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Preview: MedWorm: Gene Therapy

MedWorm: Gene Therapy News



MedWorm.com provides a medical RSS filtering service. Thousands of medical RSS feeds are combined and output via different filters. This feed contains the latest news in Gene Therapy



Last Build Date: Tue, 24 Apr 2018 15:41:13 +0100

 



Pfizer doses first patient using investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy

Mon, 23 Apr 2018 08:00:00 +0100

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)

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Myonexus Therapeutics receives FDA orphan drug designation for LGMD type 2E treatment

Mon, 23 Apr 2018 04:00:00 +0100

(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)



Are cures bad for the drug business?

Fri, 20 Apr 2018 16:41:13 +0100

A glimpse at the cold calculations required to make profitable drug investments in new genetic technologies such as gene therapy. (Source: CBC | Health)



Spark Therapeutics' ground-breaking gene therapy now being administered at select hospitals

Fri, 20 Apr 2018 11:12:52 +0100

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)



GE Rides Gene Therapy Wave With Ready-made Viral Drug Factories GE Rides Gene Therapy Wave With Ready-made Viral Drug Factories

Thu, 19 Apr 2018 20:58:03 +0100

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)



Krystal Biotech receives approval in Europe for gene therapy treatment

Thu, 19 Apr 2018 16:31:39 +0100

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)

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Gene Therapy Found to Be Promising for & #946;-Thalassemia

Thu, 19 Apr 2018 12:00:50 +0100

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)



Penn Medicine's Carl June named one of Time Magazine's most influential people

Thu, 19 Apr 2018 04:00:00 +0100

(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)



Thalassaemia gene therapy trial shows 'encouraging' results

Thu, 19 Apr 2018 02:23:08 +0100

The treatment meant some patients stopped needing blood transfusions altogether. (Source: BBC News | Health | UK Edition)



Gene Therapy May Be Cure for Rare Blood Disorder

Wed, 18 Apr 2018 22:15:18 +0100

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)



Gene therapy for blood disorder offers 'hope' in new study

Wed, 18 Apr 2018 21:31:39 +0100

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)

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Gene Therapy For Inherited Blood Disorder Reduced Transfusions

Wed, 18 Apr 2018 21:01:29 +0100

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)



Gene Therapy May Be Cure for Some With Rare Blood Disorder

Wed, 18 Apr 2018 17:00:26 +0100

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)



First gene therapy treatment to stave off childhood degenerative brain disease wins national clinical excellence award

Wed, 18 Apr 2018 04:00:00 +0100

(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)



New CDC chief stepped down from four groups to comply with ethics rules

Tue, 17 Apr 2018 19:39:31 +0100

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 smallest mechanical heart valve (Source: Washington Post: To Your Health)



GE rides gene therapy wave with ready-made viral drug factories

Tue, 17 Apr 2018 11:03:55 +0100

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)

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GSK Slims Portfolio With Sale of Rare Disease Gene Therapy Drugs GSK Slims Portfolio With Sale of Rare Disease Gene Therapy Drugs

Fri, 13 Apr 2018 19:57:22 +0100

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)



Pfizer takes big gene therapy step after acquisition in Chapel Hill

Fri, 13 Apr 2018 18:02:55 +0100

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)



GSK signs strategic agreement to transfer rare disease gene therapy portfolio to Orchard Therapeutics

Fri, 13 Apr 2018 08:00:00 +0100

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)



No Limb-Ischemia Wound-Healing Gains From Gene Therapy: STOP-PAD No Limb-Ischemia Wound-Healing Gains From Gene Therapy: STOP-PAD

Thu, 12 Apr 2018 16:58:20 +0100

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)

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Pfizer launches Duchenne gene therapy study, joining Sarepta and Solid

Thu, 12 Apr 2018 16:22:06 +0100

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)



GSK divests its rare disease gene therapy portfolio

Thu, 12 Apr 2018 15:48:51 +0100

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)



UK gene therapy firm Orchard plans stock offer after GSK deal

Thu, 12 Apr 2018 11:55:38 +0100

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)



GSK slims portfolio with sale of rare disease gene therapy drugs

Thu, 12 Apr 2018 08:51:45 +0100

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)



