Clinical Programs

Our Clinical Programs are focused on the development of the HuCNS-SC® (purified human neural stem cells) platform technology as a treatment for multiple disorders of the central nervous system (CNS).

Learn more about HuCNS-SC platform technology…

Learn more about our clinical approach…

Clinical Pipeline

  • Preclinical
  • IND
  • Phase I or I/II
  • Phase II
Spinal Cord Injury
Thoracic Injury
Cervical Injury
Retinal Disorders
Dry Age-Related Macular Degeneration (AMD)
Neurodegenerative Diseases
Neuronal Ceroid Lipofuscinosis (NCL) 
Pelizaeus-Merzbacher Disease (PMD)
  • ■ Trial completed
  • ■ Trial suspended*
  • ■ Currently underway
  • *Pending funding TBD

The Challenge of CNS Disorders

Diseases and disorders of the central nervous system (CNS) afflict tens of millions of people worldwide, and the underlying pathology in many conditions is secondary to loss of or damage to CNS cells, which leads to neurological impairment and disability. For many of these conditions, there are no curative treatment options and even therapies aimed at effective symptom relief remain limited. Cell-based therapeutics offers a promising new approach for the preservation or restoration of CNS function, which could fundamentally alter the prognosis for disorders involving all three components of the CNS: the brain, the spinal cord and the eye. We believe that our HuCNS-SC platform technology holds the potential to treat, and in some cases possibly cure, a wide range of such diseases that continue to challenge conventional medical approaches.

Because neural stem cells are capable of self-renewal, a single transplantation may have the potential to meaningfully and durably restore function for the patient.

Clinical Trials

Spinal Cord Injury (SCI)

Age-related Macular Degeneration (AMD)

Pelizaeus-Merzbacher Disease (PMD)

Neuronal Ceroid Lipofuscinosis (NCL)