Canada to Ship Experimental Ebola Vaccine to World Health Organization

Canada said it would start shipping 800 vials of an experimental Ebola vaccine to the World Health Organization on Monday to combat the deadly viral outbreak in Africa. The WHO is the international coordinating body for the Ebola outbreak, which has claimed almost 4,500 lives, mostly in Liberia, Sierra Leone and Guinea. “Canada views this experimental Ebola vaccine as a global resource and in the interest of global public health, we are sharing it with our international partners to help address the Ebola outbreak in West Africa,” Canadian Health Minister Rona Ambrose said on Saturday.
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Roche, Merck extend immunotherapy fight to breast cancer

Swiss drugmaker Roche and U.S. group Merck & Co are to present data on their rival immunotherapy drugs in breast cancer in December, extending the novel approach to fighting tumors to another cancer type. Early clinical results with Roche's drug, known as MPDL3280A, in so-called triple negative breast cancer (TNBC) will be revealed at the Dec. 9-13 San Antonio Breast Cancer Symposium, the firm said after announcing third-quarter results. TNBC does not respond to either of two kinds of hormonal therapy or drugs that target HER2 receptors, such as Roche's own Herceptin.
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Selecta, JDRF, Sanofi Collaborate on SVP Immunotherapy for Type 1

Selecta Biosciences Inc., and JDRF, announced that they have extended their joint research collaboration and attracted Sanofi as a partner in support of their program to develop a Synthetic Vaccine Particle (SVP) immunotherapy with the long-term goal to treat and potentially prevent the underlying cause of type 1 diabetes. This collaborative research program is aimed at accelerating the advancement of an SVP immunotherapy designed to reset the immune system and restore tolerance to substances (antigens) that cause the immune attacks on insulin-producing cells in the pancreas. Selecta’s proprietary tolerogenic SVP products show potential to re-educate the immune system to stop or suppress pro-inflammatory responses against a specific antigen, thereby halting the undesirable immune reaction without causing harmful global immune suppression common with general immune system modulating drugs.
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Astellas Dives into Gene Therapy with a Harvard Expert in Tow

Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease. The company has signed a deal with Harvard Medical School investigator and gene therapy expert Constance Cepko to collaborate on a gene therapy approach to retinitis pigmentosa (RP), a degenerative eye disease that can damage vision and lead to blindness. The plan is to investigate whether harmless adeno-associated virus (AAV) vectors can be used to deliver corrective genes to RP patients, Astellas said, in the process mapping out the mutations at the heart of the disease.
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DNAvaccine.com Presents: A Royalty-Free Way to Generate Functional Therapeutic Antibodies

Title: A Royalty-Free Way to Generate Functional Therapeutic Antibodies
Date: Tuesday, October 21, 2014
Time: 9:00 AM - 10:00 AM CDT

Space is limited! Reserve your Webinar Seat at: https://www4.gotomeeting.com/register/713139919

This complimentary 45-minute presentation with Dr. John Thompson, of Aldevron, will be live and followed by a 15 minute Q&A session. Register today to reserve your seat or to receive notification of the available recording.
Click below to read the complete webinar description.
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Gene Therapy Effective to Treat ‘Bubble Boy’ Syndrome

An advance in gene therapy may provide safe treatment to children with a fatal genetic disorder that leads to no functioning immune system. The majority of children with X-linked severe combined immunodeficiency, or X-SCID, die before the age of one and must live in a "bubble" for protection. During a clinical trial, nine baby boys were given healthy versions of the faulty gene that codes for the disease.

Eight of the boys were still alive up to 43 months after the treatment. They have now been able to live a life outside an isolated, sterile room, or a "virtual bubble" of constant medication to try to prevent infections. The disease affects about one in 250,000 children in the UK. Scientists in the US, Britain and France led the clinical trial, which built upon an earlier study where a gene was transferred into children with X-SCID.
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Medigene Receives Patent for TCR-Modified T cell Immunotherapy in Australia

Genocea Biosciences, Inc. GNCA, a biopharmaceutical company developing T cell-directed vaccines and immunotherapies against serious infectious diseases, announced today the presentation of final data from the Phase 1/2a study of GEN-003, the company’s immunotherapy candidate for treatment of genital herpes. The oral presentation, titled “Therapeutic HSV-2 Vaccine (GEN-003) Results in Durable Reduction in Genital Lesions at 1 Year “, will be given on Saturday, Oct. 11 at IDWeek 2014™ in Philadelphia.
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UConn Researchers Develop Personalized Ovarian Cancer Vaccines

Researchers at the University of Connecticut have found a new way to identify protein mutations in cancer cells. The novel method is being used to develop personalized vaccines to treat patients with ovarian cancer. “This has the potential to dramatically change how we treat cancer,” says Dr. Pramod Srivastava, director of the Carole and Ray Neag Comprehensive Cancer Center at UConn Health and one of the principal investigators on the study. “This research will serve as the basis for the first ever genomics-driven personalized medicine clinical trial in immunotherapy of ovarian cancer, and will begin at UConn Health this fall,” Srivastava says.
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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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