Sensorion Sas · EURONEXT:ALSEN

Sensorion Sas — Sensorion — Investor Day (April 2023)

82 slides from Sensorion Sas's investor day presentation, Apr 6, 2023

  1. Slide 1: Sensorion - Gene Therapy - R&D Day

    Sensorion - Gene Therapy - R&D Day

  2. Slide 2: Welcome and Introduction

    WELCOME AND INTRODUCTION Nawal Ouzren CEO, Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  3. Slide 3: Agenda

    Agenda Nawal Ouzren - Sensorion • Welcome and Introduction Barbara Kelley - HLAA • Incorporating the Patient Voice for Tomorrow’s Care Christine Petit - The Institut Pasteur • Deafness: from Genetic Architecture to Gene Therapy Laurent Désiré - Sensorion • GJB2-GT Program: Data to Drive Next Steps G

  4. Slide 4: Agenda

    Agenda Natalie Loundon - Necker Hospital • Otoferlin Defiency: Approaches Towards Hearing Restoration Laurent Désiré - Sensorion • OTOF-GT Program: SENS-501 Sensorion’s Lead Gene Therapy Program Christine Le Bec - Sensorion • Enabling Reliable Gene Therapy Manufacturing and Analytical Control Nawal

  5. Slide 5: Transforming Lives, Connecting People

    Transforming Lives, Connecting People Our vision is to help people with inner ear hearing disorders to live life with unlimited connections Sensorion - Gene Therapy - R&D Day

  6. Slide 6: Hearing Loss is a Massive Global Health Issue

    Hearing Loss is a Massive Global Health Issue Hearing loss is the most frequently occurring congenital sensory deficit, the largest modifiable risk factor for dementia and has a significant impact as people live longer.1-4 Number of people in millions 3000 RESTORE 2497 2500 Nonsyndromic 2206 (OTOF,

  7. Slide 7: Sensorion's Pipeline: A Comprehensive Franchise Of Advanced Hearing Loss Therapies

    Sensorion’s Pipeline: A Comprehensive Franchise Of Advanced Hearing Loss Therapies Next Milestones Product Indication Discovery In-vivo POC Preclinical Phase 1 Phase 2 Phase 3 (estimated timelines) Clinical Trial OTOF-GT* Otoferlin Deficiency Application Q2 2023 Adult onset GJB2-GT* Candidate Select

  8. Slide 8: Our Strategic Partnerships Support the Franchise

    Our Strategic Partnerships Support the Franchise • EU reference center for monogenic forms of deafness CLINICAL French Military Biomedical Research Institute RESEARCH • Natural History Study currently running for all monogenic • Access to a military population at risk of noise-induced hearing loss f

  9. Slide 9: We Have Established Strong Internal Capabilities to Ensure Successful Execution

    We Have Established Strong Internal Capabilities to Ensure Successful Execution PRECLINICAL CAPABILITIES CLINICAL CMC GENE THERAPY REGULATORY FOR SMALL MOLECULES EXPERIENCE FACILITIES EXPERTISE & GT PROGRAMS • Cell Model Platform: • 400 people enrolled in • Process development: non- • Develop regula

  10. Slide 10: Our Team has Significant Experience in Gene Therapy Clinical Development

    Our Team has Significant Experience in Gene Therapy Clinical Development The team has been involved in 15+ programs … using different technologies… from preclinical to BLA filing… 10 4 1 15 1 1 Preclinical Clinical BLA filing Gene therapy Cell therapy Gene editing (AAVs / LVs) … across different org

  11. Slide 11: We Have Established a Robust, Global Clinical Network

    We Have Established a Robust, Global Clinical Network 54 EUROPEAN UNION & TURKEY 2 CANADA 3 6 USA SOUTH KOREA MEXICO CITY 13 ISRAEL ISRAEL 2 AUSTRALIA Sensorion - Gene Therapy - R&D Day

  12. Slide 12: Our Vision: A Global Franchise Establishing Leadership in The Hearing Space

    Our Vision: A Global Franchise Establishing Leadership in The Hearing Space PORTFOLIO Future Projects (Institut Pasteur, Other…) GJB2-GT OTOF-GT SENS-401 ECOSYSTEM Natural History Key Opinion Centers of • EU Patient Groups Partners • US Studies Leaders Excellence • ROW Sensorion - Gene Therapy - R&D

