Category Archives: Gene Therapy Clinics

FDA approves first cell-based gene therapy for use in the United States – Gears Of Biz

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, said FDA Commissioner Scott Gottlieb, M.D. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, were committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patients own T-cells, a type of white blood cell known as a lymphocyte. The patients T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, with B-cell the most common. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research (CBER). Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.

The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent.

Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events. Both CRS and neurological events can be life-threatening. Other severe side effects of Kymriah include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Most symptoms appear within one to 22 days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

The FDA today also expanded the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events. Additionally, the certified health care settings are required to have protocols in place to ensure that Kymriah is only given to patients after verifying that tocilizumab is available for immediate administration. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Fast Track, Priority Review, and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDAs Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

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FDA approves first cell-based gene therapy for use in the United States - Gears Of Biz

FDA Approves New Cancer Treatment – Alive For Football

Analyst John Newman of Canaccord takes yesterday's FDA approval of Novartis' chimeric antigen receptor T-cell (CAR-T) therapy Kymriah as a meaningful victory for Kite Pharma Inc's (NASDAQ:KITE) fellow CAR-T therapy candidate Axi-cel.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL. While this was a groundbreaking approval, the therapy will cost $475,000 for 1 treatment, according to Kaiser Health News.

The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the USA market - and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. Once re-injected into the patient's body, the T cells modified to replicate quickly, and the remission is rapid.

The therapy is being licensed and brought to market by the pharmaceutical company Novartis.

"Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month".

The price of the therapy is tagged at around $500,000. An immune overreaction called "cytokine release syndrome" can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Because Kymriah can have life-threatening side effects, including unsafe drops in blood pressure, the FDA is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

For now, it is only approved for use on ALL.

Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society, is excited by the drug's approval, but he has some reservations. This is elegant science. "There is still much more work to do", Lichtenfeld told Healthline. The safety and effectiveness of Kymriah was tested in one small clinical trial of 63 children and young adults with relapsed or refractory B-cell precursor ALL. Even then, it is prescribed only as a last resort when other forms of treatment have failed.

Still, "a far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia", said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites.

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FDA Approves New Cancer Treatment - Alive For Football

Global Cancer Biological Therapy Analysis & Forecast 2016 to 2023 – Digital Journal

Cancer Biological Therapy Analysis & Forecast: Global Industry Analysis, Capacity, Production, Price, Revenue, Size, Share, Growth, Trends, and Forecasts 2017-2022

This press release was orginally distributed by SBWire

Lawndale, CA -- (SBWIRE) -- 09/04/2017 -- Introduction Biological therapy treatment is done with the help of living organisms, parts of living organisms or laboratory manufactured version of such content. There are various types of biological therapies, which inhibit specific molecules involved in development and growth of cancer tumor. Such therapies known as; cancer targeted therapies.

The global cancer biological therapy market is expected to reach USD 82,276.8 million by 2023 at a CAGR of 4.7% during the forecasted period.

The global cancer biological therapy market is segmented on the basis of phases, types, end users and regions. On the basis of phases, the market is segmented into phase I, phase II and phase III. In stage I & II the real impact of these therapies is seen and giving a success rate of 35% in Phase 1 and 20% in Phase II. The success rate of phase I is 35%.

On the basis on types, the global cancer biological therapy market is segmented into monoclonal antibodies, cancer growth blockers, interferons, interleukins, gene therapy, targeted drug delivery, colony stimulating factor, cancer vaccines and others. Monoclonal antibodies accounted for the largest market share of the global cancer biological therapy market. Colony stimulating factor is the fastest growing market at a CAGR of 5.2% during the forecasted period.

On the basis on end users, hospitals & clinics dominates the global cancer biological therapy market. Registering USD 26,790.6 million in 2016 and expected to reach at USD 38,471.9 million by 2023 at the rate of 4.4 % from 2016-2023.

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On the basis of regions, the market is segmented into North America, Europe, Asia-Pacific and the Middle East & Africa. North America has the dominating market for cancer biological therapy. The cancer biological therapy market for North America is estimated at USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10%.

