Presentation Overview:
Dr. John Thompson will be discussing some of the key factors of antibody development for human therapy.
For immunotherapeutic development, generating promising antibodies is a critical step as these must specifically recognise their target proteins in their native conformation. Aldevron proposes its proprietary GENOVAC Antibody Technology as an important tool to generate specific custom-made antibodies against native proteins. Several examples of the success of this technology will be shown including the recent collaboration with OMT to directly generate human antibodies. The presentation will be roughly 30 minutes in length and will include an open Q&A session for all attendees.
Title: Antibody Development for Human Therapy with DNA Immunisation
Date: Tuesday, April 29th, 2014
Time: 8:00 AM – 9:00 AM CDT
Hearing Quality Restored with Bionic Ear Technology Used for Gene Therapy
April 24, 2014 – 10:17 am
Researchers at UNSW Australia have for the first time used electrical pulses delivered from a cochlear implant to deliver gene therapy, thereby successfully regrowing auditory nerves. The research also heralds a possible new way of treating a range of neurological disorders, including Parkinson's disease, and psychiatric conditions such as depression through this novel way of delivering gene therapy.
Loss of Memory in Alzheimer’s Mice Models Reversed Through Gene Therapy
April 23, 2014 – 10:30 am
Alzheimer's disease is the leading cause of dementia and affects some 400,000 people in Spain alone. However, no effective cure has yet been found. One of the reasons for this is the lack of knowledge about the cellular mechanisms which cause alterations in nerve transmissions and the loss of memory in the initial stages of the disease. Researchers from the Institute of Neuroscience at the Universitat Autònoma de Barcelona have discovered the cellular mechanism involved in memory consolidation and were able to develop a gene therapy which reverses the loss of memory in mice models with initial stages of Alzheimer's disease. The therapy consists of injecting a gene called Crtc1 (CREB regulated transcription coactivator-1) into the hippocampus - a region of the brain essential to memory processing. The protein, restored through gene therapy, gives way to the signals needed to activate the genes involved in long-term memory consolidation.
Massive $28.5 billion Big Pharma swap switches cancer drug, vaccine ownership
April 22, 2014 – 9:53 am
Novartis AG will focus more on cancer, GlaxoSmithKline Plc on vaccines and Eli Lilly & Co. on animal health as the drug-makers announced a series of deals Tuesday for a total of as much as $28.5 billion.
The transactions, as well as a plan to form a consumer- health joint venture with Glaxo, are part of an overhaul of the pharmaceutical industry spurred by the loss of sales as best-selling medicines lose patent protection.
Broad Institute Gets Patent on Revolutionary Gene-Editing Method
April 18, 2014 – 9:15 am
One of the most important genetic technologies developed in recent years is now patented, and researchers are wondering what they will and won’t be allowed to do with the powerful method for editing the genome.
On Tuesday, the Broad Institute of MIT and Harvard announced that it had been granted a patent covering the components and methodology for CRISPR—a new way of making precise, targeted changes to the genome of a cell or an organism. CRISPR could revolutionize biomedical research by giving scientists a more efficient way of re-creating disease-related mutations in lab animals and cultured cells; it may also yield an unprecedented way of treating disease.
Immunotherapy Data Marks New Era for Treating Lung Cancer
April 17, 2014 – 8:57 am
A new era of lung cancer therapy is close to dawning, using drugs that can prevent tumor cells from evading the immune system, experts said at the Fourth European Lung Cancer Congress (ELCC) in Geneva, Switzerland.
For decades, scientists and doctors thought immunotherapy, which uses treatments that harness the immune system to fight a disease, was of marginal benefit in lung cancer, said Jean-Charles Soria, MD, PhD, Institute Gustave Roussy in Paris, France.
However, Soria explained that a new class of drugs, known as “immunocheckpoint regulators,” has shown huge potential. New data on several of these drugs were presented at the conference.
Plasmid DNA Vaccines: Tissue Distribution and Effects of DNA Sequence, Adjuvants and Delivery Method on Integration into Host DNA
April 16, 2014 – 10:25 am
This interesting research article was used in Dr. Richard Stout's October webinar 'Delivering Your DNA Vaccine Without a Needle'. During the study, the team developed a sensitive assay for the detection of integration in vivo, which involves separation of high-molecular-weight genomic DNA from extrachromosomal plasmid by gel electrophoresis, followed by detection of potentially integrated plasmid in the gel-purified genomic DNA by polymerase chain reaction. They demonstrated that intramuscular injection of three different plasmid DNA vaccines in mice did not result in a significant level of detectable integration in the injected muscle.
