StemCell Therapy

Carrying a $475,000 price-tag

AP

By Mallory Locklear

A CAR-T treatment a type of gene therapy for cancer has been approved for use in the US. Announced by the US Food and Drug Administration (FDA) on Wednesday, this is the first approval anywhere in the world for a type of CAR-T therapy, although the techniques have been used experimentally for some time.

CAR-T therapy made headlines earlier this year, when it was announced a CAR-T approach had saved the life of Layla, a young child in the UK who had leukaemia. The approach involves reprogramming a persons own immune cells to make them better at targeting cancerous ones.

The drug that has been approved by the FDA is Kymriah, a treatment for B-cell acute lymphoblastic leukaemia, the most common childhood cancer in the US.

Advertisement

To synthesise Kymriah, a patient first has a type of immune cell, called T-cells, removed from their body and transported to a facility in New Jersey operated by the pharmaceutical firm Novartis. Here, viruses will be used to insert a gene into these cells. The gene codes for a protein called a chimeric antigen receptor (CAR).

These cells are then reinfused back into the person. The added protein helps these modified T-cells home in on and fight leukemia cells.

In a trial, this approach achieved an 83 per cent remission rate over a period of three months in people who hadnt responded to other treatment options. The FDA has approved Kymriah for people aged 25 or under who have not responded to other treatments, or who have relapsed.

Nearly half the people in the trial experienced a side effect caused by an unwanted immune response triggered by the altered T-cells. Because of this, the FDA is requiring staff at the 32 facilities approved for this treatment to undergo specific training to recognise this response, called cytokine release syndrome.

Kymriah will cost $475,000. This sounds high, but its lower than some analysts expected, and unlike many expensive cancer drugs, it is a one-off treatment that could result in years, not months, of extended lifespan.

The FDAs decision has been hailed as the first approval for a gene therapy in the US. Some argue that this isnt a true gene therapy, as the genes introduced into the T-cells are not the treatment themselves it is the transformed T-cells that go on to fight the cancer. But the FDA defines human gene therapy as products that introduce genetic material into a persons DNA to treat a disease, so has classified Kymriah as such.

Europe has already approved two gene therapies for inherited diseases, while China approved a gene therapy for cancer treatment in 2004.

As for CAR-T therapies, other firms have similar treatments in the works, while Novartis also plans to get Kymriah approved for treating lymphoma.

More on these topics:

Continued here:
Pioneering gene therapy approved for leukaemia in the US - New Scientist

MarketWatch rounded up 10 of its most interesting topics over the past week.

Campbell Soup Co. CPB, -0.22% had a rough quarter, but the company is facing a dire long-term problem.

Novartis AG NVS, -0.08% received FDA approval for the first cancer gene therapy available in the U.S. Emma Court explained how important this is for young people suffering from a type of acute lymphoblastic leukemia (ALL), and she interviewed Janney analyst Paul Knight, who made recommendations for investors on how to play a potential decade-long growth cycle for gene therapy.

Here are charts that will help you sift through a boatload of absurdity spouted every day by self-styled stock-market gurus.

Nissan Motor Co. 7201, +0.27% is about to launch a redesigned Leaf electric car. The company has a big advantage over Tesla Inc. TSLA, -0.15% because of its huge manufacturing scale, but one big question is the new Leafs battery range, as Claudia Assis reports.

The startling increase in value for bitcoin rivals that of other types of assets that have bubbled and burst. Andrew Left believes the Bitcoin Investment Trust GBTC, -12.94% is a very dangerous investment.

The damage from Hurricane Harvey to the Houston area has been devastating. The coming flurry of activity as the damage is repaired might cause a rise in U.S. GDP, but Caroline Baum calls claims of real economic benefits predictable nonsense.

Amazon.com Inc. AMZN, +0.11% was called the weakest major U.S. retailer this week by Moodys Investors Service. But T. Rowe Price Media and Telecommunications Fund PRMTX, +0.69% is a big believer. The fund, which had more than quadrupled the S&P 500s return over the past 15 years, had more than 10% of its assets in Amazons shares as of July 31.