Key Players The leading market players in the global cancer biological therapy market include Merck Inc., F. Hoffmann-La Roche Ltd, Novartis AG, Amgen Inc., Bristol-Myers Squibb, Celgene, ELI Lilly and Company, EnGeneIC, and Pfizer Study objectives - To provide detailed analysis of the market structure along with forecast of the various segments and sub-segments of the global cancer biological therapy market - To provide insights about factors influencing and affecting the market growth - To provide historical and forecast revenue of the market segments and sub-segments with respect to countries - To provide historical and forecast revenue of the market segments based on channels, applications, and regions for the global cancer biological therapy market. - To provide strategic profiling of key players in the market, and comprehensively analysing their market share, core competencies, and drawing a competitive landscape for the market - To provide economical factors that influences the global cancer biological therapy market - To provide detailed analysis of the value chain and supply chain of the global cancer biological therapy market Target Audience - Pharmaceutical Companies - Pharmaceutical Suppliers - Cancer Research Organizations - Potential Investors - Key Executive (CEO and COO) and Strategy Growth Manager - Reaserch Companies Key Findings - North America accounted for the largest market share in the global cancer biological therapy market, USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10% - Colony stimulating factor is the fastest growing segment with a CAGR of and 5.2% in the global cancer biological therapy market, by types - Hospitals and clinics is contributing remarkable share in the market registering 47.8% in the global cancer biological therapy market, by end users in 2016

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Global Cancer Biological Therapy Analysis & Forecast 2016 to 2023 - Digital Journal

New ‘hit-and-run’ gene editing tool temporarily rewrites genetics to treat cancer and HIV – GeekWire

Nanoparticles (orange) deliver temporary gene therapy to immune cells (blue) to give them disease-fighting tools. (Fred Hutch Illustration / Kimberly Carney)

CAR T immunotherapies are all the rage in the medical community, reprogramming a patients immune system to fight cancer. For some patients, theyve produced near-miraculous recoveries, and they could be a huge breakthrough in cancer treatment.

The business community is taking note as well: Kite Pharma, a biotech company developing these therapies, announced a deal to be acquired for $11.9 billion on Monday, sending stock prices of Seattle immunotherapy developer Juno Therapeuticsskyrocketing.

But there are still giant pitfalls to using the therapies on a large scale because they are incredibly complex and expensive to produce. Researchers from Seattles Fred Hutchinson Cancer Research Center are taking the problem head-on with new hit-and-run gene editing technology.

In a study published Wednesday in the journal Nature Communications, researchers led by Dr.Matthias Stephan reported they have developed a nanoparticle delivery system that can temporarily alter cells so they are able to fight cancer and other diseases.

The best part? The treatment is a powder that just needs to be mixed with water to activate and even better, it could be an essential breakthrough in making cutting-edge medical technology affordable for patients.

Stephan told GeekWire in a previous piece on the technology that his goal is to make immunotherapy so easy to access that it replaces chemotherapy as the front-line treatment for cancer.

What I envision is like the Walgreens flu shot scenario, or you go to your doctor and you get hepatitis B shot, he said at the time. You go there every Friday, and thats it.

We realized in order to outcompete chemotherapy, we have to design something that is at least as affordable and can be manufactured at large scale by one biotech company and shipped out to local infusion centers, Stephan said. At the moment, CAR T cell therapies must be made individually for each patient in specialized labs.

Heres how the new tech works: The nanoparticles designed by Stephan and his team act like shipping containers for bundles of mRNA, the molecules that tell cells how to build disease-fighting proteins. The nanoparticles also have molecules attached to the outside to help them find the right kind of cells, like a shipping label on a package.

When the mRNA is delivered to the cell, it prompts the cell to grow disease-fighting features, like the chimeric antigen receptor in CAR T cells that help them identify and kill cancer.Researchers said the technology could potentially be used to develop treatments for HIV, diabetes and other immune-related diseases.

In the short run, the tech could help researchers discover new treatments and therapies in the lab. It could one day be used in hospitals and clinics around the world, but will first need to undergo extensive clinical trials to ensure the tech is effective and safe to use in humans.

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New 'hit-and-run' gene editing tool temporarily rewrites genetics to treat cancer and HIV - GeekWire

How one California county is fighting high-priced surgeries – Los Angeles Times

Retiree Leslie Robinson-Stone and her husband enjoyed a weeklong, all-expenses-paid trip to a luxury resort all thanks to the county she worked for.

The couple also received more than a thousand dollars in spending money and a personal concierge, who attended to their every need. For Santa Barbara County, it was money well spent: Sending Robinson-Stone 250 miles away for knee replacement surgery near San Diego saved the government $30,000.

