March 1, 2012 (Vol. 32, No. 5)

  • Cardium Therapeutics reported preclinical study results showing that induced transient ischemia, using a standard angioplasty balloon catheter, combined with an intracoronary co-infusion of nitroglycerin, substantially enhanced adenovector-mediated gene delivery to the heart. Increasing cell-transfection efficiency allowed for effectively obtaining additional targeted expression of growth factors within the ischemic heart, where resulting blood vessel growth brought about improved blood flow throughout the ischemic myocardium. Intracoronary infusion of an adenovector directly to the ischemic region of the left ventricle caused no myocardial inflammation or other adverse effects.
  • Shire will develop therapeutics for hemophilia and other monogenic diseases based on the zinc finger DNA-binding protein (ZFP) technology of Sangamo BioSciences. Under the collaboration and license agreement, Shire will pay Sangamo $13 million up front, plus research, regulatory, development and commercial milestone payments, and royalties on product sales. Shire will receive exclusive worldwide rights to ZFP Therapeutics® designed to target genes for blood clotting Factors VII, VIII, IX and X, for use in investigating therapies for hemophilia A and B. Shire will also receive the right to designate three additional gene targets. Sangamo is responsible for all activities through submission of IND applications and European Clinical Trial Applications (CTA) for each product. Shire will reimburse Sangamo for internal and external research program-related costs, and oversee clinical development and product commercialization.
  • A research team published results showing that gene therapy could prevent, or reverse, the retinal disease X-linked Retinitis Pigmentosa (XLRP) in dogs—the first proof of treatment in animals for the most common form of RP. In a study in Proceedings of the National Academy of Sciences, the team detailed its single subretinal injection of a viral vector to deliver the therapeutic RPGR gene to diseased rods and cones. Researchers successfully treated dogs with two different RPGR mutations. The mutations disrupt photoreceptors, causing them to become useless for vision—a form of blindness rare in dogs, but common in humans. William Beltran, assistant professor of ophthalmology at University of Pennsylvania’s School of Veterinary Medicine, was the lead author.
  • Mesoblast received FDA clearance to begin a Phase III trial of its adult stem cells for type 2 diabetes. Mesoblast’s Mesenchymal Precursor Cell (MPC) product is the first of the firm’s biologic therapies deliverable intravenously. The Phase II trial will compare the effects of a single intravenous injection of one of three escalating doses of allogeneic MPCs compared with placebo in type 2 diabetes patients with elevated blood glucose levels. Primary endpoint will be safety of all three doses over three months. Secondary endpoints include per-dose effects on blood glucose control, changes in inflammatory markers and hormones that may be abnormal in type 2 diabetes patients, and effects on C-reactive protein.
  • Great Britain’s Human Fertilisation and Embryology Authority launched a public consultation on a policy to allow the first clinical tests of in vitro fertilization techniques now allowed only for research, with the goal of preventing women from passing genetic diseases on to their children. One technique entails transferring the pronuclei from an embryo with unhealthy mitochondria into a new embryo containing DNA of the father and mother, and the mitochondria from an egg donor. The other technique involves transferring the nuclear DNA from an egg (instead of an embryo) with unhealthy mitochondria and placing it into an egg with healthy mitochondria.

New This Year: Human Gene Therapy Methods

The field of gene therapy is rapidly evolving and holds great promise for its applications in treating human diseases. Human Gene Therapy, the premier journal in the field, launched 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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