Geron (www.geron.com) eliminated 66 full-time positions, representing 38% of its workforce, after dropping its embryonic stem cell (ES) program, despite being first to go into clinical trials with an ES 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 2 clinical trials of imetelstat and GRN1005,” CEO John A. Scarlett, M.D., said. Imetelstat is being evaluated in four Phase 2 studies for non–small cell lung cancer, breast cancer, essential thrombocythemia, and multiple myeloma.

Neuralstem (www.neuralstem.com) advanced its ongoing Phase I safety trial of its spinal cord stem cells in the treatment of amyotrophic lateral sclerosis (ALS; Lou Gehrig's disease) at Atlanta's Emory University to the final two cohorts of patients with ALS, all of whom will be transplanted in the cervical region of the spine. The company received permission from the FDA to advance transplantation in the cervical (upper back) region. Previously, patients have received injections in the lumbar (lower back) 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 Japan's Takara Bio Inc. (Takara) 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 (a type of white blood cell) 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 (adeonapharma.com) will develop and commercialize a DNA-based therapeutic using the UltraVector platform and RheoSwitch Therapeutic System of Intrexon (www.intrexon.com) 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 (PGIS), 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 1 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% 4 months after the infusion. Among eight patients who completed a year of follow-up, LVEF rose from 39.2% at 4 months to 42.5% at 1 year. Infarct size in seven patients fell 24% at 4 months and 30% at 1 year. Results were published in The Lancet and presented at the American Heart Association 2011 Scientific Sessions.

A pair of studies showed successful results in neutralizing strains of HIV. A team from the United States, 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, NIH45–46^G54W, neutralized six NIH45–46-resistant strains. The potency and breadth of the new antibody suggest that “NIH45–46^G54W should be among a small number of characterized HIV antibodies considered for clinical testing,” the researchers concluded.

Factor IX gene therapy in six patients with hemophilia B has converted severe hemophilia to moderate or even mild disease, a research team reported in the New England Journal of Medicine. Following a single Factor IX treatment, four patients started making enough of their own clotting factor to no longer require regular protein injections, whereas the other two patients needed protein injections much less frequently than before. In a commentary in the January 2012 issue of Human Gene Therapy, Thierry VandenDriessche, Ph.D., methods editor for the journal and its sister journal Human Gene Therapy Methods, joined colleague Marinee Chuah, Ph.D., in concluding that the clinical trial “demonstrates unequivocally that gene therapy can result in a sustained therapeutic effect in hemophilia B patients.” Drs. VandenDriessche and Chuah are from the Department of Gene Therapy & Regenerative Medicine, Free University of Brussels, and the Center for Molecular and Vascular Biology, University of Leuven, Belgium.

A single injection of DNA for an HIV-neutralizing antibody into the muscle cells of live mice completely protected them against HIV transmission, a research team from two California institutions reported in Nature. David Baltimore of the California Institute of Technology and colleagues used a genetically altered adenovirus to infect muscle cells and deliver DNA that codes for antibodies isolated from the blood of people infected with HIV. The DNA is incorporated into the muscle cells' genome and programs the cells to manufacture the antibody, which is then secreted into the bloodstream. The technique builds on earlier work by researchers at the Children's Hospital of Philadelphia.