Realizing the full promise of gene editing to transform lives
Corporate Presentation
June 2022
Forward looking statements
Statements in this Presentation that are not statements of historical fact are forward-looking statements. Such forward-looking statements include, without limitation, statements regarding our research and clinical development plans, the clinical and therapeutic potential of our product candidates and platform technology, our expected manufacturing capabilities, strategy, regulatory matters, market size and opportunity, future financial position, forecasted expenses and cash runway, prospects, plans, objectives, and our ability to achieve certain milestones, and the timing thereof. Words such as "believe," "anticipate," "plan," "expect," "intend," "will," "may," "goal," "potential" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements necessarily contain these identifying words. These forward-looking statements are based on the beliefs of the management team at Graphite Bio, Inc. ("Graphite Bio") as well as assumptions made by and information currently available to us. Such statements reflect the current views of Graphite Bio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about Graphite Bio, including, without limitation, risks inherent in developing therapeutic products, future results from our ongoing and planned clinical trials and preclinical research, our ability to obtain adequate financing to fund our planned clinical trials and other expenses, trends in the industry and competitive landscape, the legal and regulatory framework for our industry, our future expenditures and overall market conditions. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. The actual results may vary from the anticipated results and the variations may be material. These forward-looking statements should not be taken as forecasts or promises nor should they be taken as implying any indication, assurance or guarantee that the assumptions on which such forward-looking statements have been made are correct or exhaustive or, in the case of the assumptions, fully stated in this presentation. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this presentation is given. This presentation discusses product candidates that are or will be under clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration (the "FDA"). No representation is made as to the safety or effectiveness of these product candidates for the therapeutic use for which such product candidates are being or will be studied.
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Graphite Bio, Inc.
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Graphite Bio: Realizing the full promise of gene editing
Powerful Next-Generation
UltraHDR™ Gene Editing Platform
- Harnessing the power of high- efficiency homology directed repair to fulfill the original goal of CRISPR gene editing
- "Find & replace" genes anywhere in the genome - correct, replace, insert
- Preclinical validation across a wide range of cell types and diseases
Robust Pipeline of
Potential One-Time Cures
- Initial focus on HSC-based cures for serious and life-threatening diseases
- First-in-industryapproach to directly correct the sickle cell mutation
- R&D programs designed to validate broad platform capabilities
Poised to Deliver
for Patients
- Founded by Stanford University genetic medicine pioneers
- Experienced management team and board with track record of developing innovative therapies
-
$352.1 million in cash, cash equivalents and investments in marketable securities (as of 3/31/2022);
cash runway into 4Q 2024
Graphite Bio, Inc.
HSC, hematopoietic stem cell; R&D, research and development. | 3 |
Harnessing the power of homology directed repair to unleash the full potential of CRISPR gene editing
Cutting is easy | Precision repair is hard | |
CRISPR Nuclease | 2nd gen CRISPR Nuclease |
Non-homologous end joining | Homology directed repair |
(Default error-prone repair) | (Precision repair) |
** | Donor DNA template |
Method to | Method to |
break and disrupt | find and replace |
Homology directed repair
- The original goal of gene editing and CRISPR technology
- The most precise DNA editing system in nature
-
Takes CRISPR beyond cutting and knock-outs - able to fix genetic lesions anywhere
in the genome - Has been historically difficult to achieve at high efficiencies until now
Graphite Bio, Inc.
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Our UltraHDR™ Platform: Building on CRISPR technology to 'find & replace' any gene
FIND: | REPLACE: | OPTIMIZE: | ||
Proprietary HiFi Cas9 RNP / modified guide | AAV6 delivers donor DNA template | Stem cells prepared | ||
RNA finds gene and precisely cuts | to drive high-efficiency HDR | and optimized for HDR | ||
R691A
sgRNA
ITR | ITR | |||
Homologous DNA | ||||
donor template | ||||
- Retains high activity
- Reduced off-target edits by 30- to 100-fold
- Minimized cellular response
- Non-integrating
- Up to 4kb template
- Enables high-efficiency HDR
- One lot may treat 1000s of patients
- HSC biology expertise and culture optimization
- Unprecedented editing efficiencies as high as 70%
- Successful GMP manufacturing
Graphite Bio, Inc.
GMP, good manufacturing practices; HDR, homology directed repair; HiFi Cas9, high-fidelity Cas9; HSC, hematopoietic stem cell; RNP, ribonucleoprotein. | 5 |
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Graphite Bio Inc. published this content on 29 June 2022 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 29 June 2022 21:41:11 UTC.