Our platform utilizes cutting-edge technologies to efficiently deliver one-time precision treatments to the central nervous system (CNS). Our therapeutic antibodies are delivered as a transgene to the target cells by a viral vector, resulting in long-term (years) expression after a single administration. The antibodies are designed to specifically bind the toxic variant of the protein or lipid and not the normal variant, these targets can be located inside or outside of a cell. Vectorization of the antibody enables us to overcome the limitations of current therapies including wide-spread CNS transduction, durability, and (intracellular) target accessibility.

With the application of VecTab technology a pathogenic protein or toxic lipid is cleared from the CNS. An adeno-associated viral (AAV) vector delivers the VecTab transgene to the neuron where the therapeutic antibody (VecTab) is subsequently produced. Binding of the VecTab clears the toxic variant to modify the disease.

The VecTron technology comprises of a vectorized antibody fused with a degron sequence that directs the target towards pathway specific degradation. An adeno-associated viral (AAV) vector delivers the VecTron transgene to the neuron where the therapeutic antibody-degron fusion protein (VecTron) is subsequently produced. Binding of the VecTron directs the toxic variant towards a specific degradation pathway (in this example ubiquitination and subsequent proteasome degradation).

For the delivery of the antibody transgene, we use AAV vectors. This validated delivery tool is widely used in gene therapy but, significant challenges remain in its clinical use, including the broad tropism and the poor penetration of the blood brain barrier. Therefore, we developed a modular platform to generate new viral capsids (VeCaps) with superior tropism for the central nervous system (CNS).

We have established a modular platform aimed at generating VeCaps, next generation tissue specific recombinant AAV (rAAV) capsids. We have designed a rational approach to engineer the rAAV capsid utilizing protein structural engineering to optimize the transduction of a specific tissue type. With this technology we aim to optimize our AAV delivery system for highly efficient manufacturing and​ wide-spread CNS transduction, resulting in long-term (intracellular) expression of the antibody after a single administration.

Manufacturability​ of AAV-based therapies remains an industry challenge as current systems do not allow for large scale production. We have developed scalable processes on a novel production system (ManuVec) to consistently produce high vector yields and reduce cost.

Our AAV capsid production system on the Baculovirus/insect cell platform generates viral vectors of high quality and potency. Production can be scaled to large quantities (up to 2000L) with far higher yields and at lower cost than the industry standard.