GSK sells rare disease gene therapy portfolio

Thu, 12 Apr 2018 08:34:40 +0100

Biotech group Orchard Therapeutics acquires €594,000 treatment for ‘bubble boy’ syndrome (Source: FT.com - Drugs and Healthcare)

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GSK divests rare disease gene therapy drugs to Orchard Therapeutics

Thu, 12 Apr 2018 07:18:46 +0100

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)



Lonza's Virus Factory Shows Gene Therapy's Texas-sized Promise Lonza's Virus Factory Shows Gene Therapy's Texas-sized Promise

Wed, 11 Apr 2018 19:32:30 +0100

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)



Genprex Appoints Julien Pham, MD, MPH As President To Lead Commercialization Of Oncoprex Immunogene Therapy

Wed, 11 Apr 2018 15:04:52 +0100

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)



Vision Restored: The Latest Technologies to Improve Sight

Tue, 10 Apr 2018 16:45:00 +0100

Cell implants, gene therapy, even optogenetics are making headway in clinical trials to treat various forms of blindness. (Source: The Scientist)



Lonza's virus factory shows gene therapy's Texas-sized promise

Tue, 10 Apr 2018 09:36:14 +0100

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)

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Novartis/gene therapy: corporate DNA

Mon, 09 Apr 2018 16:07:22 +0100

The control premium for AveXis is hefty, but should be given the benefit of the doubt (Source: FT.com - Drugs and Healthcare)



Why Did Novartis Spend $9 Billion On Gene Therapy? CEO Says:'A Journey To Focus On Data And Digital '

Mon, 09 Apr 2018 14:41:00 +0100

“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)



Novartis buys US gene therapy group AveXis for $8.7bn

Mon, 09 Apr 2018 09:17:25 +0100

Deal is latest strategic move by Swiss drugmaker’s new chief executive (Source: FT.com - Drugs and Healthcare)



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

Mon, 09 Apr 2018 08:00:00 +0100

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)



Gene therapy may help astronauts going to Mars resist deadly radiation

Sat, 07 Apr 2018 10:00:59 +0100

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)

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Bracco hits Jubilant with patent complaint on rubidium PET

Fri, 06 Apr 2018 23:16:51 +0100

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)



Viral gene therapy not long-term solution for CAH

Fri, 06 Apr 2018 12:00:00 +0100

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)



UCLA research could be first step toward healing the hearts of children with Duchenne

Fri, 06 Apr 2018 03:02:41 +0100

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 pursuing new treatments and cures.The scientists are trying to use minuscule nanoparticles to carry bits of DNA to the hearts of people with Duchenne. Inside heart cells, ...



Bertarelli Symposium 2018 to tackle the mysteries of the senses

Thu, 05 Apr 2018 04:00:00 +0100

(Harvard Medical School) Scientists will discuss latest research, new gene therapies at international sensory biology conference. (Source: EurekAlert! - Medicine and Health)



Abeona Therapeutics Appoints Carsten Thiel, Ph.D., as Chief Executive Officer

Mon, 02 Apr 2018 15:12:07 +0100

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)

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Exclusive: Penn patient shares the story of her CAR-T cell therapy journey

Thu, 29 Mar 2018 15:02:02 +0100

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)



New work from Ron Crystal's lab on treating hereditary adrenal disorders

Thu, 29 Mar 2018 04:00:00 +0100

(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)



Investors sue Solid Biosciences, say they were duped about Duchenne drug ’s safety

Wed, 28 Mar 2018 20:19:44 +0100

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)



Genes in songbirds hold clues about human speech disorders, UCLA biologists report

Tue, 27 Mar 2018 21:37:00 +0100

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 integrative biology and physiology and senior author of the study, thinks FoxP2 and the changes it causes could be a part of the molecular basis for vocal learning. In both ...

