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F.D.A. Speeds Review of Gene Therapies, Vowing to Target Rogue Clinics
The agency plans to speed approval of treatments to get them to the market faster, signaling the quickened pace of advancements in this field. (Source: NYT Health)
Source: NYT Health - November 17, 2017 Category: Consumer Health News Authors: SHEILA KAPLAN and DENISE GRADY Tags: Stem Cells Genetic Engineering Immunotherapy Food and Drug Administration Source Type: news

$1 million price tag in spotlight as gene therapy becomes reality
LONDON (Reuters) - Battle lines are being drawn as the first gene therapy for an inherited condition nears the U.S. market, offering hope for people with a rare form of blindness and creating a cost dilemma for healthcare providers. (Source: Reuters: Health)
Source: Reuters: Health - November 16, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Scientists Have Made Their First Attempt at Gene Editing Inside a Human Patient
(OAKLAND, Calif.) — Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person’s DNA to try to cure a disease. The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot. “It’s kind of humbling” to be the first to test this, said Madeux, who has a metabolic disease called Hunter syndrome. “I’m willing to take that risk. Hopefully it will help me and other people.” Signs of whether i...
Source: TIME.com: Top Science and Health Stories - November 15, 2017 Category: Consumer Health News Authors: Marilynn Marchione / AP Tags: Uncategorized gene editing Genetics health Innovation onetime overnight Research Source Type: news

Knowing Too Much about Your Genes Might Be Risky
Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Mental Health (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 13, 2017 Category: Consumer Health News Source Type: news

The Forbes Healthcare Summit 2017: The Draft Agenda
The time is drawing near: our sixth annual Forbes Healthcare Summit will be held on Nov. 29 and Nov. 30. The event will take attendees through the whole spectrum of the healthcare business, from the cutting edge of gene therapy and to the hard economic choices every health system must make. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 10, 2017 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:BLUE NYSE:PFE NYSE:MRK NASDAQ:ALNY Source Type: news

Genetic treatment for blindness may soon be reality
(American Academy of Ophthalmology) Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting of the American Academy of Ophthalmology. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 10, 2017 Category: International Medicine & Public Health Source Type: news

Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products
(Source: What's New at CBER)
Source: What's New at CBER - November 9, 2017 Category: Biomedical Science Source Type: news

Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products; Guidance for Industry
(Source: What's New at CBER)
Source: What's New at CBER - November 9, 2017 Category: Biomedical Science Source Type: news

Boy with rare disease gets new skin with gene therapy
"We were forced to do something dramatic because this kid was dying," his doctor said (Source: Health News: CBSNews.com)
Source: Health News: CBSNews.com - November 9, 2017 Category: Consumer Health News Source Type: news

Stem Cell Gene Therapy Replaces Boy's Entire Epidermis Stem Cell Gene Therapy Replaces Boy's Entire Epidermis
Ex vivo gene therapy of epidermal cells from a boy with junctional epidermolysis bullosa enabled grafts to completely replace his epidermis.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - November 9, 2017 Category: Consumer Health News Tags: Dermatology News Source Type: news

Boy with rare disease gets most of skin replaced through gene therapy
Desperate to save a seven-year-old boy's life, doctors used experimental gene therapy to create new skin in a lab after skin graft attempts had failed. (Source: CBC | Health)
Source: CBC | Health - November 8, 2017 Category: Consumer Health News Tags: News/Health Source Type: news

Gene Therapy Creates Replacement Skin to Save a Dying Boy
Doctors grew sheets of healthy skin that were transplanted onto a boy with a genetic disease that caused blistering and tearing all over his body. (Source: NYT Health)
Source: NYT Health - November 8, 2017 Category: Consumer Health News Authors: DENISE GRADY Tags: Skin Epidermolysis Bullosa Genetic Engineering Nature (Journal) Source Type: news

Gene Therapy Creates New Skin to Save a Dying Child
Doctors grew sheets of healthy skin that were transplanted onto a boy with a genetic disease that caused blistering and tearing all over his body. (Source: NYT Health)
Source: NYT Health - November 8, 2017 Category: Consumer Health News Authors: DENISE GRADY Tags: Skin Epidermolysis Bullosa Genetic Engineering Nature (Journal) Source Type: news

Medical News Today: How gene therapy saved a little boy's life
Epidermolysis bullosa gives rise to painful blisters and chronic wounds. Doctors were sure that one little boy was going to lose his life — but he didn't. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 8, 2017 Category: Consumer Health News Tags: Dermatology Source Type: news

