Complex problems, bold solutions
Sania's science is positioned at the leading edge of the evolving neural genetic therapy landscape, and is set apart by an innovative approach to therapeutic development. Sania pioneers a novel approach called precision genetic neuromodulation to precisely and selectively target dysfunctional neurons and regulate their electrical activity for long-term therapeutic benefit. We harness our approach to build therapies that unlocks a broad range of diseases within and beyond neurology with the highest unmet needs.
Our pipeline leverages a combination of patented technology platforms that enable precision delivery of proteins for therapeutic neuromodulation of neural circuits. Key to this research and development is our novel human-centric approach, where the initial stages of therapeutic development occur entirely in human neural circuits. This permits breakthrough efficacy and ensures we can make the translational leaps from research in the lab to patients in the clinic.
At the root of many neurological dysfunctions lies faulty electrical signaling within specific neuronal pathways, or circuits, the fundamental components of the nervous system. Stabilising this anomalous activity without effecting healthy neurons has incredible potential to help treat neural dysfunction. With this in mind, Sania applies chemogenetics, a powerful technique that safely and accurately alters neuronal activity.
Identifying suitable chemogenetic candidates for different types of neurons means that Sania can build an adjustable 'remote control' for treating various nervous systems dysfunctions with minimal side effects.
PRECISION & TRANSLATION
Safe and effective genetic therapies require the precise delivery of genetic material to specific cells. Unfortunately, many promising therapies in this area fail to make the translational leap to humans.
Sania harnesses the power and safety of adeno-associated viral vectors (AAVs) and combines this with our own unique suite of human-centric technology platforms designed to drive high levels of translation from research in the lab to patients in the clinic.
By utilising a proprietary system of human stem cells, neural circuits and organoids to engineer novel AAV capsids and promotors, we can selectively and precisely target our therapeutics to specific cell types.
Sania's research and development emphasises translation into the clinic with programs to normalise disease states with pinpoint precision and without effecting neighbouring healthy neurons. Sania aims to maximise the impact of its technology by assessing where it can have the most clinical benefit in the largest number of patients. This is exemplified by our lead program in hyperexcitable motor dysfunction such as spasticity.
Sania's ability to target specific neural subtypes is our defining edge. We can harness this ability as an 'engine' that powers development of precision neuromodulation therapies for diseases beyond the current scope of genetic medicines where neural innervation plays a regulative role. Our initial focus is on dysfunctions that can be treated by local access to components of the peripheral nervous system, such as in spasticity and other undisclosed indications with high unmet need.