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Penn gene therapy spinout raises $9.2M

Tue, 27 Mar 2018 11:37:10 +0100

The company is operating a stealth mode for now. (Source: bizjournals.com Health Care News Headlines)



Gene therapy may help brain heal from stroke, other injuries

Tue, 27 Mar 2018 04:00:00 +0100

(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)



Boy gets gene therapy to reverse blindness in Miami

Thu, 22 Mar 2018 22:34:43 +0100

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)



Boy Hopeful Groundbreaking Surgery In Boston Will Improve Vision

Wed, 21 Mar 2018 21:29:47 +0100

BOSTON (CBS) – A medical breakthrough in Boston with far reaching implications offers a young boy a chance to see better. It’s the first time an FDA approved gene therapy has been used to fight an inherited disease. And after groundbreaking surgery at Mass Eye and Ear, both patient and doctor are beaming, with hope. “This is just the beginning. I’m the first one. There’s going to be more,” says 13-year-old Jack Hogan from New Jersey who has had serious vision problems his whole life. “I have trouble seeing at night. Probably around 6 o’clock, I’ll go inside and I can’t see outside at all.” Jack also has no peripheral vision. But Tuesday at Mass Eye and Ear in Boston, Jack was the first person outside of clinical trials to receiv...

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Mass. Eye & Ear makes history with first gene therapy procedure for inherited blindness

Wed, 21 Mar 2018 13:00:51 +0100

Photo courtesy of Mass. Eye & Ear Massachusetts Eye and Ear Infirmary made history this week, becoming the first institution to use an FDA-approved gene therapy to treat a patient with an inherited disease. Spark Therapeutics‘ (NSDQ:ONCE) Luxturna gene therapy, which was approved by the FDA in December, is designed to improve vision in patients with inherited retinal disease caused by a particular genetic mutation. Yesterday, Dr. Jason Comander injected the treatment into the eye of a 13-year-old boy from New Jersey. Get the full story at our sister site, Drug Delivery Business News. The post Mass. Eye & Ear makes history with first gene therapy procedure for inherited blindness appeared first on MassDevice. (Source: Mass Device)



Children's Hospital Los Angeles treats first patient on the west coast using gene therapy

Wed, 21 Mar 2018 04:00:00 +0100

(Children's Hospital Los Angeles) Surgeons of The Vision Center at Children's Hospital Los Angeles completed a revolutionary gene replacement procedure on Tuesday, March 20 at 9:20 AM, to restore vision in a patient with retinal degeneration, using the first gene therapy ever approved for a genetic condition in the United States. (Source: EurekAlert! - Medicine and Health)



Mass. Eye and Ear makes history, injecting $850,000 gene therapy for first time

Tue, 20 Mar 2018 20:24:29 +0100

Philadelphia-based biotech Spark Therapeutics made history in December by winning FDA approval for the first gene therapy for an inherited disease. Now, Massachusetts Eye and Ear says it has performed the first post-approval injection of the drug, which treats a form of blindness and costs $850,000. The hospital announced on Tuesday that Jason Comander, the associate director of its inherited retinal disorders service, had performed the procedure on a 13-year-old boy from New Jersey. He is the first… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)



Surgeons at Children's Hospital Los Angeles treat the hospital's first patient with gene therapy

Tue, 20 Mar 2018 04:00:00 +0100

(Children's Hospital Los Angeles) On March 20, 2018, surgeons at Children's Hospital Los Angeles, treated the hospital's first patient with gene therapy. The complex eye surgery was performed by dedicated retina specialist, Aaron Nagiel, MD, PhD , of The Vision Center at CHLA, on an adult patient with Leber's Congenital Amaurosis, a form of childhood blindness that affects the retina. (Source: EurekAlert! - Medicine and Health)

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Mass. Eye and Ear performs first FDA-approved gene therapy procedure for inherited disease

Tue, 20 Mar 2018 04:00:00 +0100

(Massachusetts Eye and Ear Infirmary) Massachusetts Eye and Ear made medical history on Tuesday by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. The occasion marks the beginning of a new era in medicine, as it is the first time any FDA-approved gene therapy has been given to a patient for any inherited disease. (Source: EurekAlert! - Medicine and Health)



Study finds the heart can terminate atrial fibrillation itself after local gene therapy

Mon, 19 Mar 2018 04:00:00 +0100

(European Society of Cardiology) The heart is capable of terminating arrhythmias itself after local gene therapy, potentially avoiding the need for patients to undergo painful electric shocks, according to a proof-of-concept study presented today at EHRA 2018, a European Society of Cardiology congress. (Source: EurekAlert! - Social and Behavioral Science)



