January 1, 2012 (Vol. 32, No. 1)

  • Geron eliminated 66 full-time positions, representing 38% of its workforce, after dropping its embryonic stem cell (ES) program, despite being the first to go into clinical trials with an ES cell product. The move, which repositions the company into a developer of first-in-class oncology drugs, followed what Geron called a strategic review of the costs, value inflection timelines and clinical, manufacturing, and regulatory concerns associated with its research and clinical-stage assets. “In the current environment of capital scarcity and uncertain economic conditions, we intend to focus our resources on advancing our Phase II clinical trials of imetelstat and GRN1005,” CEO John A. Scarlett, M.D., said.
  • Neuralstem advanced the ongoing Phase I safety trial of its spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS; Lou Gehrig disease) at Emory University to the final two cohorts of patients with ALS. The company received permission by FDA to advance transplantation in the cervical region. Previously, patients have received injections in the lumbar region only.The trial is under the direction of principal investigator Eva Feldman, M.D., Ph.D., director of the A. Alfred Taubman Medical Research Institute, and director of research of the ALS Clinic at the University of Michigan Health System; and Jonathan D. Glass, M.D., director of the Emory ALS Center.
  • TMUCIH, one of China’s largest biotherapy centers, has inked an agreement with Takara Bio to launch a research project on T-cell receptor (TCR) gene therapy against refractory cancer, for which surgery is ineffective and which is initially unresponsive to chemo- or radiation therapy. A patient’s lymphocytes are transduced in vitro with the retrovirus vector having the TCR gene, which recognizes the tumor cells. The gene-modified lymphocytes are then grown in vitro and reintroduced to patients. These adapted lymphocytes recognize and attack patients’ tumors. The treatment helps lung cancer, esophageal cancer, and malignant melanoma patients.
  • Adeona Pharmaceuticals will develop and commercialize a DNA-based therapeutic using the UltraVector® platform and RheoSwitch Therapeutic System® from Intrexon for pulmonary arterial hypertension. Adeona will use Intrexon’s advanced transgene engineering platform for controlled, precise, and continuous in vivo cellular production of prostaglandin synthase, a specific effector enzyme that regulates the production of prostacyclin.Intrexon will be responsible for technology discovery efforts and managing the patent estate as well as for certain aspects of manufacturing. Adeona will be responsible for conducting preclinical and clinical development of candidates, as well as for other aspects of manufacturing and the commercialization of the candidate product.
  • Autologous cardiac stem cells show promise in the treatment of ischemic cardiomyopathy, based on results from a Phase I study by a research team led by Robert Bolli of the University of Louisville, Kentucky. Among 14 patients who were analyzed, the left ventricular ejection fraction (LVEF) increased from 30.3% before the intracoronary infusion of cardiac stem cells to 38.5% four months after the infusion. Among eight patients who completed a year of follow-up, LVEF rose from 39.2% at four months to 42.5% at one year. Infarct size in seven patients fell 24% at four months and 30% at one year.
  • A pair of studies showed successful results in neutralizing strains of HIV. A team from the U.S., United Kingdom, Japan, and the Netherlands found that the antibody PGT 128 can neutralize about 70% of HIV strains by blocking their ability to infect cells, and do so more potently, in smaller concentrations, than any previously reported broadly neutralizing anti-HIV antibody. In a separate study, a team from the California Institute of Technology modified the antibody NIH45-46 to increase contact with the HIV-1 spike protein gp120, using structure-based rational design. The new antibody, NIH45-46G54W, neutralized six NIH45-46 resistant strains. The potency and breadth of the new antibody “suggests that NIH45-46G54W should be among a small number of characterized HIV antibodies considered for clinical testing,” the researchers concluded.

Launching in 2012: Human Gene Therapy Methods

The field of gene therapy is evolving and and holds great promise for its applications in treating human disease. Human Gene Therapy, the premier in the field, is launching Human Gene Therapy Methods to complement the flagship publication. HGT Methods answers the growing need for a central forum for nurturing, promoting, and advancing new technologies and methods that will ultimately pave the way for product development. The Editor in Chief is James M. Wilson, M.D., Ph.D.


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