DOCSLIB.ORG

INTELLIA THERAPEUTICS, INC. (Exact Name of Registrant As Specified in Its Charter)

Total Page：16

File Type：pdf, Size：1020Kb

Download full-text PDF Read full-text
INTELLIA THERAPEUTICS, INC. (Exact Name of Registrant As Specified in Its Charter) Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2016 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-37766 INTELLIA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 36-4785571 (State or other jurisdiction of (I.R.S. Employer incorporation or organization) Identification No.) 130 Brookline Street, Suite 201, Cambridge, Massachusetts 02139 (Address of principal executive offices) (Zip code) 857-285-6200 (Registrant’s telephone number, including area code) Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐ Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files). Yes ☒ No ☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. See definitions of “accelerated filer,” “large accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act. (Check one): Large accelerated filer ☐ Accelerated filer ☐ Non-accelerated filer ☒ (Do not check if a smaller reporting company) Smaller reporting company ☐ Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒ The number of shares outstanding of the registrant’s common stock as of October 28, 2016: 35,995,331 shares. Table of Contents PART I—FINANCIAL INFORMATION Item 1. Financial Statements. Consolidated Balance Sheets as of September 30, 2016 and December 31, 2015 3 Consolidated Statements of Operations for the Three and Nine Months Ended September 30, 2016 and 2015 4 Consolidated Statement of Stockholders’ Equity (Deficit) for the Nine Months Ended September 30, 2016 5 Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2016 and 2015 6 Notes to Consolidated Financial Statements 7 Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations 16 Item 3. Quantitative and Qualitative Disclosures About Market Risk 25 Item 4. Controls and Procedures. 25 PART II—OTHER INFORMATION Item 1A. Risk Factors 25 Item 2. Unregistered Sales of Equity Securities and Use of Proceeds 58 Item 6. Exhibits 59 Signatures 60 2 Table of Contents PART I—FINANCIAL INFORMATION Item 1. Financial Statements INTELLIA THERAPEUTICS, INC. CONSOLIDATED BALANCE SHEETS (UNAUDITED) (Amounts in thousands except share and per share data) September 30, December 31, 2016 2015 ASSETS Current Assets: Cash and cash equivalents $ 290,618 $ 75,816 Accounts receivable 1,112 1,000 Prepaid expenses and other current assets 972 810 Total current assets 292,702 77,626 Property and equipment, net 5,492 2,708 Other assets 3,275 1,805 Total Assets $ 301,469 $ 82,139 LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT) Current Liabilities: Accounts payable $ 657 $ 1,360 Accrued expenses 4,810 2,788 Current portion of deferred revenue 17,166 6,547 Total current liabilities 22,633 10,695 Deferred revenue, net of current portion 60,406 3,765 Other long-term liabilities 306 323 Commitments and contingencies (Note 6) Convertible preferred stock (Series B, Series A-2, Series A-1, Junior and Founder), $0.0001 par value; 5,000,000 shares and 36,500,000 shares authorized, respectively; 0 shares and 36,316,628 shares issued and outstanding, respectively — 88,557 Stockholders’ Equity (Deficit): Common stock, $0.0001 par value; 120,000,000 shares and 50,000,000 shares authorized, respectively; 35,995,074 shares issued and outstanding and 2,558,755 shares issued and outstanding, respectively 4 — Additional paid-in capital 261,127 735 Accumulated deficit (43,007) (21,936) Total stockholders’ equity (deficit) 218,124 (21,201) Total Liabilities and Stockholders’ Equity (Deficit) $ 301,469 $ 82,139 See notes to consolidated financial statements. 