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IND Cleared for Adrenomyeloneuropathy Gene Therapy

SwanBio previously presented positive preclinical data at the ESGCT 2021 meeting.

SwanBio Therapeutics' investigational new drug (IND) application for gene therapy SBT101 has been cleared by the FDA for the potential treatment of adrenomyeloneuropathy (AMN), an adult-onset degenerative spinal cord disease that affects people living with adrenoleukodystrophy.1

SBT101 is an adeno-associated virus (AAV) gene therapy that contains a corrected ABCD1 gene designed to increase ABCD1 expression levels and to reduce very long chain fatty acid (VLCFA) levels in AMN. SwanBio plans to initiate a randomized, placebo-controlled, phase 1/2 clinical trial to evaluate the safety and efficacy of SBT101 in the second half of 2022.

“Today’s IND clearance is a formative milestone for SwanBio, enabling us to evolve from a preclinical company to a truly integrated research and development organization, underscoring the expertise of our team and potential of our technology platform,” Tom Anderson, chief executive officer and director, SwanBio Therapeutics, said in a statement.1 “SBT101 has the potential to become the first disease-modifying treatment for patients with AMN, a devastating and progressive disease with no approved treatments. We look forward to initiating clinical development of SBT101 later this year, bringing us closer to our ultimate goal of delivering life-changing treatments to patients.”

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The decision was based on preclinical findings seen with SBT101, positive data from which were announced by SwanBio in October 2021 at the European Society for Gene and Cell Therapy (ESGCT) Virtual Congress.2 Preclinical studies demonstrated dose-dependent expression of hABCD1 and reduction of VLCFA levels in mouse models. The therapy was shown to be well-tolerated in non-human primates at six months post-treatment.

“We are excited to share the first data on SBT101, which demonstrate its potential to directly address the underlying cause of AMN by establishing ABCD1 expression in the spinal cord,” Karen Kozarsky, PhD, chief scientific officer, SwanBio, commented at the time.2 “AMN is a devastating disease with no approved treatments, and we are pleased to have observed improvements in markers of AMN disease as well as good tolerability in both mice and NHPs. Taken together, these findings support the continued evaluation of SBT101 as a potential disease-modifying treatment for AMN as we advance towards the clinic.”

SwanBio is also conducting the natural history CYGNET study (NCT05008874) in adult men with AMN to inform future research and clinical development of therapies targeting the disease.

REFERENCES
1. SwanBio Therapeutics Announces FDA investigational new drug clearance for first AAV-based gene therapy for the treatment of adrenomyeloneuropathy. News release. SwanBio Therapeutics. January 25, 2022. https://www.businesswire.com/news/home/20220125005388/en/SwanBio-Therapeutics-Announces-FDA-Investigational-New-Drug-Clearance-for-First-AAV-Based-Gene-Therapy-for-the-Treatment-of-Adrenomyeloneuropathy
2. SwanBio Therapeutics announces new preclinical data demonstrating proof of principle for SBT101 gene therapy as a potential treatment of adrenomyeloneuropathy. News release. SwanBio Therapeutics. October 19, 2021. https://swanbiotx.com/investors-and-media/swanbio-therapeutics-announces-new-preclinical-data-demonstrating-proof-of-principle-for-sbt101-gene-therapy-as-a-potential-treatment-of-adrenomyeloneuropathy/