Gene Therapy

Deriving genomic diagnoses without revealing patient genomes

Although data-sharing is crucial for making the best use of genetic data in diagnosing disease, many individuals who might donate data are concerned …

Genetics

Doctors Plan Bold Test of Gene Therapy on Boys with Muscular Dystrophy - MIT Technology Review

In 1990, a British muscular dystrophy researcher, Kay Davies, described the unusual case of a 61-year-old man who by rights should not have been …

Genetics

Sangamo Therapeutics: The Breakthrough In Gene Therapy

Sangamo has introduced more capital and expertise to the roster.<p>Satisfying clinical results, industry-wide appraisal and the leader in ZFN gene …

Business

Fate lands new U.S. patent covering gene therapy

The USPTO issues U.S. Patent No. 9,675,641 to Fate Therapeutics (FATE -1.3%) covering the use of prostaglandins as viral transduction enhancers for …

Business

Voyager Therapeutics: What's Next For This 'Busted IPO'?

Today we look at a 'Busted IPO' named Voyager Therapeutics.<p>This early stage gene therapy concern has multiple 'shots on goal' and is well-funded.<p>A …

Business

OMF - Dominic Deluca, 20, acute myloid leukemia, (AML – a blood cancer), Forestdale, with Dr. Richard Stone, chief of staff, Program Director, Adult Leukemia, Dana-Farber 8-15-17

Dominic was diagnosed with acute myloid leukemia in July 2016. After working six days in one week and playing a pick-up basketball game, Dominic had …

Cancer

Gene Therapy - Abstract of article: Human genomics projects and precision medicine

Review<p><i>Gene Therapy</i> accepted article preview 14 August 2017; doi: 10.1038/gt.2017.77<p>This is an unedited manuscript that has been accepted for …

Genetics

Gene Therapy Is Now Available, but Who Will Pay for It?

By Ben Hirschler<p>LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce …

Medicine

US biotech Spark hires UK team as cure for inherited blindness edges closer

US biotech Spark Therapeutics has set up a UK office as it targets a rapid launch of its under-review gene therapy for a form of inherited blindness.<p>The Nasdaq-listed firm, which has a market cap of $2.4bn (£1.8bn), is at the forefront of the emerging global field of gene therapies for serious and …

Genetics

£2m UK consortium to tackle gene therapy

A new consortium, led by Oxford BioMedica, will embark on a two-year, £2 million project focused on gene and cell therapy manufacturing.<p>Other …

Genetics

A Novel Form of Gene Therapy Can Treat Diabetes With Genetically Modified Skin Transplants

“The human skin is a promising conduit for genetic engineering, as it is the largest and most accessible organ.” <br>Read More

Genetics

Looking Beyond Factors In Hemophilia

The hemophilia market is set for a shake-up - in the near term with Roche's (OTCQX:RHHBY) bispecific antibody emicizumab and in the longer term with …

Business

Researchers Developed a New Device Capable of Fixing Damaged Tissue with Just One Press

Just a Single Touch<p>Science fiction has often featured technology that’s capable of healing wounds and injuries almost instantly. Such devices could …

Breakthroughs

A revolutionary leukaemia treatment could soon be approved – here's what it means for patients

A revolutionary gene therapy treatment for leukaemia has been recommended by an advisory panel for approval by the Food and Drug Administration …

Cancer

Pfizer to invest $100 million in N Carolina for gene therapy | Miami Herald

Biopharmaceutical giant Pfizer Inc. plans to invest $100 million in a North Carolina plant to focus on a process by which genetic material is …

Business

Gene therapies getting closer to delivering on promise

Reuters reports that the time is fast approaching for gene therapies to deliver on their much-ballyhooed promise (therapeutically and commercially), …

Business

Bioverativ: Advancing Hemophilia Treatments

Despite strong competition in the hemophilia market, the company is increasing its market share.<p>Promising pipeline with recent True North acquisition.<p>…

Stocks

Leading US biotech Bluebird eyes UK launch for pioneering gene therapies

One of America’s leading biotechs, Bluebird Bio, is in talks with UK medicine regulators about a potential launch of its pioneering range of gene therapies for rare diseases.<p>Bluebird has a market value of over $4bn (£3bn) on New York’s Nasdaq stock exchange and specialises in bone marrow …

Genetics

Genome editing and the AMA Code of Medical Ethics

An international team of researchers recently published, in the journal <i>Nature</i>, their study using genome editing to correct a heterozygous mutation in …

Genetics

Scientists treat diabetes and obesity with genetically altered skin grafts

A group of scientists have used CRISPR-gene editing methods to create a skin graft capable of reducing the symptoms of diabetes and obesity in …

Genetics

CRISPR Gene Therapy via Skin Grafts Treats Obesity and Diabetes in Mice | GEN

Click Image To Enlarge +<p>When fed a high-fat diet, mice with a skin graft (left), genetically altered to secrete GLP-1 in response to the antibiotic …

Diabetes

Gene therapy reverses muscular dystrophy symptoms in dogs

Why it matters to you<p>Duchenne muscular dystrophy is a horrible disease which slowly attacks every muscle in the body. Gene therapy could help treat …

Genetics

Behind The Hype, Crispr Is A Story Of Slow Progress And Acrimony

Some people reading this morning's mainstream press headlines about a Nature paper describing the correction of a germline mutation using Crispr …

Genetics

Recent Advancements in the Regeneration of Auditory Hair Cells and Hearing Restoration

Introduction<p>Hearing loss is a serious global morbidity that affects 360 million people worldwide (WHO, 2013). Hearing loss is a neurological …

Neuroscience

Here's Why Spark Therapeutics Inc. Is Soaring Today

What Happened<p>After reporting second-quarter results that contained upbeat clinical news, <b>Spark Therapeutics</b> (NASDAQ: ONCE), a clinical-stage company focused on gene therapy, rose 18% as of 12:30 p.m. EDT on Wednesday.<p>So what<p>Here's a review of the key highlights from the quarter that have traders …

Stocks

Spark Therapeutics Q2 loss narrows; gene therapy candidate SPK-8011 shows promise in hemophilia A; shares ahead 10% premarket

Spark Therapeutics (NASDAQ:ONCE) Q2 results ($M): Revenues: 1.3 (-13.3%); Net Loss: (28.7) (+61.4%); Loss/Share: (1.04) (+56.7%).<p>Gene therapy …

Business

Philly drug maker seeks approval in EU for gene therapy

Philadelphia gene therapy company Spark Therapeutics has applied to the European Medicines Agency for approval to sell its treatment of rare …

U.S. Food and Drug Administration

Transient gene therapy may help youngsters with a premature ageing syndrome

Kick-starting the growth of cells with telomerase messenger RNA may slow the ageing of the cardiovascular system

Longevity

Maryland scientists research gene linked to depression

<b>By Carrie Wells</b>, <i>The Baltimore Sun</i>BALTIMORE — Although there are medications to treat depression, many scientists aren’t sure why they’re effective …

Depression

From Medicine to Modern Ag, GMOs Continue to Improve Our World

I was excited to see the lead story headline in the July 13, 2017 <i>New York Times</i>: “F.D.A. Panel Urges New ‘Living Drug’ To Fight Cancer, “Threshold In Medicine: Treatment That Alters a Patient’s Own Cells to Fight Back.”<p>And interestingly, that same day, the <i>Times</i> also ran a story with this headline: …

Agriculture