The only difference between our two hospitals is one is expensive and the other is exorbitant, said Andreas Pyper, assistant director of human resources for Santa Barbara County, referring to the local options.

After years of hospital industry consolidation, frustration with sky-high hospital bills and a lack of local competition is common to many employers and consumers across the country. Fed up with wildly different price tags for routine operations, some private employers are steering patients they insure to top-performing providers who offer bargain prices. Santa Barbara County, with about 4,000 employees, is among a handful of public entities to join them.

The county has saved nearly 50% on four surgery cases since starting its out-of-town program last year, officials said. The program is voluntary for covered employees.

At a Scripps Health hospital in the San Diego area, the county paid $61,600 for a spinal fusion surgery that would have cost more than twice as much locally. It avoided two other operations altogether after patients went outside the area for second opinions.

Typically, employers are seeking deals through bundled payments in which one fixed price covers tests, physician fees and hospital charges. And if complications arise, providers are on the hook financially. Medicare began experimenting with this method during the Obama administration.

Santa Barbara County is among about 400 employers on the West Coast working with Carrum Health, a South San Francisco, Calif., start-up that negotiates bundled prices and chooses surgeons based on data on complications and readmissions.

Not all surgeons are equal. We dont want to give Scripps a blank check. That defeats the whole purpose, said Sachin Jain, Carrums chief executive.

Santa Barbara officials try to persuade workers and their family members to participate in its program by waiving co-pays and deductibles. The county pays about $2,700 in travel costs and still comes out way ahead.

If that doesnt speak to the inefficiencies in our healthcare system, I dont know what does, Pyper said. Its almost like buying a Toyota Corolla for $50,000 and then going to San Diego to buy the same Corolla for $16,000. How long would the more expensive Toyota dealership last?

Even as more employers and insurers embrace bundled payments, the Trump administration is applying the brakes.

In August, Medicare officials proposed canceling mandatory bundled payments for certain surgeries and scaling back the program for knee and hip replacements. Health and Human Services Secretary Tom Price, when he was still a member of Congress, accused Medicare of overstepping federal authority and interfering in the doctor-patient relationship. Hospital trade groups have voiced similar objections.

That leaves some health-policy experts dismayed.

These bundled payments put pressure on medical providers and the savings are astonishing, said Bob Kocher, a former health official in the Obama administration and now a partner in the venture capital firm Venrock.

Santa Barbara County officials said they had no choice after seeing their medical costs soar by 15% in each of the last two years. Like many local governments, it has an older workforce prone to chronic illness, blocked arteries and bum knees.

But health costs run higher than the state average in this scenic coastal county of about 450,000 people, according to data from Oakland-based Integrated Healthcare Assn. By one measure, the average health insurance premium in the individual market runs $660 a month in Santa Barbara, 27% higher than in Los Angeles.

Heidi de Marco / Kaiser Health News

Maya Barraza is Santa Barbara Countys manager for employee benefits.

Maya Barraza is Santa Barbara Countys manager for employee benefits. (Heidi de Marco / Kaiser Health News)

Still, Maya Barraza, the countys manager for employee benefits and rewards, knew the program would be a hard sell to workers. You dont want to be away from your family and whats familiar, she said.

Cottage Health, the countys largest health system, says it wants to keep patients in town for treatment and follow-up care.

Established in 1891, its grown from a single hospital to more than 500 beds across three hospitals, and annual revenue hit $746 million last year.

Valet attendants greet visitors at two entrances outside the groups white, Spanish-style hospital in the city of Santa Barbara. In the main lobby, the names of wealthy donors are splashed across one wall, including billionaire investor and Donald Trump confidant Thomas Barrack.

We are continually looking at reducing costs and improving quality, said Cottage Health spokeswoman Maria Zate. Cottage Health has some of the top surgeons in California.

Sixty miles north in Santa Maria, the states largest hospital chain, Dignity Health, offers another option: Marian Regional Medical Center.

Both Cottage and Dignity hospitals in Santa Barbara County have quality scores of fair to excellent for joint replacements, spinal procedures and coronary bypass surgeries, according to three years of Medicare data analyzed by research firm Mpirica Health.

Dignity Health didnt respond to requests for comment.

