Market Deep Dive Report CRISPR Therapeutics June 2020
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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 -
Tissue-Specific Delivery of CRISPR Therapeutics
International Journal of Molecular Sciences Review Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors Karim Shalaby 1 , Mustapha Aouida 1,* and Omar El-Agnaf 1,2,* 1 Division of Biological and Biomedical Sciences (BBS), College of Health & Life Sciences (CHLS), Hamad Bin Khalifa University (HBKU), Doha 34110, Qatar; [email protected] 2 Neurological Disorders Research Center, Qatar Biomedical Research Institute (QBRI), Hamad Bin Khalifa University (HBKU), Doha 34110, Qatar * Correspondence: [email protected] (M.A.); [email protected] (O.E.-A.) Received: 12 September 2020; Accepted: 27 September 2020; Published: 5 October 2020 Abstract: The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing system has been the focus of intense research in the last decade due to its superior ability to desirably target and edit DNA sequences. The applicability of the CRISPR-Cas system to in vivo genome editing has acquired substantial credit for a future in vivo gene-based therapeutic. Challenges such as targeting the wrong tissue, undesirable genetic mutations, or immunogenic responses, need to be tackled before CRISPR-Cas systems can be translated for clinical use. Hence, there is an evident gap in the field for a strategy to enhance the specificity of delivery of CRISPR-Cas gene editing systems for in vivo applications. Current approaches using viral vectors do not address these main challenges and, therefore, strategies to develop non-viral delivery systems are being explored. Peptide-based systems represent an attractive approach to developing gene-based therapeutics due to their specificity of targeting, scale-up potential, lack of an immunogenic response and resistance to proteolysis. -
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). -
CRISPR THERAPEUTICS AG (Exact Name of Company As Specified in Charter)
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): September 10, 2018 CRISPR THERAPEUTICS AG (Exact Name of Company as Specified in Charter) Switzerland 001-37923 Not Applicable (State or Other Jurisdiction (Commission (IRS Employer of Incorporation) File Number) Identification No.) Baarerstrasse 14 6300 Zug Switzerland +41 (0)41 561 32 77 (Address, Including Zip Code, and Telephone Number, Including Area Code, of Registrant’s Principal Executive Offices) 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 (see General Instruction A.2. below): ☐ 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)) 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). Emerging growth company ☒ If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. -
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. -
2020: Growth & Resilience in Regenerative Medicine
2020: Growth & Resilience in Regenerative Medicine 2020 ANNUAL REPORT Table of Contents The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies. ARM promotes legislative, regulatory and reimbursement ARM Annual Report initiatives to advance this innovative and transformative sector, which includes cell therapies, gene therapies and tissue-based therapies. 2 Letter from the CEO 24 ARM Advances Alongside Maturing Sector Early products to market have demonstrated profound, durable and potentially curative benefits that are already helping thousands of patients worldwide, many 4 Investor Perspective 25 Virtual Programming of whom have no other viable treatment options. Hundreds of additional product candidates contribute to a robust pipeline of potentially life-changing regenerative 5 Record Financings Bolster Maturing Sector 26 Capacity Building medicines and advanced therapies. 10 Real Life Miracles: The Price Family 26 What’s Next? In its 11-year history, ARM has become the global voice of the sector, representing the interests of 380+ members worldwide, including small and large companies, 12 academic research institutions, major medical centers and patient groups. The View From Science & Industry Affairs 27 Parting Letter From ARM Chair To learn more about ARM or to become a member, visit www.alliancerm.org. 14 Progressing Through the Pipeline 28 2021 ARM Officers & Board 19 The View From Public Affairs 30 ARM Staff 20 Europe Policy & Regulatory Update 32 Upcoming Events 2021 22 U.S. Policy & Regulatory Update 1 Letter from the CEO: Growth & Resilience hen we issued the Alliance for Regenerative Medicine (ARM) 2019 Annual Report a year Beyond the new approvals, there were extraordinary scientific accomplishments and ago, we never imagined what 2020 would throw at the world, the regenerative medicine recognitions. -
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. -
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. -
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). -
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. -
CRISPR Therapeutics AG Annual Report 2021
CRISPR Therapeutics AG Annual Report 2021 Form 10-K (NASDAQ:CRSP) Published: February 16th, 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 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 001-37923 CRISPR THERAPEUTICS AG (Exact name of Registrant as specified in its Charter) Switzerland Not Applicable (State or other jurisdiction of (I.R.S. Employer incorporation or organization) Identification No.) Baarerstrasse 14 6300 Zug, Switzerland Not Applicable (Address of principal executive offices) (Zip Code) Registrant’s telephone number, including area code: + 41 (0)41 561 32 77 Securities registered pursuant to Section 12(b) of the Act: Trading Title of each class Symbol(s) Name of each exchange on which registered Common Shares, nominal value CHF 0.03 CRSP 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 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. -
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.