Ernexa Therapeutics Inc. · NASDAQ:ERNA
Ernexa Therapeutics — Brooklyn ImmunoTherapeutics — Investor Presentation (November 2021)
30 slides from Ernexa Therapeutics's investor presentation, Nov 12, 2021
Slide 1: mRNA Engineered Cell & Genetic Medicines
mRNA Engineered Cell & Genetic Medicines November 2021 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 is Led by a Strong, Experienced Management Team
Howard FederoffMD, PhDChief Executive Officer and President Kevin D’AmourPhDChief Scientific Officer Ron Guido, MS Pharm. Med.Chief Development Officer Roger SidhuMDChief Medical Officer Jay SialMBAChief Administrative Officer Sandra GurrolaVP of Finance BTX is Led by a Strong, Experienced Managemen
Slide 4: BTX Transforms into Regenerative Medicine Company with Platform Technology
BTX Transforms into Regenerative Medicine Company with Platform Technology Reverse merger Exclusive license to mRNA and LNP patents Secured $55M in Capital* Acquired Novellus Therapeutics Recruited new Exec team Nasdaq listing(from NYSE American) 2021 * Runway into 2023 4
Slide 5: Leveraging In-licensed Patent Portfolio to Advance Medicine
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 a competitive advantageMajor platform components: mRNA Cell Reprogramming (25 patents, extensive cell
Slide 6: BTX has a Broad Technology Landscape
mRNA therapeutics Non-viralgenetic medicines Footnote: doesn’t represent ex vivo gene editing space In Vivo gene editing iPSC-derivedtherapies BTX has a Broad Technology Landscape 6
Slide 7: Brooklyn's Licensed mRNA-Based and LNP Technologies
Brooklyn’s Licensed mRNA-Based and LNP Technologies 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 7
Slide 8: The Foundational mRNA Cell Reprogramming Platform
The Foundational mRNA Cell Reprogramming Platform Typical Results Highest efficiency generation of iPSC: 5 factors, rapid protein expressionLow toxicity, high percentage transfectedCustom reprogramming mediaSafe: no chance of genome integrationCan combine reprogramming with gene editing to streamlin
Slide 9: Unique mRNA-Based Delivery of a Novel Gene Editing Platform
Unique mRNA-Based Delivery of a Novel Gene Editing Platform Gene Knockout Gene Repair Safe-harbor Insertion Use of mRNA for Delivering Gene-Editing ProteinsRapid, high expression (efficiency)Transient expression (specificity)Amenable to non-viral deliveryNo risk of vector insertionMultiple in-licens
Slide 10: Novel Lipid with Effective mRNA Delivery In Vitro and In Vivo
Novel Lipid with Effective mRNA Delivery In Vitro and In Vivo U.S. Pat. No. 10,501,404 A compound of Formula (I) Wherein n is 1, 2, 3, 4, 5, 6, 7, 8, 9,10, 11, 12, 13, 14, or 15. Novel, single lipid component LNPNovel lipid, composition of matter IPLow toxicity enables repeated transfectionEfficient
Slide 11: Platform Technology Deployed in Four Regenerative Medicine Pillars
Platform Technology Deployed in Four Regenerative Medicine Pillars Allogenic Stem CellPlatform (iMSC)ARDSBone marrow transplant failure/poor functionSolid tumors Autologous, Gene Edited PlatformHemoglobinopathiesOpportunities in many genetic diseases Autologous iPSCPlatformParoxysmal nocturnal hem
Slide 12: Allogeneic iMSC Therapies
Fil Brooklyn Allogeneic iMSC Therapies
Slide 13: Allogeneic iMSC Product Platform
Allogeneic iMSC Product Platform iPSC-derived Mesenchymal Stem Cells (iMSC)Low risk of toxicity, proven across many clinical studiesLow immunogenicity, no need for immunosuppressive drugsLeveraging decades of work with MSC process development and manufacturingA single Drug Product can be used across
Slide 14: Brooklyn's iMSC Address Issues with Tissue-derived MSC
Brooklyn’s iMSC Address Issues with Tissue-derived MSC Historical Issues with MSC field Tissue-derived MSC Brooklyn’s iMSC Donor to donor variability Tissue source variability Manufacturing variability Limited or inconsistent characterization Poor mechanistic understanding Capacity to precisely gene
Slide 15: Extensive Characterization of iMSC to Ensure Consistency