  13. Slide 13: Incorporating the Patient Voice for Tomorrow's Care

    INCORPORATING THE PATIENT VOICE FOR TOMORROW’S CARE Barbara Kelley Executive Director, HLAA April 6, 2023 Sensorion - Gene Therapy - R&D Day

  14. Slide 14: The mission of HLAA

    The mission of HLAA is to open the world of communication to people with hearing loss by providing information, education, support and advocacy. hearingloss.org Sensorion - Gene Therapy - R&D Day

  15. Slide 15: Technology Users - Staying in the Hearing World

    Technology Users - Staying in the Hearing World Sensorion - Gene Therapy - R&D Day

  16. Slide 16: Because the Comfort of Community Is More Than Something You Hear

    Because the Comfort of Community Is More Than Something You Hear Sensorion - Gene Therapy - R&D Day

  17. Slide 17: Sow Joy. Nurture Community

    Sow Joy. Nurture Community BELONGING IT MAKES ALL THE DIFFERENCE Sensorion - Gene Therapy - R&D Day

  18. Slide 18: Film: Here We Go

    FILM Here we GO Sensorion - Gene Therapy - R&D Day

  19. Slide 19: Increased Access to Influencing Change

    Increased Access to Influencing Change Sensorion - Gene Therapy - R&D Day

  20. Slide 20: Externally-Led Patient-Focused Drug Development Meeting

    With permission from the U.S. Food and Drug Administration May 25, 2021 EXTERNALLY-LED PATIENT-FOCUSED DRUG DEVELOPMENT MEETING FOR PEOPLE AND FAMILIES LIVING WITH SENSORINEURAL HEARING LOSS Sensorion - Gene Therapy - R&D Day

  21. Slide 21: Qualitative Data - Hearingloss.org and Fda.gov

    Qualitative Data - Hearingloss.org and Fda.gov Sensorion - Gene Therapy - R&D Day

  22. Slide 22: Voice of the Patient: Most Troublesome Hearing Loss Related Concerns

    Voice of the Patient: Most Troublesome Hearing Loss Related Concerns Topic 1, Q2. Select the TOP 3 most troublesome hearing loss-related health concerns that you have or have had Sensorion - Gene Therapy - R&D Day

  23. Slide 23: Voice of the Patient: Top Struggles of Daily Life

    Voice of the Patient: Top Struggles of Daily Life Due To Hearing Loss Topic 1, Q3. What specific activities of daily life are most important to you are NOT able to do or you struggle with due to hearing loss? Select TOP 3 Sensorion - Gene Therapy - R&D Day

  24. Slide 24: Voice of the Patient: Worries About the Future

    Voice of the Patient: Worries About the Future Topic 1, Q4. What worries you most about you or your loved one’s condition in the future? Select TOP 3 Sensorion - Gene Therapy - R&D Day

  25. Slide 25: "I'm sorry" she said. You Could Have Heard a Pin Drop…

    “I’m sorry” she said. You Could Have Heard a Pin Drop… Sensorion - Gene Therapy - R&D Day

  26. Slide 26: Conclusions

    Conclusions • People with hearing loss described many worries for the future • Hearing loss profoundly impacts all activities of daily life • Hearing loss leads to social exclusion and diminished quality of life • Hearing loss is heavily stigmatized • Many hide their hearing loss, are intentionally

  27. Slide 27: Thank You

    Today. Tomorrow. Together CREATING A FUTURE OF PROMISE THANK YOU Barbara Kelley Executive Director bkelley@hearingloss.org Hearing Loss Association of America hearingloss.org Sensorion - Gene Therapy - R&D Day

  28. Slide 28: Deafness: From Genetic Architecture to Gene Therapy

    DEAFNESS: FROM GENETIC ARCHITECTURE TO GENE THERAPY Pr. Christine Petit Director of the Laboratory for Innovation in hearing therapies The Institut Pasteur, Hearing Institute April 6, 2023 Sensorion - Gene Therapy - R&D Day

  29. Slide 29: Hearing Institute

    Hearing Institute Sensorion - Gene Therapy - R&D Day

  30. Slide 30: Causal Gene for Deafness

    Causal Gene for Deafness Sensorion - Gene Therapy - R&D Day

  31. Slide 31: Genetic Architecture of Sensorineural Deafness

    Genetic Architecture of Sensorineural Deafness Casual genes for monogenic forms of deafness (early & late onset) and predisposing genes to age-related hearing loss, noise induces hearing loss… CAUSAL GENES: • ~ 130 causal genes for monogenic isolated forms of deafness (congenital/prelingual/ pre- ad

