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Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy
Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy (DMD). The first boy received an infusion of the mini-dystrophin gene on March 22nd, administered under the supervision of principal investigator, Edward Smith, MD, Associate Professor of Pediatrics and Neurology at Duke University Medical Center. (Source: World Pharma News)
Source: World Pharma News - April 23, 2018 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news

Myonexus Therapeutics receives FDA orphan drug designation for LGMD type 2E treatment
(CincyTech) Myonexus Therapeutics , a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children's Hospital announce the US Food and Drug Administration (FDA) has granted Orphan Drug designation to Myonexus' lead candidate, MYO-101 for the treatment of limb girdle muscular dystrophy (LGMD) type 2E. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 23, 2018 Category: International Medicine & Public Health Source Type: news

Are cures bad for the drug business?
A glimpse at the cold calculations required to make profitable drug investments in new genetic technologies such as gene therapy. (Source: CBC | Health)
Source: CBC | Health - April 20, 2018 Category: Consumer Health News Tags: News/Health Source Type: news

Spark Therapeutics' ground-breaking gene therapy now being administered at select hospitals
Spark's first product, Luxturna, was approved by the FDA in December to treat an inherited retinal disorder that leads to blindness if untreated. The first patients have been treated with the gene therapy, which is now available at a select group of medical centers. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 20, 2018 Category: Pharmaceuticals Authors: John George Source Type: news

GE Rides Gene Therapy Wave With Ready-made Viral Drug Factories GE Rides Gene Therapy Wave With Ready-made Viral Drug Factories
General Electric is raising its bet on biotechnology with the launch of prefabricated manufacturing units for producing virus-based gene and cell therapies, novel anti-cancer treatments and vaccines.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - April 19, 2018 Category: Consumer Health News Tags: Medscape Today News Source Type: news

Krystal Biotech receives approval in Europe for gene therapy treatment
A gene therapy company has received a designation for one of its treatments from the European Medicines Agency. Krystal Biotech, developing and commercializing novel treatments for dermatological diseases, announced that KB103, a treatment for a severe skin disorder, was granted an Orphan Medicinal Product Designation by the agency, according to a news release. In November 2017, the FDA granted the same de signation for the treatment, which is meant for dystrophic epidermolysis bullosa, an incurable… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 19, 2018 Category: Biotechnology Authors: Stacey Federoff Source Type: news

Gene Therapy Found to Be Promising for & #946;-Thalassemia
THURSDAY, April 19, 2018 -- For patients with severe β-thalassemia, the need for red-cell transfusions can be reduced or eliminated by transducing cells with the LentiGlobin BB305 vector, according to a study published in the April 18 issue of the... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 19, 2018 Category: Pharmaceuticals Source Type: news

Penn Medicine's Carl June named one of Time Magazine's most influential people
(University of Pennsylvania School of Medicine) TIME named University of Pennsylvania cancer and HIV gene therapy pioneer Carl June, MD, to the 2018 TIME 100, its annual list of the 100 most influential people in the world. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - April 19, 2018 Category: Cancer & Oncology Source Type: news

Thalassaemia gene therapy trial shows 'encouraging' results
The treatment meant some patients stopped needing blood transfusions altogether. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 19, 2018 Category: Consumer Health News Source Type: news

Gene Therapy May Be Cure for Rare Blood Disorder
There are an estimated 288,000 cases of beta-thalassemia across the world, making it one of the most common genetic diseases, according to an editorial accompanying the study. (Source: WebMD Health)
Source: WebMD Health - April 18, 2018 Category: Consumer Health News Source Type: news

Gene therapy for blood disorder offers 'hope' in new study
An experimental gene therapy for blood disorders was shown to be safe and effective in helping beta thalassemia patients avoid blood transfusions in a new study. However, more research is needed, and if approved for use, it could come with high costs. (Source: CNN.com - Health)
Source: CNN.com - Health - April 18, 2018 Category: Consumer Health News Source Type: news