Extensive Characterization of iMSC to Ensure Consistency Characterization to include:Stability of phenotypes with progressive passagesChanging responses to hypoxia and other stimuli Single cell RNA-seq Mass Cytometry Secretome Kehl et. A. (2019) Hutton et. A. (2021) Liu et. A. (2019) 15
Slide 16: iMSC Application in Graft Failure/Poor Graft Function
MSCs modulate immunological responses, support hematopoiesis, and repair bone marrow stromaClinical applications in hematopoietic stem cell transplant (HSCT)Treating engraftment failure or poor graft functionPromoting HSC engraftmentWorking with world class KOLs in HSCT to focus on best clinical p
Slide 17: Developing a Family of Gene-edited iMSC Products for Solid Tumors
Gene-editing iPSC and thorough characterization; followed by differentiation to iMSCMultiple engineered iMSC products to deliver agents locally and avoid systemic toxicitiesIL-7 & IL-15 drive expansion and engraftmentCombination with CAR-T and checkpoint inhibitors Developing a Family of Gene-ed
Slide 18: Autologous Cell Therapies
Fis Brooklyn Oia ImmunoTherapeutic: Autologous Cell Therapies
Slide 19: Autologous Cell Therapy, Efficient mRNA Gene Editing
Autologous Cell Therapy, Efficient mRNA Gene Editing Autologous HSC-based gene therapy for many addressable indicationsHemoglobinopathies Primary immunodeficienciesCongenital cytopeniasLeverages 30 years of clinical experience and isolation techniques for CD34+ HSC; robust engraftment and safetymRNA
Slide 20: Autologous iPSC Therapies
Fil Brooklyn Autologous iPSC Therapies
Slide 21: Autologous iPSC Cell Therapy Platform
Autologous iPSC 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 toxicityEfficient: Can combine
Slide 22: Autologous HSC from iPSC
Autologous HSC from iPSC Autologous iPSC / Gene-modified autologous iPSC for: Genetic disease (e.g. hemoglobinopathies)Paroxysmal nocturnal hemoglobinuria (PIG1A)Infectious disease 22
Slide 23: In Vivo Gene Editing Therapies
In Vivo Gene Editing Therapies
Slide 24: Second Pillar is a Genetic Medicine Product Platform
Second Pillar is a Genetic Medicine Product Platform Proprietary lipid nanoparticle for nucleic acid deliveryNovel lipid with composition of matter IPProperties can be tuned to target different cell types and tissuesCan deliver RNA or DNA; facilitates gene correction approachesDelivery of mRNA encod
Slide 25: 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 target is knock-out of TTR for Familial Transthyretin Amyloidosis (ATTR)
Slide 26: Amyloidosis Caused by Transthyretin (ATTR)
Amyloidosis Caused by Transthyretin (ATTR) Caused by misfolded transthyretin (TTR), aggregating into toxic oligomeric formsDeath 5-15 years after onset of symptoms is typicalNon-Familial (200,000-500,000 worldwide)Owing to tissue deposition of normal TTR amyloidCommon clinical feature is periph
Slide 27: Clinical Features of Familial ATTR
Three sites of TTR synthesis, determine distinct clinical phenotypesLiver: cardiomyopathy, peripheral neuropathyChoroid plexus: LeptomeningealNeuroretina: Ocular toxicity Current treatments address only liverFull phenotypic recovery requires both hepatic and CNS/choroid plexus interventionBrook
Slide 28: BTX In Vivo Editing Addresses all ATTR Manifestations
Polyneuropathy Cardiomyopathy Ocular depositions Leptomeningeal depositions Subtype Observed across subtypes (except leptomeningeal) Observed across subtypes (except leptomeningeal) Chiefly leptomeningeal subtype Restricted mutations + observed across subtypes Epidemiology Most common
Slide 29: BTX Cell Therapy and Gene-Editing Pipeline Summary
BTX Cell Therapy and Gene-Editing Pipeline Summary Indication Genetargets Delivery Discovery Preclinical IND-enabling Clinical Comments iMSC: iPSC-derived mesenchymal stem cells ARDS (all etiologies) n/a I.V. injection NoveCite program BMT/HSCT setting n/a I.V. injection
Slide 30: BTX Will Leverage Best In Class mRNA-based Technologies
BTX Will Leverage Best In ClassmRNA-based Technologies to Deliver Transformative Regenerative Medicines Exclusive license to foundational mRNA & gene editing IPDiversified product strategy for multiple clinical applicationsExperienced management team with deep expertise in C>Strong partnership
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