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1 What is CRISPR? A guide to what CRISPR is and its far-reaching applications in healthcare, agriculture, & more. 2 The presentation will also be sent to you. Feel free to share with colleagues. The resolution of some slides may be suboptimal due to the webinar software. Those slides will look fine in the presentation that we send you. Briefing recording will be distributed http://support.citrixonline.com/en_US/Webinar W I T H I N 2 4 H O U R S 3 @cbinsights @ja_lee2 #WhatIsCRISPR J O I N T H E C O N V E R S A T I O N O N T W I T T E R 4 W H O W E A R E The technology market intelligence platform. CB Insights software lets you predict, discuss, and communicate emerging technology trends using data in ways that are beyond human cognition. We are a leader in the Expert Automation & Augmentation Software (EAAS) space. 5 T R U S T E D B Y T H E W O R L D ' S L E A D I N G C O M P A N I E S "We use CB Insights to find emerging trends and interesting companies that might signal a shift in technology or require us to reallocate resources." Beti Cung, Corporate Strategy, Microsoft 6 PARTICIPATING COMPANIES CBI Councils bring together experienced executives to accelerate their success through the sharing of experience-based insights that inform business decisions and help solve complex strategic, organizational, and growth challenges. If you are an EVP, SVP, or C-level executive at a company doing $1B+ in revenue, request an invite by emailing councils@cbsinghts.com. LEARN MORE HERE events.cbinsights.com/councils councils@cbinsights.com cbinsights.com 7 Ja Lee Ja Lee is an intelligence analyst at CB Insights, where she produces data-driven analysis and research reports on trends in biotechnology and medical devices. Prior to joining CB Insights, Ja worked with clinical trials at Children's National Medical Center. She is a graduate of the University of North Carolina at Chapel Hill with majors in Biology and Spanish. She received her Master's degree in Physiology from North Carolina State University. @Ja_Lee2 | jlee@cbinsights.com Intelligence Analyst A B O U T T H E A N A L Y S T 8 Contents 9 12 18 23 27 What is CRISPR? How does it work? Startups to watch Applications & limitations What lies ahead 9 CRISPR ushers in a new wave of gene editing G E N E E D I T I N G 2 . 0 10 CRISPR leads among other gene editing techniques CRISPR gene editing technology leads against older methods. Before there was CRISPR, there were other gene-editing techniques known as Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs). CRISPR TALENs ZFNs 11 CRISPR-Cas9 system has 3 main players C R I S P R = C L U S T E R E D R E G U L A R L Y I N T E R S P A C E D S H O R T P A L I N D R O M E R E P E A T S 1 Guide RNA aka "GPS coordinates" 2 3 Cas9 protein aka "molecular scissors" DNA containing the sequence we want to insert 12 How does CRISPR work? B R E A K I N G C R I S P R D O W N 13 Understanding How The CRISPR Gene-editing Process Works W H A T I S C R I S P R ? 14 Understanding How The CRISPR Gene-editing Process Works W H A T I S C R I S P R ? 14 15 Understanding How The CRISPR Gene-editing Process Works W H A T I S C R I S P R ? 16 Understanding How The CRISPR Gene-editing Process Works W H A T I S C R I S P R ? 17 Understanding How The CRISPR Gene-editing Process Works W H A T I S C R I S P R ? 18 More CRISPR startups are on the horizon K E Y S T A R T U P S 19 Incumbents begin CRISPR clinical trials in 2018 Funding: $163M Select investors: Google Ventures, Khosla Ventures, Third Rock Ventures Clinical trial focus: Genetic eye disease Leber Congenital Amaurosis type 10 phase 1 Funding: $162M Select investors: Versant Ventures, Celgene, Bayer Clinical trial focus: Beta- thalassemia (Europe) & sickle cell disease (US) phase 1/2 trials Funding: $85M Select investors: Atlas Venture, Novartis Venture Funds, OrbiMed Advisors Clinical trial focus: Transthyretin Amyloidosis (Pre-clinical) T O P 3 P U B L I C C O M P A N I E S 20 " it might be better to describe CRISPR not as scissors but as a Swiss army knife, a tool with a panoply of functionalities that all stem from the action of a single molecular machine." Jennifer Doudna, PhD A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution 21 Mammoth Diagnostics provides a CRISPR-based platform for disease detection. It uses a simple paper-based test in which a liquid sample (e.g. saliva) is applied and subsequently, a color change becomes visible. A smartphone app can be used to upload that data to the cloud, in which an analysis is delivered in 30 minutes. Images: Mammoth Diagnostics N E W A P P L I C A T I O N : A T - H O M E D I A G N O S T I C S K I T Disclosed Funding: $120K Key team members: Select Investors: NFX Guild Trevor Martin Co-Founder, CEO Jennifer Doudna Co-Founder, Chair of Scientific Advisory Board 22 Beam Therapeutics develops therapeutics using a more precise method of CRISPR called base-editing. By tethering a modified CRISPR enzyme and an editing enzyme together, this technology allows editing to occur without cutting into the DNA at all. Images: Beam Therapeutics P R E C I S I O N G E N E T I C M E D I C I N E : B A S E - E D I T I N G Disclosed Funding: $87M Key team members: Select Investors: ARCH Venture Partners, F-Prime Capital David Liu, PhD Co-Founder Feng Zhang, PhD Co-Founder, Board Member 23 CRISPR will spur cross-industry innovation A P P L I C A T I O N S & L I M I T A T I O N S 24 Faster Cheaper Precision medicine Drug discovery process could be transformed Image: P H A R M A & B I O T E C H Current process With CRISPR 25 CRISPR could transform food supply & quality F O O D & A G R I C U L T U R E April 2016 1st CRISPR-edited organism to bypass USDA The white button mushroom, edited to not brown as quickly, bypassed USDA regulations. Sept 2017 Another USDA approval Yield10 Bioscience's CRISPR plant Camelina sativa with enhanced omega-3 oil bypasses regulations. October 2017 Leaner livestock Scientists at the Chinese Academy of Sciences in Beijing used CRISPR to genetically engineer pig meat with 24% less body fat Jan 2018 CRISPR saving chocolates Innovative Genomics Institute at Berkeley is working with candy company Mars to genetically engineer cacao plants 26 Immune system complications Yet, there are still some hurdles ahead L I M I T A T I O N S Mosaic generation Off-target activity 1 2 3 Different cutting enzymes (molecular scissors) Edit stem cells in egg or sperm cells Use a different Cas protein or ex-vivo therapies to bypass immune system 27 What lies ahead for CRISPR C O N T R O V E R S I E S & F U T U R E O F C R I S P R 28 Designing-your-own- baby Germline modifications CRISPR applications present ethical concerns C O N T R O V E R S I E S L I E A H E A D Bringing back extinct species Biohacking 29 Xenotransplantation Future applications of gene-editing Images: eGenesis; E. OTWELL AND M. TELFER, ScienceNews.org; DARPA F U T U R E O F C R I S P R Gene drive Genome editing as a weapon of mass destruction? 30 Questions? Twitter: @ja_lee2 jlee@cbinsights.com 31 The CB Insights platform has the underlying data included in this report W H E R E I S A L L T H I S D A T A F R O M ? CLICK HERE TO SIGN UP FOR FREE 32 cbinsights.com @cbinsights