Gene Therapy For Inherited Blood Disorder Reduced Transfusions
A small study finds promise for using gene therapy to treat patients with beta-thalassemia, a blood condition that can cause severe anemia. The experimental treatment is in early development.(Image credit: Power and Syred/Science Photo Library/Getty Images) (Source: NPR Health and Science)
Source: NPR Health and Science - April 18, 2018 Category: Consumer Health News Authors: Rob Stein Source Type: news

Gene Therapy May Be Cure for Some With Rare Blood Disorder
WEDNESDAY, April 18, 2018 -- Wanda Sihanath didn't like the fact that her inherited blood disorder would not allow her to travel far from Chicago to attend college, but what could she do? Without regular transfusions and blood testing, the... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 18, 2018 Category: General Medicine Source Type: news

First gene therapy treatment to stave off childhood degenerative brain disease wins national clinical excellence award
(Burness) The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation's leading academic health centers, today awarded its most prestigious honor to a Massachusetts General Hospital research team for its discovery of the first successful gene therapy treatment for a fatal brain disease, cerebral adrenoleukodystrophy (ALD). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 18, 2018 Category: International Medicine & Public Health Source Type: news

New CDC chief stepped down from four groups to comply with ethics rules
Centers for Disease Control and Prevention Director Robert Redfield has resigned from positions at four groups, including a gene therapy biotechnology company and a conservative AIDS organization, to comply with government ethics rules, according to his financial disclosures. Redfield, a longtime HIV/AIDS researcher who started the job March 26, succeeded Brenda Fitzgerald, the former Georgia […]Related:These are the top priorities for the nation’s top cancer doctorFor advanced lung cancer, immune therapy plus chemo prolongs survivalThis 3-year-old has ‘a sparkle’ in her heart: The world’s sm...
Source: Washington Post: To Your Health - April 17, 2018 Category: Consumer Health News Source Type: news

GE rides gene therapy wave with ready-made viral drug factories
LONDON (Reuters) - General Electric is raising its bet on biotechnology with the launch of prefabricated manufacturing units for producing virus-based gene and cell therapies, novel anti-cancer treatments and vaccines. (Source: Reuters: Health)
Source: Reuters: Health - April 17, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

GSK Slims Portfolio With Sale of Rare Disease Gene Therapy Drugs GSK Slims Portfolio With Sale of Rare Disease Gene Therapy Drugs
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.Reuters Health Information (Source: Medscape Pathology Headlines)
Source: Medscape Pathology Headlines - April 13, 2018 Category: Pathology Tags: Pathology & Lab Medicine News Source Type: news

Pfizer takes big gene therapy step after acquisition in Chapel Hill
Pharmaceutical giant Pfizer (NYSE: PFE) has made a big step in the gene therapy space following its acquisition of Chapel Hill-based Bamboo Therapeutics in a deal worth up to $645 million back in August 2016.   Pfizer has initiated its “first new clinical trial from the Bamboo acquisition,” confirms Bob Smith, senior vice president of the global gene therapy business at Pfizer.  The new trial underway is a Phase 1b clinical trial for a gene therapy candidate for the treatment of boys with… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - April 13, 2018 Category: American Health Authors: Jennifer Henderson Source Type: news

Pfizer takes big gene therapy step after acquisition in Chapel Hill
Pharmaceutical giant Pfizer (NYSE: PFE) has made a big step in the gene therapy space following its acquisition of Chapel Hill-based Bamboo Therapeutics in a deal worth up to $645 million back in August 2016.   Pfizer has initiated its “first new clinical trial from the Bamboo acquisition,” confirms Bob Smith, senior vice president of the global gene therapy business at Pfizer.  The new trial underway is a Phase 1b clinical trial for a gene therapy candidate for the treatment of boys with… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 13, 2018 Category: Biotechnology Authors: Jennifer Henderson Source Type: news

GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics
GSK and Orchard Therapeutics have announced a strategic agreement, under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard, securing the continued development of the programmes and access for patients. This acquisition strengthens Orchard's position as a global leader in gene therapy for rare diseases. (Source: World Pharma News)
Source: World Pharma News - April 13, 2018 Category: Pharmaceuticals Tags: Featured GlaxoSmithKline Business and Industry Source Type: news