  32. Slide 32: A Genetic Continuum from Early Onset Deafness to Age-Related Hearing Loss

    A Genetic Continuum from Early Onset Deafness to Some Forms of Age-Related Hearing Loss (Presbycusis) Prelingual Monogenic deafness forms of DFNB forms presbycusis Young adult onset deafness DFNA forms Sensorion - Gene Therapy - R&D Day

  33. Slide 33: Sensorion - Gene Therapy - R&D Day

    Sensorion - Gene Therapy - R&D Day

  34. Slide 34: Five Major Groups of Cochlear Functions Each Defective in Deafness Forms

    Five Major Groups of Cochlear Functions Each Defective in Several Deafness Forms Gene symbol Protein name Deafness form OMIM identifier MYO6 Unconventional myosin-VI DFNB37/DFNA22 600970 MYO7A* Unconventional myosin-VIIa DFNB2/DFNA11 USH1B 276903 AUDITORY MECHANOELECTRICAL TRANSDUCTION MACHINERY MYO

  35. Slide 35: Petit, C et al., Nature genetics reviews, in press

    Petit, C et al., Nature genetics reviews, in press Sensorion - Gene Therapy - R&D Day

  36. Slide 36: Gene Therapy Approaches of Sensorineural Deafness

    Gene Therapy Approaches of Sensorineural Deafness 46 POCs; 41 POCs with positive effects 21 deafness genes DFNB forms: Cabp2, Gjb2, Kcne1, Kcnq1, Lhfpl5(Tmhs), Msrb3, Otof, Pcdh15, Pjvk, Slc26aA4, Slc26aA5, Strc, Syne4, Tmc1, Ush1c, Ush1g, Ush3a, Whrn and DFNA forms: Kcnq4, Myo6, Slc17a8(Vglut3) Gen

  37. Slide 37: Vestibule: the balance organ

    VESTIBULE: the balance organ Retzius 1884 COCHLEA: the hearing organ Sensorion - Gene Therapy - R&D Day

  38. Slide 38: Carson et al., 2023, JAMA Otolaryngol Head Neck Surg

    Carson et al., 2023, JAMA Otolaryngol Head Neck Surg Sensorion - Gene Therapy - R&D Day

  39. Slide 39: GJB2: The Most Frequent Causal Deafness Gene in North Africa

    GJB2: The Most Frequent Causal Deafness Gene in North Africa Sensorion - Gene Therapy - R&D Day

  40. Slide 40: From Connexins to Connexons and Gap Junction Channels

    From Connexins to Connexons and Gap Junction Channels heteromeric E1 E2 M1 conserved M3 M2 M4 variable homomeric • 428 pathogenic or likely pathogenic GJB2 variants have been reported. Topology of a generic connexin connexins connexon • 90% of these variants are associated with early onset DFNB1 for

  41. Slide 41: Variations in Audiogram Thresholds by Genotype and Progressivity

    Variations in Audiogram Thresholds by Genotype and Progressivity of Hearing Loss GJB2-ASSOCIATED HEARING LOSS HEARING LOSS PROGRESSION WITH AGE IN GJB2 TR/TR TR: truncating variant miss: missense variant Carson et al., 2023, JAMA Otolaryngol Head Neck Surg Sensorion - Gene Therapy - R&D Day

  42. Slide 42: Connexin 26 Expression in Mouse Cochlea

    Connexin 26 Expression in Mouse Cochlea Sensorion - Gene Therapy - R&D Day

  43. Slide 43: Neuroepithelial, Fibrocyte and Stria Vascularis Cx26 Networks

    Neuroepithelial Cx26 network Fibrocyte Cx26 network Stria vascularis Cx26 network Sensorion - Gene Therapy - R&D Day

  44. Slide 44: Identification of Cochlear Cell Types: Transcriptomic Profiling and RNAscope

    Identification of Cochlear Cell Types: Transcriptomic Profiling and RNAscope Analysis Basilar membrane Tympanic border cells Jean P. Wong V. ….Petit C* & Michalski N* submitted Sensorion - Gene Therapy - R&D Day

  45. Slide 45: Clustering of Genes Key to Cochlear Development and Function

    Clustering of Genes Key to Cochlear Development and Function to their Temporal and Spatial Patterns of Expression Jean P. Wong V. ….Petit C* & Michalski N* submitted Sensorion - Gene Therapy - R&D Day