Carrum tries to help employers like Santa Barbara County find more affordable options. It has struck bundled price deals for various procedures with Scripps hospitals in the San Diego area, Stanford Health Care in the Bay Area and Swedish Medical Center in Seattle, part of the Providence Health chain.

Some companies have gone so far as to send patients overseas for cheaper care, but most employers favor a more regional approach, experts say. Workers still rely on local physicians for follow-up care.

For some hospitals, there are advantages in offering deep discounts: They get patients they otherwise would never see and are paid in full right after the patient is discharged, avoiding the onerous billing and collections process.

They also have the financial capacity to offer such sharply reduced prices.

Michael Bark, assistant vice president of payer relations at Scripps Health, said most hospitals significantly mark up their commercial rates for orthopedic procedures and cardiac surgeries to compensate for lower government reimbursements.

There are immense profit margins built into those cases, Bark said.

Robinson-Stone, a former county sheriffs deputy and a computer support specialist, was initially wary of traveling for her surgery. But the 62-year-old had ongoing pain that kept her from biking, walking her dogs and tending to her fruit trees. Medication and cortisone shots didnt work, and she had no ties to local surgeons. So she signed up online and was given a choice of six orthopedic surgeons at Scripps Green Hospital in La Jolla.

In June 2016, she and her husband, Frank Stone, checked in at the Estancia La Jolla Hotel and Spa.

Robinson-Stone met the surgeon on a Wednesday, had the operation the next day and returned to her hotel room by Saturday. She continued physical therapy at the hotel and returned to the hospital a few days later to have the staples removed.

She was back on her bike within two months and eventually lost about 20 pounds.

I just celebrated one year from surgery, she said, and Im a happy camper.

Chad Terhune is a senior correspondent for Kaiser Health News, an editorially independent publication of the Kaiser Family Foundation. Heidi DeMarco contributed to this story.

ALSO

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Knocking on doors, climbing through fences: How L.A. County's health investigators are out trying to stop syphilis

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How one California county is fighting high-priced surgeries - Los Angeles Times

Australian Market Declines – Markets Insider

(RTTNews) - The Australian stock market is declining on Monday after the banking regulator's announcement of the launch of an independent inquiry into Commonwealth Bank dragged down banking stocks. In addition, weakness in base metal prices weighed on mining stocks.

In late-morning trades, the benchmark S&P/ASX 200 Index is declining 39.80 points or 0.69 percent to 5,704.10, off a low of 5,700.50. The broader All Ordinaries Index is down 36.70 points or 0.63 percent to 5,766.70.

The big four banks - ANZ Banking, Westpac, Commonwealth Bank and National Australia Bank - are lower in a range of 1.1 percent to 1.7 percent.

The Australian Prudential Regulation Authority or APRA said it will launch an inquiry to look at governance, culture and accountability practices at Commonwealth Bank after recent allegations that it violated laws.

In the mining space, Rio Tinto is declining more than 1 percent and Fortescue Metals is losing almost 2 percent, while BHP Billiton is up 0.2 percent.

Gold miners are advancing after gold prices gained on Friday. Newcrest Mining is edging up less than 0.1 percent and Evolution Mining is adding almost 1 percent.

Oil stocks are also higher after crude oil prices rose on Friday. Santos is rising more than 2 percent, Oil Search is adding 0.3 percent and Woodside Petroleum is up 0.2 percent.

Australian Pharmaceutical Industries, the operator of Priceline pharmacies, has withdrawn from the potential acquisition of Laser Clinics Australia saying the sale price was too high. Shares of API are adding 0.2 percent.

Amaysim Australia reported a 6.5 percent decline in net profit for the full year, while its underlying earnings rose 22.9 percent. The mobile service provider's shares are gaining almost 6 percent.

CSL has agreed to acquire U.S. biotechnology company Calimmune, which is developing a stem cell gene therapy to treat rate conditions such as sickle cell disease, for an up-front payment of $91 million. Shares of CSL are adding 0.3 percent.

Ten Network has been acquired by U.S. media giant CBS Corp. Shares of Ten Network are in a trading halt since June 13 after it was placed into voluntary administration.

In the currency market, the Australian dollar rose against the U.S. dollar, which weakened after the Jackson Hole speeches by global central bankers. In early trades, the local unit was trading at US$0.7925, up from US$0.7904 on Friday.

