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Toward a first-in-class gene therapy

Fighting the genetic causes of AMN

There is currently no treatment to slow or alter the progression of AMN, a severe neurodegenerative disease. Our gene therapy program could be the first.

A progressive condition without a true treatment

Adrenomyeloneuropathy (AMN) is a neurodegenerative disease characterized by mobility loss, incontinence, extreme pain, and sexual dysfunction. Symptoms worsen as the condition progresses, leading to a diminished quality of life.

The current standard of care for AMN is symptom management, physical therapy, and mobility aids. There is no treatment available that can alter the course of the disease.

Addressing the underlying genetic causes

AMN is caused by a mutation in the ABCD1 gene. These mutations disrupt the functioning of cells in the spinal cord.

Our product candidate, SBT101, is designed to counteract these mutations by delivering a functional ABCD1 gene into spinal cord cells. To deliver the gene to the right cells, SBT101 uses an adeno-associated virus (AAV) capsid, which has been shown to effectively deliver genetic material to cells in the central nervous system.

Moving toward a safe and effective treatment

Preclinical studies for SBT101 have demonstrated its clinical promise, showing elevated ABCD1 expression and decreases in toxic substrates in the spinal cord. We’re also continuing to build on our unique understanding of AMN through an ongoing natural history study into how AMN progresses.

SBT101 is currently in a Phase 1/2 first-in-human clinical study.

Published results and data

Ongoing clinical trials