Update from INGLAXA Phase 1/2 Clinical Trials
- Development Plans for 4D-310 Genetic Medicine for Fabry Disease Cardiomyopathy
February 22, 2023
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© 2022 4D Molecular Therapeutics. All Rights Reserved. | 2 |
We are Boldly Innovating to Unlock the Full Potential of Genetic Medicines for Millions of Patients
COMPANY | Emeryville, | ~140 Employees | |
FDMT | GMP Facilities | ||
CA | |||
R&D Headquarters | |||
PLATFORM | Directed Evolution | ~1 BILLION | Targeted & Evolved Vectors |
Payload Design | |||
capsid sequences | |||
GMP Manufacturing | |||
PRODUCT | Vector Modularity | Clinical-Stage Vectors in 3 Therapeutic Areas | |
ENGINE
CLINICAL DEVELOPMENT
5 Clinical Candidates for 7 Patient Populations
STRATEGY | Fully Integrated Large Market Genetic Medicines Company |
© 2022 4D Molecular Therapeutics. All Rights Reserved. | CONFIDENTIAL | 3 |
Pipeline: 5 Clinical-Stage Product Candidates incl. Large Markets
THREE THERAPEUTIC AREAS, RARE & LARGE SUSTAINABLE PATIENT POPULATIONS
VECTOR | PRODUCT | INDICATION | RESEARCH | IND-ENABLING | PHASE 1 / 2 | PHASE 3 | PRODUCT |
Delivery | CANDIDATE | CANDIDATE | RIGHTS | ||||
R100 | OPHTHALMOLOGY | ||||||
Intravitreal | |||||||
4D-150 | Wet AMD | ||||||
Diabetic Macular Edema | |||||||
4D-125 | XLRP | ||||||
4D-110 | CHM | ||||||
4D-175 | Geographic Atrophy | ||||||
A101 | PULMONOLOGY | ||||||
Aerosol | |||||||
4D-710 | CF modulator-ineligible | ||||||
CF eligible for modulators | |||||||
4D-725 | A1AT Deficiency | ||||||
C102 | CARDIOLOGY | Today's Focus | |||||
IV | Fabry Disease | ||||||
4D-310 | |||||||
Cardiomyopathy | |||||||
© 2022 4D Molecular Therapeutics. All Rights Reserved. | CONFIDENTIAL | 4 |
Key Takeaways for Today: 4D-310
- Heart: Clinical POC for single low dose IV delivery, transgene expression & efficacy o 3rd 4DMT Vector with Clinical POC
- Safety: Generally well-tolerated with no liver, heart or DRG toxicity observed
- Transient acute atypical hemolytic uremic syndrome ("aHUS"): o Understood & manageable
- Approval pathway clear
© 2022 4D Molecular Therapeutics. All Rights Reserved. | CONFIDENTIAL | 5 |
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4D Molecular Therapeutics Inc. published this content on 22 February 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 22 February 2023 22:55:32 UTC.