Genes May Explain Why Some Don't Respond to Bipolar Drug
Source: HealthDay Related MedlinePlus Pages: Bipolar Disorder, Genes and Gene Therapy, Medicines (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 8, 2017 Category: Consumer Health News Source Type: news

Spark Therapeutics and Pfizer amend license agreement for investigational SPK-9001 in hemophilia B
Spark Therapeutics (NASDAQ:ONCE) and Pfizer Inc. (NYSE:PFE) have entered into an amendment to their license agreement for SPK-9001, an investigational gene therapy for hemophilia B. Spark Therapeutics will enroll up to five additional participants in the current Phase 1/2 clinical trial who will receive SPK-9001 manufactured using an enhanced process to test its comparability to the SPK-9001 received by the first 10 participants enrolled in the ongoing trial. (Source: World Pharma News)
Source: World Pharma News - November 8, 2017 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news

Boy is given new skin thanks to gene therapy
(Ruhr-University Bochum) A medical team at the Ruhr-Universit ä t Bochum's burn unit and the Center for Regenerative Medicine at the University of Modena (Italy) were the first ever to successfully treat a child suffering from extensive skin damage using transplants derived from genetically modified stem cells. The boy is a so-called butterfly child: he suffers from epidermolysis bullosa, a genetic skin disease that had destroyed approximately 80 percent of his epidermis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 8, 2017 Category: International Medicine & Public Health Source Type: news

Spark, Pfizer amend agreement for experimental hemophilia gene therapy
Spark Therapeutics and Pfizer Inc. have amended their license agreement for an experimental gene therapy for hemophilia B — which could result in additional payments of up to $25 million for Spark. Under the amended agreement, Spark Therapeutics, a Philadelphia gene therapy company spun out of Children’s Hospital of Philadelphia, will enroll up to five additional participants in the current phase-I/II clinical tri al testing the treatment known as SPK-9001. The new participants will receive… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - November 7, 2017 Category: American Health Authors: John George Source Type: news

CRISPR technology adapted to edit RNA
Researchers designed a highly specific RNA editing system based on the CRISPR/Cas gene editing technology. The study suggests a potential alternative to gene therapy. (Source: NIH Research Matters from the National Institutes of Health (NIH))
Source: NIH Research Matters from the National Institutes of Health (NIH) - November 7, 2017 Category: Consumer Health News Source Type: news

Medical News Today: Gene therapy may protect brain from age-related cognitive decline
One dose of a gene therapy that raises levels of the protein Klotho in the brain protected younger mice against age-related cognitive decline. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 2, 2017 Category: Consumer Health News Tags: Seniors / Aging Source Type: news

Gene Therapy, New Drug Battle a Rare but Deadly Disease in Kids
Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Spinal Muscular Atrophy (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - November 2, 2017 Category: Consumer Health News Source Type: news

Gene Therapy, New Drug Battle a Rare But Deadly Disease in Kids
Title: Gene Therapy, New Drug Battle a Rare But Deadly Disease in KidsCategory: Health NewsCreated: 11/1/2017 12:00:00 AMLast Editorial Review: 11/2/2017 12:00:00 AM (Source: MedicineNet Kids Health General)
Source: MedicineNet Kids Health General - November 2, 2017 Category: Pediatrics Source Type: news

Roswell Park Cancer Institute Launches New Gene-Therapy Study Using...
Cancer center begins clinical study open to patients with several cancer types, involving a unique two-pronged immunotherapy strategy(PRWeb November 02, 2017)Read the full story at http://www.prweb.com/releases/2017/11/prweb14874396.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - November 2, 2017 Category: Pharmaceuticals Source Type: news

Gene Therapy, New Drug Fight Rare Disease in Kids
Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven highly effective in clinical trials, researchers report. (Source: WebMD Health)
Source: WebMD Health - November 1, 2017 Category: Consumer Health News Source Type: news

Baby gene therapy study offers hope for fatal muscle disease
Researchers report a first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe extended the tots' lives (Source: ABC News: Health)
Source: ABC News: Health - November 1, 2017 Category: Consumer Health News Tags: Health Source Type: news

Gene Therapy, Antisense Show Gains in Spinal Muscular Atrophy Gene Therapy, Antisense Show Gains in Spinal Muscular Atrophy
An antisense oligonucleotide drug and gene therapy each extended survival and led to attainment of motor milestones in some children with spinal muscular atrophy.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - November 1, 2017 Category: Consumer Health News Tags: Pediatrics News Source Type: news

