Pipeline

New Era of Genetic Medicine

A Robust and Diverse Clinical Pipeline

Our diversified product pipeline spans three therapeutic areas, each of which leverages a unique and proprietary customized vector invented at 4DMT. Each product candidate is administered by one of three different routes of administration based on the disease target: intravitreal (ophthalmology), aerosol delivery (pulmonology) and intravenous (cardiology).

Vector Delivery

Product Candidate

Indication

Research Candidate

IND-Enabling

Phase 1/2

Phase 3

Clinical Trial

Product Rights

Ophthalmology R100 Intravitreal

4D-150

Wet AMD Phase 1/2

Ophthalmology R100 Intravitreal

4D-150

Diabetic Macular Edema Phase 1/2

Spectra

Ophthalmology R100 Intravitreal

4D-125

XLRP Phase 1/2

Excel

Ophthalmology R100 Intravitreal

4D-110

Choroideremia Phase 1/2

Chorus

Ophthalmology R100 Intravitreal

4D-175

Geographic Atrophy Research Candidate

Gaze

Ophthalmology R100 Intravitreal

Undisc.

Vector licensed to Astellas

Undisclosed Rare Monogenic Ophthalmic Disease Research Candidate

Pulmonology A101 Aerosol

4D-710

Cystic Fibrosis not modulator-amenable Phase 1/2

aerow

Pulmonology A101 Aerosol

4D-710

Cystic Fibrosis modulator-amenable Phase 1/2

Pulmonology A101 Aerosol

4D-725

A1AT Deficiency Research Candidate

Cardiology C102 IV

4D-310

Fabry Disease Phase 1/2

Inglaxa

CNS B SERIES Multiple

Unnamed

Led by Arbor

Amyotrophic Lateral Sclerosis Research Candidate

50/50 WW

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Our Product Candidates

4D-150

4D-150 is currently being evaluated in the Phase 2 stage of the Phase 1/2 PRISM clinical trial in adults with wet AMD and in the Phase 2 SPECTRA clinical trial in adults with DME. We believe this product candidate is highly differentiated because it is designed as a single, routine, low-dose intravitreal injection to induce continuous expression of two therapeutic transgenes from retinal cells, resulting in durable suppression of four different vascular endothelial growth factor (VEGF) family members at the site of disease activity.

4D-125

4D-125 is an investigational genetic medicine using the R100 vector for the treatment of XLRP related to mutations in the RPGR gene. 4D-125 is currently being evaluated in a Phase 1/2 study. We believe this product is highly differentiated because it utilizes the primate evolved and customized intravitreal R100 vector for transgene (RPGR) delivery to the retina with the goal of transgene expression in all regions of the retina after a single dose.

4D-110

4D-110 is an investigational genetic medicine using the R100 vector for the treatment of choroideremia related to mutations in the CHM gene. 4D-110 is currently being evaluated in a Phase 1/2 study. We believe this product is highly differentiated because it utilizes the primate evolved and customized intravitreal R100 vector for transgene (Rep1) delivery to the retina with the goal of transgene expression in all layers and all regions of the retina after a single dose.

4D-175

4D-175 combines the customized and evolved intravitreal vector, R100, and a codon-optimized transgene encoding a highly functional shortened form of human complement factor H (sCFH). The genetic medicine candidate is designed to provide durable transgene expression in the retina without significant inflammation following a single, low-dose intravitreal injection. The sCFH payload is designed to restore normal complement regulation, which has the potential to slow progression of disease.

4D-710

4D-710 is an investigational genetic medicine candidate using the A101 vector for the treatment of cystic fibrosis lung disease. 4D-710 is currently in a Phase 1/2 study for patients with cystic fibrosis who are ineligible for CFTR modulator therapy or who have discontinued therapy due to adverse effects. We believe this product is highly differentiated because it is designed for a single dose aerosol delivery of a corrective transgene (CFTR∆R) throughout the airways and alveoli of the lungs for a mutation agnostic (or mutation independent) disease-modifying benefit.

