4D Molecular Therapeutics, Inc. · NASDAQ:FDMT

4D Molecular Therapeutics — Technical Presentation (2021)

9 slides from 4D Molecular Therapeutics's technical presentation, 2021

  1. Slide 1: Identification and Characterization of a Novel AAV Capsid & Product for the Treatment of Cystic Fibrosis Lung Disease

    Identification and Characterization of a Novel AAV Capsid & Product for the Treatment of Cystic Fibrosis Lung Disease Melissa A. Calton, Ph.D. 1, Melissa Kotterman, Ph.D.1, Christopher Schmitt, Ph.D.1, Austin Klein1, Christian Burns1, Roxanne Croze, Ph.D.1, Julie Nye1, Ghezal Beliakoff1, Melissa Que

  2. Slide 2: Disclosure to Learners

    Disclosure to Learners • Financial relationships with relevant companies within the past 24 months: – 4D Molecular Therapeutics, Inc., Full-time Employee

  3. Slide 3: Unmet Need & Limitations of Conventional AAV Vectors for CF Lung Disease

    Unmet Need & Limitations of Conventional AAV Vectors for CF Lung Disease DURABLE REPLACEMENT OF CFTR BY AAV GENE THERAPY HOLDS PROMISE Background: ▪ Cystic Fibrosis (CF): monogenic disorder caused by mutation in CFTR ▪ High unmet medical need in CF lung disease remains despite modulator therapy ▪ Ae

  4. Slide 4: Therapeutic Vector Evolution in Primate Lung: A101 Invention

    Therapeutic Vector Evolution in Primate Lung: A101 Invention 4DMT AAV DISCOVERY PROGRAM FOR AEROSOL DELIVERY TO LUNG AIRWAYS ▪ Industrialized Therapeutic Vector Evolution platform ▪ ~ 1 BILLION synthetic AAV capsid sequences ▪ Primate (NHP) model most relevant ▪ Aerosol delivery with clinically appr

  5. Slide 5: Human Antibody Resistance & Efficient Transduction of Human Cells

    Human Antibody Resistance & Efficient Transduction of Human Cells A101 DRIVING EXPRESSION OF REPORTER OR microCFTR (4D-710) TRANSGENE A101 HUMAN ANTIBODY RESISTANCE 4D-710 CFTR PROTEIN EXPRESSION 4D-710 CFTR mRNA EXPRESSION ▪ Compared to conventional AAV ▪ Dose-dependent & cell membrane ▪ Dose-depen

  6. Slide 6: A101 Aerosol Delivery & Transgene Expression in Primates

    A101 Aerosol Delivery & Transgene Expression in Primates VECTOR CHARACTERIZATION & 4D-710 PILOT SAFETY/DOSE FINDING NHP STUDIES A101-EGFP: BIODISTRIBUTION 4D-710: TOLERABILITY & CFTR TRANSGENE EXPRESSION ▪ Single aerosol delivery with clinical ▪ Single aerosol dose of product candidate 4D-710 (3E13

  7. Slide 7: Conclusions

    Conclusions 4D-710 FOR THE TREATMENT OF CYSTIC FIBROSIS LUNG DISEASE ▪ Therapeutic Vector Evolution used in primates to invent A101 vector ▪ A101 matches Target Vector Profile: o Aerosol delivery throughout NHP airways o Mucus barrier penetration in NHP o Resistance to pre-existing human antibodies

  8. Slide 8: Acknowledgments

    Acknowledgments ▪ Our special thanks to the Cystic Fibrosis Foundation ▪ 4DMT Process & Analytical Development ▪ 4DMT Project Management © 2020 4D Molecular Therapeutics. All Rights Reserved.

  9. Slide 9: Thank You

    Thank You 5858 Horton Street, Suite 455 Emeryville, CA 94608 (510) 505-2680 | [email protected] 4DMT.com © 2020 4D Molecular Therapeutics. All Rights Reserved. 9

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