Gene Therapy

Trump, Tasty Pigs and Hurricanes: The Top Medical Stories of the Year

Health 2017 health Donald Trump CRISPR<p>From advances in genetic research that could have been lifted from a Michael Crichton novel to hard-nosed …

Genetics

New drug may combat deadly Huntington's disease

The results of the first trial are "a triumph" for Huntington's patients and their families since existing medications only help to manage the …

Medicine

Five Winners -- And One Loser -- From Medicine's Big Meeting About Blood

For the past two days, doctors who treat blood diseases have been meeting at the annual convention of the American Society of Hematology in Atlanta. There’s a lot of innovation coursing through this field. Here’s a guide to companies that are standing out.<p><b>Bluebird Bio Hits A Grand Slam</b><p>Researchers …

Cancer

Bluebird's gene therapy shows strong results in two rare blood disorders

Biotech<p>Bluebird’s gene therapy shows strong results in two blood disorders in small trials<p>A<p>TLANTA — Bluebird Bio (BLUE) only discloses gene therapy …

Genetics

Gene therapy gives 'bubble babies' immune systems

Initial results from a new gene therapy technique suggest it could open the doors to a cure for "bubble baby" disease. Lacking the ability to ward off even the most common infections, infants born with the genetic disorder -- known as severe combined immunodeficiency (SCID) -- usually die before …

Technology

Excitement Builds Around Gene Therapy Cures For Hemophilia

How enthralled are scientists by the prospects for gene therapy in hemophilia, the deadly disease of uncontrolled bleeding? Ask the usually staid New England Journal of Medicine, which just ran this headline: “A Cure for Hemophilia [is] within Reach.”<p>It would be one of medicine’s holy grails — and …

Medicine

San Francisco Is Really, Really Worried About Robots - MIT Technology Review

The Download<p>What's up in emerging technology<p>Today<p>San Francisco Is Really, Really Worried About Robots<p>Well, this seems ironic. It may be home to some …

The New England Journal of Medicine

Sangamo Therapeutics: Engineering Genomic Solutions For Rare Diseases

Sangamo just became the first company to do in vivo genome editing in the clinic.<p>One of the four trials Sangamo has in the clinical is SB-525. Pfizer …

Business

Groundbreaking Gene Therapy Drastically Reduces Bleeding Episodes In Hemophilia B Patients

A hemophilia expert called the results "striking" and just another example of a gene therapy renaissance that has brought patients with rare …

Molecular Biology

Gene Therapy for Factor IX Deficiency — NEJM

Quick Take Video Summary from The New England Journal of Medicine — Gene Therapy for Factor IX Deficiency

Medical Journals

College gets grant for research into depression treatments

A $2.3 million federal grant will fund University of Arizona College of Medicine-Phoenix research aimed at developing new treatments for …

Business

Gene therapy stops bleeding episodes in hemophilia trial

Researchers have scored their first clear success in using gene therapy to treat hemophilia, an inherited blood disorder. Ten men received a single …

Medicine

Routing gene therapy directly into the brain

Researchers report that transplanting genetically-engineered cells directly into the brain could help treat a variety of central nervous system …

Neuroscience

Gene therapy makes a big advance treating hemophilia B blood disorder

Jay Konduros used to rush home several times a year after accidentally cutting or bumping himself. There he would inject himself with refrigerated blood-clotting factor to prevent internal bleeding and extensive bruising and swelling. “I was walking on eggshells all the time,” said the former …

Medicine

2017 - Professor Elizabeth Rakoczy

<b>Professor Elizabeth Rakoczy is modifying viruses to use their powers for good. She’s created a new gene therapy for wet age-related macular</b> …

Medicine

Gene Therapy Shows Promise Against Blood-Clotting Disease

sGene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can …

Medicine

Catalyst Rich Pipelines - Strongest Biotech Plays For 2018

Small biotech catalysts may provide significant returns in 2018.<p>Wide breath of biotech companies provides diversity within the sector.<p>A couple large …

Stocks

FDA warns biohackers over DIY gene therapy

Biohackers were on the receiving end of an FDA warning this week, one that advised DIYers that publicly available gene therapy kits are illegal. Gene …

Genetics

Biohackers Disregard FDA Warning on DIY Gene Therapy - MIT Technology Review

Despite a warning from the federal government about do-it-yourself gene therapy, two companies say they’ll continue offering DNA-altering materials …

Genetics

Gene Therapy Is Booming, But How Will We Manage The Costs?

Jeffrey Marrazzo, the co-founder and chief executive officer of Spark Therapeutics, has an approach to valuing his company’s gene therapy treatment for a rare form of blindness that even he realizes may be controversial. The treatment is expected to win approval from the FDA in January, and the …

Genetics

Hemophilia Gene Therapy – A Pipeline Analysis Report – article directory

<b>“The Report Hemophilia Gene Therapy – A Pipeline Analysis Report provides information on pricing, market analysis, shares, forecast, and company</b> …

Molecular Biology

A promising gene therapy will be tried in living humans next year

Another Chance to Treat Heart Failure<p>According to the Centers for Disease Control and Prevention, nearly 6 million adults in the U.S. live with heart …

Medicine

Benitec advancing gene therapy candidate BB-301; shares ahead 15% premarket

Thinly traded nano cap Benitec Biopharma (NASDAQ:BNTC) is up 15% premarket on increased volume in response to its announcement that it is on track …

Business

This Week in Science: Nov 18 – 24, 2017

Researchers genetically modified beetles that grew a third fully functional eye.<p>A stable plasma ring was created in open air, without the need of a …

Science

The Feds Are Officially Cracking Down on Basement Biohackers

The Food and Drug Agency has issued a stern warning to anyone who might be crazy enough to undertake gene therapy in the do-it-yourself fashion. …

Molecular Biology

One way to end meth addiction? Alter the DNA of addicts using gene therapy

Addiction Antibody<p>In a major step forward in addiction treatment, a group of scientists at the University of Arkansas for Medical Sciences has …

Molecular Biology

Information About Self-Administration of Gene Therapy

Gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties …

Genetics

The Promise and Challenges of CAR-T Gene Therapy

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Genetics

Premarket Gainers as of 9:05 am

• DCIX +37% on Q3 result.<br>• DGLY +28% on Axon patent ruling.<br>• ARAY +10%.<br>• CZZ +10% on repurchase of shares.<br>• XGTI +10% on $12.5M Army contract.<br>• EGLT +9% on …

Stock Markets

ZIOPHARM's gene therapy candidate Ad-RTS-hIL-12 shows positive effect in treatment-resistant brain cancer in early-stage study; shares ahead 13% premarket

ZIOPHARM (NASDAQ:ZIOP) is up 13% premarket on average volume on the heels of its announcement of positive results from a Phase 1 clinical trial …

Business