CRISPR THERAPEUTICS AG (Exact Name of Company As Specified in Charter)
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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. -
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. -
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. -
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. -
Uncertainty, Institutions and Regulatory Responses to Emerging Technologies: CRISPR Gene Editing in the US and the EU (2012–2019)
Regulation & Governance (2020) doi:10.1111/rego.12335 Uncertainty, institutions and regulatory responses to emerging technologies: CRISPR Gene editing in the US and the EU (2012–2019) Alberto Asquer Senior Lecturer in Public Policy and Management, School of Finance and Management, SOAS University of London, London, UK Inna Krachkovskaya Research Associate, School of Finance and Management, SOAS University of London, London, UK Abstract This study aims to improve theoretical accounts of regulatory responses to emerging technologies by proposing a model of regulatory development, which incorporates a role for types of uncertainty and for existing regulatory institutions. Differently from existing theories of regulatory development, the model proposed here posits a sequence of cyclical activities where regu- latory responses arise in incremental fashion out of efforts to make sense of emerging technologies and to ponder the applica- bility of existing regulatory tools. The model is discussed on the basis of the comparison between regulatory responses to the emergence of CRISPR gene editing in the US and the EU in the period 2012–2019. The comparison between the two cases suggests how regulatory responses to emerging technologies are affected by expectations of future technological and regulatory developments and by existing regulatory institutions. Keywords: CRISPR, emerging technology, regulatory institution, uncertainty. 1. Introduction The increased pace of scientific discoveries and technological advances of the last decades poses novel challenges for regulators. Emerging technologies - here defined as new technologies characterized by radical novelty, rela- tively fast growth, coherence, prominent impact, and uncertainty (Rotolo et al. 2015) –pose new sort of dangers and hazards to individuals and to the natural environment, build on mechanisms whose underlying cause-effect relationships are not fully understood, and lend themselves – as “technological platforms”–to a plethora of pos- sible uses (Levi-Faur & Comaneshter 2007; Brownsword 2008; Hodge et al. -
Investigating the Role of Microglia in Myelin Development by Liang Li B.Eng
Investigating the role of microglia in myelin development By Liang Li B.Eng. Chemical and Biomolecular Engineering National University of Singapore, 2014 Submitted to the Institute for Data, Systems, and Society in partial fulfillment of the requirements for the degree of Master of Science in Technology and Policy and Master of Science in Electrical Engineering and Computer Science at the MASSACHUSETTS INSTITUTE OF TECHNOLOGY February 2021 © Massachusetts Institute of Technology 2021. All rights reserved. Author . Institute for Data, Systems, and Society January 27, 2021 Certified by. .. Guoping Feng Poitras Chair Professor of Neuroscience McGovern Institute for Brain Research Thesis Supervisor Certified by. .. Thomas Heldt Associate Professor of Electrical and Biomedical Engineering, Department of Electrical Engineering & Computer Science Thesis Reader Accepted by . Noelle Eckley Selin Director, Technology and Policy Program Associate Professor, Institute for Data, Systems, and Society and Department of Earth, Atmospheric and Planetary Sciences Accepted by . Leslie A. Kolodziejski Professor of Electrical Engineering and Computer Science Chair, Department Committee on Graduate Students Investigating the role of microglia in myelin development By Liang Li B.Eng. Chemical and Biomolecular Engineering National University of Singapore, 2014 Submitted to the Institute for Data, Systems, and Society in partial fulfillment of the requirements for the degree of Master of Science in Technology and Policy and Master of Science in Electrical Engineering and Computer Science at the MASSACHUSETTS INSTITUTE OF TECHNOLOGY February 2021 © Massachusetts Institute of Technology 2021. All rights reserved. Abstract We investigate the role of microglia in myelin development. Myelination is an essential process in early development that consists of the ensheathment of axons by myelin, which ensures rapid conduction of action potentials. -
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”). -
Transformative Gene-Based Medicines for Serious Human Diseases
ANNUAL REPORT 2018 Transformative Gene-Based Medicines For Serious Human Diseases CRISPR Therapeutics AG Baarerstrasse 14 6300 Zug Switzerland Confidential 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, 2018 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: Common shares, nominal value CHF 0.03 per share The Nasdaq Global Market Title of each class Name of each exchange on which registered 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.