4D Molecular Therapeutics

Company at a glance

• Late-stage biotechnology company focused on targeted genetic medicines using adeno-associated virus (AAV) vectors.
• Core platform: Therapeutic Vector Evolution (TVE), a directed-evolution approach that creates customized AAV capsids to deliver transgenes to specific tissues with improved performance and reduced immune concerns.

Therapeutic focus and product areas

• Retinal diseases
  • 4D-150: Lead retina program using the R100 vector; encodes anti-VEGF biologics (aflibercept) and VEGF-C RNAi for a potentially long-lasting backbone therapy delivered by in-office intravitreal injection.
  • 4D-175: Geographic atrophy (GA) program; IND is open; further development planned after financing or partnerships.
• Pulmonary diseases
  • 4D-710: Cystic fibrosis (CF) lung disease program using the A101 vector with aerosol delivery; designed for durable and broad CFTR restoration; currently in Phase 1/2 (AEROW trial).
  • 4D-725: Alpha-1 antitrypsin deficiency (AAT) lung disease program in preclinical development; IND filing supported by funding from the California Institute for Regenerative Medicine (CIRM).
• Platform and pipeline
  • TVE has produced hundreds of synthetic AAV capsids and is being applied to multiple tissue targets, including retina, lung, and heart, with parallel capabilities to support additional product candidates.

Clinical development status (highlights)

• 4D-150 (retina)
  • PRISM Phase 1/2 data and two Phase 3 4FRONT trials (4FRONT-1 in North America; 4FRONT-2 global) for wet AMD and DME.
  • RMAT designation in the U.S. for wet AMD and DME; PRIME designation in the EU for 4D-150.
  • 4FRONT-1 enrollment completed ahead of projections with >500 randomized; topline data anticipated in 2027 (52-week primary endpoint). 4FRONT-2 enrollment expected to complete in 2026; topline data expected in 2027.
• 4D-150 for DME (SPECTRA)
  • Phase 1/2 results selected 3E10 vg/eye as the Phase 3 dose. A single global Phase 3 trial is planned with 300–400 patients and a primary endpoint of BCVA noninferiority versus aflibercept; initiation planned for 2026 with topline data in 2027.
• 4D-710 (CF)
  • AEROW Phase 1/2 ongoing. December 2025 interim data showed dose-dependent CFTR transgene RNA expression and early clinical activity. A dose of 2.5E14 vg was selected for Phase 2. Data indicate durable CFTR expression for up to about one year in biopsies.
• Regulatory progress
  • As of March 2026, seven INDs have been submitted and received FDA clearance to proceed to clinical development.
  • The company maintains regulatory engagement and alignment across jurisdictions for its Phase 3 and late-stage programs.

Intellectual property and collaborations

• Intellectual property
  • Solely owned portfolio: 18 granted U.S. patents and 135 granted foreign patents; patent expirations roughly 2037–2042 (subject to extensions). Pending: 16 U.S. non-provisional and 169 foreign applications.
  • In-licensed (UC Berkeley): 5 granted U.S. patents and 21 granted foreign patents related to AAV vectors (including A101) and related technologies.
  • In-licensed (UPenn): 2 granted U.S. patents and 10 foreign patents related to the sCFH payload for 4D-175.
• Licenses and collaborations
  • Otsuka: Exclusive rights to develop and commercialize 4D-150 for retinal vascular diseases in Japan, China, Australia, and APAC. Otsuka provided $85.0 million upfront, funds development, and may pay up to $335.5 million in regulatory/commercial milestones plus double-digit royalties. 4DMT retains global rights outside APAC.
  • Cystic Fibrosis Foundation (CFF): Multi-year support including grants and convertible investments; 2025 CFF activity included purchase of 776,398 shares for $7.5 million and a new $3.6 million investment contingent on milestones. A Joint Steering Committee guides 4D-710 development.
• Other IP considerations
  • The company depends on multiple licenses that include milestones, royalties, and diligence obligations; terminations or breaches could affect development.

Manufacturing and operations

• In-house cGMP capabilities
  • Emeryville, California site with upstream and downstream manufacturing, fill/finish, analytical development, QC, and GLP/toxicology support.
  • Experience producing over 300 unique AAV vectors and more than 500 vector lots for research and clinical use; 28 clinical trial material lots released for six product candidates.
• Third-party manufacturing
  • CDMOs engaged to supplement internal capacity. Tech transfer is underway with a leading global commercial CDMO to support potential 4D-150 commercialization.
• Process and supply risks
  • Dependence on plasmid suppliers and other third parties for raw materials; supply interruptions could affect development timelines.
• Workforce
  • As of March 6, 2026: 196 full-time employees (144 in R&D; 39 with MD or PhD degrees).

Financial position and liquidity

• Cash position
  • $514.0 million in cash, cash equivalents, and marketable securities as of December 31, 2025.
• Revenue and profitability
  • No product revenue to date. Recurring net losses driven by R&D and operating expenses.
  • Net losses: $140.1 million (2025) and $160.9 million (2024).
  • Accumulated deficit: $716.3 million as of December 31, 2025.
• Runway and funding outlook
  • Existing resources were expected to fund operations for at least one year from the date of the financial statements.
  • Further fundraising and collaborations are anticipated to finance continued development; additional financing could dilute existing stockholders.
• Dependence on external funding
  • The business plan contemplates continued reliance on collaborations, equity offerings, debt financing, and government or charitable support to fund ongoing development and future commercialization.

Corporate information

• Founded: 2013
• Headquarters: Emeryville, California
• Public filings: Information summarized here is drawn from the company’s Form 10-K and public disclosures.