On Wall Street, stocks closed mixed on Friday after seeing early strength that partly reflected optimism about tax reform following comments from President Donald Trump's chief economic adviser Gary Cohn.

The Nasdaq edged down 5.68 points or 0.1 percent to 6,265.64, while the Dow inched up 30.27 points or 0.1 percent to 21,813.67 and the S&P 500 ticked up 4.08 points or 0.2 percent to 2,443.05.

The major European markets moved modestly lower on Friday. While the French CAC 40 Index slipped by 0.2 percent, the U.K.'s FTSE 100 and the German DAX Index both edged down by 0.1 percent.

Crude oil prices gained on Friday as the dollar fell and the U.S. petroleum industry braced for Hurricane Harvey. WTI crude rose $0.44 or 0.9 percent to close at $47.87 a barrel on the New York Mercantile Exchange.

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Australian Market Declines - Markets Insider

New Stanford drug saves child with deadly genetic disease – The Mercury News

At 7 months old, Zoe Harting got a shot at Lucile Packard Childrens Hospital Stanford that changed the course of her life.

A few months earlier, during a family Christmas vacation, Zoes parents, John and Eliza Harting of El Granada, realized something was wrong with their newborn.

Zoe was not developing at the same rate as her cousin even though the two were born just a week apart.

Her cousin was very mobile: wriggling around, pushing stuff, John Harting said. Zoe wasnt doing any of that. She was very quiet.

The Hartings got the difficult news in early 2013 that Zoe had a deadly genetic disease: spinal muscular atrophy type 1, or SMA-1. Nationwide, about 250 babies are annually diagnosed with the rare disease, or about one in 10,000.

They learned that their first child was expected to die before she turns 2.

Without effective treatment, Zoes muscles would progressively weaken, taking away her ability to walk, eat and, ultimately, breathe.

The Hartingswere told there was nothing they could do. Distraught and frustrated, they joined an SMA support group, now called Cure SMA, and found a new pediatrician.

It was a good thing we did, John Harting said, because our pediatrician happened to attend a conference where she met John Day.

Dr. Day, director of the Neuromuscular Division and Clinics at Stanford University, was about to conduct a clinical trial using nusinersen as the first drug for SMA-1.

Zoe was the first baby in the world to receive the drug.

Day emphasized to the Hartings that he didnt know if the treatment would work but they knew this was their only option.

In December 2016, the Food and Drug Administration approved Spinraza, developed by Biogen, as the first-ever sanctioned therapy for pediatric and adult patients with SMA.

Patients with SMA dont produce enough of a protein called survival motor neuron, or SMN, which helps send signals from the spinal cord to muscles. When the muscles dont get the signals, they atrophy.

Patients with SMA are missing the main gene, SMN1, that produces the protein. Patients have a second gene, SMN2, that also can produce the protein, but it only makes 5 to 10 percent of the amount needed.

The new drug works by acting like a patch to cover up the flawed portion of the SMN2 gene, which then spurs production of the protein.

What we need to do is get a person up to about 50 percent of the normal amount of protein, Day said. Its a 15-or-20 nucleic long signal that ends up being precisely paired with RNA. Thats what gives us this power. Make something incredibly focused on that flaw and it will fix that flaw but not have any other side effect.

Day said its important that families now know there is something doctors can do if they see the infants early enough.

Day said theres minimal awareness of the genetic disease largely because many patients die so young and pediatricians may not have updated information that treatment is available.

Today, a pediatrician gets a genetic test back and they might very well tell the family, Go home and love your child as long as you have them, Day said.

By the time a family does research and come across Days comprehensive care clinic, the child might be six or nine months old with irreversible muscular atrophy.

If we see them early enough, before they see any symptoms, the child may not see any muscular impact, Day said. Its potentially that effective of a treatment if we see the patient early enough.

Day is an advocate for newborn genetic screening so SMA is identified at birth and treatment can begin before the child shows signs of the disease.

Babies are not yet being treated in utero, but such treatment is under development, Day said.

The Hartings shared their story this month as part of SMA Awareness Month, because they want families to know the importance of early detection and that there is treatment. About one in 50 parents are carriers of the recessive gene disorder.

Every four months, Zoe, who is now 4 years old, goes to Stanford for a 12 mg dose of the drug through a lumbar puncture, similar to an epidural. She gets physical therapy in between shots.