Award time: Life Sciences Pennsylvania hands out annual industry awards

Fri, 16 Mar 2018 19:32:53 +0100

Life Sciences Pennsylvania, a Wayne-based trade group,  handed out its annual industry awards this week. Here is who was honored: Three organizations received patient impact awards. CSL Behring of King of Prussia was honored for Haegarda, a treatment that reduces painful swelling attacks in different body parts including the abdomen, face and the lar ynx in patients with hereditary angiogram. Spark Therapeutics of Philadelphia was honored for Luxturna, a one-time gene therapy treatment developed… (Source: bizjournals.com Health Care:Physician Practices headlines)



Scientists create nanostructures that could improve gene therapies

Thu, 15 Mar 2018 18:16:27 +0100

UCLA scientists have developed news microscopic structures called " nanospears " that could make gene therapies safer, faster and more cost-effective. (Source: Health News - UPI.com)

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The Future is Here: What ’s New and Exciting in Medical Materials

Thu, 15 Mar 2018 16:45:42 +0100

The medical device industry is at the peak of innovation, believes Asmita Khanolkar, manager, manufacturing engineering, CeQur. To meet the requirements of today’s complex devices, new materials are needed, she said. Key areas of research include advances in biomaterials, medical electronics, soft materials, and new raw materials for rapid prototyping and additive and subtractive manufacturing, she told MD+DI. Khanolkar will be exploring emerging materials in the upcoming BIOMEDevice Boston April 18 session, “What’s New & Exciting in Medical Materials.” She’ll look at 5- and 10-year trends in biomaterials, medical electronics, plastics, and soft materials, and explore cutting-edge applications being used by today’s medical device engineers. Khanolkar has more than 22 years...



Nanostructures created by UCLA scientists could make gene therapies safer, faster and more affordable

Thu, 15 Mar 2018 06:03:06 +0100

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules such as genes straight to patient cells. These magnetically guided nanostructures could enable gene therapies that are safer, faster and more cost-effective.The research waspublished in the journal ACS Nano by senior author Paul Weiss, UC Presidential Chair and distinguished professor of chemistry and biochemistry, materials science and engineering, and member of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.Gene therapy, the process of adding or replacing missing or defective genes in patient cells, has shown great promise as a treatment for a host of diseases, including hemophilia, muscula...



Shares of Solid Biosciences crater as FDA halts Duchenne gene therapy trial

Wed, 14 Mar 2018 22:28:01 +0100

Less than two months after pulling off one of the biggest initial public offerings for a local biotech in recent years, Cambridge-based Solid Biosciences said that the FDA had halted a trial of its gene therapy treatment for Duchenne muscular dystrophy, sending shares of the company into a tailspin. Solid (Nasdaq: SLDB) said Wednesday that the FDA had stopped an early-stage study of the drug after one patient was hospitalized due to reduced platelet and red blood cell count. The company added that… (Source: bizjournals.com Health Care:Physician Practices headlines)



Nanostructures created at UCLA could make gene therapies safer, faster & more affordable

Wed, 14 Mar 2018 04:00:00 +0100

(University of California - Los Angeles Health Sciences) UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called 'nanospears' for the targeted delivery of biomolecules such as genes straight to patient cells. (Source: EurekAlert! - Biology)

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Gene Therapy Flops for Critical Limb Ischemia

Mon, 12 Mar 2018 19:30:00 +0100

(MedPage Today) -- Non-viral biologic agent didn't improve wound healing (Source: MedPage Today Cardiovascular)



Chemically induced proximity in biology and medicine

Thu, 08 Mar 2018 00:00:00 +0100

Proximity, or the physical closeness of molecules, is a pervasive regulatory mechanism in biology. For example, most posttranslational modifications such as phosphorylation, methylation, and acetylation promote proximity of molecules to play deterministic roles in cellular processes. To understand the role of proximity in biologic mechanisms, chemical inducers of proximity (CIPs) were developed to synthetically model biologically regulated recruitment. Chemically induced proximity allows for precise temporal control of transcription, signaling cascades, chromatin regulation, protein folding, localization, and degradation, as well as a host of other biologic processes. A systematic analysis of CIPs in basic research, coupled with recent technological advances utilizing CRISPR, distinguishes...