Gene Therapy, New Drug, Spinraza, Battle Rare Spinal Muscular Atrophy in Kids
WEDNESDAY, Nov. 1, 2017 -- Babies born with a previously untreatable degenerative nerve disease now have two fresh sources of hope for their future. Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven highly effective... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - November 1, 2017 Category: General Medicine Source Type: news

NICE draft guidance approves gene therapy for rare syndrome
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - November 1, 2017 Category: Drugs & Pharmacology Source Type: news

Gene Therapy May Fight Brain Cancer's Return
Title: Gene Therapy May Fight Brain Cancer's ReturnCategory: Health NewsCreated: 10/27/2017 12:00:00 AMLast Editorial Review: 10/30/2017 12:00:00 AM (Source: MedicineNet Cancer General)
Source: MedicineNet Cancer General - October 30, 2017 Category: Cancer & Oncology Source Type: news

Gene Therapy May Fight Brain Cancer's Return
A new form of gene therapy shows promise in battling recurrent brain cancer. (Source: WebMD Health)
Source: WebMD Health - October 27, 2017 Category: Consumer Health News Source Type: news

Gene Therapy May Fight Brain Cancer ’ s Return
(Source: The Doctors Lounge - Oncology)
Source: The Doctors Lounge - Oncology - October 27, 2017 Category: Cancer & Oncology Tags: Oncology, Research, News, Source Type: news

Gene Therapy May Fight Brain Cancer's Return
FRIDAY, Oct. 27, 2017 -- A new form of gene therapy shows promise in battling recurrent brain cancer. The phase 1 clinical trial included 56 patients with recurrent high-grade glioma brain cancer. Three years after the gene therapy treatment, more... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - October 27, 2017 Category: General Medicine Source Type: news

Gene Therapy May Fight Brain Cancer's Return
(Source: Cancercompass News: Other Cancer)
Source: Cancercompass News: Other Cancer - October 27, 2017 Category: Cancer & Oncology Source Type: news

FDA Grants Breakthrough Therapy Designation for BioMarin's Valoctocogene Roxaparvovec (formerly BMN 270), an Investigational Gene Therapy for Hemophilia A
SAN RAFAEL, Calif., Oct. 26, 2017 -- (Healthcare Sales & Marketing Network) -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S. Food and Drug Administration (FDA) granted valoctocogene roxaparvovec (formerly BMN 270) Breakthrough ... Biopharmaceuticals, FDA BioMarin Pharmaceutical, Valoctocogene, Roxaparvovec, Hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - October 26, 2017 Category: Pharmaceuticals Source Type: news

When It Comes to Obesity, Genes Just Partly to Blame
Source: HealthDay Related MedlinePlus Pages: Genes and Gene Therapy, Obesity, Weight Control (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - October 26, 2017 Category: Consumer Health News Source Type: news

NICE approves gene therapy for rare ‘bubble baby syndrome’
Strimvelis, a treatment for an ultra-rare inherited immune deficiency condition that has been dubbed ‘bubble baby syndrome’, has been approved by NICE in draft guidance. (Source: NHS Networks)
Source: NHS Networks - October 24, 2017 Category: UK Health Source Type: news

The Guardian view on gene therapy: money well spent | Editorial
A rare and fatal disease will be now treated on the NHS. But the real problems come from common and unglamorous complaintsThe NHS is tofund a very expensive treatment for a very rare but terrible childhood disorder that leaves babies condemned to life in a sterile bubble. This is a triumph for medical science but it should also provoke some deep and careful thought. The treatment in question, strimvelis, qualifies as the second most expensive drug ever put on the market (the only one more expensive waswithdrawn due to lack of demand). A single dose costs nearly £500,000 plus VAT, and can only be administere...
Source: Guardian Unlimited Science - October 23, 2017 Category: Science Authors: Editorial Tags: Medical research Stem cells Genetics Biology Science NHS Health Society Children UK news Source Type: news

Gene therapy for 'bubble baby' syndrome approved on NHS
GlaxoSmithKline ’s Strimvelis is the first such treatment to be funded in the UKThe NHS will fund gene therapy for the first time after the UK ’s healthcare cost watchdog approved treatment for the so-called “bubble baby” syndrome, despite a price tag of more than £500,000.The treatment is used against adenosine deaminase deficiency, or ADA-SCID, which disables the immune system and means that children with the illness have to be kept in isolation to avoid infection – hence the “bubble baby” name.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - October 23, 2017 Category: Science Authors: Reuters Tags: NHS The National Institute for Health and Care Excellence (Nice) Genetics Biology Science GlaxoSmithKline Immunology Medical research UK news Pharmaceuticals industry Society Source Type: news