No Limb-Ischemia Wound-Healing Gains From Gene Therapy: STOP-PAD No Limb-Ischemia Wound-Healing Gains From Gene Therapy: STOP-PAD
Although the novel treatment didn't improve ulcers or other wounds from critical limb ischemia, the study highlights the microvasculature's important role in peripheral artery disease.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - April 12, 2018 Category: Consumer Health News Tags: Cardiology News Source Type: news

Pfizer launches Duchenne gene therapy study, joining Sarepta and Solid
Pfizer has officially entered the race to cure Duchenne muscular dystrophy with gene therapy, joining Cambridge-based Sarepta Therapeutics and Solid Biosciences as the only companies to use the experimental technology on a boy with the muscle-wasting disease. Pfizer (NYSE: PFE) announced on Thursday that the first patient in a small, early-stage trial of its gene therapy drug had been dosed on March 22. Earlier this year, both Sarepta (Nasdaq: SRPT) and Solid Biosciences (Nasdaq: SLDB) began their… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 12, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

GSK divests its rare disease gene therapy portfolio
GlaxoSmithKline is getting out of the rare disease gene therapy business. The London-based pharmaceutical company, which has large operations in Philadelphia and Montgomery County, said Thursday it has entered into an agreement to transfer its portfolio of approved and investigational rare disease gene therapies to Orchard Therapeutics. Under the terms of the deal, GSK will receive a 19.9 percent equity stake in Orchard — which is based in London and has U.S operations in California — and receive… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 12, 2018 Category: Pharmaceuticals Authors: John George Source Type: news

UK gene therapy firm Orchard plans stock offer after GSK deal
LONDON (Reuters) - Britain's Orchard Therapeutics, which has already raised more than $140 million to fund its work in gene therapy, plans another private sale of shares following its acquisition of a portfolio of GlaxoSmithKline rare disease medicines. (Source: Reuters: Health)
Source: Reuters: Health - April 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

GSK slims portfolio with sale of rare disease gene therapy drugs
LONDON (Reuters) - 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. (Source: Reuters: Health)
Source: Reuters: Health - April 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

GSK sells rare disease gene therapy portfolio
Biotech group Orchard Therapeutics acquires €594,000 treatment for ‘bubble boy’ syndrome (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - April 12, 2018 Category: Pharmaceuticals Source Type: news

GSK divests rare disease gene therapy drugs to Orchard Therapeutics
LONDON (Reuters) - GlaxoSmithKline said on Thursday is was transferring its rare disease gene therapy drugs to Orchard Therapeutics as Chief Executive Emma Walmsley makes good on her promise to prune the drugmaker's pharmaceuticals portfolio. (Source: Reuters: Health)
Source: Reuters: Health - April 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Lonza's Virus Factory Shows Gene Therapy's Texas-sized Promise Lonza's Virus Factory Shows Gene Therapy's Texas-sized Promise
Swiss drugmaker-for-hire Lonza is betting that trillions of customized viruses made at a giant factory in Texas will be the lucrative raw materials of a medical revolution.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - April 11, 2018 Category: Consumer Health News Tags: Medscape Today News Source Type: news

Genprex Appoints Julien Pham, MD, MPH As President To Lead Commercialization Of Oncoprex Immunogene Therapy
AUSTIN, Texas and CAMBRIDGE, Mass., April 11, 2018 -- (Healthcare Sales & Marketing Network) -- Genprex, Inc. (NASDAQ: GNPX), a clinical stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology plat... Biopharmaceuticals, Oncology, Drug Delivery, Personnel Genprex, Oncoprex, immunogene therapy, non-small cell lung cancer (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 11, 2018 Category: Pharmaceuticals Source Type: news

Vision Restored: The Latest Technologies to Improve Sight
Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness. (Source: The Scientist)
Source: The Scientist - April 10, 2018 Category: Science Tags: News Analysis Source Type: news

