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Juno And Other Gene Therapy Players Are Hot Despite Ongoing Pricing Debates
The rumored acquisition of CAR-T developer Juno by Celgene has ignited investor enthusiasm for gene therapy. But in the background, concerns about how the healthcare system will afford these pricey new treatments persist. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - January 18, 2018 Category: Pharmaceuticals Authors: Arlene Weintraub, Contributor Tags: NASDAQ:JUNO Source Type: news

Stem Cell Gene Therapy Might Guard Against HIV Stem Cell Gene Therapy Might Guard Against HIV
Research in monkeys suggests that hematopoietic stem/progenitor Cclls (HSPCs) in concert with chimeric antigen receptor (CAR) T-cells could provide long-lasting protection against HIV.Reuters Health Information (Source: Medscape Hiv-Aids Headlines)
Source: Medscape Hiv-Aids Headlines - January 16, 2018 Category: Infectious Diseases Tags: HIV/AIDS News Source Type: news

Nonprofit agency says Spark's gene therapy $425K price tag is too high
Spark said the analysis is faulty because it excludes indirect costs associated with vision loss. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 16, 2018 Category: Biotechnology Authors: John George Source Type: news

Study advances gene therapy for glaucoma
(University of Wisconsin-Madison) In a study published today in the scientific journal Investigative Ophthalmology and Visual Science, Kaufman and Curtis Brandt, a fellow professor of ophthalmology and visual sciences at UW-Madison, showed an improved tactic for delivering new genes into the eye's fluid drain, called the trabecular meshwork. It could lead to a treatment for glaucoma. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 16, 2018 Category: International Medicine & Public Health Source Type: news

Spark's price for Luxturna blindness gene therapy too high: ICER
(Reuters) - The proposed price for Spark Therapeutics Inc's groundbreaking one-time gene therapy for a rare form of blindness is far too high, an independent U.S. nonprofit organization that evaluates clinical and cost effectiveness of new medicines said on Friday. (Source: Reuters: Health)
Source: Reuters: Health - January 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Non-Profit Says $850,000 Gene Therapy Is At Least Twice As Expensive As It Should Be
The non-profit Institute for Clinical Evaluation and Review (ICER) says that undisputed breakthrough should cost $400,000 at most, and perhaps $200,000. At that price, would it even exist? (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - January 12, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:ONCE Source Type: news

Spark's planned price for Luxturna gene therapy for rare blindness too high: ICER
(Reuters) - The proposed price for Spark Therapeutics Inc's groundbreaking one-time gene therapy for a rare form of blindness is far too high, an independent U.S. nonprofit organization that evaluates clinical and cost effectiveness of new medicines said on Friday. (Source: Reuters: Health)
Source: Reuters: Health - January 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

The coming of age of gene therapy: A review of the past and path forward
No longer the future of medicine, gene therapy is part of present-day clinical treatment. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - January 12, 2018 Category: American Health Source Type: news

Drugmakers see a pricing blueprint in an $850,000 gene therapy
SAN FRANCISCO (Reuters) - Global drugmakers are looking to a tiny biotech's $850,000 therapy for a rare type of blindness as a model for getting paid for highly expensive – and effective – new medicines. (Source: Reuters: Health)
Source: Reuters: Health - January 12, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

The coming of age of gene therapy: A review of the past and path forward
(NIH/National Heart, Lung and Blood Institute) After three decades of hopes tempered by setbacks, gene therapy -- the process of treating a disease by modifying a person's DNA -- is no longer the future of medicine, but is part of the present-day clinical treatment toolkit. The Jan. 12 issue of the journal Science provides an in-depth and timely review of the key developments that have led to several successful gene therapy treatments for patients with serious medical conditions. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 11, 2018 Category: International Medicine & Public Health Source Type: news

Gene therapy: The power of persistence
(Source: ScienceNOW)
Source: ScienceNOW - January 11, 2018 Category: Science Authors: Kiberstis, P. A. Tags: Medicine, Diseases twis Source Type: news