Investigating CAR T-Cell Therapy in Patients With DLBCL, FL, and Other Lymphomas

Wed, 07 Mar 2018 23:45:00 +0100

In this interview, Dr. Frederick Locke discusses the promise of CAR T-cell therapy for lymphomas, and how this gene therapy could offer hope for patients who don't respond to standard treatments. (Source: CancerNetwork)



Bioengineering team's 'circuit' work may benefit gene therapy

Tue, 06 Mar 2018 05:00:00 +0100

(University of Texas at Dallas) Researchers at The University of Texas at Dallas have designed genetic 'circuits' out of living cellular material in order to gain a better understanding of how proteins function, with the goal of making improvements. (Source: EurekAlert! - Biology)



New gene therapy corrects a form of inherited macular degeneration in canine model

Mon, 05 Mar 2018 05:00:00 +0100

(University of Pennsylvania) Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable. (Source: EurekAlert! - Biology)

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‘Holy grail’ of gene therapy still a long way off for cystic fibrosis

Wed, 28 Feb 2018 04:00:27 +0100

Conventional treatments developed by Vertex help many sufferers, but they have a hefty price tag (Source: FT.com - Drugs and Healthcare)



Enabling technology for emerging gene therapies

Tue, 27 Feb 2018 05:00:00 +0100

(Washington University in St. Louis) For years, researchers have attempted to harness the full potential of gene therapy, a technique that inserts genes into a patient's cells to treat cancer and other diseases. However, inserting engineered DNA molecules into cells is difficult. A team of engineers at Washington University in St. Louis has developed a new method that could make the process easier. (Source: EurekAlert! - Cancer)



McKesson Specialty Health and Cryoport Collaborate to Bolster Logistics Services to Support Commercialization of Cell and Gene Therapies

Mon, 26 Feb 2018 08:00:00 +0100

The Woodlands, Texas& Irvine, Calif. (February 26, 2018) – McKesson Specialty Health, a division of McKesson Corporation, announced a strategic collaboration with Cryoport, Inc. (NASDAQ: CYRX, CYRXW) designed to further strengthen its robust logistics services to support the delivery of cell and ge... (Source: McKesson News)



New advances in medication for muscle disease in children

Mon, 26 Feb 2018 05:00:00 +0100

(University of Gothenburg) Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of neuromuscular disease, spinal muscular atrophy (SMA), which afflicts children from 6 to 18 months of age. That is shown by a study published in the New England Journal of Medicine (NEJM). (Source: EurekAlert! - Medicine and Health)



Gene therapy found safe in trial of Pompe disease treatment

Wed, 21 Feb 2018 20:04:01 +0100

The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers. (Source: Health News - UPI.com)

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AMD Treat-and-Extend Regimens OK: Ophthalmology Times

Tue, 20 Feb 2018 22:00:00 +0100

(MedPage Today) -- Also, gene therapy approved for retinal dystrophy (Source: MedPage Today Primary Care)



Cambridge's Voyager, AbbVie to pursue Alzheimer's gene therapy under $69M deal

Tue, 20 Feb 2018 15:52:28 +0100

Voyager Therapeutics said Tuesday that it will work with AbbVie to develop gene therapy treatments for Alzheimer ’s disease in a deal worth at least $69 million for the Cambridge biotech. The announcement by Voyager (Nasdaq: VYGR) and Chicago-based AbbVie (NYSE: ABBV) comes as drug developers are exploring new approaches to Alzheimer’s following a string of recent clinical setbacks. Gene therapies — dru gs that work by inserting healthy genes into a person’s cells to replace faulty or missing… (Source: bizjournals.com Health Care News Headlines)



Chinese research advances highlighted in special issue of Human Gene Therapy

Fri, 16 Feb 2018 05:00:00 +0100

(Mary Ann Liebert, Inc./Genetic Engineering News) China is helping to advance gene and cell therapy and genome editing research and clinical development by creating novel viral and nonviral vectors for gene delivery and innovative applications of CRISPR technology in a broad range of disease areas. (Source: EurekAlert! - Biology)



