Ernexa Therapeutics Inc. · NASDAQ:ERNA
Ernexa Therapeutics — Brooklyn ImmunoTherapeutics — Investor Presentation (September 2021)
21 slides from Ernexa Therapeutics's investor presentation, Sep 20, 2021
Slide 1: mRNA Engineered Cell & Cytokine Medicines
September 20, 2021 mRNA Engineered Cell & Cytokine Medicines A platform company in cell, gene-editing & cytokine therapies
Slide 2: Disclaimer
Disclaimer This presentation is intended to provide summary information about the business of Brooklyn ImmunoTherapeutics, Inc. (“BTX”). The information in this presentation is in no respects complete, comprehensive or exhaustive, and it should be read in conjunction with BTX’s public filings with t
Slide 3: BTX: World's Only mRNA Cell-Engineering Platform
BTX: World’s Only mRNA Cell-Engineering Platform What is mRNA cell-engineering? Messenger RNA (mRNA) is a special class of molecules that contain the instructions that determine how cells function. BTX’s platform uses synthetic mRNA with minimized immune response to engineer cells to treat disease b
Slide 4: BTX: Leveraging In-licensed Patent Portfolio to Advance Medicine
BTX: Leveraging In-licensed Patent Portfolio to Advance Medicine BTX has an exclusive license from Factor Bioscience to a portfolio of granted patents around mRNA-based cell engineering that will provide BTX with a competitive advantageBTX’s major platform components:mRNA Cell Reprogramming (25 pate
Slide 5: Brooklyn's Licensed mRNA-Based and LNP Technologies
Brooklyn’s Licensed mRNA-Based and LNP Technologies 5 Cell reprogramming Gene editing mRNA and LNP are toolsto make engineered cell medicine Nucleic acid delivery mRNA and LNP are the drugas in vivo gene-editing medicine
Slide 6: Fields of Medicine Addressable with BTX Technology
Fields of Medicine Addressable with BTX Technology 6
Slide 7: ToRNAdo Delivery Offers Superior Results
ToRNAdo™ Delivery Offers Superior Results 7 Vector toxicity
Slide 8: An Investment in BTX Today Offers Future Value
An Investment in BTX Today Offers Future Value Breadth and depth of BTX technology offer numerous opportunities for successful clinical resultsBTX in-licensed patent portfolio offers potential for licensing and royalty revenue in coming yearsBTX technology platform offers predictable path to clinica
Slide 9: BTX Technology: Competitive Landscape
9 9 BTX CRISPR1 TALENs2 Zinc-Finger Nucleases3 mRNA Vaccines4 CAR-T5 Primary Technologies mRNA Cell ReprogrammingmRNA Gene EditingNoveSlice™ToRNAdo™ CRISPR(including base-editing) TALENs Zinc-Finger Nucleases None Viral Vectors for CAR-T mRNA Gene Editing Yes Some No No N/A No mRNA Cell Reprogrammin
Slide 10: First Product Tier is a Stem Cell Product Platform
First Product Tier is a Stem Cell Product Platform Mesenchymal Stem Cells (MSC)Multipotent stem cells capable of expansion and differentiation into different kinds of tissuesSources of trophic factors modulating the immune system and inducing repair of tissuesProduct development leveraging extensive
Slide 11: The iPSC Technology Addresses Issues with Adult Sources
The iPSC Technology Addresses Issues with Adult Sources 11 iPSC-derived MSC advantagesAmenable to more scaled manufacturing due longer proliferative life-spanOne of the easiest iPSC-derived cell products to manufactureMore consistent characteristics and improved therapeutic properties relative to ad
Slide 12: First Targeted Clinical Indication for iMSC Products
First Targeted Clinical Indication for iMSC Products 12 Multiple clinical indications in setting of bone marrow transplant (BMT)Working with world class KOLs in BMT to focus on best clinical population(s) and trial designPreclinical development path elucidated by the iMSC program in ARDS (NoveCite p
Slide 13: Developing Gene-edited Versions of iMSC Products Targeting Solid Tumors
Developing Gene-edited Versions of iMSC Products Initially Targeting Poorly Addressed Solid Tumors 13 Rationale for gene-edited iMSC in oncologyHarnessing MSC homing to tumors to locally deliver immune stimulating proteinsAddition of specific cytokines counters the pro-tumor effect that sometimes ob
Slide 14: Second Product Tier is a Genetic Medicine Product Platform
Second Product Tier is a Genetic Medicine Product Platform Proprietary lipid nanoparticle for nucleic acid deliveryProperties can be tuned to access different cell types and target tissuesCan deliver RNA or DNA; facilitates gene correction approachesDelivery of mRNA encoding for proprietary site-spe
Slide 15: Developing In Vivo Gene-editing Products Addressing Rare Disease Indications
Developing In Vivo Gene-editing Products Addressing Rare Disease Indications (Orphan Designation) Direct gene editing in the liver, brain or eye for monogenic disordersAbility to knock-out or correct the target geneInitial gene targets includeTTR for Familial Transthyretin Amyloidosis (ATTR)ABCA4 fo
Slide 16: Third Product Tier is a Personalized Cell Therapy Platform
Third Product Tier is a Personalized Cell Therapy Platform Licensed technology is the safest, most efficient, and fastest method for iPSC derivation Safe: Non-integrating method using synthetic mRNA to produce reprogramming factorsEfficient: Uses LNP for repeated in vitro delivery with low toxicityE
Slide 17: Autologous Cell Therapy Applications of Licensed Technology
Autologous Cell Therapy Applications of Licensed Technology Autologous iPSC / Gene-modified autologous iPSC for: Genetic disease (e.g. Sickle cell disease)Infectious disease (e.g. HIV)Other cell therapy indicationsAutologous programs represent opportunities for future BTX expansion or partnering 17
Slide 18: BTX Cytokine, Cell Therapy, and Gene Editing Pipeline
BTX Cytokine, Cell Therapy, and Gene Editing Pipeline 18 Indication Approach Discovery Preclinical IND-enabling Early clinical Late clinical Comments IRX-2: human cell-derived mixed cytokine Head & Neck Cancer SubQ injections Phase 2b Various IST SubQ injections Phase 1/2 iMSC: iPSC-derived mesenchy
Slide 19: IRX-2 Mixed Cytokine Product Invigorates the Immune Response to Tumors
IRX-2 Mixed Cytokine Product Invigorates the Immune Response to Tumors Multiple cytokines in mixture can act locally and potentially in systemic fashionHuman cell-derived source addresses toxicity observed with other IL-2 cancer therapiesCurrently in Phase 2b for Neoadjuvant Head and Neck Cancer, da
Slide 20: BTX Most Advanced Asset: IRX-2 Human-Derived Cytokines
BTX Most Advanced Asset: IRX-2 Human-Derived Cytokines Phase 2 Company Sponsored Study in 1 IST Indication targeted to begin in 2022Phase 3 Study in Neoadjuvant Head and Neck Cancer targeted to begin in 2023 20 Renal Cell CancerLiver CancerHead and Neck CancerGastrointestinal Cancer Currently in Pha
Slide 21: A platform company in cell, gene-editing & cytokine therapies
September 20, 2021 A platform company in cell, gene-editing & cytokine therapies mRNA Engineered Cell & Cytokine Medicines
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