Medical Reading

StemCells, Inc. To Present On Batten Disease To The California Institute Of Regenerative Medicine's Oversight Committee

May 21, 2017

StemCells, Inc. (NASDAQ:STEM) announced that it has accepted an invitation by the California Institute of Regenerative Medicine (CIRM) to present on Batten Disease at the upcoming meeting of CIRM's Independent Citizens Oversight Committee (ICOC). At the meeting, Ann Tsukamoto, Ph.D., the Company's Chief Operating Officer, and Stephen Huhn, M.D., F.A.C.S., F.A.A.P., Vice President and Head of the Company's CNS Program, will describe the Company's proprietary human neural stem cells and their potential application to Batten Disease, a fatal neurodegenerative disorder. The presentation, entitled Spotlight on Batten Disease, will take place at the Arrillaga Alumni Center, McCaw Hall, on the campus of Stanford University, Palo Alto, on Wednesday, August 13, 2008, at 8:15 a.m. PDT. The presentation is one of a series of educational events organized by CIRM to educate the ICOC and the general public about specific diseases or medical conditions that could be treated with stem cells or related research.

StemCells is currently conducting a Phase I clinical trial of its proprietary HuCNS-SC® product candidate (purified human neural stem cells) as a treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL), which is often referred to as Batten Disease. All six patients planned for this trial have been enrolled and transplanted with HuCNS-SC cells, and the Company expects the trial to be completed in January 2009.

About Neuronal Ceroid Lipofuscinosis (Batten Disease)

Neuronal ceroid lipofuscinosis is a fatal neurodegenerative disorder that afflicts infants and young children. The disorder, often referred to as Batten disease, is caused by genetic mutations, and children who inherit the defective gene are unable to produce enough of an enzyme that processes cellular waste substances that accumulate in a part of cells known as the lysosome. Without the enzyme, the cellular waste builds up, and eventually the cells cannot function and die. Children with NCL appear healthy when born, but as their brain cells die, they begin to suffer seizures and progressively lose motor skills, sight and mental capacity. Eventually, they become blind, bedridden and unable to communicate or function on their own. There currently is no cure for the disease. The infantile and late infantile forms of NCL are caused by different genetic mutations. As the names imply, the two forms begin to afflict patients at different stages of infancy, but both have similar disease progression and outcomes.

About HuCNS-SC Cells

StemCells' HuCNS-SC product candidate is a purified composition of normal human neural stem cells that are expanded and stored as banks of cells. The Company's preclinical research has shown that HuCNS-SC cells can be directly transplanted; they engraft, migrate, and differentiate into neurons and glial cells; and they survive for as long as one year with no sign of tumor formation or adverse effects. The Company has demonstrated that HuCNS-SC cells, when transplanted in a mouse model of infantile NCL, engraft; migrate throughout the brain; produce the missing lysosomal enzyme; measurably reduce the toxic storage material in the brain; protect host neurons so that more of them survive; and extends the lifetime of the mice compared to a control (non-transplanted) group. Based on this preclinical data, StemCells believes that transplantation of HuCNS-SC cells into the brain of an infantile or late infantile NCL patient may provide the prospect of long-term production and delivery of the missing lysosomal enzyme, which would help clear the cellular waste and protect the patient's brain cells.

About StemCells, Inc.

StemCells, Inc. is a clinical-stage biotechnology company focused on the discovery, development and commercialization of cell-based therapeutics to treat diseases of the nervous system and liver. The Company's programs seek to repair or repopulate neural, liver or other tissue that has been damaged or lost as a result of disease or injury. StemCells has pioneered the discovery and development of HuCNS-SC cells, its highly purified, expandable population of human neural stem cells. StemCells owns or has exclusive rights to more than 50 issued or allowed U.S. patents and more than 150 granted or allowed non-U.S. patents. Further information about the Company is available on its web site at: stemcellsinc.

Apart from statements of historical fact, the text of this press release constitutes forward-looking statements regarding, among other things, the future business operations of StemCells, Inc. (the "Company") and its ability to conduct clinical trials as well as its research and product development efforts. These forward-looking statements speak only as of the date of this news release. The Company does not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Such statements reflect management's current views and are based on certain assumptions that may or may not ultimately prove valid. The Company's actual results may vary materially from those contemplated in such forward-looking statements due to risks and uncertainties to which the Company is subject, including uncertainty as to whether the FDA or other applicable regulatory agencies will permit the Company to continue clinical testing in NCL or in future clinical trials of proposed therapies for other diseases or conditions despite the novel and unproven nature of the Company's technologies; uncertainties regarding the Company's ability to obtain the increased capital resources needed to continue its current research and development operations and to conduct the research, preclinical development and clinical trials necessary for regulatory approvals; uncertainty regarding the validity and enforceability of the Company's patents; uncertainty as to whether HuCNS-SC and any products that may be generated in the future in the Company's cell-based programs will prove safe and clinically effective and not cause tumors or other adverse side effects; uncertainties regarding the Company's manufacturing capabilities given its increasing pre-clinical and clinical commitments; uncertainties as to whether the Company will achieve revenues from product sales or become profitable; and other factors that are described under the heading "Risk Factors" in Item 1A of Part II of the Company's Annual Report on Form 10-K.

StemCells, Inc.