4D Molecular Therapeutic Inc.

• US
• 4D Molecular Therapeutic Inc.
• Health-Care

FDMT

We are a development stage precision gene therapy company dedicated to pioneering the development of targeted therapies based on our next-generation AAV vectors. Our proprietary Therapeutic Vector Evolution platform enables our “disease first” approach to product discovery and development, thereby allowing us to customize our AAV vectors to target specific tissue types associated with the underlying disease.

Our proprietary AAV vectors are designed to provide targeted delivery by routine clinical routes, efficient transduction, reduced immunogenicity and resistance to pre-existing antibodies, which we believe will enable us to develop gene therapies that could overcome known limitations of conventional AAVs. As a result, we believe these key attributes will enable us to develop gene therapies with improved therapeutic profiles, pursue previously untreatable diseases and address a broad range of rare and large market diseases. Our product candidates that are in or have completed Investigational New Drug (IND)-enabling studies include a wholly-owned asset in development for the treatment of Fabry disease, as well as two assets in ophthalmology. In ophthalmology our product candidate for the treatment of X-linked Retinitis Pigmentosa (XLRP), is wholly-owned subject to an exclusive option for Roche to develop and commercialize the asset, and our product candidate for the treatment of Choroideremia is licensed to Roche. In addition our wholly-owned product candidate for the treatment of Cystic Fibrosis has completed lead optimization and we expect to initiate IND-enabling studies in the first half of 2020. We expect to initiate our first clinical trials for these product candidates in 2020. We also have a pipeline of product candidates in the lead optimization stage, including for the treatment of Duchenne Muscular Dystrophy (DMD) and Wet Age-Related Macular Degeneration (Wet AMD).

While gene therapy holds tremendous promise as a transformative therapeutic class, the majority of gene therapeutic approaches utilize naturally-occurring or conventional AAV vectors that have encountered limitations such as high dose requirements, limited efficacy, inflammation and toxicity, and neutralization by pre-existing antibodies. As a result of these shortcomings, current efforts with conventional AAV vectors have focused on diseases in which a surgical technique is used for direct cell access, such as subretinal injection for the retina, or a low level of gene expression may be sufficient for patient benefit, such as Hemophilia A or B treatment. Rather than using conventional AAV vectors for our products, we instead take a “disease first” approach creating proprietary customized AAV vectors for each disease indication through our Therapeutic Vector Evolution platform. Our Therapeutic Vector Evolution platform is based on directed evolution, a high-throughput biological engineering and screening approach harnessing the power of natural selection to identify novel AAVs with desirable characteristics. As of September 1, 2019 we have a total of 37 distinct libraries, with an estimated over one billion vector sequences. We select customized AAVs based on the Target Vector Profile (TVP) for the disease and we subsequently characterize each lead vector through extensive studies in non-human primates (NHPs) and proprietary human cell and organotypic tissue assays. Based on our preclinical data to date from NHPs and human cell models, including preclinical head to head comparisons with conventional AAVs, we observed that our precision AAVs were well-tolerated and achieved enhanced delivery, increased transgene expression, reduced immunogenicity and/or improved antibody resistance when compared to therapies using conventional AAVs. 

Offering Team

Pre-IPO Investors

Deal Highlights

less

More

Deal Tracker

Stock Price