EX-99.1 2 brhc10030727_ex99-1.htm EXHIBIT 99.1

Exhibit 99.1
mRNA Engineered Cell & Genetic Medicines November 2021 A platform company in cell, ​gene-editing & cytokine therapies

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 the Securities and Exchange Commission, including information set forth in those filings under “Risk Factors” and similar headings. ‎​Forward-Looking Statements. Certain statements presented below on pages 4, 8-11, 13, 16-17, 19, 21-22, 24-28 and 30 are forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are any statements that are not statements of historical fact and may be identified by terminology such as “expect,” “plan,” “potential,” “project” or “will” or other similar words. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results may vary significantly from BTX’s expectations based on a number of risks and uncertainties, including but not limited to the following: (i) the evolution of BTX’s business model into a platform company focused on cellular, gene editing and cytokine programs; (ii) BTX’s ability to successfully, cost effectively and efficiently develop its technology and products; (iii) BTX’s ability to successfully commence clinical trials of any products on a timely basis or at all; (iv) BTX’s ability to successfully fund and manage the growth of its development activities; (v) BTX’s ability to obtain regulatory approvals of its products for commercialization; and (vi) uncertainties related to the impact of the COVID-19 pandemic on the business and financial condition of BTX, including on the timing and cost of its clinical trials. BTX cannot guarantee any future results, levels of activity, performance or achievements. The industry in which BTX operates is subject to a high degree of uncertainty and risk due to variety of factors, including those described in BTX’s public filings with the Securities and Exchange Commission, including its Current Report on Form 8-K filed with the Securities and Exchange Commission on May 11, 2021 and any subsequently filed Quarterly Reports on Form 10-Q for a more complete discussion of these factors and other risks, particularly under the heading “Risk Factors.” BTX expressly disclaims any obligation to update forward-looking statements after the date of this presentation. 2

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 Management Team 3 Lynn Sadowski– Mason, MS EVP of Clinical Operations

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

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 cellular data)mRNA Gene Editing (15 patents, extensive cellular data)NoveSlice™ Gene-Editing Protein (15 patents, extensive cellular data)ToRNAdo™ mRNA Delivery (4 patents, extensive cell and animal data) NoveSlice™ and ToRNAdo™ are trademarks of Factor Bioscience Limited.​ 5

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

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

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 streamline autologous therapies in genetic diseaseExtensive in-licensed patent protection 8

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-licensed patents cover mRNA encoding CRISPR, TALEN, ZFN, etc Chromatin Context-Sensitive Gene Editing EndonucleaseNovel nuclease (clear IP landscape)High specificity (36-40 base site)Blocked by histone modifications (specificity)Unlimited genomic sites (no PAM) 9

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 transfection in context of serumTransfection of many cell types demonstrated 10

Platform Technology Deployed in Four Regenerative Medicine Pillars Allogenic Stem CellPlatform (iMSC)ARDS​Bone marrow transplant failure/poor functionSolid tumors​ Autologous, Gene Edited PlatformHemoglobinopathiesOpportunities in many genetic diseases Autologous iPSCPlatformParoxysmal nocturnal hemoglobinuria​Partnership opportunities​ In Vivo Gene EditingPlatformTransthyretin amyloidosisStargardt disease​Non-syndromic hearing loss​Opportunities in many genetic diseases​ 11

Allogeneic iMSC Therapies

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 multiple and varied indicationsiPSC can be gene edited to program the iMSC with additional properties, expanding indicationsMSC therapies have had inconsistent clinical efficacy due to product heterogeneity 13

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 genetically modify 14

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

MSCs modulate immunological responses, support hematopoiesis, and repair bone marrow stromaClinical applications in hematopoietic stem cell transplant (HSCT)Treating engraftment failure or poor graft function​Promoting HSC engraftment​Working with world class KOLs in HSCT to focus on best clinical population(s) and trial designAnticipate FIH in 4Q-2023 iMSC Application in Graft Failure/Poor Graft Function Zhou, T et al. (2021) Challenges and advances in clinical applications of mesenchymal stromal cells. J Hematol Oncol 14:24, 1-24 16

