Novartis and Janssen Take Top Prizes at this Year’s UK Prix Galien

Swiss drug major Novartis (NOVN: VX) and US health care giant Johnson & Johnson’s (NYSE: JNJ) Europe-based subsidiary Janssen have won the two top awards at the UK Prix Galien awards. The prize was established to recognize the pharma industry’s outstanding achievement in the evolution of medicines.

Novartis took home the innovative product award for its breakthrough meningitis vaccine Bexsero (multicomponent meningococcal Group B vaccine [recombinant, adsorbed]), while Janssen won orphan drug award for tuberculosis medication Sirturo (bedaquiline). Sirturo is the first new treatment in this indication for four decades.
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Gene Therapy Approach to FAP: in Vivo Influence of T119M in TTR Deposition in a Transgenic V30M Mouse Model

Familial amyloidotic polyneuropathy (FAP) is a neurodegenerative disorder characterized by extracellular deposition of amyloid fibrils composed by mutated transthyretin (TTR) mainly in the peripheral nervous system. At present, liver transplantation is still the standard treatment to halt the progression of clinical symptoms in FAP, but new therapeutic strategies are emerging, including the use of TTR stabilizers. Here we propose to establish a new gene therapy approach using adeno-associated virus (AAV) vectors to deliver the trans-suppressor TTR T119M variant to the liver of transgenic TTR V30M mice at different ages. This TTR variant is known for its ability to stabilize the tetrameric protein. Analysis of the gastrointestinal tract of AAV-treated animals revealed a significant reduction in deposition of TTR non-fibrillar aggregates in as much as 34% in stomach and 30% in colon, as well as decreased levels of biomarkers associated with TTR deposition, namely the endoplasmic reticulum stress marker BiP and the extracellular matrix protein MMP-9. Moreover, we showed with different studies that our approach leads to an increase in tetrameric and more stable forms of TTR, in favor of destabilized monomers. Altogether our data suggest the possibility to use this gene therapy approach in a prophylactic manner to prevent FAP pathology.
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Local Biotech Company to Begin Human Testing for Ebola Vaccine


Without any effective treatments for Ebola, health officials in West Africa are in desperate need of a vaccine to keep the virus from spreading. Stepping into that void, among others, is Inovio Pharmaceuticals of Plymouth Meeting, Pennsylvania. President and CEO of Inovio, Joseph Kim, said beginning early next year their DNA-based vaccine will be given to 30 volunteers. The phase I human trial will test for safety and preliminary signs of efficacy based on analysis of blood samples for the appropriate immune responses.

The Inovio vaccine contains bits of Ebola virus DNA that correspond to its surface sugar coating, or glycoprotein. Once delivered into cells, those sequences can trigger the body to make protective antibodies and T cells that can go on the attack in case of infection. The technology was pioneered by David Weiner, a researcher at the University of Pennsylvania, who currently serves on Inovio's scientific advisory board and also helped co-found a precursor to the company.
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Up to 18 Exposed to U.S. Ebola Patient, Including Children

Health experts were observing up to 18 people, including children, who had contact with the first person to be diagnosed with the deadly Ebola virus in the United States, officials said on Wednesday. Confirmation that a man who flew to Texas from Liberia later fell ill with the hemorrhagic fever prompted U.S. health officials to take steps to contain the virus, which has killed at least 3,338 people in Liberia, Sierra Leone and Guinea, the World Health Organization said.
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Press Release: Compugen Discloses New Results for Novel Target Candidate for Cancer Immunotherapy

Compugen Ltd. today disclosed results from recent studies further confirming CGEN-15049 as a promising target candidate for cancer immunotherapy. These recent studies evaluated the function of this Compugen-discovered immune checkpoint candidate on immune cells derived from the tumor environment of melanoma patients. Based on these and earlier experimental results, CGEN-15049, which is expressed on various cancers including lung, ovarian, breast, colorectal, gastric, prostate and liver, is further advancing in the Company’s Pipeline Program, with ongoing therapeutic antibody development activities against this novel target.
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Liver Gene Therapy Corrects Heart Symptoms in Model of Rare Enzyme Disorder

