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SwanBio Therapeutics Initiates First-in-Human Study of AAV Gene Therapy for Adrenomyeloneuropathy


Phase 1/2 study builds on momentum of over-subscribed and fully enrolled natural history study

PHILADELPHIA–(BUSINESS WIRE)–SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today announced the initiation of its PROPEL clinical trial. The first-in-human trial will study the company’s lead candidate, SBT101, an investigational AAV-based gene therapy intended to treat the progressive and debilitating neurodegenerative disease adrenomyeloneuropathy (AMN).

“As we wrap up a pivotal year for SwanBio, I am pleased to announce that we have successfully initiated PROPEL, our first interventional clinical study,” said Tom Anderson, chief executive officer and director, SwanBio. “This milestone comes on the heels of completing enrollment and over-subscribing our natural history study, CYGNET. These two achievements not only demonstrate Swan’s ongoing commitment to the AMN community, but also highlight the exceptional creativity and execution capabilities of our team. We expect to dose the first patient in PROPEL in early 2023 and are well positioned to meet our recruitment goals for this trial.”

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PROPEL is a multinational Phase 1/2 clinical trial assessing the safety and efficacy of SBT101, an investigational therapy designed to compensate for the ABCD1 mutation that causes AMN. The clinical trial will incorporate learnings from CYGNET, which pilots the use of wearable technologies to reduce onsite patient visits and explores more efficient ways to capture disease progression.

Visit the SwanBio website for more information about PROPEL, which is actively recruiting patients.

About SBT101

SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated in non-human primates through six months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, plus SwanBio’s deep understanding of the underlying pathophysiology of AMN and the AMN patient experience, including new insights being gathered in the ongoing, proprietary natural history study, CYGNET.

In early 2022, the FDA cleared SwanBio’s Investigational New Drug application for SBT101 and granted SBT101 Fast Track and Orphan Drug Designation.

About Adrenomyeloneuropathy

Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Patients often experience adrenal gland dysfunction as well. Between 8,000-10,000 men in the United States and European Union are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications – spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBio’s lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit


Media Contact:
Lara Furst


Investor Contact:
Jan Case

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