  46. Slide 46: Sensorion - Gene Therapy - R&D Day

    Sensorion - Gene Therapy - R&D Day

  47. Slide 47: GJB2-GT Program: Data to Drive Next Steps

    GJB2-GT PROGRAM DATA TO DRIVE NEXT STEPS Dr. Laurent Désiré Head of Preclinical Development Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  48. Slide 48: We Aim To Develop Best-in Class and First-in Class Gene Therapy

    We Aim To Develop Best-in Class and First-in Class Gene Therapy CRITERIA SENSORION AAV capsid selected for high-level of target cells specificity GT product showing high-level of target cells transduction Limited off-target tissue biodistribution Surgical approach developed and mastered by ENT surge

  49. Slide 49: GJB2 Related Hearing Loss Affects More Than 300,000 Patients

    We Estimate That GJB2 Related Hearing Loss Affects More Than 300,000 Patients in the US, EU and Japan We have identified 3 forms of hearing loss associated with GJB2 gene mutations (Connexin 26). Prevalence of Congenital and Childhood onset forms estimated to be around 200k patients and around 50% o

  50. Slide 50: GJB2 Plays a Critical Role in Inner Ear

    GJB2 Plays a Critical Role in Inner Ear GJB2 IS THE GENE ENCODING CONNEXIN 26 PROTEIN IS INVOLVED FOR THE CONNEXIN 26 PROTEIN IN THE K+ HOMEOSTASIS GJB2 IS NOT NATURALLY EXPRESSED IN HAIR CELLS BUT IS EXPRESSED IN SUPPORTING CELLS V Stria Vascularis I Perilymph B (Scala Vestibuli) 0 mV, 5 mM [K+] En

  51. Slide 51: Our Lead Candidate Is Designed to Ensure Broad Coverage of Relevant Cochlear Cells

    Our Lead Candidate Is Designed to Ensure Broad Coverage of Relevant Cochlear Cells While Detargeting Hair Cells WT MOUSE WT NON-HUMAN PRIMATE AAV - RegSeq Negative control AAV - RegSeq AAV + RegSeq IHC OHC GFP+ cells (%) 100 80 30 AAV + RegSeq 15 0 RegSeq - + - + Sensorion - Gene Therapy - R&D Day

  52. Slide 52: Our Lead Candidate Prevents Hearing Loss in Relevant Mouse Model

    Our Lead Candidate Prevents Hearing Loss in Relevant Mouse Model PROOF OF CONCEPT IN PROGRESSIVE MOUSE MODEL Conditional knock-out mouse model leading to 2 phenotypes ABR threshold - 1 month after birth ABR threshold - 6 months after birth HEARING LOSS PREVENTION CORRELATES WITH CONNEXIN 26 EXPRESSI

  53. Slide 53: Our Lead Candidate Is Moving Into IND/CTA Enabling Studies

    Our Lead Candidate Is Moving Into IND/CTA Enabling Studies CRITERIA LEAD CANDIDATE AAV capsid selected for high-level of target cells specificity GT product showing high-level of target cells transduction Limited off-target tissue biodistribution Surgical approach developed and mastered by ENT surge

  54. Slide 54: GJB2-GT Program: Natural History Studies to Prepare Execution of Clinical Trials

    GJB2-GT PROGRAM NATURAL HISTORY STUDIES TO PREPARE EXECUTION OF CLINICAL TRIALS Dr. Géraldine Honnet Chief Medical Officer Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  55. Slide 55: We are Running Two Studies to Better Understand Early Onset Presbycusis Patients

    We are Running Two Studies to Better Understand the Profile of Early Onset Presbycusis (EOP) Patients and to Design the GT Trials EOP • Collaboration with the Institut Pasteur/CERIAH AUDIOLOGIC Genetic screening • Performing a battery of tests to identify the PROFILE most discriminating test to char

  56. Slide 56: AUDIOGENAGE - EOP Audiologic Profile Study

    AUDIOGENAGE - EOP Audiologic Profile Study Study description Qualification of audiological tests for the accurate diagnosis and follow-up of age-related hearing loss Sponsor: The Institut Pasteur (NCT05312983) Population • Participants over the age of 40 with hearing loss about 20 years ahead of sch

  57. Slide 57: SONG - EOP Natural history study

    SONG - EOP Natural history study Study description Longitudinal prospective study of the Natural History of Early Onset Form of Presbycusis caused by mutations in GJB2 Gene in adult subjects Sponsor: Sensorion Population • Participants with no cochlear implant at baseline, aged from 30 to 55 years a

