Our Pipeline

Dravet syndrome (DS) is a severe, developmental and epileptic encephalopathy that begins in infancy, and is characterized by frequent, treatment-resistant and prolonged seizures, significant developmental delays, movement and balance issues, and sleeping and behavioral difficulties. DS occurs in approximately 1 in 16,000 births worldwide, with most cases resulting from loss-of-function variants in the SCN1A gene.

SCN1A

Cell Selective targeting (GABAergic neurons), endogenous gene upregulation (eTF)

Dravet Syndrome Foundation.

Angelman syndrome (AS) is a severe neurodevelopmental disorder that becomes noticeable by the age of 6 – 12 months, and is characterized by limited to no speech, walking and balance disorders, disturbed sleep, and seizures. AS occurs in approximately 1 in 15,000 births worldwide and is caused by the loss-of-function of the maternally-inherited UBE3A gene.

UBE3A antisense transcript (UBE3A-ATS)

Unsilencing

Angelman Syndrome Foundation.

Alzheimer’s disease is a progressive neurodegenerative condition that affects memory, thinking and behavior. The symptoms of dementia associated with Alzheimer’s disease gradually worsen over a number of years. In its early stages, memory loss is mild, but with late-stage disease, individuals lose the ability to communicate and respond to their environment. Alzheimer’s disease is estimated to affect 6 million people in the U.S.

Intracellular tau

miRNA knockdown

Alzheimer’s Association.

Chronic neuropathic pain affects millions of patients in the U.S., with prevalence estimates between 3% and 17% of the general population. It may be caused by spinal vertebral compression or radiculopathy, diabetes, infections (such as herpes zoster), and nerve damage. Patients experience constant, burning pain as well as stabbing, sudden attacks. Unlike acute pain, chronic neuropathic pain is poorly responsive to anti-inflammatory drugs and opioids. Chronic pain can be very severe, intractable, and disabling, and is associated with significant psychological and social consequences, which contribute to a reduction in quality of life.

SCN9A (Na_V1.7)

miRNA knockdown

Van Hecke, O., Austin, S. K., Khan, R. A., Smith, B. H., & Torrance, N. (2014). Neuropathic pain in the general population: A systematic review of epidemiological studies. Pain, 155(4), 654–662.

Brewer, R. P., Reznik, M., & Zajaczkowski, W. J. (2004). Contemporary management of neuropathic pain for the primary care physician. Mayo Clinic Proceedings, 79(12), 1533–1545.

Lucas, J. W., & Sohi, I. (2024, November). Chronic pain and high-impact chronic pain in U.S. adults, 2023 (NCHS Data Brief No. 518). Centers for Disease Control and Prevention. (Accessed April 12, 2024)