29 June 2022 - - US-based gene therapy company SwanBio Therapeutics presented details about the company's ongoing natural history study of adrenomyeloneuropathy at the 8th Congress of the European Academy of Neurology in Vienna.

The intention of the CYGNET study is to assess disease progression in patients with AMN to inform the research and development of potential treatments, including SwanBio's lead candidate, SBT101, the first clinical-stage AAV-based gene therapy for AMN.

SwanBio's CYGNET natural history study will track a number of variables over a two-year period, including:

Body sway, which has been shown to be an early predictor of disease progression and likelihood of falls in AMN, even in asymptomatic patients.

Both traditional motor tasks and novel activity and sleep outcomes, using wearable technology.

Quality of life and several disease severity and functional impairment measures.

More details about the CYGNET study, including demographics and disease characteristics of the first 21 patients enrolled, were presented during a virtual poster session at EAN.

SwanBio anticipates recruiting approximately 80 patients for the CYGNET natural history study; as of early June, the study was over 40% enrolled across five different global sites.

SBT101 is the first clinical-stage adeno-associated virus -based gene therapy candidate for people with adrenomyeloneuropathy.

SBT101 was designed to compensate for the disease-causing ABCD1 mutation.

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 at six months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, plus the company's already deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN.

SwanBio expects to initiate a randomized, controlled Phase 1/2 clinical trial designed to assess the safety and efficacy of SBT101 in patients with AMN in the second half of 2022.

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

Adrenomyeloneuropathy 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.

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.

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. SwanBio is supported by long-term investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures.