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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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:

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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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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