4D Molecular Therapeutics

What 4D Molecular Therapeutics does

4D Molecular Therapeutics (NASDAQ: FDMT) is a clinical-stage biotechnology company founded in 2013 and headquartered in Emeryville, California, that designs, develops, and intends to commercialize AAV-based gene therapies for large-market diseases with significant unmet need, primarily in ophthalmology and pulmonology. The company's foundation is its proprietary Therapeutic Vector Evolution platform, which uses directed evolution to invent customized viral vectors with superior tissue targeting, lower dose requirements, and resistance to pre-existing human antibodies compared to conventional AAV; this platform has produced two flagship vectors, R100 (designed for routine low-dose intravitreal delivery to the retina) and A101 (designed for efficient aerosol delivery throughout the lung airway).

The clinical pipeline is anchored by 4D-150, an intravitreal gene therapy expressing two anti-VEGF transgenes for wet age-related macular degeneration and diabetic macular edema, which is being evaluated in the 4FRONT-1 and 4FRONT-2 Phase 3 trials and showed up to a 92% reduction in anti-VEGF injection burden in earlier data. 4D-710 is a mutation-agnostic aerosolized CFTR gene therapy for cystic fibrosis lung disease in Phase 2 with positive interim Phase 1 data; 4D-310 targets Fabry disease cardiomyopathy in Phase 1/2; and preclinical assets 4D-175 (geographic atrophy) and 4D-725 (alpha-1 antitrypsin deficiency lung disease) extend the franchise across the same vectors.

The business model is currently pre-commercial: revenue consists of partnership and licensing income rather than product sales. The company has executed a $85 million upfront (plus up to $336 million in milestones) Asia-Pacific license to Otsuka Pharmaceutical for 4D-150, a separate up-to-$962 million Astellas license for a rare ophthalmology vector, and a $11 million grant plus Joint Steering Committee partnership with the Cystic Fibrosis Foundation supporting 4D-710. Future revenue is expected to shift toward specialty pharmaceutical distribution of gene therapy products priced in the $60,000–$350,000 per-patient range, distributed via specialty pharmacies, hospital systems, and specialty distributors, complemented by regional partnerships such as Otsuka for Asia-Pacific markets.

Name

4D Molecular Therapeutics

Legal name

4D Molecular Therapeutics, Inc.

Website

https://4dmoleculartherapeutics.com

Company type

Public

Founded year

2013

Operating status

Operating

Headcount range

101–250 employees

Short description

4D Molecular Therapeutics (NASDAQ: FDMT) is a clinical-stage biotechnology company that designs and develops customized AAV-based gene therapies for large-market retinal and pulmonary diseases using its proprietary Therapeutic Vector Evolution platform, currently advancing lead candidates 4D-150 and 4D-710 through Phase 3 and Phase 2 trials.

Ownership category

akta.pro rank

Product category

Gene Therapy / Genetic Medicines

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Ophthalmology Disorder Therapies (HLAIAIAK)

akta.pro secondary industries

Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG), Personalized Therapeutic Design (neoantigen vaccines, individualized cell/gene therapies) (HLAAANAK), Alpha-1 Antitrypsin (AAT) Therapies (HLAIAHAD)

Keywords

• Gene therapy products
• AAV vector platform
• Retinal disease therapeutics
• Cystic fibrosis treatment
• Ophthalmic gene therapy

Headquarters

HQ city

Emeryville

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales, Supply Chain

Revenue model

1. Gene Therapy Product Sales: Pre-commercial company developing gene therapies. Primary revenue will come from commercializing lead product 4D-150 for wet AMD and DME, and 4D-710 for cystic fibrosis. As a late-stage biotech, revenue model will shift from partnership/milestone payments to product sales upon regulatory approval.
2. Partnership & Licensing Revenue: Strategic licensing deals including $85M upfront from Otsuka for Asia-Pacific rights to 4D-150, with potential milestone payments up to $336M. Includes upfront payments, development milestones, regulatory milestones, and sales royalties.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

4D Molecular Therapeutics (4DMT) is a clinical-stage biotechnology company that designs, develops, and commercializes AAV-based gene therapy products using its proprietary Therapeutic Vector Evolution platform. The company's pipeline includes 4D-150 for wet AMD and diabetic macular edema (Phase 3), 4D-710 for cystic fibrosis lung disease (Phase 2), 4D-310 for Fabry disease cardiomyopathy (Phase 1/2), and preclinical candidates 4D-175 for geographic atrophy and 4D-725 for alpha-1 antitrypsin lung disease.

Product overview

4D Molecular Therapeutics is a clinical-stage genetic medicines company developing a diversified pipeline of AAV-based gene therapies using its proprietary Therapeutic Vector Evolution Platform. The company's clinical-stage products include 4D-150 (wet AMD and DME, Phase 3), 4D-710 (cystic fibrosis, Phase 2), and 4D-310 (Fabry disease, Phase 1/2). Preclinical candidates include 4D-175 for geographic atrophy and 4D-725 for A1AT lung disease. The platform enables invention of customized vectors (R100 for retina, A101 for lung) that overcome limitations of conventional AAV vectors through directed evolution.

