June 1, 2012 (Vol. 32, No. 11)

  • Amsterdam Molecular Therapeutics (AMT) lost its appeal to EMA’S Committee for Medicinal Products for Human Use (CHMP), which maintained a previous recommendation that the company’s gene therapy Glybera® (alipogene tiparvovec) should not be granted marketing authorization in Europe for lipoprotein lipase deficiency. CHMP re-evaluated Glybera in patients with severe or multiple pancreatitis attacks, concluding that the drug’s benefits did not outweigh its risks. CHMP said evidence of Glybera’s efficacy was not convincing since data from only 12 patients were available, and the reduced risk of pancreatitis seen in some patients could have been due to other factors. Despite favorable recommendations from the Committee for Advanced Therapies and Scientific Advisory Group, CHMP voted 16–15 for Glybera “under exceptional circumstances,” but 17 votes were required. AMT said it will liquidate and delist itself by summer, following completion of its sale to uniQure BV.
  • EMA released for public consultation its “Reflection Paper on Classification of Advanced Therapy Medicinal Products.” The paper clarifies the legal basis for the classification of medicines as advanced therapies and provides information on how these medicines are classified—as gene therapy, somatic-cell therapy, tissue-engineered, or combined medicines. It also gives details on the types of information applicants need to submit to EMA when applying for classification of their medicine as an advanced therapy, and discusses some borderline cases, as well as areas where scientific knowledge is limited or evolving rapidly. The reflection paper is open for comments until July 31.
  • ReGenX BioSciences received an orphan drug product designation from FDA for its program to treat homozygous familial hypercholesterolemia (HoFH). ReGenX uses a NAV rAAV8 vector expressing a normal copy of the human low-density lipoprotein receptor (hLDLR) gene. Research from ReGenX academic collaborators James M. Wilson, M.D., Ph.D., and Daniel J. Rader, M.D., both from the University of Pennsylvania, had shown in a mouse model of HoFH that delivery of functional hLDLR to the liver via NAV rAAV8-mediated gene delivery results in dramatic decreases in circulating LDL-derived cholesterol. ReGenX is working with academic partners to develop a clinical program intended to support licensure of an HoFH treatment.
  • A single administration of an adeno-associated virus (AAV)-based anti-cocaine vaccine dramatically reduced the need for proactive engagement by individuals abusing the drug. In a study published in GEN’s sister journal Human Gene Therapy, researchers from Weill Cornell Medical College, The Scripps Research Institute, and Cornell University found that nonhuman primate serotype AAVrh.10 can serve as a vehicle for the expression of an anti-cocaine antibody, enabling the vaccine to stimulate the body’s own immune system to block cocaine molecules from reaching the brain, thus preventing the drug’s effects before it can produce pleasurable sensations. The approach may be added to the future toolbox of therapeutic interventions for cocaine addiction, where current alternatives remain only behavioral therapies, the research team concluded. Weil Cornell signed an agreement with Immunovaccine to advance the vaccine.
  • A Phase I trial using a single intranodal injection of Ad-ISF35 in 15 patients with chronic lymphocytic leukemia (CLL) showed that the gene immunotherapy is feasible and safe. The replication-defective adenoviral vector expresses chimeric humanized CD154, the ligand for CD40 which is expressed on CLL cells. Binding of the ligand enhanced the ability of Ad-ISF35 to function as antigen-presenting cells, generating an anti-leukemia immune response.

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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