Entrada Therapeutics, Inc. · NASDAQ:TRDA
Entrada Therapeutics — MDF 2022
12 slides from Entrada Therapeutics's conference presentation, 2022
Slide 1: Introduction to Entrada and the EEV Platform in Myotonic Dystrophy Type I
Introduction to Entrada and the Endosomal Escape Vehicle (EEV™) Platform to Enhance Oligonucleotide Delivery in Myotonic Dystrophy Type I Michael S. Oldham, MD, MPH Executive Medical Director Entrada Therapeutics, Inc. WANT TO LEARN MORE? Scan the QR code for access to our recent publications and co
Slide 2: Disclaimer
DISCLAIMER This presentation includes express and implied “forward-looking statements. Forward looking statements include all statements that are not historical facts, and in some cases, can be identified by terms such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,
Slide 3: Meet Entrada
MEET ENTRADA Our Commitment Our Culture Our Team • Developing treatments for • We are building a company • ~130 employees with an people living with devastating culture where everyone can experienced leadership team diseases for which no or belong, contribute and grow insufficient options currently
Slide 4: Our Commitment to the DM1 Community
OUR COMMITMENT TO THE DM1 COMMUNITY Our partnerships with community leaders and patient advocacy organizations are at the heart of our work to bring innovative therapies to people living with devastating diseases As we progress our research program from ENTR-601WeeweWe-44ENTR-601-44 the lab to the c
Slide 5: The Challenge with Drug Delivery
THE CHALLENGE WITH DRUG DELIVERY Oligonucleotides offer a therapeutic approach to treat genetic disorders such as DM1. However, they often cannot effectively reach the disease-causing targets inside cells 5 1Verdine GL, Walensky LD. Clin Cancer Res. 2007;13(24):7264-70. MDF 2022
Slide 6: Endosomal Escape Vehicle (EEV) Platform
ENDOSOMAL ESCAPE VEHICLE (EEV™) PLATFORM We are developing EEV-oligonucleotide conjugates for the treatment of individuals with DM1 • Entrada’s EEV™ platform consists of a library of proprietary cyclic peptides with unique chemistry that enable improved uptake into the cell and escape from the endos
Slide 7: DNA and RNA Play Important Roles in the Creation of Proteins
DNA AND RNA PLAY IMPORTANT ROLES IN THE CREATION OF PROTEINS Deoxyribonucleic acid (DNA) Genes or short sections of DNA carry genetic instructions TRANSCRIPTION Disruptions at any Messenger ribonucleic acid stage of this process (mRNA) Genes are transcribed to mRNA, a can interfere with single-stran
Slide 8: DM1 Overview and Our Therapeutic Approach
DM1 OVERVIEW AND OUR THERAPEUTIC APPROACH Myotonic Dystrophy Type 1 (DM1) is a multisystemic disease caused by CUG trinucleotide repeats in DMPK mRNA that sequester MBNL proteins Healthy Individual Individual with DM1 ENTR-701 Approach in DM1 DMPK gene with few CTG repeats DMPK gene with 1000s of CT
Slide 9: Promising Efficacy Data of ENTR-701 in a DM1 Mouse Model
PROMISING EFFICACY DATA OF ENTR-701 IN A DM1 MOUSE MODEL Nuclear Foci Reduction 40 Our data in DM1 mouse Foci Positive Nuclei (%) Nuclei model showed: 30 HSA-LR • Dose-dependent reduction Vehicle Foci of nuclear foci (left) 20 **** • Reduced CUG-repeat 10 containing DMPK mRNA • Correction of aberran
Slide 10: Myotonia Correction in a DM1 Mouse Model
MYOTONIA CORRECTION IN A DM1 MOUSE MODEL A single dose of ENTR-701 ameliorated pinch-induced myotonia for at least 8 weeks HSA-LR Mouse: Non-treated HSA-LR Mouse: ENTR-701 Treated 10 ENTR-701 is the clinical candidate selected for DM1, composed of CUG-repeat blocking PMO conjugated to EEV. MDF 2022
Slide 11: Our Preclinical Data Support Advancement of ENTR-701
OUR PRECLINICAL DATA SUPPORT ADVANCEMENT OF ENTR-701 Single Dose of ENTR-701 Demonstrates Prolonged Effect for >8 Weeks Substantial reduction of nuclear foci, CUG-repeat-specific mRNA reductions Myotonia correction in a DM1 and robust splice correction, which mouse model following a single may indic
Slide 12: Thank You From the Whole Entrada Team!
THANK YOU FROM THE WHOLE ENTRADA TEAM! Please visit us during the upcoming poster and exhibitor session! WANT TO LEARN MORE? Scan the QR code for access to our recent publications and conference presentations. For questions, contact: Regan Sherman, Head of Patient Advocacy, [email protected] 12