First Stargardt's Patient in EU Trial Treated with 150,000 RPE Cells
Derived from Human Embryonic Stem Cells
Advanced Cell Technology, Inc. ("ACT"; OTCBB:
ACTC), a leader in the field of regenerative medicine, today
announced treatment of the first patient in the third dosage cohort, and
seventh patient overall, in its European Phase I clinical trial for
Stargardt's macular dystrophy (SMD) using retinal pigment epithelial
(RPE) cells derived from human embryonic stem cells (hESCs). The patient
was injected with 150,000 hESC-derived RPE cells, as compared with the
100,000-cell dose used in patients of the second cohort. The surgery was
performed on Friday, April 19, without any complications, and the
patient is recovering uneventfully.
"Europe represents a huge potential market for us, the world's largest
after the U.S., so we are particularly pleased to now be past the
halfway point in all three of our clinical trials on both continents,"
commented Gary Rabin, chairman and CEO of ACT. "SMD affects as many as
100,000 patients in the U.S. and Europe. Moreover, as previously
announced, the European Medicines Agency's (EMA) Committee for
Orphan Medicinal Products (COMP) officially granted our hESC-derived RPE
cells orphan medicinal product designation for the treatment of SMD. We
expect this will provide a number of benefits once our SMD treatment has
made the transition from the clinic to the bedside, including reduced
fees and protection from competition."
The Phase 1/2 trial is designed to determine the safety and tolerability
of hESC-derived RPE cells following sub-retinal transplantation in
patients with SMD at 12 months, the study's primary endpoint. It will
involve a total of 12 patients, with cohorts of three patients each in
an ascending dosage format.
"We are pleased to have now moved into the second-half of all our
clinical trials for macular degeneration," said Robert Lanza, M.D.,
chief scientific officer. "We are eagerly anticipating advancing toward
the final cohort in all three trials."
About Stargardt's Disease
Stargardt's disease or Stargardt's Macular Dystrophy is a genetic
disease that causes progressive vision loss, usually starting in
children between 10 to 20 years of age. Eventually, blindness results
from photoreceptor loss associated with degeneration in the pigmented
layer of the retina, called the retinal pigment epithelium, which is the
site of damage that the company believes the hESC-derived RPE may be
able to target for repair after administration.
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company applying
cellular technology in the field of regenerative medicine. For more
information, visit http://www.advancedcell.com.
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are detailed from time to time in the company's periodic reports,
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Forward-looking statements are based on the beliefs, opinions, and
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the company does not assume any obligation to update its forward-looking
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assurance that the Company's clinical trials will be successful.
James Young, 212-732-4300
Bill Douglass, 646-450-3615
David Schull, 858-717-2310