Transcriptional reprogramming of motor neurons for ALS

This technology is a gene therapy platform to reactivate two transcriptional factors, Islet1 and Lhx3, for increased resilience against ALS degeneration in motor neurons.

Unmet Need: Effective intervention for the prevention and treatment of ALS

Amyotrophic lateral sclerosis (ALS), a neurodegenerative disorder characterized by widespread motor neuron dysfunction and death, is a uniformly fatal disorder. The few treatment options available for people living with ALS provide only mild therapeutic benefits and may present off-target side effects. There are currently no cures for ALS.

The Technology: Genetic reprogramming of motor neurons to prevent ALS-induced motor neuron degeneration

This method uses AAV-based gene therapy to re-express Islet1 and Lhx3 transcription factors to increase resilience against motor neuron degeneration in people living with ALS. With this technology, targeted cell type specific increased expression of Islet1 and Lhx3 transcription factors can revert motor neurons to a state where they may be more naturally resilient to ALS disease, thus preventing the progression of the disorder and reducing off-target side effects.

This technology has been validated in mouse models of ALS.

Applications:

• Therapeutic treatment for ALS and other neurodegenerative disorders
• Preventative intervention against ALS and other neurodegenerative disorders
• Research tool for ALS pathology

Advantages:

• Directly targets Islet1 and Lhx3 transcription factors implicated in motor neuron development and degeneration
• Cell-type specific targeting of motor neurons
• Reduces off-target side effects
• Preventative intervention

Lead Inventor:

Hynek Wichterle, Ph.D.

Patent Information:

Patent Pending

Related Publications:

Tech Ventures Reference:

• IR CU23184, CU23208

• Licensing Contact: Jerry Kokoshka