[Ip-health] GeneNews on Penn Scientist ​James W. Wilson role in for profit Passage BIO

James Love james.love at keionline.org
Tue Aug 27 12:07:11 PDT 2019

See if you can tell where Penn, the non-profit University and recipient of
more than $17 billion in federal grants for health research, ends, and the
venture capital projects begin.

This article focuses on James M. Wilson, one of researchers whose patents
were licensed by Penn for the $2.1 million Zolgensma gene therapy now
marketed by Novartis, the giant Swiss pharma/biomedicine company.   Wilson
Directs the Gene Therapy Program at the U of Penn Perelman School of
Medicine, but is also engaged in a number of for profit firms that will
license Penn's NIH subsidized inventions.

Penn recently refused to provide the patent numbers in an also secret
license agreement that was used for Zolgensma, making it hard to determine
if NIH funds were used or disclosed.

It's such bad  manners to ask Penn (or Francis Collins)  what these new
treatments will cost.



Wilson among Co-Founders of Gene Therapy Developer Launched with $115.5M
February 15, 2019

A developer of gene therapies for rare monogenic CNS diseases—whose
co-founders include pioneer researcher James M. Wilson, MD, PhD—has
launched with $115.5 million in Series A financing.

Passage Bio said it will use proceeds from the financing to develop a
portfolio of five therapeutic candidates that incorporate adeno-associated
virus (AAV) technologies developed by Wilson and colleagues at the
University of Pennsylvania (Penn) and its Gene Therapy Program (GTP).

The gene therapy candidates will be developed through a research,
collaboration, and license agreement with Penn and GTP, together with the
Penn Orphan Disease Center (ODC), with the support of Wilson, who is
director of both GTP and ODC, professor of medicine and pediatrics at
Penn’s Perelman School of Medicine, and the company’s chief scientist

“Our team at Penn is extremely experienced and has been on the cutting edge
of AAV research for over 20 years. 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,” Wilson said in a

Passage Bio retains an option to fund the preclinical development of up to
seven additional rare monogenic CNS indication programs at the GTP, and
license new intellectual property arising from these programs from Penn.

. . .

Blockbuster background

Squinto is also a co-founder of Alexion Pharmaceuticals, where he most
recently served as EVP and chief global operations officer. At Alexion, he
was involved in the discovery, development, and commercialization of the
blockbuster orphan drug Soliris® (eculizumab), indicated for patients with
paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome,
and generalized myasthenia gravis. Soliris generated $3.563 billion in
sales in 2018, up 13% from $3.144 billion in 2017.

Squinto is now a Venture Partner at OrbiMed Advisors, which led the Series
A financing, joined by Frazier Healthcare Partners, Versant Ventures, New
Leaf Venture Partners, Vivo Capital, and Lilly Asia Ventures (LAV).

LAV originated in 2008 as a corporate venture subsidiary of Eli Lilly, but
later spun off and became an independent investment management company with
more than $1.2 billion of committed capital under management.

A venture partner at Frazier, Tachi Yamada, MD, is another co-founder of
Passage Bio and serves as its chairman of the board. Before joining
Frazier, he was chief medical and scientific officer at Takeda
Pharmaceuticals, and has served as chairman of research and development at

Wilson’s lab has pioneered vector technology for nearly three decades,
including the discovery and development of novel serotypes of AAV, and has
contributed to the development of a variety of nonviral and viral
platforms. He led the research team in the 1999 clinical trial in which a
patient who suffered from a liver enzyme deficiency, 18-year-old Jesse
Gelsinger, died four days after treatment with gene therapy. Gelsinger
received the highest dose in the trial, 38 trillion virus particles.

. . .

Passage Bio disclosed that Wilson and three Penn-associated
shareholders—the Wilson Family Trust, Julie Johnston, and Monique
Molloy—hold founder shares in the company. Penn and GTP will receive
sponsored research funding from Passage Bio, while Wilson and Penn may
receive additional future financial benefits under the license as an
inventor of the licensed technology.

Penn also holds an equity interest in Passage Bio, and Wilson is
compensated for his scientific advisory position, the company also

James Love.  Knowledge Ecology International
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