Sentences with phrase «as gene therapies»

The company is developing CAR T - cell immunotherapies for multiple myeloma, prostate and other cancer types, as well as gene therapies for orphan diseases.

Furthermore, the same technologies used to create genetic models of cancer show promise as gene therapies capable of repairing mutations that lead to a range of diseases.

Companies are also finding that using M&A to acquire R&D capabilities is an effective way to build positions in emerging technologies such as gene therapy and biosimilars.

So far, its trials have shown it can improve outcomes when used alongside other multiple myeloma drugs and that could offer it some insulation if the market gets disrupted by new treatment approaches, such as gene therapy.

These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth testing as gene therapy treatments for Parkinson's disease, Lu says.

This is advantageous, since dogs provide new models to investigate the disease mechanisms and to plan new therapies such as gene therapy, successfully applied to blindness in dogs and human previously,» explains Lohi.

«Both of the designed linker proteins may possibly be used in the future as a gene therapy treatment for congenital muscular dystrophy,» says Rüegg.

This achievement will also contribute to accelerating the research applications of RNAi such as to the development of RNA - based next - generation drugs, for example as gene therapy to suppress the production of a disease - causing protein.

Experimental approaches such as gene therapy are also being investigated, but Dr. Rudnicki's research suggests that these approaches will have to be modified so that they target muscle stem cells as well as muscle fibres.

As gene therapy and CRISPR / Cas9 - style gene editing research progresses, we can anticipate demand for services such as those provided by the Viral Vector Core.

For those patients without fully matched donors, researchers are working to devise better treatments, such as gene therapy.

Novel therapies such as gene therapy represent an opportunity to fix the faulty gene responsible and allow these children the chance to have a normal life.

Sophisticated cell targeting systems such as the gene therapy approach developed for senescent cell clearance by Oisin Biotechnologies could also be turned to stem cell or immune cell destruction, given suitable markers of cell chemistry.

Dr. Timothy Cripe from Nationwide Children's Hospital, joined PPMD on Wednesday, March 14 to answer some of the questions that are top of mind for our community right now, as gene therapy trials start.

PPMD recently hosted a webinar focused on understanding the different therapies that are being developed that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR / Cas9, how are they similar and different, and what are the challenges and limitations for each of them.

Find out why Ramez Naam believes that we should embrace, not fear, the biological enhancements that are increasingly available to us through the use of modern technologies such as gene therapy.

Not exact matches

The treatment, named Kymriah, was hailed by doctors and the life sciences community as a major advance in medicine and a boon to children and young adults with a certain form of leukemia (the group for whom the gene therapy is approved).

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.

LONDON, April 12 - GlaxoSmithKline is divesting its rare disease gene therapy drugs to private biotech company Orchard Therapeutics as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.

LONDON, April 12 - GlaxoSmithKline is divesting its rare disease gene therapy drugs to Orchard Therapeutics, it said on Thursday, as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio.

The Kamens claim the main thing that distinguishes their foundation from other brain tumor foundations is their focus on pediatric brain cancer specifically, as well as their close ties with pharmaceutical and biotech companies working in the fields of immunotherapy and target gene therapy.

New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.

After reporting second - quarter results that contained upbeat clinical news, Spark Therapeutics (NASDAQ: ONCE), a clinical - stage company focused on gene therapy, rose 18 % as of 12:30 p.m. EDT on Wednesday.

With major clinical successes in areas such as CAR - T, gene therapy, immune - oncology, cell therapy and gene editing, many see 2017 as the year that biotech really came of age.

«The event, the fourth of its kind, seeks to raise global awareness and create a forum for collaboration around the wide array of powerful and promising cell therapies, gene therapies, and immunotherapies emerging from medical institutions around the world, as well as the impact new technology will have on humanity and society,» a press release by the Cure Foundation explains (h / t Christian Post).

She has served as a biochemical patent agent and a research scientist for a gene - therapy company.

Kevin Whittlesey (2006 - 07 Congressional Fellow sponsored by Optical Society of America and Materials Research Society) has moved into the gene therapy field at 4D Molecular Therapeutics as director of program and alliance management.

The quantity of ctDNA, as well as the emergence of specific gene mutations, could be used in the future to steer therapy in a more precise manner.

Kotterman was 4DMT's first hire after working on its gene therapy technology as a graduate student in Schaffer's lab.

Gene therapy procedures in humans have been linked to the onset of leukemia and various tumors, as well as sudden death.

But as new methods of genetic modification emerge, both the scientific and the sports communities are becoming increasingly aware that gene therapy — the insertion of genes into an individual's cells and tissues — will make its way onto the playing field.

As a student, Arman did consultancy work for a friend who was launching a gene therapy company called Eos Neuroscience.

Gene therapy and stem cells are also being explored as ways to restore sight, but a drug would be simpler and any side effects should be reversible, says Kramer.

The study, published online today in Science Translational Medicine, further points to a master switch for these gene sets as a potential target of future therapies.

Scientists believe that is what happened during a 1999 French gene therapy trial on a group of 10 young children with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.

Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS.

Recent advances in the understanding of cancer have led to more personalized therapies, such as drugs that target particular proteins and tests that analyze gene expression patterns in tumors to predict a patient's response to therapy.

They note that targeting inflammation to treat infections offers an advantage over antibiotic therapy, as the former hinders gene transfer and the evolution of pathogens, while the latter promotes bacterial evolution and, ultimately, antibiotic resistance.

Philadelphia — Ten years ago this month the promise of using normal genes to cure hereditary defects crashed and burned, as Jesse Gelsinger, an 18 - year - old from Tucson, Ariz., succumbed to multiorgan failure during a gene therapy trial at the University of Pennsylvania.

It could be multiple things going wrong, and it's worth getting a full explanation, especially as we head closer and closer to gene therapy,» Butte said.

A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.

As Saaïd Safieddine, CNRS Director of Research at the Institut Pasteur and co-senior author of the study with Prof. Christine Petit (head of the Genetics & physiology hearing unit at the Institut Pasteur), explains, «we have just shown that it is possible to partially correct a specific form of hereditary hearing loss accompanied by balance problems using local gene therapy performed after the embryogenesis of the ear, which is primarily affected by the mutation responsible for the disorder.

It may also advance gene therapy for this disorder as well.»

To use viruses as delivery vehicles for gene therapy, researchers take all the harmful and replicative genes out of the virus and put in the therapeutic genes they want to deliver.

In the 1990s scientists such as himself, he explains, were too caught up in the promise of gene therapy to realize that they did not know enough about it to warrant human testing.

Kingsman hopes that the gene therapy technique will attack secondary cancers as well as primary ones.

These deletions also may extend to neighboring genes, an event known as «collateral lethality,» which may create new options for development of therapies for several cancers.

And in an ironic twist, the work could yield a new way to treat common ailments such as heart disease and cancer with gene therapies.

Researchers such as geneticist Richard King of the University of Minnesota and cell biologist Vitali Alexeev of Thomas Jefferson University are working on gene therapies or drugs that would fix albinism - causing mutations.

Surprisingly, the second round of gene therapy further strengthened the brain's response to the initially treated eye, as well as the newly treated one, perhaps «because the two eyes act in concert, and some aspects of vision rely on binocularity,» she says.