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1 CRISPR/Cas9 -Mediated Targeted Insertion of Human F9 Achieves Therapeutic Circulating Protein Levels in Mice and Non -Human Primates Hon-Ren Huang, Ph.D. American Society of Gene and Cell Therapy 22nd Annual Meeting April 29, 2019 Disclosure: Employee of Intellia Therapeutics,
CRISPR/Cas9 -Mediated Targeted
Insertion of Human F9 Achieves
Therapeutic Circulating Protein Levels in
Mice and Non -Human Primates
Hon-Ren Huang, Ph.D.
American Society of Gene and Cell Therapy
Disclosure: Employee of Intellia Therapeutics, Inc.2Intellia Therapeutics’ Legal Disclaimer
This presentation contains “forward -looking statements” ofIntellia Therapeutics, Inc.(“Intellia”) within themeaning ofthePrivate Securities Litigation Reform
Actof1995 .These forward -looking statements include, butarenotlimited to,express orimplied statements regarding Intellia’s ability toadvance andexpand
theCRISPR/Cas 9technology todevelop human therapeutic products, aswellasourCRISPR/Cas 9intellectual property portfolio ;ourability toachieve stable
oreffective genome editing ;ourability toeffectively administer onedose ormultiple doses ofourCRISPR/Cas 9-based product candidates ;thepotential timing
andadvancement ofourpreclinical studies, including continuing non-human primate studies forourTransthyretin Amyloidosis (“ATTR”) program andother
studies forourother programs (such as,alpha -1antitrypsin deficiency (“AATD”)), andhuman clinical trials ;thetiming andpotential achievement ofmilestones
toadvance ourpipeline, including initiation ofinvestigational new drug (“IND”) -enabling studies andfiling INDs ;ourability toreplicate results achieved inour
preclinical studies, including those inourATTR, AATD, andprimary hyperoxaluria type 1(“PH1”)programs, inanyfuture studies, including human clinical
trials ;ourability togenerate data and replicate results relating toenhancements toourproprietary lipid nanoparticle (“LNP”) technology, including its
formulation andcomponents, inpreclinical orclinical studies, orthat anyenhancements willresult inanimproved product candidate profile ;thepotential
development ofourproprietary LNP-adeno -associated virus (“AAV”) hybrid delivery system toadvance ourcomplex genome editing capabilities, such as
insertion ;thepotential development ofinvivo orexvivo celltherapeutics ofalltypes using CRISPR/Cas 9technology ;ourintent togenerate andpresent
additional data fororgans beyond theliver, andadditional insertion/repair data;theintellectual property position andstrategy ofIntellia’s licensors orother
parties from which itderives rights, aswellasthird-parties andcompetitors ;actions bygovernment agencies ;ourgrowth asacompany andtheanticipated
contribution ofthemembers ofourboard ofdirectors andourexecutives toouroperations andprogress ;theimpact ofourcollaborations onourresearch and
development programs ;thepotential commercialization opportunities, including value andmarket, forourproduct candidates ;ourexpectations regarding our
uses ofcapital, expenses, future accumulated deficit andother 2018 financial results ;andourability tofund operations into2021 .
Any forward -looking statements inthispresentation arebased onmanagement’s current expectations and beliefs offuture events, and aresubject toa
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