After a String of Gene Therapy Licensing Deals, ReGenX Says it’s Time to Shine

ReGenX Biosciences is one of the most intriguing gene therapy biotechs you never heard of. And now the executive team in charge intends to start changing that. Today the Washington D.C.-based company is talking up an early stage but important inflection point for the company. It successfully concluded a proof-of-concept animal study for a gene therapy that addresses a rare lysosomal storage disorder. Now it is working on raising a new venture round that will allow the company to complete an IND and then jumping into the clinic with its own program in 2016.
read more.-->

Allergy Immunotherapy: What Is the Evidence for Cost Saving?

Given the widespread prevalence of allergic disease, its substantially associated clinical and economic burden, the unique disease-modifying benefits of allergy immunotherapy (AIT), and increased availability of sublingual immunotherapy (SLIT), a critical update of the evidence for AIT-related cost savings [for both subcutaneous immunotherapy (SCIT) and SLIT] is particularly relevant and timely. The present article reviews the evidence for SCIT-related and SLIT-related cost savings derived from a systematic review of the published literature.

Examined were 24 publications pertaining to the health economics of AIT. Except for one early study comparing the costs of AIT to symptomatic drug treatment (SDT), the remainder provide compelling evidence for AIT cost savings (whether SCIT or SLIT) over SDT. Furthermore, of the six studies comparing cost outcomes of SLIT to SCIT, four reported cost savings favoring SLIT.
read more.-->

Startup’s Antiviral Drug Combats HPV

San Diego startup Hera Therapeutics is developing a topical antiviral drug that attacks human papillomavirus - and early studies are showing it can successfully combat three strains, including two that cause 70 percent of all cervical cancer. The drug candidate, HTI-1968, blocked the replication of HPV-16, HPV-18 and HPV-11 cells, according to the NIAID-sponsored study. The work was conducted primarily by University of Alabama researchers Louise T. Chow and Thomas Broker. The drug could be effective against more, CEO Karl Hostetler said, but these are the only three HPV strains available for screening through the NIH.
read more.-->

Exclusive: U.S. Approval of Merck Cancer Immunotherapy Expected Soon

U.S. regulators are likely to approve Merck & Co's highly anticipated immuno-oncology drug, pembrolizumab, as a treatment for melanoma well ahead of a late October deadline, according to three sources familiar with the situation. If approved by the Food and Drug Administration, the drug would be the first in a promising new class designed to help the body's own immune system fend off cancer by blocking a protein known as Programmed Death receptor (PD-1), or a related target known as PD-L1, used by tumors to evade disease-fighting cells. Companies including Bristol-Myers Squibb, Roche Holding AG and AstraZeneca Plc are racing to develop similar treatments for a variety of cancers. Some analysts expect the new class could generate more than $30 billion in annual sales worldwide by 2025.
read more.-->

Speeding up the Fight Against Ebola, Other Diseases

For vaccines and treatments aimed at vanquishing infectious diseases, it’s a long, bumpy road from the laboratory to the doctor’s office, a journey that can take years and cost hundreds of millions of dollars — if it happens at all. Now, academic researchers, biotechnology specialists, and pharmaceutical leaders in Boston and elsewhere are offering tantalizing evidence that vaccines against Ebola and other killer diseases can be made faster and cheaper than previously believed. In a study accepted for publication in the journal Human Vaccines & Immunotherapeutics, a Boston research consortium, called VaxCelerate, details how it produced a vaccine ready for animal testing against lassa fever, a hemorrhagic disease similar to Ebola, for less than $1 million and in four months.
read more.-->

Will Inovio’s Deal With Roche Lead To A Cancer Blockbuster?

Roche and Inovio had announced they would develop INO-5150, a vaccine for prostate cancer, back on September 10, 2013. The agreement, giving Roche exclusive rights, allowed for milestone payments of up to $412.5 million, including the initial payment of $10 million. "In addition, Inovio is entitled to receive up to double-digit tiered royalties on product sales." INO-5150 has only been tested so far in animal studies. It targets two antigens associated with prostate cancer, PSMA (prostate-specific membrane antigen) and PSA (prostate-specific antigen). The animal studies showed the vaccine invoked "strong and robust T-cell immune responses."
read more.-->

Madison Vaccines Rides Immunotherapy Wave With Early-Stage Prostate Drug

Madison Vaccines (MVI) is working to develop a new treatment option for early-stage prostate cancer patients—after their prostates have been removed, but before the cancer has spread to the bones or other organs. The idea is to prevent, or at least delay, the onset of metastases and the need for castration by priming the immune system to recognize cancerous cells and kill them. Building on research from the lab of Douglas McNeel, an oncologist and professor of medicine at the University of Wisconsin-Madison, MVI is creating vaccines from tiny circular bits of DNA called plasmids. The company’s lead DNA vaccine, MVI-816, contains instructions for a protein named prostatic acid phosphatase (PAP), which is made by prostate cells. When injected into the skin, the DNA is taken up by the body’s cells (the company is still researching exactly which types of cells do the absorbing), which use the DNA to make copies of the PAP protein. That protein, in turn, acts as an antigen to stimulate an immune response against PAP, the company has shown in Phase I trials.
read more.-->

First Gene Therapy Trial Launched for Heart Patients with Mechanical Pumps

newseventsimages

For the first time in the world, a patient with a mechanical heart pump is taking part in a new gene therapy trial for heart failure. This is the start of a new clinical trial that will assess gene therapy for patients with heart pumps and provide detailed insight on its impact on the heart muscle. Heart failure occurs when the heart no longer pumps blood effectively and it affects hundreds of thousands of people in the UK. Some individuals with an advanced heart failure can be fitted with a Left Ventricular Assist Device (LVAD), which supports the failing heart and aims to restore normal blood flow. The LVAD is an electrically driven pump, moving the blood from the left ventricle into the main artery (aorta) so it can circulate the oxygen-rich blood to the rest of the body. Individuals with advanced heart failure who require a transplant may be fitted with an LVAD to keep them alive until a suitable donor heart becomes available. Currently there are around 100 to 150 people in the UK living with an LVAD.
read more.-->

Immunotherapy Targets Breast Cancer

A new technique aimed at harnessing the body's immune system to attack one of the deadliest forms of breast cancer is being developed by British scientists. The team led by Dr John Maher at King's College London is working on an immunotherapy treatment that specifically attacks HER2-positive tumours. Breast cancer cells that over-produce the HER2 protein are especially aggressive and hard to treat. They account for one in five of the 50,000 cases of breast cancer diagnosed each year in the UK. The new research is supported by a grant worth around £100,000 from the charity Breast Cancer Campaign.
read more.-->

Potential ‘Universal’ Blood Test for Cancer Discovered

140728094410-large

Researchers from the University of Bradford have devised a simple blood test that can be used to diagnose whether people have cancer or not. The test will enable doctors to rule out cancer in patients presenting with certain symptoms, saving time and preventing costly and unnecessary invasive procedures such as colonoscopies and biopsies being carried out. Alternatively, it could be a useful aid for investigating patients who are suspected of having a cancer that is currently hard to diagnose. Early results have shown the method gives a high degree of accuracy diagnosing cancer and pre-cancerous conditions from the blood of patients with melanoma, colon cancer and lung cancer. The research is published online in FASEB Journal, the US journal of the Federation of American Societies for Experimental Biology.
read more.-->