Unconstrained by Nature

Beyond What Nature Created

Viruses have naturally evolved to infect cells and, in so doing, deliver their genetic contents into cells. Investigators in the field have engineered viruses for safety (removing their disease-causing capacity) and to use as shuttles for delivering genetic medicines (such genetic medicine delivery tools are called “vectors”). However, nature did not intend for viruses to be therapeutic vectors. For the past decade, 4DMT has been pioneering and industrializing the development of customized and improved viral vectors by using Nobel Prize-winning Directed Evolution technologies. Directed Evolution is a high-throughput platform approach that allows scientists to harness the power of natural selection and invent designer biologics with new and desirable characteristics. At 4DMT, we apply directed evolution principles to create our proprietary vector invention platform we refer to as Therapeutic Vector Evolution.

Therapeutic Vector Evolution Platform

The major hurdles in genetic medicine have been inefficient delivery into diseased tissues and cells, resulting in the need for high doses, expensive manufacturing, and the potential for toxicities (e.g., liver toxicity). Preexisting antibodies to conventional vectors also limit the utility of these medicines. We have leveraged our proprietary Therapeutic Vector Evolution platform to design and subsequently invent novel customized vectors that overcome the limitations encountered with genetic medicines that use conventional AAV vectors. We do this by applying the principles of Directed Evolution to invent targeted and evolved vectors with the precise features that we want for patient benefit. This enables us to deliver genetic material (e.g., transgenes) with our medicines to specific tissue types to treat a wide range of diseases.

We use our Therapeutic Vector Evolution platform to invent the novel synthetic capsid that best matches our Target Vector Profile (TVP). The TVP will define the cell types and intra-organ distribution to be targeted by the vector (and therefore the product), the route of administration, the desired dose range for delivery, and, if necessary, resistance to pre-existing antibodies in the human population.

Targeted Vectors Fit For Purpose

Through our proprietary Therapeutic Vector Evolution platform, each of our vectors in our clinical stage products has been invented with the goal of providing superior performance and clinical benefit in patients.


Designed for routine low dose intravitreal delivery to the retina (all layers and regions) for the treatment of retinal diseases. In contrast to conventional AAV vectors for the retina, R100 is a primate-evolved and customized intravitreal vector that treats the entire retinal surface at low doses and does not require sub-retinal surgery.


Designed for routine and efficient aerosol delivery to all major airway regions throughout the lung and to penetrate through the mucus barrier for transduction of lung airway cells, and for resistance to pre-existing antibodies in the human population. In contrast to conventional AAV vectors for the lung, A101 was invented for efficient delivery and transduction of human airway cells.


Designed for routine low dose intravenous (IV) delivery to muscle cells throughout the heart (cardiomyocytes). In contrast to conventional AAV vectors for the heart, C102 was invented for efficient delivery and transduction of human cardiomyocytes.

Product Design & Development Engine

Our powerful product design and development engine enables us to efficiently drive development of a diverse and deep pipeline of product candidates across multiple therapeutic areas. We have numerous open INDs, or IND equivalents, in the US and other countries. Our integrated engine seamlessly incorporates cross-functional teams in analytical and process development, GMP manufacturing, translational medicine, preclinical and clinical R&D. This engine has driven the development and progression of our product pipeline for both large market and rare disease indications. We believe the efficiency of our product engine is enabled by the modularity of our product candidates. By simply inserting different disease-specific payloads, we can leverage the same vector and manufacturing process for use in multiple products in the same therapeutic area.

Our Manufacturing Operations

Technical Operations

Our Technical Operations teams are led by experienced, innovative and team-oriented experts in manufacturing, process & analytical development. Our in-house capabilities include GMP manufacturing for clinical trial material, including Phase 3 & commercial scale production, IND-enabling studies and research candidate production.

Product Payloads

Our design and engineering team develops payloads (optimized promoters & transgenes) for insertion into our customized vectors. By inserting disease-specific payloads, we leverage our customized vectors for multiple products addressing diseases affecting the same target tissues. This modularity increases speed, reduces costs and decreases technical and regulatory risks.

Product development

Our product development teams have expertise in human cell and disease modeling, immunology, biomarkers, translational medicine and clinical R&D. These teams efficiently design and develop our preclinical and clinical stage product candidates.