Geron Corporation, a clinical stage biopharmaceutical company, focuses on the development of a telomerase inhibitor, imetelstat, in hematologic myeloid malignancies. The discovery and early development of imetelstat, the company’s sole product candidate, is based on its core expertise in telomerase and telomere biology. Telomerase is an enzyme that enables cancer cells, including malignant progenitor cells, to maintain telomere length, which provides them with the capacity for limitless, uncontrolled proliferation. Using the company’s proprietary nucleic acid chemistry, it designed imetelstat to be an oligonucleotide that targets and binds with high affinity to the active site of telomerase, thereby directly inhibiting telomerase activity and impeding malignant cell proliferation. Molecular responses in essential thrombocythemia and remission responses, including reversal of bone marrow fibrosis, in myelofibrosis suggest imetelstat has disease-modifying activity by inhibiting the progenitor cells of the malignant clone for the underlying disease in a relatively selective manner. In 2014, the company entered into an exclusive collaboration and license agreement with Janssen Biotech, Inc. to develop and commercialize imetelstat worldwide for all indications in oncology, including hematologic myeloid malignancies, and all other human therapeutic uses. Telomeres and Telomerase in Normal Development Telomerase is a naturally occurring enzyme that maintains telomeres and prevents them from shortening during cell division in cells, such as stem cells, which must remain immortalized to support normal health. Telomerase consists of at least two essential components, such as a ribonucleic acid (RNA) template (hTR), which binds to the telomere; and a catalytic subunit (hTERT) with reverse transcriptase activity, which adds a specific deoxyribonucleic acid sequence to the chromosome ends. Telomerase is active during embryonic development, enabling the rapid cell division that supports normal growth. During the latter stages of human fetal development and in adulthood, telomerase is repressed in various cells, and telomere length gradually decreases during a lifetime. In tissues that have a high turnover throughout life, such as blood and gut, telomerase could be transiently upregulated in progenitor cells to enable controlled, self-limited proliferation to replace cells lost through natural cell aging processes. In proliferating progenitor cells, relatively long telomeres are maintained by upregulated telomerase. As the progeny of progenitor cells mature, telomerase is downregulated and telomeres shorten with cell division, preventing uncontrolled proliferation. Telomeres and Telomerase in Cancer Telomerase is upregulated in various tumor progenitor cells, which enable the continued and uncontrolled proliferation of the malignant cells that drive tumor growth and progression. Telomerase expression has been found to be present in approximately 90% of biopsies taken from a range of human cancers. The company’s nonclinical studies, in which the telomerase gene is artificially introduced and expressed in normal cells grown in culture, have suggested that telomerase does not itself cause a normal cell to become malignant. Imetelstat: The First Telomerase Inhibitor to Advance to Clinical Development Imetelstat is a lipid conjugated 13-mer oligonucleotide that is designed to be complementary to and bind with high affinity to the RNA template of telomerase, thereby directly inhibiting telomerase activity. To improve the ability of imetelstat to permeate through cellular membranes, the company conjugated the oligonucleotide to a lipid group. Imetelstat's IC50, or half maximal inhibitory concentration, is 0.5-10 nM in cell free assays. Imetelstat has been shown in nonclinical studies to exhibit relatively preferential inhibition of the clonal proliferation of malignant progenitor cells compared to normal progenitors. For this reason, imetelstat has been studied as a treatment for malignant diseases. Imetelstat is the first telomerase inhibitor to advance to clinical development. The Phase 1 trials that the company completed evaluated the safety, tolerability, pharmacokinetics and pharmacodynamic effects of imetelstat. In 2014, the company received written notice from the United States Food and Drug Administration (FDA) that its Investigational New Drug application for imetelstat had been placed on full clinical hold following the FDA's review of safety data in the company’s then ongoing clinical studies. Government Regulation Human therapeutic products, such as imetelstat, are subject to rigorous preclinical and clinical testing and other approval procedures of the FDA and similar regulatory authorities in European and other countries. History Geron Corporation was founded in 1990. The company was incorporated in the state of Delaware in 1990.
geron corp (GERN:German Stock Exchange)
149 Commonwealth Drive
Menlo Park, CA 94025
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