52 THE SCIENTIST | the-scientist.com
BIO BUSINESSTHE SCIENTISTSTAFFclinical manufacturing facility for mak-
ing gene therapy treatments that could be
used in clinical trials.
With the ability to perform its own
early-stage trials, thanks to treatments
made in-house and staff dedicated to
regulatory tasks such as filing investi-
gational drug applications, Nationwide
was able to “de-risk” its discoveries,
McFarland says, by gathering prelimi-
nary clinical data and thus moving
discoveries closer to market. This de-
risking makes the prospect of licensing
Nationwide’s products for commer-
cialization more attractive to compa-
nies. “We’ ve helped prove the concept
that gene therapy can actually go frombench to bedside,” says Dennis Durbin,
Nationwide’s chief scientific officer.
“We are one of a small number of lead-
ing institutions that have really taken
it across the finish line,” he adds, refer-
ring to the 2019 approval of Novartis’s
Zolgensma, a gene therapy for spinal
muscular atrophy that stemmed from
findings made by Mendell’s team.
So far, five startups have been
launched in Ohio and elsewhere based
on prospective AAV-based therapies
developed at Nationwide. Collectively,
the companies are developing treat-
ments for 16 conditions, McFarland says.
The technology transfer office he heads,
which tries to find companies to licenseand commercialize therapies currently in
development, has expanded from 3 staff-
ers to 11 since he joined the hospital in
2012, he adds.
According to entrepreneur Mike
Triplett, who helped launch the Columbus-
based startup Myonexus in 2017 based
on technology licensed from Nation-
wide, the hospital has grown into one of
the “foundational pillars” of gene ther-
apy research, along with the University
of Pennsylvania and its affiliated Chil-
dren’s Hospital of Philadelphia, and the
University of North Carolina School of
Medicine. McFarland says the hospital’s
current position owes much to its earlier
decision to stick with gene therapy when
the technology’s future was far from
certain. “Because we had made all those
investments when gene therapy was
still a potentially risky proposition...
it put us in a place where now we have
the responsibility and the opportunity
to be leaders in the gene therapy space.”Full speed ahead
Ohio’s first gene therapy startup, Milo
Biotechnology, launched in 2012 in
Cleveland, just a couple of hours’ drive
north from Columbus. The company,
whose sole employee is its chief execu-
tive officer, Al Hawkins, is collaborating
with Mendell and others at Nationwide
on a few small clinical trials testing a
treatment, licensed from the hospital,
for a form of muscular dystrophy.
In 2013, another startup, Abeona
Therapeutics, also launched in Cleve-
land based on technologies developed at
Nationwide. Abeona has clinical-stage
gene and cell therapies for the connec-
tive tissue disorder recessive dystrophic
epidermolysis bullosa and for Sanfilippo
syndrome, in which a buildup of toxic
sugars in the body causes neurodegen-
eration and death. The company is also
conducting preclinical studies on treat-
ments for several other disorders.
Abeona opened its own manufac-
turing center in Cleveland last spring
to produce the therapies needed for its
clinical trials. However, the US Food and
Drug Administration (FDA) has askedCOLUMBUS
Sarepta Therapeutics Gene Therapy Center
Myonexus*
Nationwide Childrens Hospital
Ohio State University
Rev1 Ventures
CINCINNATI
Aruvant
University of Cincinnati
CincytechCLEVELAND
Milo Biotechnology
Abeona Therapeutics
Case Western Reserve
UniversityGENE THERAPY IN OHIO
In the last decade, Ohio has spawned several gene therapy startups as well
as outposts of more-established biotech companies such as Sarepta, all
of which benefi t from the proximity to academic centers of gene therapy
research and sources of research funding.*Myonexus was acquired by Sarepta in 2019.