February 20, 2014 – 9:58 am
When a heart attack brings blood flow to a screeching halt, that’s only the first assault on our fist-size organ. Among survivors, the recovery itself fuels more permanent damage to the heart. Scar tissue can harden once-flexible heart muscle, making it less elastic. And as tentacles of this tissue creep over the aorta the heart muscle can no longer fully contract. This long-term damage can minimize the amount of oxygen-rich blood sent throughout the body, which can send patients spiraling into heart failure. Heart transplants are one way to circumvent these scar tissue issues, but donor hearts are always in short supply. Devising other truly effective solutions has long eluded researchers. A form of gene therapy, however, is now showing promise in pigs.
February 18, 2014 – 9:25 am
Deniz Safak was 5 years old when he first displayed symptoms of the disease that would later take his life. “He started being sick and had intense, stroke-like seizures,” his mother, Ruth, recalled. Doctors were baffled by the boy’s condition, and it took months before a diagnosis was made. Ruth and her husband, Erdhal, were told that Deniz was suffering from mitochondrial disease, an incurable condition that is passed from mother to child and can often be fatal.
February 13, 2014 – 9:38 am
A Cambridge biotechnology company launching Wednesday is taking aim at Parkinson’s disease and ALS with a new gene therapy that deliberately infects patients with a virus. The firm, Voyager Therapeutics, plans to use a class of viruses known as adeno-associated viruses as carriers to deliver vital proteins to the brain. Intentional infection may be counterintuitive, but the viruses used in the therapy are harmless to humans, making them ideal vehicles for moving proteins throughout the body, without troublesome side effects. Boston venture capital firm Third Rock Ventures considered Voyager’s research so promising that it invested $45 million to get the company off the ground, an unusually big bet on such an early stage life sciences firm.
February 13, 2014 – 9:08 am
RNA interference has become a ubiquitous biological tool, and is being harnessed for therapeutic purposes as well. Therapeutic posttranscriptional gene silencing takes advantage of the endogenous RNAi pathway through delivery of either chemically synthesized siRNAs, or transgenes expressing hairpin-based inhibitory RNAs (e.g., shRNAs and artificial miRNAs). RNAi has expanded the field of viral gene therapy from gene replacement to gene knockdown. Here, we review various noncoding RNAs such as shRNAs, miRNAs, and miRNA decoys which can be utilized for therapeutic applications when expressed from recombinant adeno-associated vectors (AAV), and present examples of their basic design. In addition the basis of exploiting cellular miRNA profiles for detargeting AAV expression from specific cells is described. Finally, an overview of AAV-mediated RNAi preclinical studies is presented, and current RNAi-based clinical trials are reviewed.
February 12, 2014 – 10:21 am
Recognizing that India plays a crucial role in supplying the world with vaccines, IMAPAC will bring together, for the fourth consecutive year, players from the Indian and international vaccine industry at the 4th Annual Vaccine World Summit India 2014 ( taking place from 4th to 6th March 2014 in Hyderabad, India). The Indian economy is among the fastest growing economies in the world. The Indian vaccine industry is expected to grow at 10-13% over the next ten years. India is the major supplier of basic Expanded Programme on Immunization vaccine to the United Nations Childres’s Fund (UNICEF). Dr Suresh Jadhav, Executive Director, Serum Institute of India, showed the importance India as a global vaccine supplier, when he said, "Around 75-to-80% of vaccines developed and procured by UN agencies are from the developing world and almost 80% of these are from India”.
February 11, 2014 – 12:05 pm
Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome -- an often fatal condition that causes organ damage and other medical complications. Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).
February 10, 2014 – 9:32 am
Cancer scientists in France have made “one of the first steps” toward the creation of personalised medicines targeted to combat an individual patient’s breast cancer. By scanning all of the DNA – the entire genome – of more than 400 women with late stage breast cancer for the first time, researchers have provided “proof of principle” that the technique can be used to understand the genetic cause of cancer in an individual, and help design tailor-made drugs to target it.
Ichor Enters Into an Agreement with Pfizer to Develop Electroporation Devices for use in Therapeutic Cancer Vaccine Regimens
February 5, 2014 – 9:19 am
Ichor Medical Systems, Inc. announced today that it has entered into a Collaboration and License Agreement with Pfizer. Ichor’s TriGrid™ intramuscular electroporation technology will be used to facilitate clinical administration of DNA-based vaccines as part of Pfizer’s preclinical cancer vaccine-based immunotherapy research program. Electroporation is a method that uses electrical pulses to create temporary pores in cell membranes, potentially allowing efficient uptake of a DNA-based vaccine or other substance. SAN DIEGO, February 5, 2014 -- Ichor Medical Systems, Inc. announced today that it has entered into a Collaboration and License Agreement with Pfizer. Ichor’s TriGrid™ intramuscular electroporation technology will be used to facilitate clinical administration of DNA-based vaccines as part of Pfizer’s preclinical cancer vaccine-based immunotherapy research program. Electroporation is a method that uses electrical pulses to create temporary pores in cell membranes, potentially allowing efficient uptake of a DNA-based vaccine or other substance.
February 4, 2014 – 1:29 pm
A University of Wisconsin professor’s cancer vaccine spin-off has locked in its first venture round to carry two potential prostate cancer treatments through early-stage clinical trials. Madison Vaccines Inc. began raising its $8 million round at the end of 2013, according to a SEC filing, and closed it in January with participation from Wisconsin-based investors Venture Management LLC, Venture Investors LLC, the Wisconsin Alumni Research Foundation and the State of Wisconsin Investment Board, the company said in a statement.
January 31, 2014 – 8:35 am
Oxford scientists have set up a biotechnology company to develop the use of gene therapy in treating blindness, and the Wellcome Trust medical charity has invested 12 million pounds ($20 million) in the start-up. NightstaRx plans to develop and sell therapies for retinal dystrophies - degenerative conditions affecting vision - by building on work carried out at the University of Oxford's Nuffield Laboratory of Ophthalmology, it said on Thursday.