Our potential first-in-class gene therapy candidate for adrenomyeloneuropathy (AMN) has joined Spur’s expanding pipeline of next-generation genetic medicines. Learn more and get updates at SpurTherapeutics.com.
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May 18, 2022
PHILADELPHIA, May 18, 2022 – SwanBio Therapeutics, a gene therapy company advancing adeno-associated virus (AAV)-based therapies for the treatment of devastating, inherited neurological conditions, today announced the completion of a $56 million Series B financing round, led by founding investors Syncona Limited and Mass General Brigham Ventures. The new funding brings SwanBio’s total financing raised to date to $133 million.
“Since our initial investment, SwanBio has made excellent progress, including rapidly advancing its lead program for AMN toward the clinic. In parallel, the company has built the platform capabilities that will allow it to advance its broader pipeline of AAV gene therapies for spinal cord-related disorders toward clinical development,” said Chris Hollowood, chief investment officer, Syncona and chair, SwanBio. “We are excited by the potential we see in this business to become a leading gene therapy company, delivering life-changing treatments to patients with debilitating neurological disorders.”
SwanBio’s latest round of financing will support the company’s ongoing evolution into a fully integrated research and development organization, with an initial focus on the clinical advancement of SBT101, including plans to dose patients in a Phase 1/2 trial by the end of this year. SBT101 is the first clinical-stage AAV-based gene therapy candidate for the treatment of adrenomyeloneuropathy (AMN). The investigational drug has been designed to compensate for the disease-causing ABCD1 mutation in people living with AMN. This mutation disrupts the function of spinal cord cells and other tissues, leading to nerve degeneration, causing loss of mobility in adulthood, incontinence, debilitating pain, and sexual dysfunction. Patients often experience adrenal gland dysfunction as well. In early 2022, the FDA cleared SwanBio’s Investigational New Drug application for SBT101 and granted Orphan Drug and Fast Track designations to SBT101.
“We are determined in our pursuit of AAV-based therapies targeting the root cause of disease, and are building our pipeline to develop treatments where there is a significant unmet need,” said Tom Anderson, chief executive officer and director, SwanBio. “The strategic decisions we make as a company are motivated by our drive to help those affected by neurological diseases as soon as possible because they can’t wait – and we don’t want them to.”
Beyond SBT101, SwanBio is developing a pipeline of gene therapy candidates designed to be delivered intrathecally to targets in both the central and peripheral nervous systems, with an initial focus on spinal cord-related disorders like AMN. This approach is built on SwanBio’s proprietary insights into delivery and biodistribution, expertise in cassette design, and ability to develop novel capsids. The Series B funding will be used to further progress the pipeline and apply learnings from SBT101 to other monogenic disorders, as well as polygenic disorders.
SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people living with adrenomyeloneuropathy (AMN), 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 deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN, including new insights being gathered in SwanBio’s ongoing, proprietary natural history study, CYGNET.
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. 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.
