Phase 1/2 safety and Building ProQR blindness, dystrophic epidermolysis efficacy trial In 2017, we made important strides bullosa, cystic fibrosis and other • First patient in trial dosed in late 2017 in ProQR’s growth. -
Orphan Drug Letters
November 18, 2010 Kent Lieginger Senior Vice President, Managed Care and Customer Operations Genentech 1 DNA Way South San Franciso, CA 94080 Dear Dr. Lieginger: We are writing on behalf of a broad coalition of hospitals participating in the federal 340B drug discount program. We have been advised that Genentech has begun to withhold 340B pricing for orphan drugs purchased by a subset of our membership, primarily children’s and rural hospitals. We are aware that, under the Health Care and Education Reconciliation Act (HCERA), Congress established a statutory exclusion with respect to orphan drugs purchased by hospitals specifically added by the Patient Protection and Affordable Care Act (PPACA) to the 340B program under the Public Health Service Act, including freestanding cancer hospitals, children’s hospitals, sole community rural referral centers, and critical access hospitals. 1 However, we are deeply concerned with Genentech’s broad interpretation of the orphan drug exclusion, which Genentech has stated extends to all drugs that have been designated “orphan” by the FDA, regardless of the intended use of the drugs. We believe that, absent guidance from the Health Resources and Services Administration (HRSA) regarding the scope of the orphan drug exclusion, it is premature to withhold 340B pricing on orphan drugs. Accordingly, we request that Genentech resume its sale of orphan drugs to hospitals identified in PPACA at 340B prices until directed otherwise by HRSA. We understand that several manufacturers have decided to delay implementation until they receive further guidance from the government and ask that you follow their lead. Our concerns regarding your company’s interpretation are two-fold. -
Ultragenyx Announces Emil D. Kakkis, M.D., Ph.D. As Recipient of BIO’S 2019 Henri A
Ultragenyx Announces Emil D. Kakkis, M.D., Ph.D. as Recipient of BIO’s 2019 Henri A. Termeer Biotechnology Visionary Award May 29, 2019 NOVATO, Calif., May 29, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that the Biotechnology Innovation Organization (BIO) has awarded this year’s Henri A. Termeer Biotechnology Visionary Award to Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer, President and founder of Ultragenyx. The award, which will be presented to Dr. Kakkis on June 5 during the BIO 2019 International Convention keynote session, recognizes Dr. Kakkis’ transformative work to accelerate research and development of novel treatments for rare diseases. “Henri Termeer was a pioneer who worked relentlessly to do what many thought was impossible and bring therapies to patients with rare diseases,” said Dr. Kakkis. “I am deeply honored to receive this award from BIO.” “Dr. Kakkis’ innovative work has pushed the boundaries of our knowledge in rare diseases and through his efforts our industry better prioritizes treatments for rare and ultra-rare diseases and understands the unique needs of clinical trials in this category,” said Jim Greenwood, President and CEO of BIO. “His contributions to the study of rare diseases have been monumental for patients and families, and we’re looking forward to presenting him with this well-deserved honor at BIO 2019.” Over the last 25 years, Dr. Kakkis has spearheaded the development of many rare disease treatments, including an enzyme replacement therapy for the rare and debilitating disorder, mucopolysaccharidosis type I (MPS I), which was approved by the FDA in 2003. -
Biotechnology Summit: Putting a Human Face on Biotechnology
17Rla S. HRG. 106-677 BIOTECHNOLOGY SUMMIT: PUTTING A HUMAN FACE ON BIOTECHNOLOGY HEARING before the JOINT ECONOMIC COMMITTEE CONGRESS OF THE UNITED STATES ONE HUNDRED SIXTH CONGRESS FIRST SESSION September 29, 1999 PRINTED FOR THE USE OF THE JOINT ECONOMIC COMMITFEE U.S. GOVERNMENT PRINTING OFFICE WASHINGTON: 2000 CC 62-990 FOR SALE BY THE U.S. GOVERNMENT PRINTING OFFICE SUPERNTENDENTOF DOCUMEN CONGRESSIONALSALESOFFICEWASHINGTON, D.C20402 JOINT ECONOMIC COMMITTEE [Created pursuant to Sec. 5(a) of Public Law 304, 79th Congress] SENATE HOUSE OF REPRESENTATIVES CONNIE MACK, Florida, Chairman JIM SAXTON, New Jersey, Vice Chairman WILLIAM V. ROTH, JR., Delaware MARK SANFORD, South Carolina ROBERT F. BENNETT, Utah JOHN DOOLITTLE, California ROD GRAMS, Minnesota TOM CAMPBELL, California SAM BROWNBACK, Kansas JOSEPH R. PITTS, Pennsylvania JEFF SESSIONS, Alabama PAUL RYAN, Wisconsin CHARLES S. ROBB, Virginia PETE STARK, California PAUL S. SARBANES, Maryland CAROLYN B. MALONEY, New York EDWARD M. KENNEDY, Massachusetts DAVID MINGE, Minnesota JEFF BINGAMAN, New Mexico MELVIN L. WATT, North Carolina SHELLEY S. HYMES, Executive Director JAMES D. GWARTNEY, Chief Economist HOWARD ROSEN, Minority Staff Director (ii) CONTENTS OPENING STATEMENTS OF MEMBERS Senator Connie Mack, Chairman ........ ..................... I Representative Pete Stark, Ranking Minority Member ..... ...... 4 Senator Bill Frist . ......................................... 6 Senator Edward M. Kennedy ............................... 31 Senator Robert F. Bennett .. .............................. 33 Representative David Minge ................................ 36 Senator Jeff Sessions .............. ....................... 39 WITNESSES PANELI Statement of Carolyn Boyer-Fortier, Breast Cancer Survivor. ...... 8 Statement of Dr. Steven Shak, Staff Scientist and Senior Director of Medical Affairs, Genentech ........................... 11 Statement of Joan London, Rheumatoid Arthritis Patient .... ..... 13 Statement of Dr. Robert Bunning, Director of the Arthritis Program at National Rehabilitation Hospital. -
How Scientist/Founders Lead Successful
HOW SCIENTIST/FOUNDERS LEAD SUCCESSFUL BIOPHARMACEUTICAL ORGANIZATIONS: A STUDY OF THREE COMPANIES Lynn Johnson Langer A DISSERTATION Submitted to the Ph.D. in Leadership & Change Program of Antioch University in partial fulfillment of the requirements for the degree of Doctor of Philosophy May, 2008 This is to certify that the dissertation entitled: HOW SCIENTIST/FOUNDERS LEAD SUCCESSFUL BIOPHARMACEUTICAL ORGANIZATIONS: A STUDY OF THREE COMPANIES prepared by Lynn Johnson Langer is approved in partial fulfillment of the requirements for the degree of Doctor of Philosophy in Leadership & Change. Approved by: ______________________________________________________________________ Alan E. Guskin, Ph.D., Chair date ______________________________________________________________________ Jon Wergin, Ph.D., Committee Member date ______________________________________________________________________ Mitch Kusy, Ph.D., Committee Member date ______________________________________________________________________ Alice Sapienza, DBA, External Reader date Copyright 2008 Lynn Johnson Langer All rights reserved Acknowledgements I arrive at this place in my journey to becoming a scholar supported in love and friendship by so many people. I must thank first and foremost my husband, Eric Langer, who has supported me in many ways, but particularly with my education. I thank my daughter, Julia Annemarie, my study partner. We shout across the hall to each other, complaining and laughing about all the work. I thank her for her support and the opportunity to be a role model. She never once complained about the plays and events I missed because I was away at a residency. I thank my son, Adam and his wife, Megan who through their lives, remind me to follow my dreams. I thank my son, Benjamin, who shows me that through hard work, we can accomplish great things. -
Conference Day One Thursday, April 23Rd 2015 – Main Conference Room
Conference Day One Thursday, April 23rd 2015 – Main Conference Room 8.55 Chairperson’s Opening Remarks Hans Schikan, Former Chief Executive Officer, Prosensa 9.00 Keynote: Data driving diagnostics - Clinically relevant information from ordinary photos, and an algorithm to aid in diagnoses of rare diseases Christoffer Nellaker, Research Fellow, Medical Research Foundation’s Functional Genomics Unit, Oxford University 9.30 Keynote: Is the market sustainable? Henri Termeer, Former Chairman, President and CEO, Genzyme (on video) 10.00 Speed Networking & Networking coffee break 11.00 Good medicine is good business: Transforming lives and meeting unmet needs Stephanie Okey, Senior Vice President and Head of North America, Genetic Diseases and US General Manager, Genzyme 11.20 The Great Debate: “The Price is Too Damn High!”…or is it? FOR: The orphan population is so small the rest of the population can more than cover the cost Ram Selvaraju, Managing Director, Equity Research – Healthcare, MLV & Co. AGAINST: First there was rare, now there’s ultra-rare. And it seems every other disease is rare and every other drug orphan. WE won’t be able to afford it – period. Ed Pazalla, VP, National Medical Director for Pharmacy Policy and Strategy, Aetna 12.00 Networking Lunch 1:00 Lunch session: Pediatric vouchers to expedite rare disease treatments in children Gayatri Rao, Director, Office of Orphan Product Development, FDA 1.30 2 x 40-minute roundtables with a 10 minute break in-between. Please sign up in the exhibition hall lounge. 1: How does miscommunication -
Gene Editing Workshop