3 Table of Contents INTELLIA THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF OPERATIONS (UNAUDITED) (Amounts in thousands except per share data) Three Months Ended Nine Months Ended September 30, September 30, 2016 2015 2016 2015 Collaboration revenue $ 4,869 $ 1,688 $ 10,852 $ 4,351 Operating expenses: Research and development 7,861 3,458 20,509 6,795 General and administrative 4,705 1,531 11,680 5,474 Total operating expenses 12,566 4,989 32,189 12,269 Operating loss (7,697) (3,301) (21,337) (7,918) Interest income 215 — 266 — Loss before income taxes (7,482) (3,301) (21,071) (7,918) Income tax benefit — 282 — 766 Net loss $ (7,482) $(3,019) $(21,071) $ (7,152) Net loss per share attributable to common stockholders, basic and diluted $ (0.22) $(10.27) $ (1.16) $ (48.65) Weighted average shares outstanding, basic and diluted 34,316 294 18,098 147 See notes to consolidated financial statements. 4 Table of Contents INTELLIA THERAPEUTICS, INC. CONSOLIDATED STATEMENT OF STOCKHOLDERS’ EQUITY (DEFICIT) (UNAUDITED) (Amounts in thousands except share data) Total Additional Stockholders’ Common Convertible Preferred Paid-In Accumulated Equity Shares Amount Capital Deficit (Deficit) Shares Amount Balance at December 31, 2015 2,558,755 $ — $ 735 $ (21,936) $ (21,201) 36,316,628 $ 88,557 Conversion of convertible preferred stock 23,481,956 3 88,554 — 88,557 (36,316,628) (88,557) Issuance of common stock, net of issuance costs of $3,365 9,955,554 1 167,138 — 167,139 — — Equity-based compensation (1,191) — 4,700 — 4,700 — — Net loss — — — (21,071) (21,071) — — Balance at September 30, 2016 35,995,074 $ 4 $261,127 $ (43,007) $ 218,124 — $ — See notes to consolidated financial statements. 5 Table of Contents INTELLIA THERAPEUTICS, INC. CONSOLIDATED STATEMENTS OF CASH FLOWS (UNAUDITED) (Amounts in thousands) Nine Months Ended September 30, 2016 2015 CASH FLOWS FROM OPERATING ACTIVITIES: Net loss $ (21,071) $ (7,152) Adjustments to reconcile net loss to net cash provided by operating activities: Depreciation and amortization 699 196 Loss on disposal of property and equipment 2 7 Equity-based compensation 4,700 845 Benefit from intraperiod tax allocation — (766) Changes in operating assets and liabilities: Accounts receivable (112) — Prepaid expenses and other current assets (162) (360) Accounts payable 154 405 Accrued expenses 1,741 1,220 Deferred revenue 67,260 10,004 Other assets (2,675) (841) Other long-term liabilities (17) 170 Net cash provided by operating activities 50,519 3,728 CASH FLOWS FROM INVESTING ACTIVITIES: Purchases of property and equipment (2,760) (1,705) Net cash used in investing activities (2,760) (1,705) CASH FLOWS FROM FINANCING ACTIVITIES: Proceeds from sale of Class A-2 preferred units and Series B preferred stock — 74,661 Payments to acquire in-process research and development (600) (900) Payment of preferred unit and preferred stock issuance costs (100) (2,622) Proceeds from common stock offering 170,507 — Payment of common stock offering costs (2,764) (395) Net cash provided by financing activities 167,043 70,744 Net increase in cash and cash equivalents 214,802 72,767 Cash and cash equivalents, beginning of period 75,816 9,845 Cash and cash equivalents, end of period $ 290,618 $ 82,612 SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION: Purchases of property and equipment unpaid at period end $ 944 $ 144 Acquisition of in-process research and development unpaid at period end — 800 Financing costs incurred but unpaid at period end — 792 See notes to consolidated financial statements. 6 Table of Contents INTELLIA THERAPEUTICS, INC. NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (UNAUDITED) 1. Overview and Basis of Presentation Intellia Therapeutics, Inc. (collectively referred to with its wholly-owned, controlled subsidiary, Intellia Securities Corp., as “Intellia” or the “Company”) is a genome editing company focused on developing potentially curative therapeutics utilizing a biological tool known as CRISPR/Cas9. The consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States of America have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Prospectus that forms a part of the Company’s Registration Statement on Form S-1 (File No. 333-210689), which was filed with the SEC pursuant to Rule 424(b)(4) on May 5, 2016 (the “Prospectus”). The unaudited consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its subsidiary. All intercompany transactions and balances of the subsidiary have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure.
