Sentences with phrase «targeted disease therapies»

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One company that met her criteria is Toronto - based Deep Genomics, which applies machine learning to early disease detection to make possible more targeted therapies.

Furthermore, an early - stage study pumping up the dose of its hemophilia A treatment — which aims to be a one - time therapy for the rare blood clotting - related disease — appeared to hit its targets without serious side effects.

Luxturna is the first of a crop of treatments that target diseases caused by mutations in specific genes, and thus is referred to by many as the first gene therapy in the U.S.

This active IND enables Aura to begin initial clinical testing of AU - 011, a unique targeted therapy that could transform the primary treatment of patients with OM, a rare and life - threatening disease.

Cambridge, MA — March 30, 2017 — Aura Biosciences, a biotechnology company developing a new class of therapies to target and selectively destroy cancer cells using viral nanoparticle conjugates, announced today that it has enrolled and dosed the first patient in its Phase 1b clinical trial of light - activated AU - 011, an investigational, first - in - class targeted therapy in development for the treatment of ocular melanoma, a rare and life - threatening disease.

That therapy for treating melanoma is already in the development pipeline, along with 906 other biologics targeting over 100 diseases from autoimmune disorders to viruses.

What Stephen Hawking Missed: Small Biotechs Developing Promising Cell Therapies for Devastating Disease Source: Streetwise Reports (5/2/18) In the second of a two - part series exploring the disruptive cell therapy space, Maxim Group analyst Jason McCarthy takes a look at small - cap companies targeting big - ticket indications and their potential to drive blockbuster value for both patients and investors.

«These new insights into the complexities of epigenetic regulation are contributing to our basic understanding of this process in human health and disease and gives us new vision for how to go about targeting errors in DNA methylation with innovative drug therapies.»

Prof. Severine Vermeire (KU Leuven / UZ Leuven): «In the future, gut bacteria may be used to help identify PSC patients with a more or less aggressive disease, or patients that may benefit from microbiota - based therapies, such as fecal microbiota transplantation or targeted pre - and probiotics.»

The Southampton researchers believe that therapies to target these abnormalities may slow the progression of this disabling joint disease, but further work is required to examine this.

«Our study reveals a new mechanism that could be harnessed for biological therapies for lupus and other autoimmune diseases, where the immune system mistakenly targets the body's own cells,» says senior study author Boris Reizis, PhD, professor of Pathology and Medicine at NYU Langone.

They give some clues about why these diseases occur, but more detailed maps, such as this, will pinpoint where the problems lie — providing precise targets for therapies.

Currently, there are no targeted therapies for affected children, who are usually treated with topical steroids and, in severe disease, with immunosuppressant drugs.

Longer - term goals are therapies that more directly target the major pathway in this typically age - related disease.

«Research may provide new targets for inflammatory bowel diseases therapies.»

«Previous bacterial infection increases risk of newly - diagnosed Sjögren's syndrome: Identifying trigger will hopefully help future development of targeted therapy for patients suffering from this debilitating disease.»

He hopes that further studies may identify novel approaches to immune - based cancer therapy or reveal new drug targets for inflammatory diseases.

The researchers believe there are other disease entities, aside from cancers, that may be significantly affected by targeted epigenetic therapies.

«It is expected that this study will lay the foundation for developing a new class of potent and effective cancer therapies and the development of reagents targeting epigenetic events in immune - mediated diseases as well as other epigenetically - influenced diseases,» said Alani, who also is chief of dermatology at Boston Medical Center.

Now, by discovering a very similar condition in canines, researchers have a means to diagnose the disease, unravel its molecular intricacies, and target new therapies.

Understanding what happens with the UPR in disease is key to illuminating the normal operation of this essential pathway — and to providing insights into the development of targeted therapies.

This is important because it makes it possible to model almost any autoimmune disease, evaluate new drug therapies, and even identify novel targets in the emerging area of cancer immunotherapies.