Gene therapy comes of age
After almost 30 years of promise tempered by setbacks, gene therapies are rapidly becoming a critical component of the therapeutic armamentarium for a variety of inherited and acquired human diseases. Gene therapies for inherited immune disorders, hemophilia, eye and neurodegenerative disorders, and lymphoid cancers recently progressed to approved drug status in the United States and Europe, or are anticipated to receive approval in the near future. In this Review, we discuss milestones in the development of gene therapies, focusing on direct in vivo administration of viral vectors and adoptive transfer of genetically engi...
Source: ScienceNOW - January 11, 2018 Category: Science Authors: Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., Sadelain, M. Tags: Medicine, Diseases review Source Type: news

GenSight to launch trial of gene therapy, visual stimulation device combo in UK
The UK’s Medicines and Healthcare Regulatory Agency has approved GenSight Biologics‘ (EPA:SIGHT) application to combine gene therapy and a wearable optronic visual stimulation device in patients with retinitis pigmentosa. The first-in-man Phase I/II trial is designed to study the safety and tolerability of GenSight’s GS030 combination therapy in three groups of three patients each. Get the full story at our sister site, Drug Delivery Business News. The post GenSight to launch trial of gene therapy, visual stimulation device combo in UK appeared first on MassDevice. (Source: Mass Device)
Source: Mass Device - January 10, 2018 Category: Medical Devices Authors: Sarah Faulkner Tags: Clinical Trials Optical/Ophthalmic Pharmaceuticals Regulatory/Compliance Wall Street Beat gensightbiologics Source Type: news

Biotech startup Immusoft raises $3 million and hires new CEO
The first chunk of Immusoft's ongoing Series B round will help new CEO Sean Ainsworth move its gene therapy into human clinical trials. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - January 9, 2018 Category: American Health Authors: Casey Coombs Source Type: news

Biotech's deal-making showcase is under way: early themes and a podcast
Gene therapy and data are early themes as biotech executives and financiers gather around the J.P. Morgan Healthcare Conference. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 8, 2018 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Pricey gene therapies expected to ‘dominate’ talk at J.P. Morgan conference
If you ’re a biotech industry executive or investor, chances are you’re in San Francisco this week for the annual J.P. Morgan healthcare conference. And you can expect to hear plenty of lively discussion about gene therapies, a new class of drugs that carries both promise and perils for the biotech ind ustry. Gene therapies are intended to modify a person’s DNA — specifically, by inserting healthy genes into cells to replace faulty or missing ones. This approach, though far from foolproof,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 8, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

Medical News Today: Diabetes: Can gene therapy normalize blood glucose levels?
Researchers from the University of Pittsburgh have shown that alpha cells can be reprogrammed into beta cells to restore blood glucose levels in diabetes. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - January 8, 2018 Category: Consumer Health News Tags: Diabetes Source Type: news

Pricey gene therapies expected to ‘dominate’ talk at J.P. Morgan conference
If you ’re a biotech industry executive or investor, chances are you’re in San Francisco this week for the annual J.P. Morgan healthcare conference. And you can expect to hear plenty of lively discussion about gene therapies, a new class of drugs that carries both promise and perils for the biotech ind ustry. Gene therapies are intended to modify a person’s DNA — specifically, by inserting healthy genes into cells to replace faulty or missing ones. This approach, though far from foolproof,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 7, 2018 Category: Biotechnology Authors: Max Stendahl Source Type: news

Pricey gene therapies expected to ‘dominate’ talk at J.P. Morgan conference
If you ’re a biotech industry executive or investor, chances are you’re in San Francisco this week for the annual J.P. Morgan healthcare conference. And you can expect to hear plenty of lively discussion about gene therapies, a new class of drugs that carries both promise and perils for the biotech ind ustry. Gene therapies are intended to modify a person’s DNA — specifically, by inserting healthy genes into cells to replace faulty or missing ones. This approach, though far from foolproof,… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - January 7, 2018 Category: American Health Authors: Max Stendahl Source Type: news

Gene therapy offers long-term treatment for mice with diabetes
The newly resurgent field of gene therapy, which recently produced treatments forblood cancers andblindness, has taken a step toward fighting a scourge that is on the rise worldwide: diabetes.Inresearch reported Thursday in the journal Cell Stem Cell, scientists showed that a single infusion... (Source: Los Angeles Times - Science)
Source: Los Angeles Times - Science - January 5, 2018 Category: Science Authors: Melissa Healy Source Type: news

