Plotting clinical entry, James Wilson’s gene therapy startup brings in billionaire for $110M Series B

The gene therapy biotech that James Wilson helped found to take some rare disease programs out of Penn all the way to an approval has scored $110 million in its latest financing.

Passage Bio is getting a boost just seven months after launching with another megaround. Access Biotechnology — the therapeutic-focused investment arm of billionaire Len Blavatnik’s conglomerate — is leading the Series B, which also included all the marquee investors who provided the initial $115 million: OrbiMed, Frazier Healthcare Partners, Versant Ventures, Lily Asia Ventures, New Leaf Venture Partners and Vivo Capital. New backers include Boxer Capital of Tavistock Group, Highline Capital Management, Logos Capital and Sphera Funds Management.

The company is on track to initiate clinical trials for its two lead programs in the first half of 2020 as planned. True to its stated mission of tackling monogenic CNS disease, the therapies would treat GM1 gangliosidosis, a neuron-destroying disorder most common in infants, and frontotemporal dementia, which overwhelmingly affects the elderly, respectively.

A third program in Krabbe disease — intended to fix a genetic defect causing a shortage of galactosylceramidase, an important enzyme for making myelin — is expected to follow later in the year.

Keeping the stream of programs flowing, Passage Bio disclosed that it’s licensed two additional indications from the University of Pennsylvania while keeping an option to grab seven more.

Once Penn’s Gene Therapy Program — where Wilson is director — completes the IND-enabling preclinical work with the help of the Orphan Disease Center, the baton is passed to Passage Bio for all clinical development, regulatory strategy and eventual commercialization.

“As we continue to work toward advancing our three lead programs into the clinic over the coming year, we are focused on our mission to serve patients by offering best-in-class, life-transforming therapies,” said Stephen Squinto, the Alexion co-founder and OrbiMed partner who is running the shop as interim CEO, in a statement.

Liam Ratcliffe, the newly minted head of Access Biotechnology, is joining the board chaired by Tachi Yamada.

Yamada, a VC at Frazier formerly of GlaxoSmithKline and Takeda, was an old mentor of Wilson’s who offered crucial support in the immediate aftermath of a clinical trial that led to the death of a teenager and sent the gene therapy field into a harsh winter.