While this could not heal or replace an entire body's worth of diseased muscle, it could be used in tandem with more widely targeted genetic therapies or to heal more localized problems.

Dr. Lazaridis says this molecule therefore should be further examined as a target of therapy for the disease.

Specific therapies that target a mutation can improve outcomes, such as reducing the risk of death or lessening the severity of disease.

«Commonalities in late stages of inherited blinding diseases suggest targets for therapy.»

Nonalcoholic fatty liver disease (NAFLD)-- induced hepatocellular carcinoma (HCC) is an emerging malignancy in the developed world; however, mechanisms that contribute to its formation are largely unknown, and targeted therapy is currently not available.

The same process has been studied as a potential genetic therapy for more than a decade, because you can target any disease gene with matching dsRNA.

But therapies that target common molecular pathways active late in disease could be part of a multi-pronged treatment plan for people with these inherited conditions.

While the disease can take many forms, recent advances have better characterized how lymphoma cells proliferate and interact with other cells and tissues, leading to the development of powerful, targeted therapies with fewer side effects than traditional approaches.

With the data provided by the FLI researchers, scientists around the world may now set targeted mutations to investigate the relevance of single genes for aging, in order to better understand regulating mechanisms and, eventually, develop new approaches for therapy or prevention of aging - related diseases.

Moreover, her studies are the first to indicate that therapies targeted at controlling the properties of smooth muscle cells within lesions may be highly effective in treating a disease that is the leading cause of death worldwide.

Ben Stanger, MD, PhD, a professor in the division of Gastroenterology, and first author Ravi Maddipati, MD, an instructor in the division of Gastroenterology, say that these results may prove useful in designing better targeted therapies to stop tumor progression and provide an improved non-invasive method for detecting early disease states in this highly lethal cancer.

Unraveling the underlying mechanism could help pave the way toward making phages a targeted therapy for diseases like cholera.

Many of the GPCRs are implicated in metabolic diseases, Brady says, and are the most common targets of drug therapy.

The findings, published today in Molecular Psychiatry, offer hope that future drug therapies might be able to stop or slow the disease without acting directly on the brain, which is a complex, sensitive and often hard - to - reach target.

In cases of new infection, prompt and targeted therapy is vital, as it can prevent the disease developing into a chronic condition that can lead to inoperable liver cancer.

The emphasis now is to store samples from almost every major study with correlative science in mind, and this is essential if we are to understand disease biology, mechanism of response and resistance to therapy in the era of targeted therapy and precision medicine.»

On the flipside, targeting this growth factor or BCL - 2 could reduce NK cell numbers and offer potential therapies for immune disorders such as some types of autoimmune diseases, sepsis or graft versus host disease, a side effect of bone marrow transplants.

This may contribute to the development of stem cell therapy targeted at other diseases.

Luxturna is the first gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

«To have a therapy at our disposal that safely targets just the T cells causing graft - versus - host disease would represent a major step forward in stem cell transplantation.

Genes that increase risk for AD are potential targets for new disease - modifying AD drug therapies.

The discovery could lead to targeted therapies and interventions for Alzheimer's disease, autism and other neurological conditions.

The two cell types are important to a broad range of organ systems in the body and play active roles in diseases that could be targets for nucleic acid therapies.

In the future, the therapy will target young patients to slow or halt the disease.

In doing so, they hope to contribute to the development of more effective, precise and target - driven therapies to prevent heart disease in women.

Thus, finding new targets for drugs already in use to treat other diseases, in other words drug repurposing, is an emerging area in developing anti-cancer therapies.

«Current efforts to develop therapies against age - related diseases target these disorders one by one,» says Dr. Dan Ehninger, research group leader at the DZNE site in Bonn.

Professor Moffatt said: «The genes we identified represent new potential drug targets for allergic diseases as well as biomarkers that may predict which patients will respond to existing expensive therapies.»