01/02.2020 | THE SCIENTIST 53for more data before allowing a Phase
3 clinical trial of Abeona’s treatment
for recessive dystrophic epidermolysis
bullosa to proceed. Shareholders filed
a lawsuit late last year alleging that the
company misled them by continuing
to state that the trial would begin in
mid-2019 even after becoming aware of
problems likely to stall FDA approval of
the study.
In 2017, a few years after Abeo-
na’s founding, Triplett and Nation-
wide researcher Louise Rodino-Klapac
cofounded Columbus-based Myonexus
based on gene therapies that Rodino-
Klapac’s team had developed for five
different forms of a neuromuscular dis-
ease called limb-girdle muscular dystro-
p hy. In mid-2018, Rodino-Klapac left
Nationwide to join Sarepta as vice pres-
ident for gene therapy. The biotech had
partnered with Myonexus from its incep-
tion and last year bought the startup for
$165 million. While Rodino-Klapac had
personal reasons for wanting to stay in
the Columbus area after leaving Nation-
wide, she says it was also “what’s best for
the technology,” given the site’s proximity
to the hospital and her ability to recruit
members of her old research team to
Sarepta’s new gene therapy research cen-
ter, which she heads.
Three of the company’s limb-girdle
treatments are now in small Phase 1 clini-
cal trials, and a Duchenne muscular dys-
trophy therapy that Sarepta licensed in
2017, also developed by Rodino-Klapac’s
team while she was at Nationwide, is
being tested in a Phase 1/2 trial with 40
patients. An earlier, four-patient trial of
that therapy “showed significant prom-
ise,” Rodino-Klapac says. She adds that
there’s preclinical research in the works
on other treatments that the company
isn’t yet ready to talk about.
The relatively humble office-
park home of Sarepta’s gene therapy
research center is temporary; the com-
pany is building a new 85,000-square-
foot facility, expected to be ready later
this year, in another Columbus suburb.
Rodino-Klapac says she hopes that the
company’s Columbus outpost will help
seed an expanded local gene therapy
hub. Speaking with people at compa-
nies in other states, she is often asked:
“‘Sarepta’s in Ohio—what are we miss-
ing?’” She takes it as a good sign. “I
think [Sarepta’s presence is] going to
help create an ecosystem here... for
the gene therapy and biotech space and
maybe beyond that.”Looking forward
Nationwide’s McFarland says he thinks
Ohio has much to offer companies. For
example, state government–fostered ini-
tiatives such as Rev1 Ventures offer ser-
vices to entrepreneurs in the Columbus
area, and Nationwide has partnered with
the state to offer seed-funding programs
for early-stage companies. The low costs
to operate in Ohio, relative to more
established, coastal hubs such as Boston,
are another plus, says Severina Kraner,
the director for health care at JobsOhio,
a nonprofit company working to boost
industry in the state. Moreover, Kraner
adds, the state is home to large research
institutions such as Case Western
Reserve University in Cleveland, Ohio
State in Columbus, and the University
of Cincinnati, providing a rich source of
researchers to be recruited to up-and-
coming biotechs.
The growth of gene therapy in Ohio
is also aided by changes in the industry
more generally. From an investor’s point
of view, the outlook for gene therapy
itself has changed, says John Rice, the
director of life sciences at Ohio-based
venture capital firm Cincytech, making
it less important that particular thera-
pies be de-risked by academic institu-tions before being picked up by industry.
His company provided start-up fund-
ing for Myonexus in 2017, and having
clinical-stage technologies developed
at Nationwide “made a big difference,”
he says. “We could get in with some
confidence that we’re going to see some
value for our investment.” But now his
firm is more willing to consider pro-
jects still at the preclinical stage, Rice
says—in fact, in order to invest in novel
gene therapies, investors have to do so.
Today, “anything that was clinical stage,
here or anywhere else, has already been
partnered out—a company’s created it
and is moving along.”
Developments outside Ohio have
of course contributed to gene therapy’s
maturity as a therapeutic strategy, and
not all treatments created in the state
have stayed there. Zolgensma (onase-
mnogene abeparvovec), for example,
a therapy for spinal muscular atrophy
that received FDA approval in May 2019,
originated in research at Nationwide
and was developed in the Chicago area
by a startup and later by Novartis. And
Amicus Therapeutics, which acquired
several of Nationwide’s gene therapy
technologies for rare metabolic condi-
tions in 2018, is based in New Jersey and
has a gene therapy center in Philadel-
phia. For McFarland, having commer-
cialization proceed in other states is not
a problem. When considering potential
licensees for each of the hospital’s tech-
nologies, he says, his team asks, “What
is the best for this asset? And what gives
this asset the best chance of having an
impact on patients? And every once in a
while, that’s not going to be here.”
Ye t McFarland and others think
that in the coming years, many gene
therapy companies will find that Ohio
offers them the conditions they need to
thrive. Based on confidential conversa-
tions she’s had with business leaders,
Kraner says she hopes to see more com-
panies crop up in the state within the
next year. Looking forward, she says,
“I think there’s going to be continued
investment and opportunities to attract
and grow [gene therapy] companies.” gThere is a real chance
Columbus, Ohio, could
become the most important
place in the world for gene
therapy development.
—Doug Ingram, Sarepta Therapeutics