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Targeted Cdkn2a suppression to prevent muscle degeneration in spinal muscular atrophy

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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.

Unmet Need: Improving muscle preservation in SMA and myopathy therapies

Spinal muscular atrophy (SMA) is a pediatric muscular disorder caused by insufficient levels of survival motor neuron (SMN) protein. The current standard of care for SMA primarily focuses on restoring SMN protein levels to address motor neuron dysfunction. However, these treatments do not sufficiently address muscle degeneration caused by intrinsic muscle stem cell defects. As a result, 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.

The Technology: Targeted Cdkn2a suppression to prevent muscle degeneration

This technology is a gene-editing method to prevent the aging of muscle stem cells in spinal muscular atrophy (SMA). It works by suppressing Cdkn2a expression, a gene that has been identified to induce muscular stem cell senescence. By introducing siRNAs and gapmers targeting Cdkn2a, the method effectively reduces RNA and protein levels associated with muscle stem cell aging. The restoration of the muscle satellite cells’ ability to self-renew prevents their premature aging and exhaustion, addressing a key contributor to muscle degeneration in conditions like SMA and other myopathies.

This technology has been validated with mice models.

Applications:

  • Therapeutics for spinal muscular atrophy (SMA) and other myopathies
  • Combination therapy involving SMN protein restoration and Cdkn2a suppression
  • Drug screening for Cdkn2a-related muscle stem cell senescence
  • Research tool for investigating the role of Cdkn2a in muscle stem cell biology
  • Studying muscle regeneration mechanisms in degenerative diseases

Advantages:

  • Complementary approach for treating SMA
  • Directly targets muscle degeneration
  • Broad applicability
  • Potential for minimally invasive delivery
  • Cost-effective

Lead Inventor:

Umrao R. Monani, Ph.D.

Patent Information:

Patent Pending (WO/2024/263971)

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