Oxford BioMedica wins second $100 million gene therapy contract

Thu, 15 Feb 2018 07:52:07 +0100

LONDON (Reuters) - Britain's Oxford BioMedica has won a second $100 million contract to supply gene therapy material, this time from Bioverativ, which agreed last month to be acquired by Sanofi for $11.6 billion. (Source: Reuters: Health)



New Studies Target Gene Therapy Against AIDS

Thu, 15 Feb 2018 07:00:00 +0100

Title: New Studies Target Gene Therapy Against AIDSCategory: Health NewsCreated: 2/14/2018 12:00:00 AMLast Editorial Review: 2/15/2018 12:00:00 AM (Source: MedicineNet HIV General)

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Can gene therapy be harnessed to fight AIDS virus?

Wed, 14 Feb 2018 21:12:54 +0100

Researchers are working to improve treatment for the virus and are trying to tackle HIV ​ by editing DNA (Source: Health News: CBSNews.com)



Rewired Olympic Brains; Gene Therapy Concerns; Memory Boost from Implant

Tue, 13 Feb 2018 15:30:00 +0100

(MedPage Today) -- News and commentary from the world of neurology and neuroscience (Source: MedPage Today Public Health)



A clinical trial to test a gene therapy treatment for a rare liver disease

Mon, 12 Feb 2018 05:00:00 +0100

(AFM-T é l é thon) G é n é thon, a laboratory created by AFM-T é l é thon, starts a European phase I/II clinical trial to test a treatment for Crigler-Najjar Syndrome, a rare liver disease. The trial, sponsored by G é n é thon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by G é n é thon. (Source: EurekAlert! - Biology)



Can Microbubbles Deliver Gene Therapy?

Fri, 09 Feb 2018 23:20:36 +0100

An Italian diagnostic imaging company is teaming with a Chicago startup to study whether microbubbles can deliver personalized gene therapy to treat high LDL cholesterol. Microbubble technology, combined with contrast-enhanced ultrasound, has been used diagnostically for years and more recently has inspired research for treatment of disease. Milan-based Bracco Imaging, S.p.A. is working with Steven Feinstein, a professor of medicine at Rush University Medical Center and president of SonoGene, LLC to pursue the treatment possibilities further. A CEUS researcher, Feinstein is also co-president of the  International Contrast Ultrasound Society. “The experience Bracco has developed during the past decade allows us to understand and control the production of microbubbles as well as the inter...



Gene therapy field hit by fresh safety concern

Thu, 08 Feb 2018 00:00:00 +0100

(Source: ScienceNOW)

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Chesco gene therapy company seeks FDA OK to begin human testing of skin disorder treatment

Wed, 07 Feb 2018 16:38:25 +0100

The treatment is for a rare condition called localized scleroderma. (Source: bizjournals.com Health Care News Headlines)



Why A Gene Therapy Pioneer Is Raising Concerns About Treatments He Championed

Wed, 07 Feb 2018 15:47:00 +0100

" I recognize the fact that these are limited numbers [of animals], and in fact that studies were not designed to evaluate safety, " Wilson says. " But the fact is, we observed the toxicity with that limited number, which is, from my point of view, even more worrisome. " (Source: Forbes.com Healthcare News)



Genprex Launch IPO For Trials Of New Gene Therapy Lung Cancer Treatment

Mon, 05 Feb 2018 15:30:00 +0100

The company is raising capital to pay the costs of further clinical trials, as the cost of these trials is very high. The earliest that Oncoprex could be available to patients is two years after raising the capital in this IPO. (Source: Forbes.com Healthcare News)



How Cancer Immunotherapy Is Getting Even Better

Fri, 02 Feb 2018 22:18:47 +0100

Last year, the Food and Drug Administration approved two new immunotherapies to treat certain leukemias and lymphomas. Now, in a study published in the New England Journal of Medicine, researchers detail who is most likely to benefit from the treatments, called CAR T cell therapy. CAR T cell therapy trains the body’s immune system to target and destroy cancer cells in the blood; scientists take people’s own immune cells (T cells) and genetically engineer them to seek out and destroy cancer cells. The immune system can then attack cancer cells in the same way it does bacteria and viruses, and the therapy can lead to remissions from blood cancers of up to 80%. But Dr. Jae Park, lead author of the research, says that many of those studies only follow people for a year or so, sinc...