Differentiator

Problem solved

Functional benefit

Products and services

• 4D-150 AAV-based gene therapy for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). Delivered via intravitreal injection, designed to provide continuous expression of two anti-VEGF transgenes from retinal cells for durable suppression of VEGF family members.
• 4D-710 Investigational genetic medicine using A101 vector for cystic fibrosis lung disease. Delivered via aerosol, designed for single-dose delivery of corrective CFTRΔR transgene throughout airways and alveoli for mutation-agnostic disease-modifying benefit.
• 4D-175 AAV-based intravitreal gene therapy for geographic atrophy. Combines R100 vector with sCFH transgene encoding shortened complement factor H to restore complement regulation and slow disease progression.
• 4D-725 Investigational genetic medicine candidate using A101 vector for alpha-1 antitrypsin (A1AT) lung disease. Designed for single-dose aerosol treatment, currently in preclinical development.
• 4D-310 Gene therapy candidate for Fabry disease cardiomyopathy. Uses AAV vector to deliver functional GLA gene for treatment of Fabry disease, currently in Phase 1/2 clinical trials.

Quantifiable outcome

• 92% reduction in anti-VEGF injection burden in wet AMD patients
• +3 more outcomes

Named customers3 records

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Three partnerships are on record, tiered core and minor.

• Otsuka Pharmaceutical Co., Ltd.coreChannel Partner/ Reseller/ Distributor · 31 October 2025Exclusive license agreement for development and commercialization of 4D-150 for retinal vascular diseases in Asia-Pacific region including Japan, South Korea, Taiwan, Australia, and New Zealand. 4DMT received $85 million upfront payment with potential milestone payments up to $336 million plus double-digit royalties. Otsuka handles development, regulatory, and commercial activities in the region.
• Arbor BiotechnologiesminorStrategic or Co-development Partner · 3 January 2024Partnership to develop genetic medicines for CNS diseases using combined technologies.
• Astellas PharmaminorOEM/ Whitelabel/ Licensing Partner · 10 July 2023Licensing agreement for 4DMT's viral vector for rare ophthalmology gene therapy. Deal valued at up to $962 million including upfront, development, and commercial milestones plus royalties.

Scale indicators9 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Krystal Biotech: Commercial-stage gene therapy company (Vyjuvek) with a redosable HSV-based platform and a pipeline targeting rare diseases. Comparable in being a genetic medicines company with rare disease focus and in-house manufacturing.
• MeiraGTx Holdings: Clinical-stage gene therapy company with programs in ophthalmology (AAV for inherited retinal diseases) and rare pulmonary/CF indications. Comparable diversified AAV gene therapy pipeline with in-house manufacturing.
• uniQure N.V. Clinical-stage gene therapy company developing AAV-based therapies for rare diseases including Huntington's disease and hemophilia B. Comparable business model as a clinical-to-commercial gene therapy developer.
• REGENXBIO: Clinical-stage gene therapy company with an AAV gene delivery platform (NAV Technology) developing therapies for retinal diseases (wet AMD, DME) and rare diseases including CF. Closely comparable technology platform and pipeline overlap.
• Adverum Biotechnologies: Clinical-stage gene therapy company developing AAV-based therapies for ocular diseases, including wet AMD (ADVM-022/Ixo-vec) and other retinal indications. Direct competitor to 4DMT in intravitreal AAV gene therapy for wet AMD.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biopharmaceutical company with multiple approved enzyme replacement and gene therapy products (e.g., Roctavian for hemophilia A AAV gene therapy). Comparable as a broad rare disease player with gene therapy expertise and commercial infrastructure.
• Pfizer (Gene Therapy Division): Large pharmaceutical company with an active gene therapy portfolio (Beqvez for hemophilia B; prior 4DMT investor via Pfizer Ventures). Represents the strategic pharma landscape that licenses or competes with 4DMT in AAV gene therapy.
• Astellas Pharma (Astellas Gene Therapies): Global pharmaceutical company with a gene therapy center of excellence (formerly Audentes Therapeutics). Existing 4DMT licensing partner for rare ophthalmology AAV vector (up to $962M deal), demonstrating comparable interest in AAV gene therapy for ophthalmology and rare diseases.

Emerging players

• Editas Medicine: Clinical-stage gene editing company developing CRISPR-based therapies, with ophthalmology and rare disease focus (e.g., EDIT-101 for LCA10). Comparable as a clinical-stage genetic medicines company in ophthalmology.
• Beacon Therapeutics: Emerging gene therapy company focused on ophthalmic diseases, developing AAV-based therapies for inherited retinal diseases and wet AMD. Direct emerging competitor in retinal gene therapy space.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/4d-molecular-therapeutics-llc
• Xx.com/4dmolecular

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds9 records

Investors20 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about 4D Molecular Therapeutics