April 24, 2013 – 8:25 am
Australian doctors are using radical gene therapy to help treat a rare and aggressive childhood brain cancer, offering hope to young sufferers and their families. The clinical trial, underway at Westmead Hospital in Sydney, helps patients to tolerate higher doses of chemotherapy. While doctors are still reluctant to call it a breakthrough, the hope is that the gene therapy will eventually have a wider application in the treatment of many types of tumours.
April 22, 2013 – 1:58 pm
Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed.
DNAvaccine.com Presents: A straight-from-DNA approach to the production of antibodies to challenging targets
April 22, 2013 – 1:09 pm
Genetic immunization can be used to make antibodies using only an antigen’s DNA sequence. This allows the generation of antibodies to difficult targets in their native confirmation (e.g. GPCRs, ion channels, immunotherapy targets, cell surface receptors, etc.)
April 19, 2013 – 12:08 am
Inovio Pharmaceuticals, Inc. recently conferred with Vaccine Industry Excellence (ViE) Awards for "Best Therapeutic Vaccine" and "Best Early Stage Biotech" at the World Vaccine Congress, which is being held this week at Washington, DC. The ViE Awards recognise outstanding vaccine advancements and achievements of vaccine developers across the global industry as judged by a panel of global biotech industry stakeholders. The "Best Therapeutic Vaccine" award recognised Inovio's DNA vaccine designed to treat HPV-caused cervical dysplasia, VGX-3100. In human studies this therapeutic vaccine has demonstrated that it not only drives robust immune responses to antigens from high risk types of human papillomavirus (HPV) infection but that these immune responses display a powerful killing effect against cells changed by HPV into precancerous dysplasias. This desirable effect may ultimately contribute to the regression or elimination of cervical dysplasia, cervical cancer, and other cancers caused by HPV.
April 18, 2013 – 11:56 pm
In 1999, researchers at the University of Pennsylvania injected 19 people with a virus carrying a gene designed to correct a rare metabolic disease. Early results appeared promising: Among the first 17 adult subjects, the worst symptom was a fever, an expected response to the modified virus that carried the therapeutic gene.
April 18, 2013 – 11:52 pm
PDS Biotechnology Corporation today presented a summary of the company's preclinical data on its lead cancer immunotherapy PDS0101 at the World Vaccine Congress in Washington DC. The product is based on the company's ground-breaking and proprietary Versamune(TM) nanotechnology vaccine platform. PDS Biotechnology also announced that the company's Investigational New Drug (IND) application had been granted by the FDA allowing PDS0101 to be evaluated in human patients.
April 18, 2013 – 11:43 pm
Beat Biotherapeutics looked like it was in trouble after its co-founder and CEO died suddenly in June 2009. But the University of Washington spinout is definitely back in business, after recruiting a veteran CEO and raising another $2.5 million to advance an experimental treatment for heart failure.
April 18, 2013 – 11:30 pm
Startup biotechnology company Neuralgene (http://neuralgene.com) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS.
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
April 15, 2013 – 2:09 pm
Antibodies against adeno-associated viral (AAV) vectors are highly prevalent in humans. Both preclinical and clinical studies showed that antibodies against AAV block transduction even at low titers, particularly when the vector is introduced into the bloodstream. Here we measured the neutralizing antibody (NAb) titer against AAV serotypes 2, 5, 6 and 8 in the serum and matched synovial fluid (SF) from rheumatoid arthritis patients.
April 15, 2013 – 1:53 pm
Reports on H7N9 are increasing from individual case reports to “several” per day. Pigs and Chickens are still considered potential sources. Further geographic expansion has occurred (see graphic) suggesting that the disease would not be easily contained. The case mortality rate is high at 27-33% but the true denominator is not known.