Sania Therapeutics presents latest from AAV gene therapy platform at the American Society of Gene & Cell Therapy Annual Meeting

Abstracts outline an approach which is highly translatable, efficacious at low doses and purposefully designed to be accessible by large patient populations

First indication in spasticity a blueprint for other prevalent nervous system disorders

LONDON, UK & NEW YORK, USA – 23 April 2024, Sania Therapeutics (the “Company”), focused on developing targeted genetic medicines for prevalent disorders, will be sharing the latest on its adeno-associated viral (AAV) gene therapy approach in three posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 Annual Meeting, taking place from 7-11 May 2024 in Baltimore, MD.

Andy Murray, Ph.D, CEO & Co-Founder of Sania Therapeutics, said: “Since our official launch last May, we have been pressing ahead with developing truly differentiated AAV gene therapies. The three abstracts published today outline how our human-centric, targeted approach, underpinned by the R-Scan platform, is helping us reimagine gene therapy, in a field facing challenges of translatability and safety. We look forward to providing updates as we develop our first application in spasticity, as well as exploring how we can harness neural circuits to treat other disorders.”

Title: Generating human-evolved, cell-type-specific AAV capsids for targeted gene deliveryAbstract / Date / Time / Session: 503 / 8 May 2024 / 12:00 pm / AAV Vectors – Capsid EngineeringA major drawback of the current generation of AAV treatments is that the tropism of a particular vector can vary from one animal species to the next, which hinders its translatability. Sania’s approach, using its R-Scan platform, is focused on generating novel, targeted vectors that can be successfully and rapidly translated into the clinic. It combines human induced pluripotent stem cell derived cell types and microfluidics to perform directed AAV capsid evolution in an in vitro human system.

In the lead spasticity program, R-Scan has been used to evolve AAV vectors targeting motor neurons directly via an intramuscular injection, which show superior transduction of both human motor neurons in vitro and human neuromuscular organoids. In addition, the capsids generated by the platform show efficient transduction of mouse motor neurons following intramuscular injection, demonstrating translatability from our in vitro system to whole animal. Work is underway to develop R-Scan to generate human-evolved vectors for multiple neuronal circuits and cell types.

Title: Titratable control of neuronal activity using precision genetic neuromodulationAbstract / Date / Time / Session: 1606 / 10 May 2024 / 12:00pm / Neurologic DiseasesMany neurological diseases are linked to the abnormal electrical activity of specific neural circuits. Gene therapies could provide a route to targeting these malfunctioning neural circuits. Sania’s precision therapy for neural circuits leverages a combination of technology platforms that allow for the targeted delivery of chemogenetic proteins through engineered AAV vectors. An investigation was carried out into the efficacy of SRx-C490, its lead chemogenetic ion channel candidate, and its impact on modulating excitability in two distinct sub-types of human induced pluripotent stem cell (iPSC) derived neurons: motor neurons and sensory neurons.

The AAV-delivered SRx-C490 reduced motor neuron excitability by 80% and sensory neuron excitability by 75%, whilst having a minimal impact on baseline activity. This impact on excitability could be beneficial in a range of neurological disorders, when combined with Sania’s cell-type specific AAV capsids.

Title: A low dose, targeted, and controllable gene therapy for the treatment of spasticityAbstract / Date / Time / Session: 1140 / 9 May 2024 / 12:00 pm / Neurologic DiseasesSRx-T001 is Sania’s gene therapy in development for the treatment of spasticity. It combines an intramuscular injection of AAV gene therapy with oral medicine to selectively reduce excitability in targeted motor neurons.

Using a novel AAV vector, Sania has been able to drive overexpression of a human ion channel selectively in spastic motor neurons. This overexpression enables it to be susceptible to low doses of an oral small molecule, which in turn binds to the ion channel and reduces neuronal excitability.

By combining unique vectors engineered in human motor neurons, targeted administration, and a novel chemogenetic system, Sania is developing a gene therapy with a high safety profile and high specificity, that can be delivered cost effectively. It also provides a blueprint for future nervous system disorders.

All three abstracts are available on the ASGCT website. The posters will be made available on the Sania website following their presentation.

About Sania Therapeutics Sania Therapeutics is a ground-breaking biotechnology company pioneering new approaches for targeted and controllable gene therapies to treat prevalent disorders. Sania's approach harnesses neural circuits as a gateway to disease states. Their research and development is centred around a human-first approach to ultimately build more translatable, safer and cost-effective AAV gene therapies capable of treating millions of patients. Sania is based in London, UK. For more information visit www.saniarx.com.