Ernexa Therapeutics Inc. · NASDAQ:ERNA
Ernexa Therapeutics — Eterna Therapeutics — Investor Presentation (August 2023)
18 slides from Ernexa Therapeutics's investor presentation, Aug 2, 2023
Slide 1: Eterna Therapeutics Investor Presentation August 2023
Eterna TherapeuticsInvestor Presentation August 2023 1 © Eterna Therapeutics Inc.
Slide 2: Disclaimer and Forward-Looking Statements
This presentation (this “Presentation”) and any oral statements made in connection with this Presentation are for informational purposes only and do not constitute an offer to sell, a solicitation of an offer to buy, or a recommendation to purchase any equity, debt or other securities of Eterna Ther
Slide 3: Investment Highlights
Investment Highlights 3 © Eterna Therapeutics Inc. Novel technologies primed to generate value through collaborations and partnerships, and compete in growing, multi-billion dollar markets Gene editing and mRNA delivery technology instrumental for R&D in cell engineering Technology is protected by a
Slide 4: Primed to Compete in Growing, Multi-billion $ Markets
Primed to Compete in Growing, Multi-billion $ Markets Eterna’s technology is designed to potentially help solve a variety of industry problems, including efficient editing and delivery *Verified Market Research;**Nova1Advisor CAGR 4.7% CAGR 16.52%
Slide 5: Eterna: An Innovative RNA Cell Engineering Company
5 © Eterna Therapeutics Inc. Each platform is supported by a significant intellectual property estate with patents designed to both support and foster candidate development and trademarks designed to provide additional brand exclusivity and recognition The Eterna Platform RNA ToRNAdo™ RNA Delivery G
Slide 6: Substantial Shortcomings Remain with Industry Standard Gene Editing Technologies
Substantial Shortcomings Remain with Industry Standard Gene Editing Technologies 6 © Eterna Therapeutics Inc. Other Technologies in Use Key Shortcomings TALENs CRISPR Can be complex or can require expensive customization Immunogenic Potential cause of autoimmune diseases Potential for undesired muta
Slide 7: Eterna's platform is novel and differentiated
7 © Eterna Therapeutics Inc. Eterna’s platform is novel and differentiated with the potential to overcome the challenges of industry standard cell engineering technologies The Eterna Platform RNA ToRNAdo™ RNA Delivery Gene Editing Platforms Technology Library of Ionizable Lipids Potential Advantages
Slide 8: Potential for high-value therapeutics
8 © Eterna Therapeutics Inc Potential for high-value therapeutics CORE POTENTIAL MODALITIES POTENTIAL THERAPEUTICS Gene editing Blood cancers Gene replacement Solid tumors Cell replacement Ophthalmology, cardiology, neurology, etc. Reprogramming, Gene Editing, Delivery Cell Therapy Oncology Pipeline
Slide 9: RiboSlice: Non-Viral, DNA-Free Targeted Gene Editing
RiboSlice™: Non-Viral, DNA-Free Targeted Gene Editing 9 © Eterna Therapeutics Inc. RiboSlice™: mRNA construct that encodes gene editing proteins mRNA transiently expresses high levels of gene editing proteins within cells Does not rely on viruses or DNA-based vectors Potential to enable efficient an
Slide 10: NoveSlice: high specificity gene-editing endonuclease
NoveSlice™: high specificity gene-editing endonuclease Exhibits high efficiency on-target cutting and enhanced sensitivity to the chromatin context of the target site Designed with an array of DNA-binding repeat sequences connected by flexible linkers Designed to target cutting activity to actively
Slide 11: Eterna's LNP Delivery is Customizable Through Its Library of Novel Ionizable Lipids
The Eterna Difference: Eterna’s LNP Delivery is Customizable Through Its Library of Novel Ionizable Lipids 11 © Eterna Therapeutics Inc. Designed to effectively transfect cells Customizable constructs allow Eterna to vary the lipid head group, tail, and spacer link to fine tune the lipid transfectio
Slide 12: Ionizable Lipids Have Demonstrated Robust Transfection
Ionizable Lipids Have Demonstrated Robust Transfection 12 © Eterna Therapeutics Inc. Transfection efficiency may be increased by fine tuning with only a few highly targeted edits to the structure Eterna LNPs and lipoplexes exhibited greater in vitro transfection efficiency of THP-1 monocytes than co
Slide 13: Eterna's mRNA Delivery Capabilities to Target the Lungs
Eterna’s mRNA Delivery Capabilities to Target the Lungs 13 © Eterna Therapeutics Inc. Transgene expression observed as early as 4 h after transfection in BEAS2B cells treated with both nebulized and non-nebulized complexes and was maintained to at least 48 h(Figs. A–E) Fluorescent images of nebulize
Slide 14: Eterna's mRNA Delivery Capabilities to Target the Lungs
Eterna’s mRNA Delivery Capabilities to Target the Lungs 14 © Eterna Therapeutics Inc. IkBα and SOD3 expression significantly increased in vitro and in vivo in pulmonary cells administered respective complexes compared to those receiving scrambled mRNA control A B C D Aerosolized Pulmonary Delivery o
Slide 15: Platform to Address Challenges of Cell Reprogramming
Platform to Address Challenges of Cell Reprogramming 15 © Eterna Therapeutics Inc. Allogeneic “off-the-shelf” cell therapies can be a very powerful tool in treating a variety of diseases Eterna’s technology aims to address the challenges facing allogeneic “off-the-shelf” cell therapies including inc
Slide 16: Eterna Technology Efficiently Reprograms Cells with mRNA to iPSCs
16 © Eterna Therapeutics Inc. iPSC Markers for Confirmation Clonal iPSC Lines Generated in 2 Weeks mRNA Transfections with Eterna Technology Verified iPSC Differentiation Potential Mesenchymal Stem Cells Adipocytes Oil Red O stain Chondrocytes Alcian Blue stain Osteoblasts Alizarin Red S stain Funct
Slide 17: Combined mRNA Gene Editing and Cell Reprogramming
Combined mRNA Gene Editing and Cell Reprogramming Creation of pluripotent stem cell lines containing defined deletions in the CCR5 gene Demonstrated gene editing and dedifferentiation of human somatic cells in preclinical studies Successful demonstration of capability to generate clonal engineered p
Slide 18: Summary Highlights
Summary Highlights 18 © Eterna Therapeutics Inc. Novel technologies primed to generate value through collaborations and partnerships, and compete in growing, multi-billion dollar markets Partnership strategy is well positioned to maximize several potential revenue streams including upfront payments,
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