Karen Kozarsky, PhD: Treating the Root of AMN with Gene Therapy

Karen Kozarsky, PhD, Chief Scientific Officer and Co-founder of SwanBio Therapeutics, discussed the latest data on lead candidate SBT101, an AAV-based gene therapy to treat adrenomyeloneuropathy (AMN). The treatment is designed to compensate for the ABCD1 gene mutation found in individuals living with the rare neurodegenerative disease.

SwanBio presented new preclinical data on SBT101 at the American Academy of Neurology (AAN) 2022 Annual Meeting in Seattle. These data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile with no observed treatment-related adverse events.

Currently, there are no approved treatments for adrenomyeloneuropathy. Kozarsky explained that the standard of care is symptom control, and symptoms of AMN are deblitating. Often, people begin losing mobility in adulthood in addition to experiencing incontinence, pain, and sexual dysfunction.

"What we're trying to do with SBT101," she said, "is to actually address the root cause of the disease. So, we're trying to compensate for that disease causing mutation in the ABCD1 gene by replacing its activity for patients."

Kozarsky emphasized the amount of work and focus that the company has put into getting a good delivery of the therapeutic into the spinal cord. The additional work put in to this approach could serve as an advantage in delivering the treatment fully throughout the affected tissues.

"So, really understanding more about what can affect drug delivery to the spinal cord by delivering through the intrathecal approach, which is a fairly readily accessible approach to this," she said.

The randomized, controlled phase 1/2 clinical trial for SBT101 is expected to begin in the second half of 2022. The treatment has already received clearance for its Investigational New Drug application by the US Food and Drug Administration (FDA) earlier this year and was granted Fast Track and Orphan Drug Designation.