Gene therapy of choroideremia on the horizon
May 15, 2013 – 2:24 pm
Edmonton, Canada—Gene therapy trials in choroideremia may be on the horizon. Ian MacDonald, MSc, MD, talked about the future of this therapy.
“Choroideremia has long been seen as the next target for gene therapy. Gene therapy in this disease makes sense because the genetics of the disease are known, there is an understanding of the pathophysiology, suitable viral vectors can be used, and the vitreoretinal surgical techniques have been refined to deliver the vector to the subretinal space,” Dr. MacDonald explained. He is chair, Department of Ophthalmology, University of Alberta, Edmonton, Canada.
Cobra Biologics and Vaccibody sign agreement to manufacture DNA vaccine
May 14, 2013 – 10:33 am
Cobra Biologics Ltd, the international clinical and commercial manufacturer of biologics and pharmaceuticals and Vaccibody AS, a biotechnology company developing the next generation of vaccines against cancer and chronic infections, announces an agreement to manufacture Vaccibody’s lead DNA vaccine candidate against HPV-induced malignancies, such as precancerous lesions found in cervical cancer.
Inovio Pharmaceuticals #DNAVaccine Against Ebola & Marburg Filoviruses Provides Complete Protection in Challenge Preclinical Study
May 14, 2013 – 10:26 am
Inovio Pharmaceuticals, Inc. (NYSE MKT: INO) announced today that in a preclinical study of Inovio's SynCon®DNA vaccine against Ebola and Marburg filoviruses, labeled "Category A" bioterrorism agents by the U.S. government, the vaccine induced strong and broad immune responses and demonstrated 100% protection against death following a challenge with multiple variants of the pathogen in two animal models.
Optimization of a Der p 2-based prophylactic DNA vaccine against house dust mite allergy
May 13, 2013 – 3:45 pm
DNA vaccines encoding allergens are promising immunotherapeutics to prevent or to treat allergy through induction of allergen-specific Th1 responses. Despite anti-allergy effects observed in small rodents, DNA-based vaccines are weak immunogens in primates and humans and particularly when administered by conventional injection. The goal of the present study was to improve the immunogenicity of a prophylactic vaccine encoding the major house dust mite allergen Der p 2. In this context, we evaluated the influence of different DNA backbones including notably intron and CpG enriched sequence, the DNA dose, the in vivo delivery by electroporation as well as the heterologous prime boost regimen on the vaccine efficiency.
Safety of and Immune Response to a DNA HIV Vaccine Followed by an Adenoviral Vaccine Boost Given Three Different Ways to HIV Uninfected Adults
May 13, 2013 – 3:41 pm
One factor that may affect safety and immunogenicity to an HIV vaccine is the route of vaccine administration. Administration into the skin (intradermal) or subcutaneous tissue may be more immunogenic or provide a different pattern of immune responses than administration by the intramuscular route. Previous studies with other preventive vaccines suggest that the resulting immunogenicity following intradermal or subcutaneous vaccine administration is comparable or better than immunogenicity observed following intramuscular administration. Increased immunogenicity though use of a particular route will likely result in greater demonstrated efficacy, requiring fewer or lower doses of vaccine to elicit a sufficient immune response.
Evaluation of an ompA-based phage-mediated DNA vaccine against Chlamydia abortus in piglets
May 13, 2013 – 2:28 pm
Chlamydia abortus (C. abortus) is an obligate intracellular pathogen that causes abortion in pigs and poses a zoonotic risk in pregnant women. Although attenuated and inactivated vaccines are available, they do not provide complete protection in animals underlining the need to develop new vaccines. In this study, we tested the hypothesis that intramuscular immunization with an ompA-based phage-mediated DNA chlamydial vaccine candidate will induce significant antigen-specific cellular and humoral immune responses.
Hydrolytic Cationic Ester Microparticles for Highly Efficient DNA Vaccine Delivery #vaccinedelivery
May 13, 2013 – 2:13 pm
DNA vaccination holds great potential to be a safer and more efficient alternative to traditional vaccination strategies, but the current lack of nontoxic and effective delivery systems is the greatest impediment to its clinical implementation. In this work, a convenient one-step method is used to prepare a degradable “microgel” delivery platform, featuring hydrolytic esters. Prior to hydrolysis, these micrometer-sized gel particles can effectively condense DNA due to their positive surface charge. Upon entering antigen-presenting cells (APCs), the microgels can be hydrolyzed to nontoxic zwitterionic polymers, consequently releasing the DNA and inducing phagosomal escape. Surface charge, DNA loading, cytotoxicity, and gene transfection efficiency of the hydrolysable microparticles with different tertiary to quaternary amine ratios are systematically studied. Nonhydrolysable counterparts and commercially developed PLGA-CTAB particles are used as the control. The passive targeting effect is further evaluated by blocking the phagocytosis pathway of the cells. The hydrolytic microgels prepared in this study possess great potential to become a platform for DNA vaccine delivery.
PD1-based DNA vaccine amplifies HIV-1 GAG-specific CD8+ T cells in mice
May 7, 2013 – 11:26 am
Viral vector–based vaccines that induce protective CD8+ T cell immunity can prevent or control pathogenic SIV infections, but issues of preexisting immunity and safety have impeded their implementation in HIV-1. Here, we report the development of what we believe to be a novel antigen-targeting DNA vaccine strategy that exploits the binding of programmed death-1 (PD1) to its ligands expressed on dendritic cells (DCs) by fusing soluble PD1 with HIV-1 GAG p24 antigen. As compared with non–DC-targeting vaccines, intramuscular immunization via electroporation (EP) of the fusion DNA in mice elicited consistently high frequencies of GAG-specific, broadly reactive, polyfunctional, long-lived, and cytotoxic CD8+ T cells and robust anti-GAG antibody titers. Vaccination conferred remarkable protection against mucosal challenge with vaccinia GAG viruses.
Gene Therapy Trials Offer Hope For Inherited Retinal Diseases
May 2, 2013 – 10:28 am
TAKE HOME: A phase III trial of RPE65 gene therapy for Leber’s congenital amaurosis is under way. Phase I gene therapy trials for several other inherited retinal diseases are in progress or being planned.
