Gene therapy company Passage Bio has launched in the US with $115.5m series A funding to develop AAV-delivered therapies for the treatment of rare monogenic central nervous system (CNS) disorders.

The financing round was led by OrbiMed Advisors. Frazier Healthcare Partners, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures also joined the funding round.

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Passage Bio will use the funds to develop five therapeutic candidates under a research, collaboration and licence agreement with the University of Pennsylvania (Penn).

The partnership also involves Penn’s Gene Therapy Program (GTP) and the Penn Orphan Disease Center (ODC).

Lead candidates of the project include programmes for GM1 gangliosidosis (GM1) and frontotemporal dementia (FTD).

GM1 is an autosomal recessive genetic disorder, while FTD is characterised by progressive impairment of executive function, language and social interaction.

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“We believe this is a truly unique partnership, which gives Passage access to certain Penn AAV technologies developed at the GTP, our strong preclinical translational science capabilities and orphan drug development know-how.”

The company aims to bring these programmes into the clinic by early next year.

Passage Bio co-founder and chief scientific advisor James Wilson said: “We believe this is a truly unique partnership, which gives Passage access to certain Penn AAV technologies developed at the GTP, our strong preclinical translational science capabilities and orphan drug development know-how.

“We are confident in this team’s ability to move new treatments for rare CNS monogenic diseases through clinical development in an effort to one day provide new treatment options for patients with chronic unmet needs with high mortality.”

The company holds an option to fund the preclinical development of up to seven additional rare monogenic CNS programmes at the GTP.

In addition, Passage Bio can choose to license new intellectual property resulting from these programmes from Penn.

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