Gene-editing iPSC and thorough characterization; followed by differentiation to iMSC​Multiple engineered iMSC products to deliver agents locally and avoid systemic toxicities​IL-7 & IL-15 drive expansion and engraftment​Combination with CAR-T and checkpoint inhibitors​ Developing a Family of Gene-edited iMSC Products To Address High Unmet Need Solid Tumors Song, N et al. (2020) Mesenchymal Stem Cell Immunomodulation: Mechanisms and Therapeutic Potential. Trends in Pharm Sci 9, 653-664 17

Autologous Cell Therapies

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-based gene editing is less complex than viral methodsFuture partnering possibilities 19 GeneEditing

Autologous iPSC Therapies

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 reprogramming and gene editing in single step derivationFast: Reprogramming and iPSC colony formation within 2 weeksThe safety, reliability and speed enable autologous iPSC programsEfficiency of reprogramming permits low quantity of cells from biopsy and simultaneous correction of gene defects Can quickly produce multiple iPSC clones per patientAbsence of genome integration facilitates screening to identify and characterize a safe clone 21

Autologous HSC from iPSC Autologous iPSC / Gene-modified autologous iPSC for: Genetic disease (e.g. hemoglobinopathies)Paroxysmal nocturnal hemoglobinuria (PIG1A)Infectious disease 22

In Vivo Gene Editing Therapies

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 encoding proprietary site-specific nucleaseCan target any gene through design of protein binding domainsHigh specificity to target genomic siteAchieves high level but transient expression, enhancing safety 24

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) ToRNAdo™ is a trademark of Factor Bioscience Inc. 25

Amyloidosis Caused by Transthyretin (ATTR) Caused by misfolded transthyretin (TTR), aggregating into toxic oligomeric forms​​Death 5-15 years after onset of symptoms is typical​Non-Familial (200,000-500,000 worldwide)​Owing to tissue deposition of normal TTR amyloid​Common clinical feature is peripheral neuropathyFamilial (~50,000 worldwide)​Autosomal dominant​Owing to mutations in TTR (>140)​Mutations increase amyloidogenic property​ Brooklyn’s Focus 26

Three sites of TTR synthesis, determine distinct clinical phenotypes​Liver: cardiomyopathy, peripheral neuropathy​Choroid plexus: LeptomeningealNeuroretina: Ocular toxicity​​ Current treatments address only liverFull phenotypic recovery requires both hepatic and CNS/choroid plexus intervention​Brooklyn initial focus on unmet need: oculo/leptomeningeal disease Clinical Features of Familial ATTR 27

​ 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, hATTR and senile (WT) disease​ 25% of ATTR population over age 80​ Rare restricted mutations; small other subsets​ Rare restricted mutations; small other subsets​ Approved/ R&D synthesis inhibitors siRNA, ASO, CRISPR​ ​​​IV/SC (hepatocytes)​ ​​​IV/SC (hepatocytes)​ ​ ​ ​BTX: in vivo​gene editing​ ​​​IV (hepatocytes)​ ​​​IV (hepatocytes)​ ​​​ Subretinal injection (Retinal Pigment Epithelium)​ ​​Intracisternal injection​(Choroid Plexus)​ BTX can treat all known ATTR regardless of mutation​ BTX In Vivo Editing Addresses all ATTR Manifestations​ 28

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​ ​ ​ ​ ​ ​ TBD​ n/a​ I.V. or local​ ​ ​ ​ ​ ​ Solid tumors​ IL7, IL15​ I.V​ ​ ​ ​ ​ ​ Oncology​ Undisclosed​ I.V​ ​ ​ ​ ​ ​ Autologous HSC, gene edited​ Undisclosed​ Undisclosed​ I.V.​ ​ ​ ​ ​ ​ Autologous iPSC-derived cell therapy​ TBD​ n/a​ I.V.​ ​ ​ ​ ​ ​ In vivo gene editing​ Transthyretin Amyloidosis​ TTR​ I.V. or CNS​ ​ ​ ​ ​ ​ Stargardt Disease​ ABCA4​ Retina​ ​ ​ ​ ​ ​ 29

BTX Will Leverage Best In ClassmRNA-based Technologies to Deliver Transformative Regenerative Medicines Exclusive license to foundational mRNA & gene editing IP​Diversified product strategy for multiple clinical applications​Experienced management team with deep expertise in C&GT​Strong partnership for translational execution and future innovation​In-licensed patent portfolio offers sub-licensing opportunities​​ 30