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February 7, 2006 - BioWorld Today
International Symposium On Stem Cell
Collaboration
ReNeuron's Results Positive As Stem Cell
Meeting Starts
By Randall Osborne
West Coast Editor
SAN FRANCISCO - Just before a unique meeting on
stem cell research got under way here,
preclinical news from the UK boosted hopes for
the field as applied to stroke.
ReNeuron Group plc aims to file an
investigational new drug application and begin
dosing humans this year, based on findings from
a second study with ReN001, a therapy for
chronic disability after stroke.
"Everything is on track," said John Sinden,
Guildford, UK-based ReNeuron's chief scientific
officer, pointing out that data from the second
study in rodent models reinforce results from
the first study, with delivery method and
implant location selected to mimic as much as
possible the protocols likely to be followed in
human trials.
The new data back up the first set in another
way, he told BioWorld Today.
"We've used cells that have been banked -
they're derived from GMP lots, equivalent to the
clinical lots that will go into the patients,"
Sinden said. "The original studies were done
with, as it were, early materials."
What's more, the latest results show a
dose-ranging effect, suggesting that a higher
dose has the potential to reverse both the
sensory and motor symptoms from stroke. In
September, ReNeuron held a formal
pre-investigational new drug application meeting
with the Center for Biologics Evaluation and
Research component of the FDA.
The company's headlines hit just ahead of the
one-day International Symposium on Stem Cell
Collaboration today at the University of
California at San Francisco. Sponsored by the
Women's Technology Cluster (WTC), a business
incubator dedicated to women leaders building
technology-driven firms, the event is intended
to "help get the dialogue going early" between
academia, companies and would-be investors, said
Cori
Gorman, president of DNA Bridges, a San
Francisco-based consulting firm for biotech and
pharma.
"We're scientists with business experience,"
said
Gorman, who serves on the board of
trustees of the WTC. She spent 10 years at South
San Francisco-based Genentech Inc., and
co-founded the gene therapy firm Valentis Inc.,
of Burlingame, Calif.
Although Valentis recently reported
stock-boosting Phase IIb data with its lead
candidate VLTS 934, a nonionic block copolymer
that acts as a vascular lubricant in patients
with peripheral arterial disease, gene therapy
mostly has struggled, and that struggle partly
inspired
Gorman to find ways of advancing the
dialogue.
Planning for the stem cell event began in the
fall of 2004, but the WTC decided "it was a
little early then,"
Gorman
said. "We needed a few more successes on the
basic research side" - successes like ReNeuron's.
"What's beautiful about their work is that they
have found a way to make the cells last a long
time in the undifferentiated state,"
Gorman
told BioWorld Today . "They understand that you
need to have them stable and reproducible, and
then be able to remove whatever trigger that is
letting them remain in a stem cell-like state.
Geron [Corp., of Menlo Park, Calif.] is also
trying to do this."
ReNeuron's GMP ability with cell lines makes the
company more ready for FDA scrutiny, too, and
"that's something gene therapy missed out on a
little bit. They weren't thinking about what the
FDA would want."
Ultimately,
Gorman
said, "some of the things going on in academia"
tipped the scale in favor of putting the
symposium together. She mentioned work on rodent
spinal cord regeneration in the lab of Brian
Cummings at the University of California at
Irvine, and research on heart regeneration by
the likes of Nadia Rosenthal in Italy and
scientists at the University of Minnesota.
StemCells Inc., of Palo Alto, Calif., in July
entered a licensing deal with ReNeuron that
gives the former exclusive access to the
latter's c-mycER adult human stem cell
immortalization technology for use in developing
therapies for lysosomal storage diseases, spinal
cord injury, cerebral palsy and multiple
sclerosis. In return, ReNeuron got exclusive
access to StemCells' adult neural stem cell
patent estate for the development of its own c-mycER
conditionally immortalized neural stem cell
therapies for stroke, as well as Parkinson's
disease and Huntington's disease.
As part of that arrangement, StemCells has taken
a 7.5 percent fully diluted equity interest in
ReNeuron, and the agreement provides for
reciprocal royalties and milestone payments.
"That [deal] is a key one for us," Sinden said,
calling StemCells "a very big player in the
U.S."
After neural applications, he said, ReNeuron's
"next stage is to work with [the technology] in
other types of stem cells, and we already have
some projects under way," at the preclinical
stage.
His firm's approach is "different, because [ReNeuron
uses] clonal cell lines, so there's an element
of genetic modification, but all of that is
understood from the regulators' point of view,"
Sinden said. "They like what they see, in terms
of how we're operating."
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