4D-725

4D-725 is an investigational genetic medicine candidate using the A101 vector that is currently in preclinical development. 4D-725 is designed for single dose aerosol treatment of patients with alpha-1 antitrypsin lung disease.

4D-310

4D-310 is an investigational genetic medicine candidate using the C102 vector that received Fast Track Designation from the FDA in Q3 2020 for the treatment of classic (severe) Fabry disease cardiomyopathy. 4D-310 is being evaluated in two Phase 1/2 INGLAXA clinical trials in adult patients with classic or late-onset Fabry disease. We believe this product candidate is highly differentiated because it is designed for a single low dose intravenous delivery to, and transgene (GLA) expression within, the heart muscle cells (cardiomyocytes) in order to directly correct the leading cause of death in these patients (cardiomyopathy). Of note, cardiac benefit has not been definitively demonstrated following enzyme delivery in the blood through enzyme replacement therapy (ERT), PEGylated ERT, or gene therapy with enzyme expression from the liver.

Scientific Posters, Publications and Presentations

EURETINA Congress 2024

Extended Follow-Up in the PRISM Clinical Trial Evaluating 4D-150 in Adults with Neovascular Age-related Macular Degeneration

Arshad M. Khanani, et al. Presented at the 24th EURETINA Congress, September 19, 2024.

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Retina Society 2024

Interim Results from the PRISM Phase 1/2 Clinical Trial Evaluating Intravitreal 4D-150 in Adults with Neovascular Age-Related Macular Degeneration

David A. Eichenbaum, et al. Presented at the Retina Society 57th Annual Scientific Meeting, September 15, 2024.

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ASRS 2024

Phase 2 Population Extension Cohort in the PRISM Trial Evaluating 4D-150 in Adults With Neovascular Age-related Macular Degeneration

Raj K. Maturi, et al. Presented at the American Society of Retinal Specialists (ASRS) Annual Meeting, July 17, 2024.

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Clinical Trials at the Summit 2024

24-week Results from the Randomized Phase 2 Dose Expansion Stage of the PRISM Trial Evaluating 4D-150 in High Need nAMD Patients: Injection-free Patient Analyses

Carl Danzig, et al. Presented at the Clinical Trials at the Summit Meeting, June 8, 2024.

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47th European Cystic Fibrosis Conference

CFTR Transgene Expression in Airway Epithelial Cells Following Aerosolized Administration of the AAV-based Gene Therapy 4D-710 to Adults with Cystic Fibrosis Lung Disease

Jennifer L. Taylor-Cousar, et al. Presented at the 47th European Cystic Fibrosis Conference, June 6, 2024.

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ARVO 2024

Preclinical Characterization of 4D-175, a Novel AAV-Based Investigational Intravitreal Gene Therapy for Geographic Atrophy

Melissa A. Calton, et al. Presented at the Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), May 9, 2024.

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ARVO 2024

Interim Results from the PRISM Randomized Phase 2 Dose Expansion Trial Evaluating 4D-150 in High Anti-VEGF Need Individuals with Neovascular (Wet) Age-Related Macular Degeneration

Arshad M. Khanani, et al. Presented at the Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), May 8, 2024.

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WORLDSYMPOSIUM 2024

Phase 1/2 Clinical Trial Evaluating 4D-310 in Adults with Fabry Disease Cardiomyopathy: Interim Analysis of Cardiac and Safety Outcomes in Patients with 12–33 Months of Follow-up

Mark Thomas, et al. Presented at the 20th Annual WORLDSymposium Meeting, February 9, 2024.

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Angiogenesis, Exudation, and Degeneration 2024

First Interim Results (24 weeks) for the Randomized Phase 2 Dose Expansion Stage of the PRISM Clinical Trial of 4D-150 in High Need Patients with nAMD

Arshad M. Khanani presented at the Angiogenesis, Exudation, and Degeneration 2024 Conference, February 3, 2024.

