April 21, 2015 – 4:10 pm
Children with a devastating immune condition appear to have been cured, after becoming the first to be given a new form of gene therapy. Without the treatment, the patients who suffer from a rare condition called Wiskott-Aldrich syndrome, faced short life expectancies and prolonged periods in hospital. Doctors at Great Ormond Street Hospital, who led the successful trial, said the findings marked a turning point for gene therapy and could pave the way for similar techniques being used for more common conditions, including sickle cell disease and thalassaemia.
April 20, 2015 – 1:19 pm
Freiburg, Germany – Aldevron has recently entered into an Agreement with DaTaMabs allowing a fee-for-service offer to clients to develop humanized antibodies for therapeutic development starting from the target gene sequence. No additional downstream milestone or royalty claims will be made to the client. The core technology of Aldevron is to generate antibodies by genetic immunization. The animals are immunized intradermally with proprietary plasmid vectors allowing the target protein to be expressed by the animal to stimulate an immune response. In order to achieve this, the target protein must be brought to the cell surface to be seen by the immune system. Those proteins that normally follow such a pathway are secreted or cell surface molecules. Thus, these proteins will become correctly folded and post-translationally modified, i.e., they will be expressed in a native conformation. Aldevron Freiburg has 18 years of experience on approximately 1800 target protein targets using this technology, with an overall 84% success rate. Several of these antibodies also modulate target function. Cell-surface receptor molecules are the main targets for antibody therapy development, because these regulate cellular function through interaction with specific ligands and are accessible following systemic antibody application.
April 20, 2015 – 9:36 am
Milo Biotechnology announced the first Duchenne muscular dystrophy (DMD) patient was treated with follistatin gene therapy at Nationwide Children’s Hospital. The therapy, delivered by intramuscular injection, is designed to maintain or restore muscle function in boys affected with DMD. This six patient clinical trial follows from a trial of the therapy in Becker muscular dystrophy patients that demonstrated initial safety and efficacy. Results of that study were published in the January 2015 issue of the Journal Molecular Therapy. The trial is being led by Dr. Jerry Mendell, at Nationwide Children’s Hospital. According to Mendell, “This is the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease.” The DMD trial is being funded by the Duchenne Alliance, which coordinated funding from 15 disease foundations from around the world.
April 17, 2015 – 2:10 pm
Marking an important step in the development of immunotherapy cancer treatment, scientists have demonstrated that nanoparticle-coated bacteria can effectively deliver an oral DNA vaccine that stimulates the body's own immune system to destroy its cancer cells. This is the first time that a nanoparticle coating has been used for bacterial delivery of oral DNA vaccination in vivo. Compared with uncoated bacteria, coated bacteria can bypass many of the roadblocks that have so far limited the immune response and that currently pose the biggest challenge to DNA vaccinations against cancer. The researchers, led by Yuan Ping at Nanyang Technological University in Singapore and Guping Tang at Zhejiang University in Hangzhou, China, have published their study on using the nanoparticle-coated bacteria for oral DNA vaccines in a recent issue of Nano Letters.
April 16, 2015 – 1:06 pm
Gene therapy is superior to haploidentical hematopoietic stem cell transplantation (HSCT) to treat X-linked severe combined immunodeficiency (SCID-X1), according to a report published online April 13 in Blood. In SCID-X1 ("bubble boy disease"), impairment of the interleukin 2 receptor gamma chain causes lack of T cells and natural killer cells, which depletes B cells. The disease is X-linked. Without HSCT, most patients succumb to infection in infancy. Gene therapy introduces autologous HSCs given functional IL2RGgenes via a viral vector. Clinical trials to assess gene therapy for SCID-X1 began in 1999, but first-generation retroviral vectors led to insertional mutagenesis in five boys, one of whom died from the resulting leukemia. Retroviruses retooled to delete the enhancer elements that trigger the cancer are safe and efficacious so far, according to a report published in October 2013 in the New England Journal of Medicine.
April 13, 2015 – 2:39 pm
SAN DIEGO, APRIL 13, 2015 -- Ichor Medical Systems, Inc. (Ichor) announced today that it has entered into a product development collaboration and worldwide license agreement with Janssen Pharmaceuticals, Inc. (Janssen). Under the agreement, which was facilitated by Johnson & Johnson Innovation, the parties will work together to develop and commercialize DNA-based vaccine products for the treatment of chronic hepatitis B using Ichor’s TriGridTM electroporation technology for clinical administration. Ichor will receive an upfront payment, R&D support, and development and sales milestone payments up to a potential total of approximately $85 million USD, as well as royalty payments on any future licensed product sales. Janssen will assume responsibility for certain development costs and all commercialization costs associated with the program, including manufacturing and distribution expense for Ichor’s TriGrid Delivery System.
April 10, 2015 – 4:19 pm
Scientists at EPFL have demonstrated a new method that can be used to greatly improve the safety and efficiency of gene therapy using the patient's own stem cells. Ex vivo gene therapy is a medical technique in which stem cells are taken from the patient, and their deficient genes are switched with healthy ones. The stem cells are grown in the lab and re-inserted into the patient. However, this method often has health risks, such as leukemia and mutations. The problem is that most stem cells cannot be grown efficiently with current technologies, and the resulting cell population in the lab can contain a mix of healthy and unhealthy cells. Scientists at EPFL have developed a selection process that can detect the cells that have take up the healthy genes with great specificity, greatly reducing the risks of ex vivo gene therapy. The method, successfully tested on cells from a skin disease, is published in EMBO Molecular Medicine.
April 7, 2015 – 11:49 am
Adaptimmune Therapeutics, which is developing cancer immunotherapies based on genetically engineered T-cell receptors, filed on Monday with the SEC to raise up to $150 million in an initial public offering. The Abingdon, United Kingdom-based company, which was founded in 2008 , plans to list on the NASDAQ under the symbol ADAP. Adaptimmune Therapeutics initially filed confidentially on February 5, 2015. BofA Merrill Lynch, Cowen & Company and Leerink Partners are the joint bookrunners on the deal.
April 3, 2015 – 9:32 am
Genetic sequencing of a virus found in respiratory secretions of children in California and Colorado who suffered from paralysis or muscle weakness last fall reveals that they were infected with a mutated strain of enterovirus D68 that is closer to polio than other strains common in previous years. The study, published Monday in Lancet Infectious Diseases, sheds new light on one of the most troubling medical mysteries of recent years. Amid a nationwide outbreak of severe respiratory illness, doctors at hospitals nationwide began to report that they were seeing an alarming number of children with unexplained weakness in an arm or a leg to complete paralysis that required them to be put on ventilators. Treating physicians noted that many of the children appeared to be infected with enterovirus D68, but researchers were cautious about drawing a causal link because virus had been bouncing around the world since the 1960s and had typically only caused breathing issues such as coughing and wheezing.
April 1, 2015 – 9:31 am
Houston's MultiVir, a developer of viral vectors to deliver anticancer gene therapy, just filed for a $70 million IPO with the Securities and Exchange Commission as it seeks funding for clinical trials of its Phase I/II lead candidates for colorectal cancer and head and neck cancer, and to take the first FDA-approved gene therapy to the market. Both candidates utilize adenovirus as their specific vector. It does not typically integrate into the host cell's DNA, reducing the risk of side effects and disruptions to normal gene function. Other MultiVir candidates use a herpes simplex and vaccinia virus platform.