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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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

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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

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Key Takeaways for Today: 4D-310

  1. Heart: Clinical POC for single low dose IV delivery, transgene expression & efficacy o 3rd 4DMT Vector with Clinical POC
  2. Safety: Generally well-tolerated with no liver, heart or DRG toxicity observed
  3. Transient acute atypical hemolytic uremic syndrome ("aHUS"): o Understood & manageable
  4. Approval pathway clear

© 2022 4D Molecular Therapeutics. All Rights Reserved. | CONFIDENTIAL

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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.