She has a weak musculature, and a simple cold can immobilize her. She cant swallow or walk by herself. But after three years of treatment, she can now sit up, interact, draw and play. SMA does not affect cognitive development and there are small signs she will continue to gain muscle strength.

Day is quick to point out that the drug isnt a magic wand that makes the disease go away. But he said Zoe, who had a fairly aggressive course of SMA at three months, has strength she didnt have before treatment and theres hope for continued improvement.

She can talk, she can move her legs and arms, she even yells at me now, Day said with a chuckle. She has personality. She can throw a beach ball around. Shes going to have a life.

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New Stanford drug saves child with deadly genetic disease - The Mercury News

Orphan Diseases Market Key Players analysis … – Digital Journal – Digital Journal

"Global Orphan Diseases Market- Global Forecast To 2022"

Global Orphan diseases Market information, by Type of Diseases (autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders and Others), by Type of Treatment (gene therapy, cell therapy, drug therapy and others), by End user (hospital and clinics, research laboratory and others) - Forecast to 2022

Market Synopsis of Global Orphan diseases Market:

Market Scenario:

Global orphan diseases market also known as rare disease is growing rapidly. It affects a very small percentage of the global population. Most of the orphan diseases are genetic and is remains throughout the life of the patient. There are no exact number of diseases available but approximately there are about 7000 different rare diseases and disorders throughout the globe. Global orphan diseases market is expected to grow at the average CAGR of 24.9% constantly throughout this period 2015-2022. It is also expected that this market which was US$ 121.6 billion in 2015 will grow to US$ 576.9 billion by 2022. . However due to lack of awareness, correct diagnosis, correct treatments and availability of healthcare facilities are inhibiting the growth of the global orphan diseases market.

Key Players for Global Orphan diseases Market:

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Segments:

Global orphan diseases market has been segmented

On the basis of types of diseases which includes autoimmune disorders, genetic disorders, blood disorders, cancer, growth disorder, cardiovascular diseases, neurological disorders, respiratory disorders, digestive disorders, eye disorders and others.

On the basis of treatment type it segmented into gene therapy, cell therapy, drug therapy and others.

On the basis of end user the market is segmented into hospital and clinics, research laboratory and others.

Intended Audience

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Table of Content

1 Report Prologue 2 Market Introduction 2.1 Definition 2.2 Scope Of The Study 2.2.1 Research Objective 2.2.2 Assumptions 2.2.3 Limitations 2.3 Market Structure 3 Research Methodology 3.1 Research Process 3.2 Primary Research 3.3 Secondary Research 3.4 Market Size Estimation 3.5 Forecast Model 4 Market Dynamics 4.1 Drivers 4.2 Restraints 4.3 Opportunities 4.4 Mega Trends 4.5 Macroeconomic Indicators 4.6 Technology Trends & Assessment 5 Market Factor Analysis

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The report gives the clear picture of current market scenario which includes historical and projected market size in terms of value, technological advancement, macro economical and governing factors in the market. The report provides details information and strategies of the top key players in the industry. The report also gives a broad study of the different market segments and regions.

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Orphan Diseases Market Key Players analysis ... - Digital Journal - Digital Journal

Gene Editing in Human Embryos Leaps ForwardHere’s the Science – Singularity Hub

Imagine walking down the street with a ticking time bomb in your chest, never knowing when your heart may explode.

Or going through five decades of life, having kids, and always wondering when your mind will finally slip away from you. Or worse yet, knowing that one day the same will inevitably happen to your children and your grandchildren.

If there were a curea way to irreversibly correct the faulty biology in yourself and your offspringwould you do it? And knowing that there might be risks, would you be comfortable making that decision for generations to follow?

Last week, a remarkable study published in Nature brought these and other questions back into public discourse. For the first time, an international team led by US scientists used CRISPR, a genetic editing tool, to correct a mutation that leads to heart failure in viable human embryos.

This isnt the first time scientists have tinkered with human embryos. But it is the first that shows that certain off-target effectspreviously thought immensely challengingcan be dealt with in a relatively straightforward way.

In other words, the new technology just brought us one step closer to correcting genetic deficits in humans. And what can be used to right a disease can also be used to enhance a healthy babyartificially altering their intelligence or physical appearance.

To be clear, this study is a long way off from the complicated changes required to make designer babies. This isnt Brave New World or Gattaca.