Spark Therapeutics, Bluebird CEOs formulate a Super Bowl wager

Fri, 02 Feb 2018 14:55:43 +0100

Add one more bet to the lengthy list of "friendly" wagers politicians, business leaders and even brewery operators and art institutions in Philadelphia and Boston are making on the outcome of Super Bowl LII. Spark Therapeutics CEO Jeffrey Marrazzo bet Nick Leschly, CEO of Bluebird Bio in Cambridge, Mass., that the Philadelphia Eagles will beat the New England Patriots. Leschly accepted the wager. Here's what's at stake for leaders of the gene therapies companies. While the first part of the… (Source: bizjournals.com Health Care News Headlines)

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Trial by fire: Two Cambridge biotechs race to find a gene therapy cure for Duchenne

Thu, 01 Feb 2018 15:38:32 +0100

Gene therapy could transform the lives of boys with a deadly disease — and two local companies that are racing for a cure. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)



Chasing Genzyme, AvroBio bags $60M to fund Fabry disease gene therapy trial

Thu, 01 Feb 2018 12:00:00 +0100

Fifteen years after its approval, the Genzyme drug Fabrazyme remains the standard of care for Fabry disease, a rare disorder that can cause a wide variety of symptoms including pain in the hands and feet and vision loss. AvroBio, a Cambridge startup backed by Atlas Venture, is out to change that. The 30-employee company, which was founded in 2015, said Thursday that it had raised $60 million in new financing. The Series B round will help fund a mid-stage trial of AvroBio’s lead drug, an experimental… (Source: bizjournals.com Health Care:Physician Practices headlines)



NIDCR Science News

Wed, 31 Jan 2018 20:00:58 +0100

Having trouble viewing this email? View it as a Web page.   Pioneering Gene Therapy for Dry MouthOngoing clinical trial explores saliva-restoring gene transfer NIDCR • January 18, 2018​​   Stem Cell Study of Jaw Development Could Offer Insight into Craniofacial FlawsUniversity of Southern California • January 18, 2018 ​Researchers Get First Complete Look at Protein Behind Sense of TouchThe Scripps Research Institute • December 20, 2017 STAY CONNECTED     (Source: NIDCR Science News)



Landmark international study: CAR T-cell therapy safe and effective in youth with leukemia

Wed, 31 Jan 2018 05:00:00 +0100

(Children's Hospital Los Angeles) Results of the global, multicenter, pivotal phase 2 study that led to the first FDA approval of a gene therapy/cell therapy approach known as CAR T-cell therapy, were published today in the New England Journal of Medicine. Senior authors on the study include Stephen A. Grupp, of Children's Hospital of Philadelphia and Michael A. Pulsipher, M.D., of Children's Hospital Los Angeles (CHLA). (Source: EurekAlert! - Cancer)

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Bracco lands new FDA approval for MultiHance

Tue, 30 Jan 2018 18:56:49 +0100

Contrast developer Bracco Diagnostics has received U.S. Food and Drug Administration...Read more on AuntMinnie.comRelated Reading: Bracco targets gene therapy for ultrasound contrast Bracco declines comment on Norris gadolinium lawsuit Bracco Imaging acquires SurgVision Bracco invests in dose monitoring firm Bracco unveils colon insufflator (Source: AuntMinnie.com Headlines)



CNIO researchers cure lung fibrosis in mice with a gene therapy that lengthens telomeres

Tue, 30 Jan 2018 05:00:00 +0100

(Centro Nacional de Investigaciones Oncol ó gicas (CNIO)) Idiopathic pulmonary fibrosis is a potentially lethal disease associated with the presence of critically short telomeres, currently lacking effective treatment. The Telomere and Telomerase Group at the Spanish National Cancer Research Centre (CNIO) has succeeded in curing this disease in mice using a gene therapy that lengthens the telomeres. This work constitutes a " proof of concept that telomerase activation represents an effective treatment against pulmonary fibrosis, " the authors write in this publication. (Source: EurekAlert! - Medicine and Health)



New study shows severe toxicity with high-dose AAV9-based gene therapy in animal models

Tue, 30 Jan 2018 05:00:00 +0100

(Mary Ann Liebert, Inc./Genetic Engineering News) Researchers have described severe, life-threatening toxicity in monkeys and piglets given high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector capable of accessing spinal cord neurons. (Source: EurekAlert! - Medicine and Health)



This company is the result of Nationwide Children's reviving gene therapy in the U.S.

