Metagenomi Therapeutics, Inc. · NASDAQ:MGX
Metagenomi Therapeutics — Corporate Update (May 2024)
14 slides from Metagenomi Therapeutics's corporate update, May 1, 2024
Slide 1: Business Update Call
Nasdaq listed (MGX) Business Update Call May 1st, 2024, 5pm ET Unlocking 4 Billion Years of Microbial Evolution to Create Curative Genetic Medicines
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation includes forward-looking statements, including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this presentation, including statem
Slide 3: Today's speakers
3 Brian Thomas, PhD CEO & Founder Pamela Wapnick, MBA CFO Simon Harnest, MSc CIO & SVP of Investor Relations Sarah Noonberg, MD, PhD CMO Today’s speakers
Slide 4: Metagenomi business update: key inflection points for 2024 and beyond
Metagenomi business update 4 Key inflection points for 2024 and Beyond Hemophilia A: DC nomination on track by mid-2024 Hemophilia A: 12-month NHP durability data in 2H 2024 Additional DC in late 2024 Continued technology advancements including RIGS & CAST Mutual termination of Moderna collaboration
Slide 5: Full rights to wholly-owned gene editing technology
5 Programmable Nucleases, including ultra-small systems (Knockdown, exon skipping) Base Editors, including ultra-small systems (Single nucleotide changes) Big RIGS (>100 base pair integrations) Little RIGS (prime editing) (1-100 base pair replacement, insertion, or deletion) G A T C Full rights to w
Slide 6: Expanded targeting potential with MGX Base Editors and Ultra Small editing systems
Expanded targeting potential with MGX Base Editors and Ultra Small (SMART) editing systems MGX base editors increase the genome targetability by 5x compared to Cas9 systems Ultra small CRISPR nucleases and base editors create potential advantages for delivery, safety, manufacturing and dosing Small
Slide 7: Leading the field in conducting large, targeted genome integrations
Leading the field in conducting large, targeted genome integrations CAST (CRISPR-Associated Transposases): DNA-Mediated Integration Systems DNA Template Transposase Reverse transcriptase RNA Template Cargo Target DONOR PAM Junction between genomic target and donor template Junction between genomic t
Slide 8: Additional Base Editing and RIGS Data to be Shared at ASGCT
Additional Base Editing and RIGS Data to be Shared at ASGCT Novel CRISPR Effectors and Reverse Transcriptases Discovered from Metagenomics Enable Extensive Remodeling of the Human Genome Abstract Number: 1209 Location: Exhibit Hall Date / time: Thursday, May 9, 2024, 12:00 p.m. ET Novel and Efficien
Slide 9: Our Pipeline
Delivery Target Editing Approach Indication Discovery Lead Optimization IND-Enabling Clinical Partner Knock in HEMOPHILIA A Knock down PRIMARY HYPEROXALURIA TYPE 1 Knock down TRANSTHYRETIN AMYLOIDOSIS Knock down CARDIOVASCULAR DISEASE (AGT) RIGS/Base Editing ALPHA 1 ANTITRYPSIN DEFICIENCY RIGS WILSO
Slide 10: Durable FVIII activity demonstrated out to 4.5 months
Durable FVIII activity demonstrated out to 4.5 months DC nomination planned for mid 2024 12 month FVIII durability data to be presented in 2H 2024 Animal ID INDELS in liver (d7) FVIII gene integration frequency # (copies per 100 genomes) Mean FVIII activity % of normal (d14 to d126) 1001 45% 2.9% 75
Slide 11: AAV delivers donor DNA template
AAV delivers donor DNA template Leveraging Heme A experience to deliver and integrate a variety of target genes MG nuclease creates highly efficient cut at safe harbor locus The LNP component stays fixed and serves as a platform New programs can move quickly by simply substituting the donor DNA carg
Slide 12: Accelerating therapeutic translation
Accelerating therapeutic translation Liver Targets Knock-in & knock-down: Hemophilia A Primary Hyperoxaluria Type I Transthyretin Amyloidosis Cardiovascular Indications (AGT) Gene corrections & integrations: A1AT Deficiency Wilson’s Disease Neuromuscular + CNS Targets Ultra small systems for Knock d
Slide 13: Metagenomi continues to execute on all fronts
Metagenomi continues to execute on all fronts 13 Milestones by 2026 2 IND filings At least 2 additional DCs In vivo proof of concept for large gene integrations Additional BD opportunities Strong Cash Position $327.4 million as of March 31, 2024 Financial outlook substantially unchanged Cash runway
Slide 14: Thank you
Thank you Q&A 14 Investor Contact: Simon Harnest, MSc CIO & SVP of Investor Relations simon@metagenomi.co