GSK wins U.S. shingles vaccine approval, UK nod for gene therapy
LONDON (Reuters) - GlaxoSmithKline has won U.S. approval for a new and improved shingles vaccine, the second of three key products for which the British drugmaker expects approval this year. (Source: Reuters: Health)
Source: Reuters: Health - October 23, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Britain backs GSK's gene therapy for 'bubble boy' syndrome
(Reuters) - GlaxoSmithKline's gene therapy for the so-called "bubble boy" disease was approved by Britain's healthcare cost watchdog NICE, despite a price tag of almost 600,000 euros ($700,000). (Source: Reuters: Health)
Source: Reuters: Health - October 22, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

FDA Approves Landmark Cancer Therapy; Wilmot Positioned Among First to Offer It
UR Medicine ’s Wilmot Cancer Institute will be among the first sites in the world to offer CAR T-cell therapy —a new type of immunotherapy—to adults with aggressive lymphoma. The engineered gene therapy has been described as a revolutionary “living drug” and one of the most powerful cancer treatments to emerge in recent years. (Source: University of Rochester Medical Center Press Releases)
Source: University of Rochester Medical Center Press Releases - October 20, 2017 Category: Universities & Medical Training Authors: University of Rochester Medical Center Source Type: news

New gene therapy "gave me my life back," cancer survivor says
The FDA has approved CAR-T cell therapy for lymphoma, a form of blood cancer, after some patients showed dramatic benefits. CBS Boston's Dr. Mallika Marshall has the story. (Source: Health News: CBSNews.com)
Source: Health News: CBSNews.com - October 20, 2017 Category: Consumer Health News Source Type: news

FDA approves CAR-T cell therapy to treat adults with certain types of large B-cell lymphoma
The U.S. Food and Drug Administration approved Yescarta (axicabtagene ciloleucel), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL). (Source: World Pharma News)
Source: World Pharma News - October 20, 2017 Category: Pharmaceuticals Tags: Featured FDA Regulatory Affairs Source Type: news

Gene Therapy Approved for B-Cell Lymphoma
Title: Gene Therapy Approved for B-Cell LymphomaCategory: Health NewsCreated: 10/19/2017 12:00:00 AMLast Editorial Review: 10/20/2017 12:00:00 AM (Source: MedicineNet Cancer General)
Source: MedicineNet Cancer General - October 20, 2017 Category: Cancer & Oncology Source Type: news

FDA Approves 2nd Gene Therapy
Yescarta fights a type of lymphoma; move is heralded as helping open 'new era' in medical care (Source: Cancercompass News: Other Cancer)
Source: Cancercompass News: Other Cancer - October 20, 2017 Category: Cancer & Oncology Source Type: news

FDA Approves Gene Therapy for Large B-Cell Lymphoma (FREE)
By Amy Orciari Herman Edited by Andr é Sofair, MD, MPH, and William E. Chavey, MD, MS The FDA has for the second time approved a gene therapy — this time, for diffuse large B-cell lymphoma, … (Source: Physician's First Watch current issue)
Source: Physician's First Watch current issue - October 19, 2017 Category: Primary Care Source Type: news

US FDA approve second gene therapy for blood cancer
The Food and Drug Administration on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphomas. (Source: the Mail online | Health)
Source: the Mail online | Health - October 19, 2017 Category: Consumer Health News Source Type: news

Gene Therapy Approved for B - Cell Lymphoma
The first such treatment for non - Hodgkin form of this cancer (Source: The Doctors Lounge - Oncology)
Source: The Doctors Lounge - Oncology - October 19, 2017 Category: Cancer & Oncology Tags: Oncology, Pharmacy, FDA Approvals, Source Type: news

FDA Approves Gilead Cancer Gene Therapy
The list price will be $373,000. (Source: Healthy Living - The Huffington Post)
Source: Healthy Living - The Huffington Post - October 19, 2017 Category: Consumer Health News Source Type: news

FDA approves 2nd gene therapy targeting non-Hodgkin lymphomas
The US Food and Drug Administration has approved a second gene therapy for cancer, the first to target non-Hodgkin lymphomas. (Source: CNN.com - Health)
Source: CNN.com - Health - October 19, 2017 Category: Consumer Health News Source Type: news