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SwanBio Therapeutics to Present Preclinical Data on Lead Candidate at American Society of Gene & Cell Therapy

  • SBT101 is an investigational gene therapy for adrenomyeloneuropathy (AMN)
  • Oral presentation to showcase dose selection for Phase 1/2 clinical trial
  • Phase 1/2 trial expected to initiate in second half of 2022

SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, will present safety, efficacy, and biodistribution data from preclinical studies of its lead candidate, SBT101, for the treatment of adrenomyeloneuropathy (AMN) at the American Society of Gene & Cell Therapy (ASGCT) 2022 Annual Meeting in Washington, DC May 16-19. These data have formed the basis of SwanBio’s progress to advance SBT101 into clinical trials.

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“As we move closer to bringing SBT101 to the clinic, we are pleased to share a broader view of the preclinical data we have gathered that support our approach to target the root cause of this disease safely and effectively.”

“At this year’s ASGCT Annual Meeting, we look forward to sharing more information about the data, decisions, and guardrails that informed the design of our upcoming Phase 1/2 study of SBT101, an AAV-based therapy we’re exploring for patients with AMN, a progressive and debilitating neurodegenerative disease with no available treatment options,” said Karen Kozarsky, Ph.D., chief scientific officer of SwanBio Therapeutics. “As we move closer to bringing SBT101 to the clinic, we are pleased to share a broader view of the preclinical data we have gathered that support our approach to target the root cause of this disease safely and effectively.”

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SBT101 is the first AAV-based gene therapy candidate cleared by the FDA for human studies, designed to compensate for the disease-causing ABCD1 mutation in people living with adrenomyeloneuropathy (AMN). In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of AMN disease markers in mouse models and was 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 deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN, including new insights being gathered in an ongoing natural history study.

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