Load more

Recommended publications
  • ARK GENOMIC REVOLUTION MULTI SECTOR ETF (ARKG) HOLDINGS As of 09/27/2021
    ARK GENOMIC REVOLUTION MULTI SECTOR ETF (ARKG) HOLDINGS As of 09/27/2021 Company Ticker CUSIP Shares Market Value($) Weight(%) 1 TELADOC HEALTH INC TDOC 87918A105 3,937,797 531,208,815.30 6.99 2 EXACT SCIENCES CORP EXAS 30063P105 3,971,013 381,296,668.26 5.01 3 PACIFIC BIOSCIENCES OF CALIF PACB 69404D108 13,696,148 350,347,465.84 4.61 4 VERTEX PHARMACEUTICALS INC VRTX 92532F100 1,722,281 316,228,014.41 4.16 5 FATE THERAPEUTICS INC FATE 31189P102 4,825,395 312,926,865.75 4.12 6 IONIS PHARMACEUTICALS INC IONS 462222100 8,572,965 310,341,333.00 4.08 7 REGENERON PHARMACEUTICALS REGN 75886F107 430,742 275,201,063.80 3.62 8 TWIST BIOSCIENCE CORP TWST 90184D100 2,237,350 250,829,308.50 3.30 9 TAKEDA PHARMACEUTIC-SP ADR TAK UN 874060205 13,592,076 229,570,163.64 3.02 10 ACCOLADE INC ACCD 00437E102 5,268,242 226,850,500.52 2.98 11 INTELLIA THERAPEUTICS INC NTLA 45826J105 1,508,421 224,965,907.94 2.96 12 VEEVA SYSTEMS INC-CLASS A VEEV 922475108 741,198 222,307,516.14 2.92 13 CAREDX INC CDNA 14167L103 3,433,475 220,978,451.00 2.91 14 CRISPR THERAPEUTICS AG CRSP H17182108 1,804,041 210,044,493.63 2.76 15 INCYTE CORP INCY 45337C102 2,893,385 199,643,565.00 2.63 16 INVITAE CORP NVTA 46185L103 6,059,066 182,135,523.96 2.40 17 ADAPTIVE BIOTECHNOLOGIES ADPT 00650F109 4,888,391 178,377,387.59 2.35 18 BEAM THERAPEUTICS INC BEAM 07373V105 1,849,698 175,110,909.66 2.30 19 SIGNIFY HEALTH INC -CLASS A SGFY 82671G100 8,107,683 160,937,507.55 2.12 20 UIPATH INC - CLASS A PATH 90364P105 2,955,628 155,761,595.60 2.05 21 CASTLE BIOSCIENCES INC CSTL 14843C105 2,130,211
    [Show full text]
  • United States Securities and Exchange Commission Washington, D.C
    UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): May 30, 2020 INTELLIA THERAPEUTICS, INC. (Exact name of Registrant as Specified in Its Charter) Delaware 001-37766 36-4785571 (State or Other Jurisdiction (Commission (IRS Employer of Incorporation) File Number) Identification No.) 40 Erie Street, Suite 130 Cambridge, Massachusetts 02139 (Address of Principal Executive Offices) (Zip Code) Registrant’s Telephone Number, Including Area Code: (857) 285-6200 Not Applicable (Former Name or Former Address, if Changed Since Last Report) Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions: ☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) ☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) ☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) ☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) Securities registered pursuant to Section 12(b) of the Act: Trading Name of each exchange Title of each class Symbol(s) on which registered Common Stock (Par Value $0.0001) NTLA The Nasdaq Global Market Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
    [Show full text]
  • Questions About CRISPR
    CRISPR Questions about CRISPR A huge industry will grow from its innovation, but the IP environment around CRISPR is far from established. Marc Döring and Daniel Lim investigate The discovery of the potential of CRISPR and its first Cas system for various applications. characterisation in the form of the CRISPR/Cas9 system One group comprises Intellia, Caribou Biosciences (Caribou), represents a true revolution in the life sciences field. It is best Caribou founder Jennifer Doudna and the University of California, known as a method that allows for the accurate and targeted cutting Berkeley (UCB). That group is broadly aligned with CRISPR Tx, ERS of DNA to enable editing of genes, but variants can accomplish a Genomics and its founder Emmanuelle Charpentier, who collaborated wide range of other extremely useful effects, including modulation of with Doudna and co-owner of UCB’s foundational CRISPR patents. On gene expression. The power and versatility of the system has opened 16 December 2016, the Doudna and Charpentier camps formalised the door to advances in a wide range of industries, including human their alliance, signing a global cross-licensing and patent prosecution therapeutics, animal health, agriculture and bioindustry. CRISPR has the co-operation agreement. potential to revolutionise any industry in which molecular biology and Opposing them is Editas, its founder Feng Zhang and the Broad genetic technology play a part. Institute of MIT and Harvard (the Broad), who hold a separate, However, despite the clear potential of the technology, and leaving competing portfolio of foundational CRISPR/Cas9 patents. aside the significant amount of research and development still to be These spin-out companies have wasted no time in partnering undertaken, question marks linger over the legal and regulatory position variously with big pharma, venture capitalists and fellow disruptive of CRISPR technology and CRISPR-based products.
    [Show full text]
  • 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.
    [Show full text]
  • 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.
