01/02.2020 | THE SCIENTIST 51BIO BUSINESS© ISTOCK.COM, PAWEL.GAUL
S
arepta Therapeutics’s outpost
in Ohio occupies a collection of
offices and labs on the second
floor of a large, squat structure known
as “Building 4” in a business park out-
side of Columbus. Despite the facil-
ity’s un assuming exterior, the start of
research onsite here in spring 2019
marked a milestone in Ohio’s two-
decade-long march toward becoming a
gene therapy hub. “We’re making a very
significant commitment and investment
in Columbus,” Doug Ingram, the CEO
of Cambridge, Massachusetts–based
Sarepta, told Columbus Business First in
an article about the new division. “There
is a real chance Columbus, Ohio, could
become the most important place in the
world for gene therapy development.”
With its Ohio research center,
Sarepta joins a local gene therapy eco-
system. Bolstered by academic research
at Columbus’s Nationwide Children’s
Hospital and Cincinnati Children’s
Hospital along with public and private
funding, the state has spawned sev-
eral gene therapy startups in the past
decade, and bioscience firms currently
employ about 75,000 people across
Ohio as a whole, according to the indus-
try group BioOhio. But Sarepta’s move
marked the first time an established bio-
tech has opened a gene therapy research
center in the state, attracting attention
and, observers hope, future investment
in Ohio’s nascent gene therapy industry.Getting in on the ground floor
Most of Ohio’s commercial gene ther-
apy research programs got their start
at Columbus’s Nationwide Children’s
Hospital, which launched its Center for
Gene Therapy in 2002. It was a risky
decision, given the death of 18-year-
old Jesse Gelsinger just three years
earlier, after the teenager had a severeimmune response to a gene therapy for
a rare metabolic disorder during a clini-
cal trial run by the University of Penn-
sylvania. The incident prompted many
institutions to put the brakes on their
gene therapy programs, but Nationwide
opted to bolster its investment, devel-
oping treatments that use vectors other
than adenoviruses—which had trig-
gered Gelsinger’s immune reaction—to
deliver the gene-modifying machinery.
A pivotal figure in the center ’s
research is neurologist Jerry Mendell,
who has for decades worked to develop
adeno-associated viruses (AAVs) as vec-
tors for gene therapies that would treat
muscular dystrophies and other neuro-muscular diseases—first at Ohio State
University, an affiliate of Nationwide,
and then, from 2004, at Nationwide
itself. Mendell and Nationwide’s leader-
ship shared a vision “for investing in and
building a true bench-to-bedside trans-
lational infrastructure for gene therapy,”
says Matt McFarland, Nationwide’s vice
president of commercialization and
industry relations. To that end, Nation-
wide went on to recruit other top gene
therapy researchers and to build its ownTHE CENTER OF IT ALL: Columbus is home
to Nationwide Children’s Hospital, whose Cen-
ter for Gene Therapy has provided the starting
point for many new gene therapy products in the
last decade.The state is emerging as a nascent gene therapy hub.BY SHAWNA WILLIAMSOhio, Gene Factory