Gene Therapy

This Lawyer Is Tired Of Pharma Getting Richer And Americans Paying The Price — So He’s Suing

News outlets report on stories related to pharmaceutical pricing.<p>Stat: Meet The Lawyer Trying To Pry Drug Pricing Secrets Out Of Big Pharma <br>Class …

Pharma

Biogen's Spinraza Dazzles But Rivals Rally

From a reported ovation at a medical conference to the smashing of sales forecasts, events this week confirm that the treatment of spinal muscular …

Medicine

3 Things You Didn't Know About bluebird bio, Inc.

Going beyond LentiGlobin and CAR-T.<p>Gene-therapy leader <b>bluebird bio</b> (NASDAQ:BLUE) has made waves in the CAR-T space, although it's still trailing the …

Genetics

Three-parent babies should make us examine our instinct for offspring who share our genes

When it comes to the genetic lottery, I’ve been lucky in a lot of ways—and unlucky in others. I was bequeathed my mother’s connective tissue anomalies, the celiac disease that runs on her side, <i>and</i> my father’s asthma, along with assorted other miseries. I was born by Caesarean section because my …

Genetics

Can't Pay? We'll Take It Away! star has been diagnosed with blood cancer

Fans and co-workers are helping him find a bone marrow transplant.<p>One of the stars of Channel 5's <i>Can't Pay? We'll Take It Away!</i> has revealed he has …

Cancer

The Morning After: Monday, April 24th 2017

Welcome to a fresh new week at Engadget. Over the weekend, you may have missed Uber's latest sketchy tale, a rallying call for truth and science, and a love letter to the Galaxy S8 from a once-Samsung hater.<p>Uber's app could identify iPhones even after they'd been wiped.<br>Apple threatened to drop …

Uber

Streamlining Stem Cell Manufacturing | GEN Genetic Engineering & Biotechnology News - Biotech from Bench to Business | GEN

Stem cell therapies hold extraordinary potential to revolutionize human medicine. Indeed, the global market for cell-based therapies is expected to …

Biotechnology

First gene therapy drug proves a flop in the market

When UniQure launched the first commercial gene therapy drug, Glybera, it promised a minor revolution in medicine. By using custom viruses to deliver genes, it could reportedly cure a rare disease (lipoprotein lipase deficiency) with just one round of treatment. However, it just didn't live up to …

Genetics

Using CRISPR to Reverse Retinitis Pigmentosa and Restore Visual Function

<i>Summary: Researchers use gene editing to reverse cell degeneration and restore vision in mouse models of retinitis pigmentosa.</i><p>Source: UCSD.<p><b>In mouse</b> …

Neuroscience

The World’s Most Expensive Drugs have European Owners

<b>A new report has listed the world’s most expensive drugs. Not surprisingly, those targeting rare diseases bear the heaviest price tags – let’s have a</b> …

Medicine

With its launch fizzling out, UniQure gives up on $1M+ gene therapy Glybera

UniQure's trailblazing gene therapy has met its end—and its demise offers a lesson to other drugmakers eyeing treatments for ultrarare diseases<p>After …

Genetics

Should Californians give more money for stem cell research?

Are Californians getting their money’s worth for the $3 billion they invested in stem cell science in 2004? Is there cause for optimism that major …

Biotechnology

Firm Pulls World’s First Gene Therapy Treatment: No One Wants It

The biotech company behind the Western world's first gene therapy and the most expensive prescription medicine in history is giving up on the product because of lack of demand.<p>Only one patient has been treated commercially since the $1 million treatment was first approved in Europe nearly five …

Genetics

The world's most pricey drugs, from a $1.2M gene therapy to a $450K lymphoma med

When Biogen announced the price on its new rare disease drug Spinraza—$750,000 the first year and $375,000 after that—critics popped up immediately. …

Medicine

Liz Parrish: Ageing is a disease. Gene therapy could be the cure

After her son was diagnosed with type 1 diabetes, Elizabeth Parrish, CEO of BioViva, began researching gene therapy treatments. She then started …

Genetics

For stem cell treatments, patients, doctors, scientists must collaborate

Stem cell research begins with scientists, passes on to physicians and ends up with patients.<p>But those roles aren’t as separate as they seem, said …

Medicine

Lack of demand forces uniQure to bail on gene therapy Glybera, marketing authorization in Europe to expire October 25

Citing a lack of demand, uniQure (NASDAQ:QURE) announces that it will not pursue a renewal of its European marketing authorization for Glybera …

Genetics

Audentes Therapeutics Gears Up for Secondary Offering

Audentes Therapeutics Inc. (NASDAQ: BOLD) saw its shares drop early on Wednesday after the company announced the pricing of its secondary offering. …

Medicine

“Immortal” Cells May Bring an End to Blood Donation

[DIGEST: IFLS, Science Alert, BBC]<p>Every two seconds, someone in the United States needs blood, with nearly 21 million blood components transfused …

Breakthroughs

New technology that can help the 360 million people with hearing loss

For the 360 million people worldwide who lack some or all of their ability to hear, technological interventions have already come a long way. But …

Biomedical Engineering

Psychologists Weigh In on the "Therapy App" Trend

It's a common 21st century conundrum: You have emotional and mental stuff you need to work out in therapy, but you don't have the time or cash to do …

Mental Health

Is epigenomics the next breakthrough in precision medicine?

In the past few decades, the field of genomics has rapidly evolved. From the Human Genome Project to the Million Genomes Project, molecular insight …

Cancer

Bioverativ: Just Stop The Bleeding

Eloctate, Bioverativ's recombinant factor VIII fusion protein, provides better quality of life than competitors for patients with hemophilia A …

Stocks

Hospital throws 'Great Gatsby' prom for teen healing from bone marrow transplant

Corinne Bass’ last year in high school wasn’t going exactly as she hoped. Instead of senior skip days and pranks, she was recovering from a bone marrow transplant that she received in February. She has been stuck in a hospital room since mid-December 2016.<p>“It was sad and also really frustrating. It …

Hospitals

France Approves Trials for the First Eye Gene Therapy Free of Viral DNA

<b>Eyevensys has received regulatory approval in France to start clinical trials using eye gene therapy technology that eliminates the need for viral</b> …

Genetics

Brain cells reprogrammed to make dopamine, with goal of Parkinson’s therapy

In a pioneering study, European scientists have “reprogrammed” brain cells in mice to correct some of the movement disorders of Parkinson’s …

Neuroscience

Mystery Clot Reveals a Helpful Mutant Gene in Hunt for a Cure - Bloomberg

When he first saw the results from his 23-year-old patient with a painful leg clot, Paolo Simioni assumed the test was botched and angrily told his …

Genetics

Cycling helps pause effects of Parkinson s Disease, US experts discover

Yahoo7<p>A surprising new treatment is helping sufferers of Parkinson's Disease to not only pause the effects of the illness, but even to reverse …

Medicine

Parkinson's breakthrough as experiments show brain cells killed by the disease can be replaced

Brain cells killed off by Parkinson's disease have been replaced in a breakthrough that could offer fresh hope to sufferers.<p>The neurons produce …

Neuroscience

Scripps Scientists Create HIV-Resistant Cells That Could Lead to AIDS Cure

LA JOLLA, CA - Scientists at The Scripps Research Institute announced Monday they have found a way to tether HIV-fighting antibodies to immune cells, creating a cell population resistant to the virus.<p>Their experiments under lab conditions show that the resistant cells can quickly replace diseased …

Medicine