Privately held Swanbio Therapeutics Inc., of Bala Cynwyd, Pa., completed a $52 million expanded series A financing designed to boost the company’s R&D of adeno-associated virus (AAV)-based gene therapies to treat adrenomyeloneuropathy (AMN) and other rare, monogenic neurological diseases.

The funding round will also help the company advance its IND-enabling studies, grow its manufacturing and development capabilities and hire more staffers. In addition to Bala Cynwyd, the company has operations in Philadelphia and Cambridge, Mass.

The company uses AAVs delivered directly to the CNS and said it is focused on understanding the origin and treatment of disorders where therapeutic DNA may be able to treat neurological illnesses.

AMN is a variant of X-linked adrenoleukodystrophy, an inherited peroxisomal disorder, which leads to dysfunction of plasma, the central and peripheral nervous systems, and adrenal glands.

Swanbio’s CEO is Tom Anderson, the former chief commercial strategy officer at Sage Therapeutics Inc. and a senior operating executive with Shire Pharmaceuticals Group in a number of operational and strategic CEO-advisory roles in both rare diseases and specialty pharmaceuticals. Anderson also worked with Janssen Biotech Inc. and McNeil Pharmaceutical Inc., taking on sales, marketing and business information positions. He is on Swanbio’s board as well as on the board at Expresscells Inc., which leverages gene-editing tools and CRISPR to create customized cell lines for research in drug discovery and toxicology.

The company’s chief scientific officer, and a co-founder, is Karen Kozarsky, who has more than two decades of experience with gene therapy, primarily in developing gene therapies for inherited rare diseases using AAVs. She was president of Vector Biopartners, vice president of R&D at Regenxbio Inc, and head of gene therapy at the Glaxosmithkline Biopharmaceutical Center for Excellence for Drug Discovery.

In its drive to add heads to the company roster, the company posted job openings on its website for an associate director of program management, a scientist/senior scientist/process engineer in AAV purification and a senior scientist/principal scientist in cell culture development-AAV gene therapy, all in Philadelphia.

Syncona Ltd., of London, a life sciences investor with a portfolio of firms, and Partners Innovation Fund made a $20 million investment as part of a $52 million additional commitment to the financing.

On March 23, Syncona said its firms have made or are considering appropriate plans for delays to clinical trials due to the COVID-19 pandemic. Though Syncona said it is hard to forecast the precise impact, “we would expect delays to a number of our clinical-stage programs of at least three months.” Its previous large investment came in February when the firm and Oxford Sciences Innovation pitched in to a $35.75 million series A funding for Omass Therapeutics Ltd., of Oxford, U.K., to harness mass spectrometry and other biophysical technologies to drive drug discovery.

Partners Innovation Fund is a recent investor in Waltham, Mass.-based Affinia Therapeutics Inc.’s $60 million series A to boost research for drugs to benefit people affected by muscle and CNS conditions.

The race to treat AMN includes Minoryx Therapeutics SA, of Barcelona, Spain. In January, the FDA granted fast track designation for the company’s MIN-102 (leriglitazone), a brain-penetrant, orally bioavailable and selective PPAR-gamma agonist. The first U.S. patient in Minoryx's ongoing phase II/III trial in AMN with MIN-102 was dosed last September at Massachusetts General Hospital.

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