Blood sugar levels in diabetic mice restored with gene therapy
A new gene therapy approach can revert sugar levels back to normal in mice with type 1 diabetes, according to new findings published inCell Stem Cell.EurekAlert! (Source: Society for Endocrinology)
Source: Society for Endocrinology - January 5, 2018 Category: Endocrinology Source Type: news

NICE final draft guidance recommends 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 final draft guidance. (Source: NHS Networks)
Source: NHS Networks - January 5, 2018 Category: UK Health Source Type: news

Sangamo Partners With Pfizer to Develop Gene Therapy for ALS Sangamo Partners With Pfizer to Develop Gene Therapy for ALS
Sangamo Therapeutics Inc and Pfizer Inc said on Wednesday they would work together to develop a gene therapy to treat ALS, a disease that affects nerve cells in the brain and the spinal cord.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - January 4, 2018 Category: Consumer Health News Tags: Medscape Today News Source Type: news

West Coast ad blitz will promote Philadelphia region as 'Cellicon Valley'
Attendees at next week's J.P. Morgan Healthcare Investor Conference in San Francisco will encounter numerous electronic billboard touting Philadelphia's prowess in cell and gene therapy. (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - January 4, 2018 Category: Health Management Authors: John George Source Type: news

Atlas-backed biotech startup emerges with $25M and a plan to perfect gene therapy
Gene therapies — drugs that work by inserting healthy genes into a person’s cells to augment faulty ones — have generated significant buzz in recent years because of their potential to permanently cure a wide range of diseases. But the approach has some limitations. The viruses that are typically used to del iver the genes can trigger harmful immune responses in some patients. Even if that doesn't happen, the drugs can’t be re-dosed if and when the effects start to wear off. Generation Bio,… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 4, 2018 Category: Pharmaceuticals Authors: Max Stendahl Source Type: news

When the Lung Cancer Patient Climbs Mountains
A gene therapy clinical trial enabled a Stage IV lung cancer patient to summit a peak in the Himalayas. (Source: NYT Health)
Source: NYT Health - January 4, 2018 Category: Consumer Health News Authors: TODD BALF Tags: Lung Cancer Lungs Clinical Trials Genetics and Heredity Mountaineering Glaciers Nepal Massachusetts General Hospital Source Type: news

Could Gene Therapy Someday Eliminate HIV?
Title: Could Gene Therapy Someday Eliminate HIV?Category: Health NewsCreated: 1/3/2018 12:00:00 AMLast Editorial Review: 1/4/2018 12:00:00 AM (Source: MedicineNet HIV General)
Source: MedicineNet HIV General - January 4, 2018 Category: Infectious Diseases Source Type: news

Gene therapy restores normal blood glucose levels in mice with type 1 diabetes
(Cell Press) A study in Cell Stem Cell demonstrates that a gene therapy approach can lead to the long-term survival of functional beta cells as well as normal blood glucose levels for an extended period of time in mice with type 1 diabetes. The researchers used an adeno-associated viral (AAV) vector to deliver to the mouse pancreas two proteins, Pdx1 and MafA, which reprogrammed plentiful alpha cells into functional, insulin-producing beta cells. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - January 4, 2018 Category: Biology Source Type: news

Gene therapy for rare retinal disorder to cost $425,000 per eye
A one-time treatment with Luxturna, the first US Food and Drug Administration-approved gene therapy for an inherited disorder, will cost $425,000 per eye. (Source: CNN.com - Health)
Source: CNN.com - Health - January 3, 2018 Category: Consumer Health News Source Type: news

Could Gene Therapy Someday Eliminate HIV?
The science centers around the use of "chimeric antigen receptor" (CAR) genes. In laboratory work with monkeys, these engineered cells have destroyed HIV-infected cells for more than two years, scientists reported. (Source: WebMD Health)
Source: WebMD Health - January 3, 2018 Category: Consumer Health News Source Type: news

Gene therapy for blindness will cost $850,000  
Drug-maker Spark Therapeutics lowered the cost of its treatment for a rare form of blindness from $1 million, but at $850,000, the drug is still one of the most expensive gene therapies in the world. (Source: the Mail online | Health)
Source: the Mail online | Health - January 3, 2018 Category: Consumer Health News Source Type: news