    [Show full text]
  • Intellia Therapeutics Annual Report 2021
    Intellia Therapeutics Annual Report 2021 Form 10-K (NASDAQ:NTLA) Published: February 26th, 2021 PDF generated by stocklight.com UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2020 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number 001-37766 INTELLIA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 36-4785571 (State or other jurisdiction of (I.R.S. Employer incorporation or organization) Identification No.) 40 Erie Street, Suite 130 Cambridge, Massachusetts 02139 (Address of principal executive offices) (Zip Code) (857) 285-6200 (Registrant’s telephone number, including area code) Securities registered pursuant to Section 12(b) of the Act: Title of each Class Trade Symbol(s) Name of each exchange on which registered Common Stock, par value $0.0001 per share NTLA The Nasdaq Global Market Securities registered pursuant to Section 12(g) of the Act: None Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes ☒ No ☐ Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. Yes ☐ No ☒ Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.
    [Show full text]
  • 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).
    [Show full text]
  • News & Analysis
    NEWS & ANALYSIS NEWS IN BRIEF Intellia Therapeutics and partner Alzheimer prevention failure rattles field, anew Regeneron are working towards a 2020 investigational new drug application for Failed trials of amyloid- modulating Alzheimer disease candidates don’t come as much of a their in vivo NTLA-2001, a systemically surprise any more. But with Amgen and Novartis’s recent decision to halt two phase III trials delivered CRISPR treatment for transthyretin of the BACE1 inhibitor umibecestat for secondary prevention of the neurodegenerative amyloidosis. disease in high-risk patients, a longer shadow has now fallen on the prospects for these Asher Mullard agents in earlier lines of treatment as well. In the Generation S1 trial, the sponsors were testing umibecestat and the amyloid vaccine CAD106 in cognitively unimpaired individuals aged 60 to 75 years with two APOE4 genes, a risk factor for developing Alzheimer disease. In Generation S2, they were testing Longer- lasting HIV drugs umibecestat in cognitively unimpaired participants aged 60 to 75 years with at least one Antiretroviral therapies have revolutionized APOE4 allele. But in July the companies announced that the drug was associated with AIDS care, changing what was once a death worsening in some measures of cognitive function, and discontinued development of the sentence into a chronic and controllable BACE inhibitor. condition. But antiretroviral therapy (ART) After past failures of amyloid-modulating drugs, researchers have posited that these cocktails still have to be taken on a daily agents need to be used earlier in the course of disease — potentially years before symptoms basis, and treatment interruptions can result are detectable — to prevent neuronal death.
    [Show full text]
  • Innovative Startups 2013
    DATA PAGE Innovative startups 2013 Brady Huggett S firms, particularly companies from the Boston area, continue the Children’s Hospital of Philadelphia’s decision to support a startup Uto dominate Nature Biotechnology’s listing of innovative startups, is notable. In total, the United States accounts for about two-thirds of ranked in order of the 10 largest A rounds. This year’s list is notable firms receiving biotech A rounds in the broader life science field (Fig. 1); for the proportion of startups focusing on experimental gene thera- the United Kingdom ranks a very distant second. Although relatively pies; indeed, firms developing adeno-associated virus (AAV) platforms few biotechs in France and Switzerland received A rounds, companies garnered three of last year’s largest A rounds. A familiar group of early in those countries pulled in more on average than companies in the stage funds participated in most of the financings in Table 1, although United States (Table 2). Table 1 Top 10 A rounds in 2013 for innovative startups Company Amount raised ($M); date; investors Scientific founders Other Technology Juno $120; Dec. 4; Arch Venture Renier Brentjens, Memorial Sloan-Kettering Cancer Hans Bishop, Juno CEO and former Engineered chimeric antigen Therapeutics Partners, Crestline Investors Center (MSKCC); Phil Greenberg, Fred Hutchinson executive in residence at Warburg receptor adoptive T cell therapy (Seattle) Cancer Research Center (FHCRC) and the University of Pincus; Larry Corey, president and direc- against cancer Washington; Michael Jensen, University of Washington tor of FHCRC; Richard Klausner, senior School of Medicine; Stan Riddell, FHCRC and the vice president and chief medical officer University of Washington School of Medicine; of Illumina, chairman of Audax Health; Isabelle Rivière and Michel Sadelain, MSKCC Robert Nelsen, managing director of ARCH Venture Partners Spark $50; Oct.