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Gene Therapy for Ophthalmic Disorders Summit 2023

Targeting the Complement Pathway with AAV-Based Gene Therapy for Geographic Atrophy

Melissa Calton Presented at the Gene Therapy for Ophthalmic Disorders Summit, October 3-5, 2023.

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ASRS 2023

Interim Results of the Phase 1/2 PRISM Trial Evaluating 4D-150, a Dual-Transgene Intravitreal Genetic Medicine for Neovascular (Wet) Age-Related Macular Degeneration

Christine N. Kay, et al. Presented at the American Society of Retinal Specialists (ASRS) Annual Meeting, July 29, 2023.

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ECFS 2023

AAV mediated gene therapy for cystic fibrosis: Interim results from a Phase 1/2 clinical trial

Jennifer L. Taylor-Cousar, et al. Presented at the European Cystic Fibrosis Society (ECFS) 46th Annual Meeting, June 8, 2023.

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ASGCT 2023

AAV-Mediated Gene Therapy for Cystic Fibrosis (4D 710)

Jennifer L. Taylor-Cousar, et al. Presented at the 26th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 18, 2023.

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ARVO 2023

A Phase 1/2 Clinical Trial Evaluating 4D-150, a Dual-transgene Intravitreal Gene Therapy in Patients with Wet Age-Related Macular Degeneration: Interim Results

Arshad M. Khanani, et al. Presented at the Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), April 27, 2023.

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NACFC 2022

Update on AAV-mediated CFTR gene delivery programme (4D-710)

Jennifer L. Taylor-Cousar, et al. Presented at the North American Cystic Fibrosis Conference (NACFC), November 3, 2022.

More Video

NACFC 2022

Plenary 2: Novel CF Therapeutics & Clinical Trial Strategy to Accelerate Our Mission

Deepika Polineni, MD, MPH, and Nicole Mayer-Hamblett, PhD Presented at the North American Cystic Fibrosis Conference (NACFC), November 4, 2022.

Video

WORLDSYMPOSIUM 2022

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males with Fabry Disease

Jerry Vockley, et al. Presented at the 18th Annual WORLDSymposium Meeting, February 9, 2022.

More

ASGCT 2021

A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression & Safety in Non-Human Primates

Kevin Wittlesey, et al. Presented at the 24th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 11-14, 2021.

More

ASGCT 2021

A Multi-Mechanistic Anti-Angiogenic AAV Gene Therapy Product Candidate, 4D-150, for the Treatment of Wet Age-Related Macular Degeneration (Wet AMD) and Diabetic Macular Edema (DME): Intravitreal Biodistribution, Transgene Expression, Safety and Efficacy in Non-Human Primates

Peter Francis, et al. Presented at the 24th Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 11-14, 2021.

More

ATS 2021

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

Melissa A. Calton, et al. Presented at the American Thoracic Society (ATS) 2021 Virtual Conference, May 14-19, 2021.

More

ASGCT 2020

Directed Evolution of AAV Targeting Lung Epithelia Using Aerosol Delivery Identifies 4D-A101, a Variant Demonstrating Robust Gene Delivery in Non-Human Primates

Melissa Kotterman, et al. Presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.

More

ASGCT 2020

In Vitro Fabry Disease Correction in Patient iPSC-Derived Cardiomyocytes and Endothelial Cells Using an Evolved and Optimized AAV Gene Therapeutic (4D-310)

Chris Schmitt, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.

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ASGCT 2020

Highly-Evolved Novel AAV Gene Therapy Directly Addresses Fabry Disease Pathology In Vivo by Cell Autonomous Expression in the Heart and Other Target Organs

Kevin Wittlesey, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.

More

ASGCT 2020

4D-C102, a Novel Muscle-Tropic AAV Variant Demonstrates Superior Gene Delivery in Cardiac and Skeletal Muscle Tissues Versus Wild-Type AAV in Human Cells and Non-Human Primates

Melissa Kotterman, et al. Poster presented at the 23rd Annual American Society of Gene & Cell Therapy (ASGCT) Meeting, May 12-15, 2020.

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