Cellular Dynamics International: Awarded $1.2 million NEI contract for developing patient-derived stem cells in gear-up for age-related macular degeneration trial

MADISON, Wis., Oct. 27, 2014 (GLOBE NEWSWIRE) — Cellular Dynamics
International (CDI) (Nasdaq:ICEL), today announced that the National
Eye Institute (NEI), a division of the National Institutes of Health
(NIH), awarded the company a $1.2 million contract to manufacture
clinically compatible induced pluripotent stem cells (iPSCs) and
iPSC-derived human retinal pigment epithelial (RPE) cells. These cells
will be manufactured from individuals suffering from dry age-related
macular degeneration (AMD) and will be used for investigational new
drug (IND) enabling studies. Once the IND is approved, the same
procedures will be used to generate clinical-grade iPSC-derived RPE
tissue for transplantation into AMD patients. This process, known as
autologous cellular therapy, would be the first of its kind in the U.S.

A photo accompanying this release is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=28623

CDI will use its expertise to reprogram skin and blood samples from
individuals with AMD to create clinically compatible, autologous iPSCs,
which are genetically identical to the individual. As a genetic match
to the patient, these cells are intended to reduce the risk of
transplant rejection. NEI researchers plan to use these cells as part
of their pre-clinical process to develop the first autologous cell
transplantation treatment for dry AMD.

Key points:

— The clinically compatible RPE cells resulting from CDI’s protocol
development and optimization, when manufactured under cGMP conditions,
would likely be the first iPSC-based, autologous cellular therapeutic
candidates transplanted into humans in a Phase I clinical trial in the
U.S.

— The contract funds the manufacture of clinically compatible iPSCs and the
development and manufacture of clinically compatible iPSC-derived human
RPE cells by CDI. The RPE manufacturing process is based on iPSC-RPE
differentiation methods developed and authenticated by NEI.

— CDI will use existing NEI differentiation protocols to develop and
optimize the methods for manufacturing RPE cells and provide NEI with
cGMP-ready protocols and training for their manufacture.

— Researchers at NEI will use the RPE cells that CDI manufactures for
preclinical studies in preparation for a clinical trial that will test
these cells in patients with dry AMD. AMD is the leading cause of vision
loss in people aged 60 and older, and as many as 11 million Americans
have some form of macular degeneration, according to the Bright Focus
Foundation.

— There are currently no FDA-approved therapies for dry AMD, which accounts
for about 90% of AMD cases. Therapies for wet AMD require repeated eye
injections that slow or, at best, arrest disease progression. Revenues
for wet AMD products were more than $5.75 billion in 2013.

Quotes:

Bob Palay, chief executive officer of CDI, said, “AMD is a debilitating
disease. It begins with vision loss in the center of the visual field
and often progresses to nearly total vision loss. CDI is delighted to
be selected by the NEI to develop autologous RPEs for treatment of AMD.
Our goal is to provide Dr. Bharti and his fellow medical researchers
with transplantable manufactured RPEs for the treatment of this major
cause of blindness. We are excited to enable these researchers’ efforts
to help patients regain their vision.”

Chris Parker, chief commercial officer of CDI, said, “CDI has a unique
ability to consistently manufacture large numbers of iPSCs and human
cells to very tight specifications, including under cGMP conditions.
CDI’s reprogramming platform enables us to develop autologous
iPSC-based therapies, which would be a first in the U.S. We look
forward to working with the NEI to successfully complete this work and
progress through Phase I clinical development.”

Sheldon Miller, Ph.D., Scientific Director at the NEI, said, “This is a
first step towards our phase I IND enabling studies. There are a small
number of facilities in the U.S. with the capacity to generate iPSCs
and differentiate them into multiple lineages. We are eager to begin
testing clinically compatible patient-derived RPE cells in laboratory
models of retinal degeneration.”

Kapil Bharti, Ph.D., Earl Stadtman Investigator and lead scientist on
this project at NEI, said, “All of us at NEI, including the basic
research, preclinical, and clinical teams, are committed to the success
of this IND. We are grateful to the NIH Common Fund and the NEI
Intramural Program for their support.”

About Cellular Dynamics International, Inc.

Cellular Dynamics International, Inc. (CDI) is a leading developer and
manufacturer of fully functioning human cells in industrial quantities
to precise specifications. CDI’s proprietary iCell Operating System
(iCell(R) O/S) includes true human cells in multiple cell types (iCell
products), human induced pluripotent stem cells (iPSCs) and custom
iPSCs and iCell products (MyCell(R) Products). CDI’s iCell O/S products
provide standardized, easy-to-use, cost-effective access to the human
cell, the smallest fully functioning operating unit of human biology.
Customers use our iCell O/S products, among other purposes, for drug
discovery and screening; to test the safety and efficacy of their small
molecule and biologic drug candidates; for stem cell banking; and in
the research and development of cellular therapeutics. CDI was founded
in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell
research at the University of Wisconsin-Madison. CDI’s facilities are
located in Madison, Wisconsin, with a second facility in Novato,
California. See www.cellulardynamics.com.