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 www.geovax.com.
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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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PD-L1 Immunotherapy in Bladder Cancer

Initial excitement with PD-1 inhibitors was focused on renal cell carcinoma, lung cancer, and melanoma. However, a second wave of excitement has been generated by this approach in bladder cancer, explains Brian I. Rini, MD. Promising data from a phase I trial exploring the anti-PD-L1 therapy MPDL3280A were presented at the 2014 ASCO Annual Meeting, explains Rini. This study explored treatment with MPDL3280A in 67 patients with metastatic urothelial bladder cancer who received prior chemotherapy.

The objective response rate with MPDL3280A was 43% in PD-L1 positive patients (IHC 2/3). At the time of the data cut-off, 94% of patients had ongoing responses. This response rate is as high as chemotherapy with a promising safety profile, representing a potential "game-changer," Rini explains.
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Amgen One Step Closer To Immunotherapy Market

Amgen, Inc. (AMGN) has joined the list of big pharmaceuticals trying to enter the lucrative immunotherapy market. It submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) yesterday, for its novel cancer drug, blinatumomab. The drug is a bispecific T cell engager (BiTE) antibody construct, and is being evaluated for the indication of relapsed or refractory acute lymphoblastic leukemia (ALL). Immunotherapies are a class of cancer treatments that work by aiding the body’s own immune system to attack the cancer. This new market is expected to reach $35 billion in value over the next decade. If approved, Amgen will join the ranks of other pharmaceuticals, like Merck & Co., Inc. (MRK) and Bristol-Myers Squibb Co (BMY), which have already launched novel immunotherapies.
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Inovio Pharmaceuticals Expands HPV Immunotherapy Development To Aerodigestive Cancers

Inovio Pharmaceuticals, Inc. (NASDAQ: INO) announced today it has initiated a phase I clinical trial in patients with aerodigestive cancers. The trial will evaluate the safety, tolerability, and immunogenicity of INO-3106. This immunotherapy targets human papillomavirus type 6 (HPV-6), which causes most aerodigestive cancers. Aerodigestive cancers affect the lips, mouth, tongue, nose, throat, vocal cords, and parts of the esophagus and windpipe. The unmet need for patients suffering from this form of cancer is significant.

This study is part of Inovio's strategy to broadly expand clinical development of its DNA-based immune therapy products to treat different HPV types and the many diseases they cause. It is a phase I, open label compassionate study enrolling patients with invasive cancer who have exhausted all other treatment options (chemotherapy, radiation and surgery). The study will test Inovio's immunotherapy, INO-3106, alone or in combination with DNA-based IL-12, Inovio's proprietary immune activator, in subjects with HPV-6 associated aerodigestive malignancies. Successful results could open a path to pursuing an FDA orphan designation (special status granted for therapies for rare diseases) for aerodigestive cancers.
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Gene Therapy Helps Weak Mice Grow Strong

A virus that shuttles a therapeutic gene into cells has strengthened the muscles, improved the motor skills, and lengthened the lifespan of mice afflicted with two neuromuscular diseases. The approach could one day help people with a range of similar disorders, from muscular dystrophy to amyotrophic lateral sclerosis, or ALS. Many of these diseases involve defective neuromuscular junctions—the interface between neurons and muscle cells where brain signals tell muscles to contract. In one such disease, a form of familial limb-girdle myasthenia, people carry two defective copies of the gene called DOK7, which codes for a protein that’s needed to form such junctions. Their hip and shoulder muscles atrophy over many years, and some eventually have trouble breathing or end up in a wheelchair. Mice similarly missing a properly working Dok7 gene are severely underweight and die within a few weeks.
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Ebola, Marburg DNA Vaccines Prove Safe in Phase 1 Trial

A phase 1 clinical trial showed that 2 experimental vaccines, 1 against Ebola and 1 against the related Marburg virus, are well-tolerated and produce immunity after a course of 3 shots and a later booster. The results were published online September 14 in the Journal of Infectious Diseases. Both viruses are in the Filoviridae family, both cause hemorrhagic fever, and both rely on a glycoprotein on their surface for entry into human cells. Previous studies in nonhuman primates showed that vaccines based on this glycoprotein in its natural, wild-type form are safe and provide better protection than vaccines in which the glycoprotein had been altered.

The current trial tested each vaccine in 10 healthy adults aged 18 to 60 years who were recruited in 2008 and 2009. The vaccines were made with a strand of DNA encoding the virus's wild-type glycoprotein. The DNA does not replicate in human cells, but the patient's body makes glycoproteins from the DNA instructions, triggering an immune response. The Marburg virus vaccine was based on the glycoprotein from the Angola strain, and the Ebola vaccine was based on glycoproteins from the Zaire and Sudan species. (The current Ebola outbreak in western Africa involves the Zaire species.)
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