James M. Wilson, MD, PhD, director of the Gene Therapy Program at the University of Pennsylvania, and a co-founder of Passage Bio.

Passage Bio, a developer of gene therapies for rare monogenic CNS diseases launched seven months ago, has licensed its sixth gene therapy from the University of Pennsylvania (Penn) and its Gene Therapy Program (GTP), a preclinical treatment for Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A).

The licensing comes five days after the company announced completion of a $110 million Series B financing, boosting its total capital raised to $225.5 million.

Passage Bio said the CMT2A gene therapy is designed to restore the normal function of the MFN2 gene, which is mutated in patients with CMT2A, the most common type of CMT2. The company plans to launch a clinical trial for the gene therapy candidate in 2021, according to its website.

The trial is anticipated to be a global, open-label, multi-center, dose escalation study designed to assess the safety, tolerability, and exploratory efficacy endpoints in subjects with CMT2A.

“The Gene Therapy Program at Penn has developed AAV vectors and delivery methods to target the nerve cells that are affected in CMT2A, raising the possibility of slowing or preventing progression of the disease by tackling the underlying genetic cause,” Stephen Squinto, PhD, co-founder and interim CEO at Passage Bio, said in a statement. “We look forward to initiating a clinical trial in the near future.”

That trial will include contributions from the Charcot-Marie-Tooth Association that include preclinical and clinical assets, access to top CMT experts, and engagement of the CMT patient community, the Association’s chairman Gilles Bouchard stated.

“Just one year after we formally launched our gene therapy program, we are witnessing two major players in the field working collaboratively to develop potential treatments for one of the more common types of CMT,” Bouchard said.

Charcot-Marie-Tooth disease encompasses a heterogeneous group of inherited, progressive, chronic peripheral neuropathies. While many types of CMT affect the myelin sheath of nerves, CMT2 results from the degeneration of the axons of neurons. Patients with CMT2A usually begin to experience progressive weakness in childhood and most become wheelchair dependent.

Three lead programs

The CMT2A candidate is the latest gene therapy program Passage Bio has agreed to develop under its research, collaboration, and license agreement with Penn and GTP, whose director is pioneer gene therapy researcher James M. Wilson, MD, PhD, a co-founder of the company.

The GTP has agreed to conduct IND-enabling preclinical work, while Passage Bio has agreed to conduct all clinical development, regulatory strategy, and commercialization activities. The agreement grants Passage Bio the option to license six more gene therapy candidates.

Under that agreement, whose value has not been disclosed, Passage Bio has three lead programs, all in IND-enabling phases and slated to begin clinical development next year. During the first half of 2020, the company plans to begin clinical development of its programs—one designed to treat GM1 gangliosidosis by targeting the lysosomal enzyme β-galactosidase (GLB1); and the other designed to treat frontotemporal dementia by targeting the gene encoding the lysosomal protein progranulin (PGRN).

In the second half of 2020, Passage Bio plans to begin clinical development of its third program GTP-206, which is aimed at treating Krabbe disease by targeting the gene encoding the hydrolytic enzyme galactosylceramidase (GALC).

Passage Bio agreed in July to partner with Catalent’s Paragon Gene Therapy unit to support its supply needs for those and other programs from clinical through commercial phases through a dedicated manufacturing suite at Paragon’s Harmans, MD, facility. The cGMP suite, whose value was not disclosed, is expected to begin operation in the second half of 2020.

Just last week, Passage Bio completed a $110 million Series B financing led by Access Biotechnology. New investors Boxer Capital of Tavistock Group, Highline Capital Management, Logos Capital, and Sphera Funds Management, participated in the round, as did existing investors, including OrbiMed, Frazier Healthcare Partners, Versant Ventures, Lily Asia Ventures, New Leaf Venture Partners, and Vivo Capital.

Passage Bio was launched in February with $115.5 million in Series A financing to develop a portfolio of therapeutic candidates that incorporate AAV technologies developed by Wilson and colleagues at Penn and GTP.

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