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)

miRNA knockdown

Angelman Syndrome Foundation.

Lennox-Gastaut syndrome (LGS) is a severe epilepsy syndrome that develops in young children and may lead to lifelong disability. The core features of LGS are seizures that begin in early childhood, multiple seizure types, including tonic seizures, abnormal EEG and developmental delay and/or intellectual disability. LGS can be caused by a variety of underlying conditions, and in some cases no cause can be identified. Seizures associated with LGS are difficult to treat and often refractory to anti-seizure medications. Lennox-Gastaut syndrome is estimated to affect 50,000 people in the U.S.

Undisclosed

Cell Selective targeting (GABAergic neurons)

LGS 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.

Neuropathic pain can be caused by various etiologies affecting the nervous system, including inflammation, trauma or compression, and chemical-induced nerve damage. Independent of cause, patients suffering from neuropathic pain often display similar symptoms, consisting of a burning constant pain with stabbing sudden attacks. Neuropathic pain is poorly responsive to anti-inflammatory drugs and opioids. Since chronic neuropathic pain can be very disabling, severe and intractable, it is associated with significant psychological and social consequences, which contribute to a reduction in quality of life.

SCN9A (Na_V1.7)

miRNA knockdown

Medscape.