  58. Slide 58: One Study on Congenital and Progressive Forms of Hearing Loss in Children

    We are Running One Study to Better Understand the Profile of Congenital and Progressive Forms of Hearing Loss Related to GJB2 Mutation in Children Longitudinal study of the natural history of otoferlin and connexin 26 related deafness in children up to 10 years Sensorion - Gene Therapy - R&D Day

  59. Slide 59: Otoferlin Deficiency: Approaches Towards Hearing Restoration

    OTOFERLIN DEFICIENCY: APPROACHES TOWARDS HEARING RESTORATION Pr. Natalie Loundon Director of the Cochlear Implant and Audiology Unit Necker Hospital April 6, 2023 Sensorion - Gene Therapy - R&D Day

  60. Slide 60: Auditory Neuropathy Spectrum Disorder

    Auditory Neuropathy Spectrum Disorder ANSD clinical aspects ANSD various origins 5-10% Sensorineural Hearing loss Brain • Mild to profound hearing loss (Pure Tone • Preterm, Hyperbilirubinemia, Mitochondrial Audiometry, PTA) • Poor speech intelligibility rate Nerve • Desynchronized Auditory Brainste

  61. Slide 61: DFNB9: Good Candidate for Gene Therapy Treatment

    DFNB9: Good Candidate for Gene Therapy Treatment Audinnove project • Mostly severe to profound hearing loss • Recessive mutations in OTOF encoding otoferlin • Deep understanding of the pathophysiology #1 Patient cohort • Defective inner hair cell synapse • Preserved inner ear structures • Mouse mode

  62. Slide 62: Gene Therapy in the Inner Ear: Surgical Approach

    Gene Therapy in the Inner Ear: Surgical Approach The inner ear is a good target for gene therapy • Routine surgical access • Isolated organ limiting off-target effects Developing specific tools for an efficient injection: • Minimally invasive • Reproduceable among surgeons across the world • Round W

  63. Slide 63: We have Developed a Dedicated Surgical Approach for Gene Therapy Administration

    We have Developed a Dedicated Surgical Approach for Gene Therapy Administration Non-Human Primates injected through the round window membrane (RWI) with or without stapedotomy (stap) 1 FENESTRATION (Round window membrane) Used for cochlear implant • Overpressure • Limited volume RWI RWI + stap • Bac

  64. Slide 64: Assessing the Prevalence of DFNB9 Among Patients with ANSD

    Assessing the Prevalence of DFNB9 Among Patients with ANSD Audinnove project Audioferlin Evaluation of a Cohort of Congenital Deep Deafness Patients and/or With Auditory Neuropathy, Looking for DFNB9 Sponsor: Necker Hospital Main objectives: • To assess the prevalence of otoferlin related deafness a

  65. Slide 65: Assessing the Natural History of DFNB9

    Assessing the Natural History of DFNB9 Longitudinal study of the natural history of otoferlin (and connexin 26) related deafness Sponsor: Sensorion Main objectives: • To assess the natural course of OTOF (and GJB2) related deafness • To assess the quality of life after CI surgery (short-term and mid

  66. Slide 66: Longitudinal Study of the Natural History of Otoferlin and Connexin 26 Deafness

    Longitudinal Study of the Natural History of Otoferlin and Connexin 26 Related Deafness in Children Up to 10 Years COHORT 1: WITHOUT CI AT STUDY ENTRY COHORT 2: WITH CI AT STUDY ENTRY COHORT 1a: CI activation Follow-up visits no CI at study entry Follow-up visits Screening period Selection 6M xY (x+

  67. Slide 67: We are Excited to Get the Gene Therapy Trial Started!

    We are Excited to Get the Gene Therapy Trial Started! Sensorion - Gene Therapy - R&D Day

  68. Slide 68: OTOF-GT Program: SENS-501 Sensorion's Lead Gene Therapy Program

    OTOF-GT PROGRAM: SENS-501 SENSORION’S LEAD GENE THERAPY PROGRAM Dr. Laurent Désiré Head of Preclinical Development Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  69. Slide 69: The Gene Therapy Pediatric Indications Have Blockbuster Sales Potential

