Andrew May DPhil Chief Scientific Officer Caribou Biosciences Inc 2112016 1 Gene Editing Tools and Methods Caribou background Founded by Jennifer Doudna Rachel Haurwitz Martin Jinek and James Berger UC Berkeley to develop commercial applications ID: 769933
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Andrew May DPhilChief Scientific OfficerCaribou Biosciences, Inc. 2/11/2016 1 Gene Editing Tools and Methods
Caribou background Founded by Jennifer Doudna, Rachel Haurwitz , Martin Jinek and James Berger (UC Berkeley) to develop commercial applications of CRISPR- Cas systems Co-founded Intellia Therapeutics in 2014 to develop human gene and cell therapies using Cas9Research partnerships with DuPont, Novartis, IntelliaLocated in Berkeley, CA 2/11/2016 2
2/11/2016 3 Caribou CRISPR technology platform is being applied in multiple areas Gene and cell therapies Antimicrobials Animal health Precision breeding Novel trait generation Crop genetics Cell engineering Animal models Phenotypic screening Genomics Microbial fermentation Therapeutic bioproduction Caribou is pioneering the translation of CRISPR- Cas genome engineering for the development of new bio-based products
We can now translate genomic information into valuable solutions 2/11/2016 4 >5,000 Mendelian disorders understood at the genetic level >40 crop species with reference genome sequences >3,400 complete microbial genome sequences Correct the genetic mutation in somatic cells with gene therapy Utilize precision breeding to develop crops with improved performance Selectively kill harmful microbes and preserve ‘friendly’ microbes
Site-directed nucleases for gene editing MeganucleasesCellectis, Precision Biosciences, bluebird Zinc Finger ProteinsSangamo BioSciences TALE Nucleases CellectisCRISPR-Cas9Caribou, Intellia, Editas, CRISPR TxOther systems2/11/2016 5
Therapeutic application areas Anti-microbial therapiesInfectious diseaseEditing microbial genomes Drug discovery and developmentImproved cellular and animal models Screens for new targets and mechanisms for intervention with small molecules and antibodies New genomic tools Antibody, vaccine, viral production2/11/20166
Important considerations for gene editing SpecificityWhere can the nuclease cut?Where does it cut?DNA repair outcomes How does the cell repair the break? How do these repair outcomes modify target activity? Functional assaysHow do DNA repair outcomes impact function of the cell type of interest?Disease-relevant models and measurements for assessing activity and safety2/11/2016 7
SITE-Seq provides comprehensive measurement of Cas9/guide RNA specificity 2/11/2016 8 Comprehensive identification of all possible cut sites in the genome Measurement of identified sites at high sensitivity allows us to determine activity at each site in cell populations of interest
Analysis of Cas9-dependent double-strand break repair outcomes 2/11/2016 9
Summary Gene editing technologies hold huge promise for therapeutics and many other fieldsGenomic tools potentially enable higher precision activity measurement and detection of effects than for other therapeutic classes Current regulatory framework has been used successfully to monitor gene editing and gene therapiesSmaller patient populations may require alternative approaches 2/11/2016 10
2/11/2016 11 Gene editing technologies can provide beneficial impact in many different areas Gene and cell therapies Antimicrobials Animal health Precision breeding Novel trait generation Crop genetics Cell engineering Animal models Phenotypic screening Genomics Microbial fermentation Therapeutic bioproduction Many regulatory considerations are common across these diverse application areas