    [Show full text]
  • Editas Medicine Announces Third Quarter 2020 Results and Update
    Editas Medicine Announces Third Quarter 2020 Results and Update November 5, 2020 EDIT-101 for LCA10 ‒ BRILLIANCE trial adult low-dose cohort completed EDIT-301 for sickle cell disease ‒ on track for IND filing by end of 2020 EDIT-201 for solid tumors ‒ preclinical data to be presented at SITC and ASH Cash, cash equivalents, and marketable securities of $541 million as of September 30, 2020 CAMBRIDGE, Mass., Nov. 05, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2020. “We were pleased to regain full operating control of our ocular programs per our new agreement with AbbVie, which provides us with important flexibility. We have finished dosing the first cohort with EDIT-101 in the landmark BRILLIANCE trial and enrollment remains active,” said Cynthia Collins, Chief Executive Officer of Editas Medicine. Ms. Collins continued, “The advancements in our ocular portfolio are complemented by the strategic development of our engineered cell medicines. We are on track to file the IND for EDIT-301 for sickle cell disease in the fourth quarter and are eager to present additional data on this potentially best-in-class medicine and our large-scale manufacturing process at the upcoming American Society of Hematology Annual Meeting. Substantial progress has also been made in the development of EDIT-201, an allogeneic NK cell medicine to treat solid tumors. Preclinical data to be presented at ASH show enhanced tumor killing with EDIT-201 compared to unedited NK cells, and additional data on this program will be featured at the upcoming Society for Immunotherapy of Cancer’s Annual Meeting.” Recent Achievements and Outlook In Vivo CRISPR Medicines EDIT-101 for LCA10 BRILLIANCE Phase1/2 adult low-dose cohort completed Editas Medicine (Company) has completed dosing of the first cohort of adult patients with visual light perception only administered the low dose of EDIT-101.
    [Show full text]
  • CRISPR Genome-Editing
    CRISPR Genome-Editing The Value of CRISPR’s IP Published: August 17, 2018 Author Manisha Samy, Analyst at ARK Invest Join the conversation on Twitter @ARKinvest www.ark-invest.com The Value of CRISPR’s Intellectual Property Manisha Samy THE VALUE OF INTELLECTUAL PROPERTY Intellectual property (IP) can be critical not only to commercial success but also to platform strategies upon which more innovation can take place. A strong patent portfolio might not guarantee success, but it can serve as a strategic asset, particularly in biotech. Patents provide a company with 20 years of exclusivity to advance innovations before others can compete. In the meantime, these companies receive licensing fees and/or royalty payments, but perhaps more importantly, attract talent seeking unfettered access to cutting edge technology. Genentech is a prime example of the benefits of a strong IP position in the field of health care. In 1980, Genentech went public, largely on the strength of its IP in recombinant DNA technology.1 Attracting some of biotech’s brightest minds, it commercialized synthetic insulin two years later, four years before Roche took a majority interest. Genentech’s stock scaled 11-fold at a 47% compound annual rate in the six years between its initial public offering (IPO) and Roche taking majority interest. CRISPR’S IP LANDSCAPE While Genentech benefited from a patent claim that was unambiguous, the CRISPR IP landscape is much more convoluted. In a high-profile patent battle between the University of California (UC), Berkeley and the Broad Institute, scientists are fighting for commercial claims to the “foundational” CRISPR IP.
    [Show full text]
  • CRISPR Therapeutics, Intellia Therapeutics and Caribou Biosciences Provide Update on U.S Federal Circuit Decision Upholding the Ruling by U.S
    CRISPR Therapeutics, Intellia Therapeutics and Caribou Biosciences Provide Update on U.S Federal Circuit Decision Upholding the Ruling by U.S. Patent and Trademark Office in Interference Proceeding Relating to CRISPR/Cas9 Genome Editing Technology The interference proceeding remains terminated without any determination on inventorship of CRISPR/Cas9 genome editing application to eukaryotic cells University of California/University of Vienna/Dr. Charpentier have patent applications covering the use of CRISPR/Cas9 genome editing technology in all environments that do not interfere with the Broad’s patent claims in this proceeding Additional legal paths are available to recognize the priority of the University of California/University of Vienna/Charpentier intellectual property covering eukaryotic cells University of California/University of Vienna/Dr. Charpentier were recently issued a U.S. patent for the use of CRISPR/Cas9 genome editing that covers guide formats widely used across the human therapeutic and agricultural industries ZUG, Switzerland and CAMBRIDGE, Mass. and BERKELEY, Calif., Sept. 10, 2018 (GLOBE NEWSWIRE) -- CRISPR Therapeutics AG (NASDAQ:CRSP), Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Caribou Biosciences, Inc. announced that the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”) affirmed the decision by the U.S. Patent and Trademark Office’s (“USPTO”) Patent Trial and Appeal Board (“PTAB”) in an interference proceeding relating to CRISPR/Cas9 genome editing technology. The interference was requested by the Regents of the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively “UC”), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome engineering, against the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively “Broad”).
    [Show full text]