Mon, 29 Jan 2018 11:00:00 +0100

It ’s the stuff of science fiction – replacing defective genes in the human body with new ones that will stop a debilitating disease. But it seems to becoming reality at Michael Triplett’s new Columbus gene therapy company, which is having success controlling a rare muscular dystrophy disease tha t cripples its patients. (Source: bizjournals.com Health Care News Headlines)



James Wilson, MD, PhD, from the University of Pennsylvania, Joins the...

Fri, 26 Jan 2018 00:00:00 +0100

The Rett Syndrome Research Trust (RSRT) is pleased to announce that James Wilson, MD, PhD, director of the University of Pennsylvania Gene Therapy Program and the Rose H. Weiss Orphan Disease Center,...(PRWeb January 26, 2018)Read the full story at http://www.prweb.com/releases/2018/01/prweb15132993.htm (Source: PRWeb: Medical Pharmaceuticals)

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Gene therapy using CAR T cells could provide long-term protection against HIV

Thu, 25 Jan 2018 20:34:45 +0100

FINDINGSA UCLA-led research team has created blood-forming stem cells that can carry a gene that allows the body to produce cells that can detect and destroy HIV-infected cells. The blood-forming cells, called hematopoietic stem and progenitor cells, or HSPCs, have been engineered to carry chimeric antigen receptor, or CAR, genes that allows the production of immune cells that target cells infected with HIV.After being transplanted into the body, the engineered cells formed immune cells that not only destroyed the infected cells, but also lived for more than two years. This suggests that they have the potential to give people long-term immunity from HIV, the virus that causes AIDS.BACKGROUNDAntiviral drugs are a key element in treating HIV, because they can suppress the virus in the body t...



Spark licenses blindness gene therapy rights outside U.S. to Novartis

Wed, 24 Jan 2018 22:27:04 +0100

(Reuters) - Spark Therapeutics Inc, whose breakthrough gene therapy to treat a rare form of blindness was approved by U.S. regulators in December, has licensed rights to the drug in markets outside the United States to Switzerland-based Novartis AG, it said on Wednesday. (Source: Reuters: Health)



Novartis strikes $170m deal for ex-US rights to gene therapy for blindness

Wed, 24 Jan 2018 21:55:04 +0100

(Source: FT.com - Drugs and Healthcare)



The Cult Of DNA-centricity

Wed, 24 Jan 2018 15:10:00 +0100

All of biology lies in the genome, right? Thats a premise of modern medicine: a disease-free utopia based on gene therapy and, soon, gene editing. But what if the real answers lie your proteins? The cult of DNA-centric thinking is holding back the next wave of cures for diseases like Alzheimer's. (Source: Forbes.com Healthcare News)



Viral gene therapy could improve results from breast reconstruction after cancer treatment

Wed, 24 Jan 2018 05:00:00 +0100

(Institute of Cancer Research) A new treatment helps healthy tissue protect itself from radiotherapy damage.Viral therapy reduced damage to skin and underlying tissues in rats -- and improved their outcomes from treatment.A new type of gene therapy delivered using a virus could protect healthy tissues from the harmful side-effects of radiotherapy after cancer treatment, a new study reports.In the future, the treatment could be used to improve outcomes for breast reconstruction surgery in women with breast cancer. (Source: EurekAlert! - Cancer)

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Bracco targets gene therapy for ultrasound contrast

Tue, 23 Jan 2018 18:15:48 +0100

Contrast developer Bracco Imaging has launched an initiative aimed at expanding...Read more on AuntMinnie.comRelated Reading: Bracco declines comment on Norris gadolinium lawsuit Bracco Imaging acquires SurgVision Bracco invests in dose monitoring firm Bracco unveils colon insufflator ESR partners with Bracco on fellowships (Source: AuntMinnie.com Headlines)