    The Gene Therapy Pediatric Indications Have Blockbuster Sales Potential OTOF-GT IS THE PERFECT PILOT PROGRAM • OTOF-GT will be the pilot program demonstrating that GT is a relevant • Well understood biology and pathology of the medical approach for the inner ear otoferlin deficiency • OTOF-GT will e

  70. Slide 70: OTOF Gene Encodes Otoferlin, A Key Ca2+ Sensor Protein

    OTOF Gene Encodes Otoferlin, A Key Ca2+ Sensor Protein LOW Ca2+ INCREASE IN Ca2+ SNARE Proteins VAMP Ca2+ Ca2+ Ca2+ Ca2+ Vesicle Ca2+- Channel Otoferlin Pre-synaptic Neurotransmitters membrane release Restricted SNARE Otoferlin SNARE SNARE-complex Primed vesicle containing interaction interaction bi

  71. Slide 71: SENS-501 Leads to Long-term Hearing Recovery in a Translational Model

    SENS-501 Leads to Long-term Hearing Recovery in a Translational Model of Otoferlin Deficiency LONG-TERM HEARING RECOVERY SENS-501 DOSE EFFECT AAV-mOTOF injected in mice before hearing onset 10 months post-injection in mice after hearing onset 120 ABR Thresold (dB SPL) Untreated - no hearing 100 ABR

  72. Slide 72: SENS-501 Leads to Restoration of Efficient Sound Processing

    SENS-501 Leads to Restoration of Efficient Sound Processing in Behavioural Test White noise pulse (2-20 kHz, 115 dB) Wild type Non-injected Otof ko/ko Restored Otof ko/ko Sensorion - Gene Therapy - R&D Day

  73. Slide 73: SENS-501 Plans CTA Submission by H1 2023

    SENS-501 Plans CTA Submission by H1 2023 CRITERIA SENS-501 AAV capsid selected for high-level of target cells specificity GT product showing high-level of target cells transduction Limited off-target tissue biodistribution Surgical approach developed and mastered by ENT surgeons Regular engagement w

  74. Slide 74: Enabling Reliable Gene Therapy Manufacturing and Analytical Control

    ENABLING RELIABLE GENE THERAPY MANUFACTURING AND ANALYTICAL CONTROL Dr. Christine Le Bec Head of GT CMC Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  75. Slide 75: We are Developing In-House CMC Capabilities

    We are Developing In-House CMC Capabilities ADVANTAGES SENSORION AAV candidates supply for POC studies Early Process & Analytical development initiation Smooth and better control of the Tech Transfer to CDMOs Costs saving as process development is performed internally Reliable development timelines

  76. Slide 76: From Research to Clinical Process

    From Research to Clinical Process 50L 2L to 50L scale 200L scale 200L scale • Process Development • Process lock • Full scale GMP • Research material • Tox batch • Process Definition - DoE • Reference standard material at small scale for optimizing • Early specifications definition conditions • Earl

  77. Slide 77: Derisking Early CMC Development Steps

    Derisking Early CMC Development Steps Upstream Process platform Downstream Process platform Analytical development platform Sensorion - Gene Therapy - R&D Day

  78. Slide 78: We Have Completed the GMP SENS-501 Drug Product Manufacturing

    We Have Completed the GMP SENS-501 Drug Product Manufacturing SCHEMATIC HYBRID DUAL AAV SENS-501 Nter SENS-501 Cter ITR ITR ITR ITR Tox batches manufactured prom HR GMP Drug Substances manufactured GMP Drug Product manufactured ITR: Inverted Terminal Repeat; SD/SA: Splicing Donor/Acceptor sequence,

  79. Slide 79: We are Progressing Our GJB2-GT Lead Candidate in Manufacturing Process

    We are Progressing Our GJB2-GT Lead Candidate in Manufacturing Process ADVANTAGES GJB2-GT LEAD CANDIDATE AAV candidates supply for POC studies Early Process & Analytical development initiation Smooth and better control of the Tech Transfer to CDMOs Costs saving as process development is performed in

  80. Slide 80: Q&A Session Closure

    Q&A SESSION CLOSURE Nawal Ouzren CEO, Sensorion April 6, 2023 Sensorion - Gene Therapy - R&D Day

  81. Slide 81: Transforming Lives, Connecting People

    Transforming Lives, Connecting People Our vision is to help people with inner ear hearing disorders to live life with unlimited connections Sensorion - Gene Therapy - R&D Day

  82. Slide 82: Sensorion - Gene Therapy - R&D Day

    Sensorion - Gene Therapy - R&D Day

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