4D Molecular Therapeutics
Unlocking the full potential of genetic medicine to treat large market diseases
Key Highlights
- Sought after leader in genetic medicines, with proprietary Nobel Prize-winning technology: 4DMT is applying the principles of directed evolution to invent novel, next generation AAV vectors to combat the stigma and shortcomings of conventional vectors within the genetic medicine space. Their work is attracting top pharmaceutical companies seeking to partner with the company and to license 4DMT’s vectors, including Astellas and Arbor Biotechnologies.
- Quality synthetic vectors fit for purpose:Phase 2 clinical data show encouraging safety and efficacy results and supports the therapeutic potential of 4DMT’s genetic medicines in retinal diseases and cystic fibrosis lung disease. Each 4DMT candidate has been designed with optimized capsids that are delivered by the routine route of administration for each disease – including intravitreal for eye diseases and aerosol for cystic fibrosis.
- Key Events in 2024:
- Recent upsized public offering of $300 million supplemented cash position of $299 million, extending company operational runway into H1 2027
- Positive interim results from Phase 2 trial for wet AMD enable rapid advancement towards pivotal development, with additional FDA and EMA regulatory interactions planned in Q2 2024, updates expected in Q3 2024, and clinical trial initiation expected in Q1 2025
- Initial interim data of Phase 2 study in DME expected in H2 2024
- Dose selection and initiation of Phase 2 expansion cohort in cystic fibrosis expected in Q2 2024, with pivotal trial initiation anticipated in H2 2025
- Expected IND filing of 4D-175 for Geographic atrophy (GA) in Q2 2024; Phase 1 initiation expected in H2 2024