This is for [the] sake of saving children from horrible diseases, says lead author Dr. Shoukhrat Mitalipov at the Oregon Health and Science University, who previously worked on Dolly the sheep and three-parent babies.

And with this milestone, says Dr. George Church at Harvard University, were one step closer.

The human body runs on the tens of thousands of genes that form the code of life. Sometimes, just a single faulty gene can have devastating consequences, such as Huntingtons disease or hypertrophic cardiomyopathya condition that often leads to heart failure.

For decades, scientists have tried hacking lifes code to cure these genetic diseases at the DNA level. The process seems straightforward: like programmers decoding a bug, scientists would read through the bodys encyclopedia of genes, identify the faulty member, cut and paste the correct code into the original spotand voil, fixed!

The promise of genetic cures seemed easily within reach when a technology called CRISPR came onto the scene in 2012. CRISPR itself isnt a cure. Rather, its a pair of molecular scissors that scientists can direct to almost any point on the human genome and make a precise cut.

The cut triggers a cell to activate a DNA repair program. Almost all cells do this, but embryos go about it slightly differently. If provided with a normal copy of the gene, embryos will use the blueprint gene to reconstruct the broken piece, essentially overwriting the mistaken code. In theory, this leads to fewer mistakes than a normal cells stitch it up repair program, which doesnt use templates.

In practice, however, embryonic DNAs been hard to hack. Just two years ago, Chinese scientists reported giving up on correcting a genetic abnormality in human embryos due to off-target effects, saying that the CRISPR-based technology was too immature.

And for good reason. The safety and ethical barriers are enormous when editing embryosso-called germline editing. The reason is this: after sperm meets egg, the resulting single cell will develop into a persons entire body. This means that any changes to an embryo will (in theory) be present in every single cell in the grown human, including reproductive cells.

In other words, any changes to the embryo will not only affect the person it will become, but also his or her children, and their children and so on. If any unwanted mutation sneaks in during the procedure, the harm is multi-generational.

Then theres the problem of mosaicism. Oftentimes, an edited embryo can lead to a mosaic of genotypes in the resulting cellssome fixed, some not, and the individual still ends up with the disease.

The new study tackles both problems head-on.

Mitalipovs team decided to focus on hypertrophic cardiomyopathy, an inherited disease due to a gene called MYBPC3. People with the condition have two copies of the gene: one normal, one faulty. This means they have a 50-50 chance of passing the condition to their children.

Because the embryo already contains a normal copy of MYBPC3, explain the authors, it already has a blueprint the cell could use to repair the abnormal one. The team recruited a dozen healthy egg donors and one sperm donor that carried the faulty MYBPC3.

Normally, scientists encode all CRISPR components into an external bit of DNA called a plasmid, put that into a cell and rely on the cell to make the necessary proteins and molecules. Mitalipovs team took a more unusual route: using a tiny syringe, they directly injected the CRISPR machinery into either a fertilized embryo or into the egg cell right before fertilization.

In the first case, the CRISPR machinery sticks around for a long time. This increases the chance that it might go rogue and snip parts of the DNA it wasnt designed to cut.

By directly injecting the components, the CRISPR scissors are chewed up by the recipient cell after they do their work: less random snipping, more precision.

The tactic worked. When the team analyzed the resulting embryos at the four- or eight-cell stage, they found 72 percent contained only normal copies of the MYBPC3 gene, compared to roughly 50 percent found in non-edited controls.

Even though the yield of wild type/wild type embryos is still higher, its not 100 percent. We have room to improve, says Mitalipov.

Heres the kicker: using a variety of modern genetic sequencing techniques, the team scrutinized the embryos genomes for off-target effects. They couldnt find any. For all intents and purposes, the edited embryos looked completely healthy.

This doesnt necessarily mean the team avoided all unexpected mutations. It just means any genetic deletions or inserts didnt affect the embryos normal development.

Thats not all. The team also surprisingly found a way to minimize mosaicism. The key is to inject CRISPR components into the egg at the same time as they pumped in the sperm to fertilize it. This is much earlier in the developmental stage than anyone had previously attempted.

It worked. Out of the 58 treated eggs fertilized with the mutant sperm, 42 contained two normal copies of MYBPC3. Only one became a mosaic. In contrast, CRISPRing a fertilized embryo led to 13 out of 54 mosaics.