Spark to charge $850,000 per patient for blindness gene therapy
(Reuters) - Spark Therapeutics Inc said on Wednesday it will charge $850,000 per patient for its breakthrough gene therapy to treat a rare form of blindness, a lower than expected price that the company said reflects patient and insurer concerns about access and cost. (Source: Reuters: Health)
Source: Reuters: Health - January 3, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Gene therapy for type of blindness gets $850K price tag
Drugmaker says it lowered cost of treatment approved by the FDA last month, citing insurer concerns (Source: Health News: CBSNews.com)
Source: Health News: CBSNews.com - January 3, 2018 Category: Consumer Health News Source Type: news

Spark reveals the price tag for its groundbreaking gene therapy treatment
The Philadelphia company will offer outcomes-based rebates and the possibility for installment payments to help ensure access to the one-time therapy that treats a condition that otherwise leads to blindness. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - January 3, 2018 Category: American Health Authors: John George Source Type: news

Spark Therapeutics' vision-loss gene therapy to cost $850,000 per patient
(Reuters) - Spark Therapeutics Inc said on Wednesday it would charge $850,000 per patient for its gene therapy to treat a rare form of blindness, lesser than analysts' expectation of around $1 million. (Source: Reuters: Health)
Source: Reuters: Health - January 3, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Drug group Spark to charge $850,000 for blindness gene therapy
Luxturna is most expensive treatment on market and price raises affordability questions (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - January 3, 2018 Category: Pharmaceuticals Source Type: news

Sangamo partners with Pfizer to develop gene therapy for ALS
(Reuters) - Sangamo Therapeutics Inc and Pfizer Inc said on Wednesday they would work together to develop a gene therapy to treat ALS, a disease that affects nerve cells in the brain and the spinal cord. (Source: Reuters: Health)
Source: Reuters: Health - January 3, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Spark Therapeutics Sets Price Of Blindness-Curing Gene Therapy At $850,000
A new drug, Luxturna, literally allows blind people to see. It does it by using a virus to insert new genes into patients ’ eyes. This morning, Luxturna’s maker, Spark Therapeutics, is announcing the cost of this medical miracle: $425,000 per eye, or $850,000 a year. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - January 3, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:ONCE NASDAQ:IONS NASDAQ:BIIB NASDAQ:ALXN Source Type: news

Spark Therapeutics Sets Price Of Blindness-Treating Gene Therapy At $850,000
A new drug, Luxturna, literally allows blind people to see. It does it by using a virus to insert new genes into patients ’ eyes. This morning, Luxturna’s maker, Spark Therapeutics, is announcing the cost of this medical miracle: $425,000 per eye, or $850,000 a year. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - January 3, 2018 Category: Pharmaceuticals Authors: Matthew Herper, Forbes Staff Tags: NASDAQ:ONCE NASDAQ:IONS NASDAQ:BIIB NASDAQ:ALXN Source Type: news

Could Gene Therapy Someday Eliminate HIV?
WEDNESDAY, Jan. 3, 2018 -- Gene therapy may have the potential to eradicate HIV in people infected with the virus, new animal research suggests. The science centers around the use of " chimeric antigen receptor " (CAR) genes. In laboratory work with... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - January 3, 2018 Category: General Medicine Source Type: news

Could viruses take cancer immunotherapy to the next level?
(Alliance for Cancer Gene Therapy) A study published in Science Translational Medicine shows a combination of oncolytic viruses and checkpoint inhibitors successfully treated breast cancer with a 60-90 percent cure rate. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - January 3, 2018 Category: Cancer & Oncology Source Type: news

Monthly News Roundup - December 2017
Luxturna Gene Therapy OK ’d for Rare Form of Vision Loss A historic approval, Luxturna (voretigene neparvovec-rzyl) from Spark Therapeutics is the first directly administered gene therapy that targets a disease caused by specific gene... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - December 31, 2017 Category: Pharmaceuticals Source Type: news