Lonza's virus factory shows gene therapy's Texas-sized promise
ZURICH (Reuters) - Swiss drugmaker-for-hire Lonza is betting that trillions of customized viruses made at a giant factory in Texas will be the lucrative raw materials of a medical revolution. (Source: Reuters: Health)
Source: Reuters: Health - April 10, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Novartis/gene therapy: corporate DNA
The control premium for AveXis is hefty, but should be given the benefit of the doubt (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - April 9, 2018 Category: Pharmaceuticals Source Type: news

Why Did Novartis Spend $9 Billion On Gene Therapy? CEO Says:'A Journey To Focus On Data And Digital '
“We're in a journey to focus Novartis as a medicines company powered by data and digital, ” chief executive Vas Narasimhan said on a conference call explaining the deal to analysts. “And already this year, we're off to a strong start to realize that goal. ” (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - April 9, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NYSE:NVS NASDAQ:AVXS NASDAQ:SRPT Source Type: news

Novartis buys US gene therapy group AveXis for $8.7bn
Deal is latest strategic move by Swiss drugmaker’s new chief executive (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - April 9, 2018 Category: Pharmaceuticals Source Type: news

Novartis enters agreement to acquire AveXis Inc. for USD 8.7 bn to transform care in SMA and expand position as a gene therapy and Neuroscience leader
Novartis announced today that it has entered into an agreement and plan of merger with AveXis, Inc. to acquire the US-based Nasdaq-listed clinical stage gene therapy company for USD 218 per share or a total of USD 8.7 billion in cash. The transaction was unanimously approved by the Boards of both companies. (Source: World Pharma News)
Source: World Pharma News - April 9, 2018 Category: Pharmaceuticals Tags: Featured Novartis Business and Industry Source Type: news

Gene therapy may help astronauts going to Mars resist deadly radiation
Researchers and scientists say new discoveries and drug creation could be beneficial to future astronauts on deep space missionsAn international group of researchers has come up with a new plan to help astronauts survive high-level radiation in space – and even get them to Mars without the deadly exposure expected during three years of space travel.Related:Elon Musk: we must colonise Mars to preserve our species in a third world warContinue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - April 7, 2018 Category: Science Authors: Sarah Betancourt Tags: Space Mars Nasa Science Source Type: news

Bracco hits Jubilant with patent complaint on rubidium PET
Contrast developer Bracco Diagnostics has filed a complaint against radiopharmaceutical...Read more on AuntMinnie.comRelated Reading: Bracco lands new FDA approval for MultiHance Bracco targets gene therapy for ultrasound contrast Bracco declines comment on Norris gadolinium lawsuit PBS report targets price hike for V/Q radiotracers Jubilant Pharma taps new DraxImage president Jubilant DraxImage, Cyclopharm ink deal (Source: AuntMinnie.com Headlines)
Source: AuntMinnie.com Headlines - April 6, 2018 Category: Radiology Source Type: news

Viral gene therapy not long-term solution for CAH
According to a study, published inHuman Gene Therapy, viral gene therapy for congenital adrenal hyperplasia provides only temporary relief, and abnormal adrenal cells will return. News Medical (Source: Society for Endocrinology)
Source: Society for Endocrinology - April 6, 2018 Category: Endocrinology Source Type: news

UCLA research could be first step toward healing the hearts of children with Duchenne
After a progressive weakening of the muscles takes away their motor skills, and then their abilities to stand and walk, most males with Duchenne muscular dystrophy die of heart and respiratory failure in their 20s.Now, researchers at theDavid Geffen School of Medicine at UCLA are pursuing a cutting-edge way to stop heart disease in patients with Duchenne, which affects one in 5,000 male babies born in the United States. Their work, which is supported by a David Geffen School of Medicine Seed Grant, is just one of a number of projects underway at the medical school in which interdisciplinary groups of UCLA researchers are p...
Source: UCLA Newsroom: Health Sciences - April 6, 2018 Category: Universities & Medical Training Source Type: news

Bertarelli Symposium 2018 to tackle the mysteries of the senses
(Harvard Medical School) Scientists will discuss latest research, new gene therapies at international sensory biology conference. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 5, 2018 Category: International Medicine & Public Health Source Type: news

