Technologies
This technology is a gene therapy platform that selectively re-expresses the developmental transcription factors Islet1 and Lhx3 in adult motor neurons, restoring youthful resilience and protecting against neurodegeneration.
Motor neuron degeneration occurs in a range of disorders- including amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), hereditary motor neuropathies, and age-associated motor decline. These conditions currently lack curative, cell-type-specific therapies. Existing treatments offer limited benefits and typically fail to modify the intrinsic vulnerability of adult motor neurons, which lose resilience with age. There is a significant need for motor-neuron-targeted therapeutic strategies that restore a youthful, protective transcriptional program and avoid off-target effects in other tissues.
This technology employs an adeno-associated virus (AAV)-based gene therapy method to re-express the Islet1 and Lhx3 transcription factors, thereby enhancing resilience against motor neuron degeneration. Since Islet1 and Lhx3 are normally expressed prenatally during spinal motor neuron development and are downregulated postnatally, increasing their expression in aged or vulnerable adult motor neurons can help revert these cells to a more youthful, resilient state, thereby preventing degeneration and minimizing off-target effects.
This technology has been validated in mouse models of ALS.
Patent Pending (WO/2024/155467)
IR CU23184, CU23208
Licensing Contact: Jerry Kokoshka