Gene therapy using CAR T-cells could provide long-term protection against HIV
(University of California - Los Angeles Health Sciences) Through gene therapy, researchers engineered blood-forming stem cells (hematopoietic stem/progenitor cells, or HSPCs) to carry chimeric antigen receptor (CAR) genes to make cells that can detect and destroy HIV-infected cells. These engineered cells not only destroyed the infected cells, they persisted for more than two years, suggesting the potential to create long-term immunity from the virus that causes AIDS. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - December 28, 2017 Category: Infectious Diseases Source Type: news

‘I want to help humans genetically modify themselves’
Former Nasa biochemist Josiah Zayner became an online sensation by conducting DIY gene therapy on himself. He explains why he did itJosiah Zayner, 36,recently made headlines by becoming the first person to use the revolutionary gene-editing toolCrispr to try to change their own genes. Part way through a talk on genetic engineering, Zayner pulled out a syringe apparently containing DNA and other chemicals designed to trigger a genetic change in his cells associated with dramatically increased muscle mass. He injected the DIY gene therapy into his left arm,live-streaming the procedure on the internet.The former Nasa biochemi...
Source: Guardian Unlimited Science - December 24, 2017 Category: Science Authors: Tom Ireland Tags: Genetics Science Biology Hacking Source Type: news

Senate Democrats and more than 300 public health groups call on Trump officials to reverse HHS language restrictions
In two separate letters Thursday, a group of Senate Democrats and more than 300 public health organizations called on top Trump administration officials to lift any restrictions on how Health and Human Services employees communicate in official documents. The missives come in the wake of reports by The Washington Post that budget writers at the […]Related:FDA approves first gene therapy for an inherited diseaseDebate erupts within HHS about ‘words to avoid’ such as ‘vulnerable,’ ‘diversity’ and ‘entitlement’FDA takes more aggressive stance toward homeopathic drugs (Sou...
Source: Washington Post: To Your Health - December 21, 2017 Category: Consumer Health News Source Type: news

Senate Democrats and more than 300 public-health groups call on Trump officials to reverse HHS language restrictions
In two letters Thursday, a group of Senate Democrats and more than 300 public-health organizations called on top Trump administration officials to lift any restrictions on how Health and Human Services employees communicate in official documents. The missives come in the wake of reports by The Washington Post that budget writers at the Centers for […]Related:FDA approves first gene therapy for an inherited diseaseDebate erupts within HHS about ‘words to avoid’ such as ‘vulnerable,’ ‘diversity’ and ‘entitlement’FDA takes more aggressive stance toward homeopathic drugs (...
Source: Washington Post: To Your Health - December 21, 2017 Category: Consumer Health News Source Type: news

Gene Therapy Developer Orchard Therapeutics Raises $110 Million Gene Therapy Developer Orchard Therapeutics Raises $110 Million
Orchard Therapeutics said on Wednesday it raised $110 million in the second round of funding as the drug developer looks to launch its gene therapy to treat a rare inherited disorder and beef up manufacturing facilities.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 21, 2017 Category: Consumer Health News Tags: Allergy & Clinical Immunology News Source Type: news

FDA approves gene therapy for a type of blindness
The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease. (Source: CNN.com - Health)
Source: CNN.com - Health - December 20, 2017 Category: Consumer Health News Source Type: news

2017 ’ s Year In Health News: Medical Breakthroughs, Opioid Crisis And More
CBS Local — There’s been plenty of progress in the medical world this year, and as a result we now know that more Americans than ever have high blood pressure, but also that coffee everyday is actually good for you. Here’s a look back at the year in health. Opioid Crisis The opioid crisis has dominated much of the health news cycle. President Trump declared the opioid crisis a national emergency earlier this year. Drug overdoses are now the leading cause of death for people under 50 in the United States. New Guidelines for High Blood Pressure The American Heart Association revised its guidelines for high ...
Source: WBZ-TV - Breaking News, Weather and Sports for Boston, Worcester and New Hampshire - December 20, 2017 Category: Consumer Health News Authors: Health – CBS Boston Tags: Health News best of 2017 Samantha Lazarus Bennet Source Type: news

Harvard scientists cure deaf mice with gene therapy
The new treatment, pioneered by Harvard University researchers, used a technique called Crispr CAS-9, which modifies DNA in living cells. Deafness is genetic in over 50 percent of cases. (Source: the Mail online | Health)
Source: the Mail online | Health - December 20, 2017 Category: Consumer Health News Source Type: news