Technologies
This technology is a gene-targeting therapeutic approach that suppresses Cdkn2a expression to prevent premature muscle stem cell aging for spinal muscular atrophy (SMA) and other myopathies.
The current standard of care for spinal muscular atrophy (SMA) primarily focuses on restoring survival motor neuron (SMN) protein levels, which addresses motor neuron dysfunction but fails to adequately target muscle degeneration caused by intrinsic muscle stem cell defects. Without addressing these defects, disease progression persists, leading to diminished therapeutic outcomes and quality of life. There is a critical need for treatments that directly preserve muscle function and prevent degeneration to complement existing SMA therapies and more effectively combat myopathy.
This technology leverages gene-targeting interventions, such as siRNAs and Gapmers, to suppress Cdkn2a expression, effectively reducing RNA and protein levels associated with muscle stem cell senescence. By restoring the self-renewal capacity of muscle satellite cells, it prevents premature aging and exhaustion of these cells, addressing a key contributor to muscle degeneration in conditions like SMA and other myopathies.
This technology has been validated in mice models.
Patent Pending(US19/422,180)
Patent Pending(WO/2024/263971)
IR CU23330
Licensing Contact: Kristin Neuman