AEGiS-PRn: SyStemix Plans to Initiate Gene Therapy Trials for HIV Infection

Important note: Information in this article was accurate in 1996. The state of the art may have changed since the publication date.

SyStemix Plans to Initiate Gene Therapy Trials for HIV Infection

PR Newswire, Wednesday, 4 December 1996.

PALO ALTO, Calif., Dec. 4 /PRNewswire/ -- SyStemix, Inc. (Nasdaq: STMX) today announced that the FDA has accepted SyStemix' Investigational New Drug (IND) application to initiate a Phase I/II gene therapy trial for treatment of HIV-infected patients. According to the trial protocol, a therapeutic gene (Rev M10) will be inserted ex vivo into patients' hematopoietic stem cells (HSCs), which can both self-renew and develop into all the cells of the blood and immune system. Pre-clinical studies have shown that the gene-modified stem cell passes on its protective gene to new stem cells it produces and to its "daughter" cells, including T-cells, macrophages and all other cells that might otherwise be vulnerable to attack and destruction by HIV. These studies also indicate that Rev M10 gene prevents HIV from replicating within cells, trapping the virus and limiting its spread to uninfected cells. SyStemix' trial will be the first using HSCs (also referred to as CD34+Thy-l+ cells) as delivery vehicles for a therapeutic gene against HIV infection.

"Stem-cell-based gene therapy for HIV may be able to provide long-term therapeutic benefit from only a single treatment, and could be used alone or in combination with multi-drug anti-viral therapies," said Christopher Juttner, F.R.A.C.P., Vice President Medical Affairs at SyStemix. "A number of major benchmarks have already been achieved in this program, and we expect to announce further advances at this week's annual meeting of the American Society of Hematology."

The program at SyStemix is a 50/50 collaboration with Sandoz Pharmaceuticals, and was initiated in 1993 to design and evaluate a series of anti-HIV gene therapies using the HSC, the common source of all cells in the blood and immune system. The first product ready for clinical trials is the gene-modified hematopoietic stem cell isolated by SyStemix, using the Rev M10 gene from Sandoz (licensed from Duke University). The collaboration utilizes SyStemix' patented hematopoietic stem cell technology, scientific and clinical experience in hematopoietic cellular therapies and its unique SCID-hu mouse model which mimics the human immune system. Sandoz has provided anti-HIV genes, retroviral vectors, and cytokines to facilitate gene transfer.

Key pre-clinical accomplishments in the SyStemix-Sandoz HIV gene therapy program have included:

* Successful isolation and gene-modification of HSCs (CD34+Thy-l+ cells) from HIV-infected patients;

* Development of a safe and proprietary "packaging cell line" derived from human cells that enables insertion of genes into the patient's HSCs with high efficiency;

* Demonstration that gene-modified stem cells produce gene-modified progeny, and that these modified cells express the gene in sufficient quantities to prevent viral replication.

SyStemix, Inc. is engaged in the development of advanced cellular and cell-based gene therapies for cancer, infectious, autoimmune and genetic diseases based on the use of isolated, expanded and gene-modified human stem cells.

The statements in this press release that are not purely statements of historical fact are forward-looking statements that are subject to risks and uncertainties that could cause the actual results to differ materially from those projected. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. The company assumes no obligation to update the information in this release.

CONTACT: Wendy R. Hitchcock, Chief Financial Officer of SyStemix, Inc., 415-813-4108; or Justin Jackson or Reagan Codner, both of Burns McClellan, 212-505-1919, for SyStemix, Inc.

Keywords: HIV; IMMUNE SYSTEM; CLINICAL TRIAL

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