Neurological Disorders

Inherited neurological disorders are conditions that can be progressive and debilitating, causing problems with balance, walking, sensation (numbness), and other symptoms.

Historically, neurological disorders have presented a unique challenge for drug development. Creating safe and effective drugs for diseases involving the spinal cord requires identifying highly targeted therapies and delivering them to nervous tissues and compartments in the body.

Treatments have been limited and those that are available focus on symptom management, rather than targeting the root cause of the disease.

Our initial focus is on spinal cord-related disorders, beginning with adrenomyeloneuropathy (AMN).

Our vision is to enhance our understanding of the origin and management of spinal cord-related disorders where therapeutic DNA has the potential to treat these debilitating illnesses.


AMN is a progressive and debilitating neurodegenerative disease caused by alterations in the ABCD1 gene. These genetic changes disrupt the function of spinal cord cells and other tissues.

AMN is characterized by loss of mobility in adulthood, incontinence, debilitating pain, and sexual dysfunction, which all affect quality of life.

Symptoms of AMN typically begin in men between the ages of 20 and 30. Women can experience symptoms later in life.

  • Early symptoms for people living with AMN include leg weakness, such as difficulty walking and running and balance issues.
  • They may experience frequent falls and may be unable to walk even short distances.
  • As the disease progresses and becomes more debilitating, most people with AMN require a wheelchair.
  • Later in the progression of AMN, bladder and bowel dysfunction have a significant impact on quality of life.
  • Beyond physical symptoms, people living with AMN report economic hardship and loss of dignity and self-esteem as the disease progresses over time.
  • Our studies of real-world data shows that men and women with AMN experience more comorbidities, higher rates of hospitalization, substantially more prescription fills, higher healthcare costs, and poorer clinical outcomes than their peers.

There are currently no approved treatments to slow or alter the progression of AMN. Standard of care is symptom management, physical therapy, and mobility aids.

Gene Therapy

Our approach to treating disease builds on advances in the delivery of gene therapy and targets the root cause of disease.

Living With AMN

Through our team’s collective decades of experience, we know that people living with neurological disorders can’t wait.