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.

Chronic pain can affect many aspects of life, often limiting physical function and overall quality of life. It is frequently associated with mood changes, including anxiety and depression, sleep disturbances, and often impacts social relationships and employment.

Post-amputation neuromas are a well-recognized source of chronic neuropathic pain in the more than 2 million Americans living with limb loss. A neuroma forms when a severed nerve attempts to heal without its original anatomical pathway, resulting in a disorganized bundle of nerve fibers. Neuromas can become hyperexcitable, generating signals, spontaneously or in response to light touch, that may be perceived as burning, electric or stabbing pain.

Because this pain is driven by over-activity of damaged nerves, it is often poorly responsive to conventional pain medication such as anti-inflammatory drugs. Neuropathic pain is also poorly responsive to opioids. For many individuals, the condition significantly interferes with daily life, including the ability to comfortably use a prosthetic limb, and may impact mobility, independence, and overall well-being.

SCN9A (Na_V1.7)

Cell-selective targeting (nociceptors), miRNA knockdown

Colloca L, Ludman T, Bouhassira D, et al. Nat Rev Dis Primers. 2017;3:17002.

Hsu E, Cohen SP. J Pain Res. 2013;6:121–136.

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.