AAVLife Gets $12M to Bring Gene Therapy to Rare Ataxia
April 16, 2014 – 9:03 am
Today, AAVLife is emerging from stealth with the help of a $12 million Series A round led by San Francisco-based Versant Ventures and the Inserm Transfert Initiative, a venture arm of the French Institute of Health and Medical Research. The funds will help Paris and New York-based AAVLife build on an animal study just published in Nature in which researchers used gene therapy to curb the cardiological dysfunction associated with Friedreich’s Ataxia, a rare, often fatal, genetic neuromuscular disorder that causes a progressive loss of motor function, among other things. AAVLife has been built to advance that work into clinical trials and hopes to get its first human study up and running in early- to mid-2015, according to CEO Amber Salzman.
DNAvaccine.com Presents: ‘Antibody Development for Human Therapy with DNA Immunisation’
April 11, 2014 – 10:14 am
Presentation Overview:
Dr. John Thompson will be discussing some of the key factors of antibody development for human therapy. For immunotherapeutic development, generating promising antibodies is a critical step as these must specifically recognise their target proteins in their native conformation. Aldevron proposes its proprietary GENOVAC Antibody Technology as an important tool to generate specific custom-made antibodies against native proteins. Several examples of the success of this technology will be shown including the recent collaboration with OMT to directly generate human antibodies. The presentation will be roughly 30 minutes in length and will include an open Q&A session for all attendees.
Title: Antibody Development for Human Therapy with DNA Immunisation
Date: Tuesday, April 29th, 2014
Time: 8:00 AM – 9:00 AM CDT
Dr. John Thompson will be discussing some of the key factors of antibody development for human therapy. For immunotherapeutic development, generating promising antibodies is a critical step as these must specifically recognise their target proteins in their native conformation. Aldevron proposes its proprietary GENOVAC Antibody Technology as an important tool to generate specific custom-made antibodies against native proteins. Several examples of the success of this technology will be shown including the recent collaboration with OMT to directly generate human antibodies. The presentation will be roughly 30 minutes in length and will include an open Q&A session for all attendees.
Title: Antibody Development for Human Therapy with DNA Immunisation
Date: Tuesday, April 29th, 2014
Time: 8:00 AM – 9:00 AM CDT
Resource: ‘Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s ataxia’
April 11, 2014 – 9:09 am
Abstract:
Cardiac failure is the most common cause of mortality in Friedreich's ataxia (FRDA), a mitochondrial disease characterized by neurodegeneration, hypertrophic cardiomyopathy and diabetes1, 2, 3. FRDA is caused by reduced levels of frataxin (FXN), an essential mitochondrial protein involved in the biosynthesis of iron-sulfur (Fe-S) clusters4, 5, 6, 7, 8. Impaired mitochondrial oxidative phosphorylation, bioenergetics imbalance, deficit of Fe-S cluster enzymes and mitochondrial iron overload occur in the myocardium of individuals with FRDA9, 10, 11, 12. No treatment exists as yet for FRDA cardiomyopathy13, 14. A conditional mouse model with complete frataxin deletion in cardiac and skeletal muscle (Mck-Cre-FxnL3/L– mice) recapitulates most features of FRDA cardiomyopathy, albeit with a more rapid and severe course15, 16. Here we show that adeno-associated virus rh10 vector expressing human FXN injected intravenously in these mice fully prevented the onset of cardiac disease.
Cardiac failure is the most common cause of mortality in Friedreich's ataxia (FRDA), a mitochondrial disease characterized by neurodegeneration, hypertrophic cardiomyopathy and diabetes1, 2, 3. FRDA is caused by reduced levels of frataxin (FXN), an essential mitochondrial protein involved in the biosynthesis of iron-sulfur (Fe-S) clusters4, 5, 6, 7, 8. Impaired mitochondrial oxidative phosphorylation, bioenergetics imbalance, deficit of Fe-S cluster enzymes and mitochondrial iron overload occur in the myocardium of individuals with FRDA9, 10, 11, 12. No treatment exists as yet for FRDA cardiomyopathy13, 14. A conditional mouse model with complete frataxin deletion in cardiac and skeletal muscle (Mck-Cre-FxnL3/L– mice) recapitulates most features of FRDA cardiomyopathy, albeit with a more rapid and severe course15, 16. Here we show that adeno-associated virus rh10 vector expressing human FXN injected intravenously in these mice fully prevented the onset of cardiac disease.