Abeona Therapeutics Appoints Carsten Thiel, Ph.D., as Chief Executive Officer
Company strengthens management team with biopharmaceutical veteran bringing extensive commercial experience in life-threatening rare diseases Tim Miller, Ph.D. continues as president and assumes chief scientific officer role NEW YORK and CLEVELAND, A... Biopharmaceuticals, Personnel Abeona Therapeutics, gene therapy, epidermolysis bullosa, Sanfilippo syndrome (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 2, 2018 Category: Pharmaceuticals Source Type: news

Exclusive: Penn patient shares the story of her CAR-T cell therapy journey
Once given five weeks to live, Nicole Gularte's prognosis changed after she received a gene therapy treatment developed at Penn Medicine's Abramson Cancer Center. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 29, 2018 Category: Pharmaceuticals Authors: John George Source Type: news

New work from Ron Crystal's lab on treating hereditary adrenal disorders
(Mary Ann Liebert, Inc./Genetic Engineering News) A new study has definitively shown that a single treatment with gene therapy using adeno-associated viral (AAV) vector gene delivery to replace the defective gene responsible for congenital adrenal hyperplasia (CAH) will only temporarily alleviate the hereditary disorder. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - March 29, 2018 Category: Biology Source Type: news

Investors sue Solid Biosciences, say they were duped about Duchenne drug ’s safety
Cambridge-based Solid Biosciences is facing legal claims from investors after disclosing safety issues with its lead drug, a gene therapy treatment for Duchenne muscular dystrophy. Solid (Nasdaq: SLDB) revealed on March 14 that the FDA had halted an early-stage study of the drug after one patient was hospitalized. While Solid said that the patient had recovered, the disclosure sent shares of the newly-public biotech tumbling more than 60 percent. The FDA setback caught the attention of a handful… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 28, 2018 Category: Health Management Authors: Max Stendahl Source Type: news

Investors sue Solid Biosciences, say they were duped about Duchenne drug ’s safety
Cambridge-based Solid Biosciences is facing legal claims from investors after disclosing safety issues with its lead drug, a gene therapy treatment for Duchenne muscular dystrophy. Solid (Nasdaq: SLDB) revealed on March 14 that the FDA had halted an early-stage study of the drug after one patient was hospitalized. While Solid said that the patient had recovered, the disclosure sent shares of the newly-public biotech tumbling more than 60 percent. The FDA setback caught the attention of a handful… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 28, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

Genes in songbirds hold clues about human speech disorders, UCLA biologists report
Insights into how songbirds learn to sing provide promising clues about human speech disorders and may lead to new ways of treating them, according to new researchpublished in the journal eLife.There are about 9,000 species of birds, about half of which are songbirds. When these birds sing, the activity of a master gene called FoxP2 declines in a key region of the brain involved in vocal control known as Area X. The decrease in FoxP2 produces changes in the activity of thousands of other genes.Reed Hutchinson/UCLAStephanie WhiteFoxP2 also plays an important role in speech in humans. Stephanie White, a UCLA professor of int...
Source: UCLA Newsroom: Health Sciences - March 27, 2018 Category: Universities & Medical Training Source Type: news

Penn gene therapy spinout raises $9.2M
The company is operating a stealth mode for now. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - March 27, 2018 Category: Health Management Authors: John George Source Type: news

Penn gene therapy spinout raises $9.2M
The company is operating a stealth mode for now. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 27, 2018 Category: Biotechnology Authors: John George Source Type: news

Gene therapy may help brain heal from stroke, other injuries
(UT Southwestern Medical Center) Scientists have found a genetic trigger that may improve the brain's ability to heal from a range of debilitating conditions, from strokes to concussions and spinal cord injuries. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 27, 2018 Category: International Medicine & Public Health Source Type: news

Boy gets gene therapy to reverse blindness in Miami
Nine-year-old Creed Pettit of Florida became one of the first people to receive a new a newly-approved gene therapy for his blindness at the University of Miami's Bascom Eye Institute yesterday. (Source: the Mail online | Health)
Source: the Mail online | Health - March 22, 2018 Category: Consumer Health News Source Type: news