In the second of two papers outlining new gene-therapy approaches to treat a rare disease called MPS I, researchers from Perelman School of Medicine at the University of Pennsylvania examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with this disorder. The second paper, which is published online in the Proceedings of the National Academy of Sciences this week, describes how an injection of a vector expressing the IDUA enzyme to the liver can prevent most of the systemic manifestations of the disease, including those found in the heart.
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Failed Cancer Vaccines Might Live Again with New Immune Drugs

Using vaccines to fight cancer is a field littered with failures but experts believe it is possible the approach could get a new lease of life if such shots are combined with a new class of drugs called checkpoint inhibitors. Unlike traditional preventative vaccines, therapeutic cancer vaccines are designed for people with established disease and are supposed to boost the patient's immune system to keep tumors at bay.

Unfortunately, the theory has not worked out in practice because, while the vaccines are successful at triggering a response from the "foot soldiers" of the immune system, cancer cells still manage to escape detection. The result has been a series of failures with high-profile experimental cancer vaccines such as Merck KGaA's Stimuvax and GlaxoSmithKline's MAGE-A3.
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GeoVax’s Chief Scientific Officer Featured as Lecturer at American Society for Microbiology Conference

GeoVax Labs, Inc., a biotechnology company developing HIV/AIDS vaccines, today announced that its Chief Scientific Officer, Harriet L. Robinson, Ph.D., presented a virtual lecture entitled "Working Towards an HIV Vaccine," on August 29, 2014 at the African Center for Integrated Laboratory Training in Sandringham, South Africa, sponsored by the American Society for Microbiology.

Dr. Robinson's lecture focused on the development of vaccines for control of the clade C pandemic in Africa and the clade B pandemic in the Americas. She reviewed clinical studies and vaccine responses that had correlated with control of infection in the first and only partially successful vaccine trial, conducted in Thailand. The lecture ended with a comparison of the distinctive features of the clade C vaccine advancing in human trials in Africa and the clade B vaccine that GeoVax is advancing in the Americas and the promise of these trials for success.
Dr. Robinson's lecture is available for viewing at
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Genzyme Collaborates on Gene Therapy for Rare Disease that Causes Childhood Blindness

Genzyme, a Sanofi company, today announced the establishment of a research collaboration with the University of Florida and the University of Pennsylvania to develop a gene therapy for the treatment of a rare genetic disease that causes childhood blindness. Leber congenital amaurosis type 1 (LCA-1) is usually diagnosed in children who are less than a year old, and patients remain severely visually impaired for the rest of their lives.

Genzyme is providing $900,000 in funding over three years to Dr. Shannon Boye, assistant professor of ophthalmology at the University of Florida, to fund her LCA-1 research. Dr. Boye’s research is focused on a gene called guanylate cyclase (GUCY2D) that is mutated in LCA-1 patients. The GUCY2D gene normally makes GC1, a protein expressed in photoreceptors, which are located in the retina of the eye and convert light into electrical signals that the brain interprets as vision. Genzyme’s funding supplements current funding from the Foundation Fighting Blindness. The majority of the early stage LCA-1 research is being conducted at the University of Florida, with Genzyme taking on increasingly more activities as the program advances toward clinical trials. Genzyme has the option to in-license the potential treatment before it enters clinical trials.
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Inovio, GeneOne to Advance Ebola Vaccine to Human Trials

Inovio Pharmaceuticals Inc. and GeneOne Life Science Inc. plan to advance Inovio's DNA vaccine for the Ebola virus into human trial next year, joining the ranks of other firms trying to fend off the rapidly spreading virus. The two companies are currently conducting pre-investigational-new-drug activities and plan to start a Phase 1 clinical trial in the first half of 2015, they said. Inovio and GeneOne, an international DNA vaccine manufacturer based in Seoul, said they would jointly seek additional third-party support and resources to further develop and commercialize this product upon successful completion of the Phase 1.

The companies' plans to co-develop the vaccine through clinical trial come as a number of pharmaceutical firms are speeding up their efforts to develop vaccines to prevent the deadly virus.
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