UNC Gene Therapy Spinout Bamboo Therapeutics Raises $49.5M Series A

North Carolina gene therapy startup Bamboo Therapeutics has raised a stunning $49.5 million, according to an SEC filing. The startup is focused on advancing the work of Dr. Richard Jude Samulski, director of the gene therapy center at the University of North Carolina, into the clinic to treat rare neurologic diseases like Duchenne’s muscular dystrophy. Bamboo says that Samulski was the first person to use adeno-associated viruses to replace defective genes with healthy ones; as a result, the company holds more than 20 patents in using AAV for therapeutic applications.
read more.-->

Researchers Identify Way Radiation May Fight Cancer Cells Escaping Immune System

A team of Georgia State University researchers is fighting cancers using a combination of therapies and recently found ways that radiation could maximize responses to novel immune-based therapeutic approaches to fight cancer. "Radiation can increase the expression of genes, which allows the immune system to attack tumor cells that may have previously escaped elimination," said Charlie Garnett Benson, assistant professor in the Department of Biology at Georgia State and lead author on the study, whose findings have been published in the International Journal of Oncology.
read more.-->

Gene Therapy for Rare Bleeding Disorder Achieves Proof-of-Concept

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder. "Our finding has great clinical relevance for patients with factor VII deficiency," said study leader Paris Margaritis, D. Phil., a hematology researcher at the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at The Children's Hospital of Philadelphia (CHOP). "These dogs have the type of mutation found in the majority of patients with this disorder, so this approach could lead to a sustained gene therapy in people." The study appeared online Dec. 23 in Blood.
read more.-->

GeneOne Life Science and Inovio Pharmaceuticals to Collaborate on Zika Virus Vaccine Development

SEOUL, South Korea, Jan. 22, 2016 (GLOBE NEWSWIRE) -- GeneOne Life Science today announced the initiation of a collaborative research program with Inovio Pharmaceuticals to test and advance a DNA-based vaccine for preventing and treating the emerging and virulent Zika virus infection. Inovio and its collaborators are leveraging their past experience in designing and testing novel DNA-based vaccines for related viruses including the West Nile, dengue, and chikungunya viruses. Moreover, Inovio and GeneOne are currently collaborating on two phase I stage vaccine candidates for severe infectious diseases: INO-4212, a vaccine for Ebola infection and GLS-5300, a vaccine for MERS infection. The Zika virus vaccine candidate is currently undergoing preclinical animal studies to evaluate its immunogenicity.
read more.-->

Magnetically-Guided Gene Therapy Heals Blood Vessels

A technique that combines gene therapy and magnets could someday provide a new tool for treating cardiovascular disease, which puts millions of lives at risk every year. Researchers have produced cells that carry magnetic nanoparticles linked to a therapeutic gene. With an external magnet to direct the cells, the researchers used them to repair damaged arteries in mice (ACS Nano 2015, DOI: 10.1021/acsnano.5b04996).
read more.-->

OrbiMed-backed LogicBio Raises $4M for Hemophilia Gene Therapy

LogicBio Therapeutics, a gene therapy startup backed quietly by OrbiMed Advisor’s Israeli arm, is in the midst of an early capital raise: It’s brought in $4 million of a proposed $5.4 million round, according to a regulatory filing. Looks like LogicBio uses recombinant adeno-associated virus-mediated gene targeting (rAAV) to target inherited diseases like hemophilia. The applications of this technology in association with CRISPR gene editing seem promising.
read more.-->

Gene Therapy to Treat HIV in Clinical Trial

HIV affects over 30 million people worldwide (over 1.2 million in the U.S.), and while there is no cure yet, there are various drug treatments to contain the virus, which attacks the immune system. Antiretroviral treatments consist of so-called cocktails of chemicals that mainly attack the virus’s ability to replicate in the bloodstream. They have to be taken daily and can have short term and long term side effects.But what if there were a means of rendering the virus harmless by altering the accessibility of the patient’s own white blood cells? That’s the goal of Calimmune’s treatment, called Cal-1. HIV attacks the immune system by destroying CD4+ T-lymphocytes, subsets of white blood cells that are crucial to the health of the immune system. A key landing point for the virus is the CCR5 receptor on the outer surface of these white blood cells.Cal-1 is designed to block the virus by preventing it from binding to CCR5. It achieves this by replacing the CCR5 receptor with a natural variant of the protein found only in the very small percentage of people that are born resistant to HIV.
read more.-->

UniQure’s Shares Rise on Early Gene Therapy Data for Hemophilia

UniQure this morning presented results from an early study of AMT-060, a gene therapy it’s developing for patients with hemophilia B. To be clear, these are numbers from just the first handful of patients in UniQure’s study, treated with a low dose of the gene therapy. And they’re early—the patient treated the longest is now 20 weeks post-treatment. For gene therapy to become a viable option for hemophilia, the effects will have to hold up for a long period of time, stop the dangerous, spontaneous bleeds that patients can suffer, and eliminate the need for frequent infusions of the recombinant factors that patients have to take to clot their blood. But the early results are the kind that can make a meaningful impact if they continue to hold up. The first two hemophilia patients treated with AMT-060, about 12 and 20 weeks after treatment, are now producing 4.5 percent and 5.5 percent, respectively, of normal Factor IX. To put that in context, these patients have severe or moderately severe hemophilia, meaning they typically produce less than 1 to 2 percent of these levels, and rely on frequent infusions to get those numbers up.
read more.-->

Gene Therapy Treats Duchenne Muscular Dystrophy in Mice

Gene therapy has long struggled with a delivery problem: How do you distribute an edited gene to all of the parts of the body where it needs to go? Now scientists have used the CRISPR genome editing tool in mice to solve that problem, a promising finding on the long path to a human therapy. Three papers, published Thursday in the journal Science, show that scientists can use CRISPR’s molecular scissors to treat mice that have been genetically modified to have Duchenne muscular dystrophy, a rare and fatal disease. A similar feat had been accomplished previously in a Petri dish with human cells or mouse embryos, but for the new work, scientists injected the therapy into juvenile and adult mice.
read more.-->

Discovery Finds Possible New Route to Malaria Vaccine

Oxford University researchers across the globe are working to beat Malaria. Now, a team of Oxford scientists in the UK and Kenya, working with colleagues in three Swiss institutes, have found two people who could reveal a new approach to targeting the malaria parasite. Malaria still claims over 400,000 lives every year, mostly due to infection by a single species of malaria parasite called Plasmodium falciparum. However, people living in areas where malaria is common naturally acquire immunity that protects them during their adult lives. So one route to finding new vaccines against malaria is by getting down to the molecular level to identify the processes behind this natural immune response.
read more.-->