Gene Editing Workshop 21st Annual Meeting ASGCT CHICAGO, ILLINOIS 2018 MAY 16-19 May 15, 2018 Hilton Chicago Continental C Chicago Gene Editing Workshop Table of Contents Workshop Supporters . p 2 Committee Listing . p 3 Faculty Listing . p 3 Faculty Bios . p 4 Disclosure of Relevant Financial Relationships . p 8 Program Schedule . p 9 Notes . p 11 American Society of Gene & Cell Therapy 1 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Gene Editing Workshop Supporters The American Society of Gene & Cell Therapy is honored to acknowledge the following organizations for their support of the Gene Editing Workshop: American Society of Gene & Cell Therapy 2 May 15, 2018 Chicago Hilton Chicago Gene Editing Workshop Committee Listing Co-Chairs J. Keith Joung, MD, PhD Matthew H. Porteus, MD, PhD Massachusetts General Hospital Stanford University School of Medicine Charlestown, MA Stanford, CA Members Paula M. Cannon, PhD Toni Cathomen, PhD Charles A. Gersbach, PhD University of Southern California Medical Center - University of Freiburg Duke University Los Angeles, CA Freiburg, Germany Durham, NC Faculty Listing Omar Abudayyeh Beverly L. Davidson, PhD J. Keith Joung, MD, PhD Broad Institute of MIT and Harvard Children’s Hospital of Philadelphia Massachusetts General Hospital Cambridge, MA Philadelphia, PA Charlestown, MA Charlie Albright, PhD Suk See De Ravin, MD, PhD Alexis C. Komor, PhD Editas Medicine National Institutes of Health, NIAID UC San Diego Cambridge, MA Bethesda, MD La Jolla, CA Leonela Amoasii, PhD Daniel Dever, PhD Vikram Pattanayak, MD, PhD UTSW Medical Center Stanford University Medical Center Massachusetts General Hospital Dallas, TX Stanford, CA Boston, MA Thomas Barnes, PhD Justin Eyquem, PhD Krishanu Saha, PhD Intellia Therapeutics Memorial Sloan Kettering Cancer Center University of Wisconsin-Madison Cambridge, MA New York, NY Madison, WI Mark A. -
Market Deep Dive Report CRISPR Therapeutics June 2020
Market Deep Dive Report CRISPR Therapeutics June 2020 Contents 1. Summary 3 2. Market Overview 3 2.1 Industry Challenges 4 2.2 Intellectual Property 5 2.3 Regulatory Requirements 5 2.4 Pitchbook Market Statistics 6 3. Technology Overview 6 3.1 Basic Summary 6 3.2 Base Editing 7 3.3 Prime Editing 8 3.4 Ex-Vivo CRISPR 9 3.5 In-Vivo CRISPR 10 3.6 Alternative techniques and platforms 11 3.7 Late stage privates & publics 13 3.8 Key People and Labs 13 4. Therapeutic Landscape 14 4.1 Present day status 14 4.2 Future Indications 16 4.3 Startups to Watch 17 5. Conclusions 18 5.1 Vertical Strengths 18 5.2 Vertical Weaknesses 18 5.3 Opportunity Cost of Capital 19 5.4 Investment Theses 19 6. References 20 2 1. Summary Since its initial discovery as a genetic reprogramming tool in 2012, CRISPR has widely been regarded as a breakthrough scientific discovery and quickly become one of the hottest new biotechnologies in the industry. However, the CRISPR therapeutics market is still in early stages, with leading therapies only just reaching human clinical trials. Furthermore, the industry is highly defended by IP, and currently addresses a narrow set of indications. Top companies including Beam Therapeutics, Editas Medicine, and Prime Medicine have been founded by the same scientists, and have formed a highly collaborative moat. Remaining companies have partnered with large pharmaceutical companies to fund operations. The future of CRISPR therapeutics development is dependent on technological advances in delivery methods and editing safety and granularity. -
Editas Medicine Announces Second Quarter 2019 Results and Update
Editas Medicine Announces Second Quarter 2019 Results and Update August 6, 2019 Cynthia (Cindy) Collins named President and Chief Executive Officer Initiated Brilliance Phase 1/2 clinical trial of EDIT-101(AGN-151587) for LCA10 Patient dosing in Brilliance trial on track for 2H19 EDIT-301 for hemoglobinopathies pre-clinical in vivo data to be presented by YE19 Cash, cash equivalents, and marketable securities of $317.9 million as of June 30, 2019 CAMBRIDGE, Mass., Aug. 06, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the second quarter of 2019. "I am pleased and honored to be appointed CEO at this exciting time for the company," said Cynthia Collins, President and Chief Executive Officer of Editas Medicine. “Our team is making history with enrollment underway and patient dosing anticipated in the first ever clinical trial of an in vivo CRISPR medicine. As we enter this new phase in our company’s development, we are also advancing our broader pipeline of in vivo CRISPR medicines, including our Usher syndrome program, as well as engineered cell medicines for hemoglobinopathies and cancers.” Recent Achievements and Outlook In Vivo CRISPR Medicines EDIT-101 for LCA10 Patient screening initiated with dosing planned for 2H19 Editas Medicine and its partner, Allergan, expect to enroll approximately 18 patients, aged 3 years and above, in the Brilliance Phase 1/2 clinical trial. The Brilliance clinical trial is a multi-center, open label, dose escalation study to evaluate the safety, tolerability, and efficacy of EDIT-101 as a treatment for Leber congenital amaurosis 10 (LCA10).