It makes previous work look pretty amateurish in terms of mosaicism and in terms of off-target effects, says Church.

Surprisingly, rather than bolstering a designer baby future, the study may have inadvertently doused a cold case of biological reality on the sci-fi idea.

Dr. Robin Lovell-Badge, a developmental biologist at the Francis Crick Institute in London, pointed out that the most unexpected result of the study is how the embryo chose to repair the gene.

In one experiment, the team tried introducing an artificial template of MYBPC3 in addition to the normal copy already present in the cell (from the healthy moms). But the cells completely ignored the researchers template, instead exclusively opting to use the maternal MYBPC3 to repair the mutation.

This suggests that you couldnt add anything that wasnt already there, says Lovell-Badge.

To Mitalipov, the crux of the conversation should be solidly based in therapy. My goal has always been to treat genetic diseases that have no cures, to save children, he says.

And there are still a lot of kinks that need ironing out before CRISPR could enter clinics. For one, scientists still hope to increase precision and accuracy. For another, IVF clinics already have solid screening protocols in place to weed out genetic abnormalities before implantation. While CRISPR can, in theory, boost the number of healthy embryos, it would have to work better to justify the cost.

To Dr. Richard Hynes, a cancer researcher at MIT who co-led a national committee that recently published a new guideline for editing embryos, the study is a big breakthrough.

What our report said was, once the technical hurdles are cleared, then there will be societal issues that have to be considered and discussions that are going to have to happen. Nows the time, he says.

Image Credit: University of Michigan via Flickr

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Gene Editing in Human Embryos Leaps ForwardHere's the Science - Singularity Hub

Global Cancer Biological Therapy Market 2017 Size, Development Status, Type and Application, Segmentation … – Digital Journal

""Cancer Biological Therapy Market""

WiseGuyReports.com adds Cancer Biological Therapy Market 2017 Global Analysis, Growth, Trends and Opportunities Research Report Forecasting to 2023reports to its database.

Cancer Biological Therapy Market:

Executive Summary

Biological therapy treatment is done with the help of living organisms, parts of living organisms or laboratory manufactured version of such content. There are various types of biological therapies, which inhibit specific molecules involved in development and growth of cancer tumor. Such therapies known as; cancer targeted therapies.

The global cancer biological therapy market is expected to reach USD 82,276.8 million by 2023 at a CAGR of 4.7% during the forecasted period.

The global cancer biological therapy market is segmented on the basis of phases, types, end users and regions. On the basis of phases, the market is segmented into phase I, phase II and phase III. In stage I & II the real impact of these therapies is seen and giving a success rate of 35% in Phase 1 and 20% in Phase II. The success rate of phase I is 35%.

On the basis on types, the global cancer biological therapy market is segmented into monoclonal antibodies, cancer growth blockers, interferons, interleukins, gene therapy, targeted drug delivery, colony stimulating factor, cancer vaccines and others. Monoclonal antibodies accounted for the largest market share of the global cancer biological therapy market. Colony stimulating factor is the fastest growing market at a CAGR of 5.2% during the forecasted period.

On the basis on end users, hospitals & clinics dominates the global cancer biological therapy market. Registering USD 26,790.6 million in 2016 and expected to reach at USD 38,471.9 million by 2023 at the rate of 4.4 % from 2016-2023.

On the basis of regions, the market is segmented into North America, Europe, Asia-Pacific and the Middle East & Africa. North America has the dominating market for cancer biological therapy. The cancer biological therapy market for North America is estimated at USD 19,481.2 million in 2016 and expected to reach by USD 29,516.9 million by 2023 at a fastest CAGR of 5.10%.

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Key Players

The leading market players in the global cancer biological therapy market include Merck Inc., F. Hoffmann-La Roche Ltd, Novartis AG, Amgen Inc., Bristol-Myers Squibb, Celgene, ELI Lilly and Company, EnGeneIC, and Pfizer

Study objectives

Target Audience

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Key Findings

The reports also covers regional analysis

o US

o Canada

o Germany

o France

o U.K.

o Italy

o Spain

o Rest of Europe

o Japan

o China

o India

o Republic of Korea

o Rest of Asia-Pacific

o Middle East

o Africa

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Global Cancer Biological Therapy Market 2017 Size, Development Status, Type and Application, Segmentation ... - Digital Journal