If you are retired, you might think it will be very difficult to get a mortgage loan because of low income. But there are many financing options available for those without a steady monthly income, according to Darrow Kirkpatrick.

Jeff Reeves weighs the pros and cons of scooping up shares of Apple Inc. AAPL, +0.05% right now.

If you get excited by Labor Day sales, you might be missing out on bigger savings later.

Want more from MarketWatch? Check out our Personal Finance Daily or other newsletters, and get the latest news, personal finance and investing advice.

See original here:
Weekend roundup: Campbell in the soup | New cancer gene therapy | Exposing bad investment advice - MarketWatch

Because gene therapy involves making changes to the bodys set of basic instructions, it raises many unique ethical concerns. The ethical questions surrounding gene therapy include:

How can good and bad uses of gene therapy be distinguished?

Who decides which traits are normal and which constitute a disability or disorder?

Will the high costs of gene therapy make it available only to the wealthy?

Could the widespread use of gene therapy make society less accepting of people who are different?

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

Current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed on to a persons children. Gene therapy could be targeted to egg and sperm cells (germ cells), however, which would allow the inserted gene to be passed on to future generations. This approach is known as germline gene therapy.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they cant choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people.

Read the original here:
What are the ethical issues surrounding gene therapy ...

In 1990, geneticist William French Anderson injectedcells with altered genes into a four-year-old girl with severe immunodeficiency disorder. This was the first sanctioned test of gene therapy, in which genetic material is used to treat or prevent disease.

If were lucky, Anderson told The Chicago Tribune, with this little girl weve opened the door for genetic engineering to attack major killers and cripplers, particularly AIDS, cancer and heart disease.

Gene therapy has never fulfilled these grand hopes. In the decades since Andersons experiment, thousands of clinical trials of gene therapies have been carried out. But the first gene therapy was only approved for sale in the U.S. this week. The Food and Drug Administration announced its approval of Kymriah, a gene therapy produced by Novartis for a form of childhood leukemia. A few gene therapies have previously become available in Europe and China.

An FDA press release emphasizes the historic nature of the approval. Were entering a new frontier in medical innovation with the ability to reprogram a patients own cells to attack a deadly cancer, FDA Commissioner Scott Gottlieb says.

As I have noted previously, for gene-therapy proponents have long predicted that it will eliminate diseases such as cystic fibrosis and early-onset breast cancer, which are traceable to a defective gene. Enthusiasts also envisioned genetically engineered "designer babies" who would grow up to be smarter than Nobel laureates and more athletic than Olympians.

Gene therapy turned out to be extremely difficult, because it can trigger unpredictable, fatal responses from the body's immune system.The National Institutes of Health warnsthat gene therapy can have very serious health risks, such as toxicity, inflammation, and cancer.

Kymriah is a case in point. The FDA press release warns that Kymriah can cause life-threatening immune reactions and neurological events, as well as serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). According to The New York Times, the FDA is requiring that hospitals and doctors be specially trained and certified to administer [Kymriah], and that they stock a certain drug needed to quell severe reactions.

Kymriah illustrates another problem with gene therapy: high cost. Novartis estimates the cost of its treatment at $475,000 per patient. As a recent Reuters article notes, over the past five years two gene therapies have been approved for sale in Europe, one for a rare blood disease and the other for the bubble-boy immunodeficiency disorder. The therapies cost $1 million and $700,000, respectively. So far, the companies that make the therapies have achieved a total of three sales.

As journalist Horace Freeland Judson points out in this excellent 2006 overview, The Glimmering Promise of Gene Therapy, most individual diseases caused by single-gene defectsthe kind that seem most likely to be cured by gene therapyare rare. (Sickle-cell anemia and some other hemoglobin disorders are among the few exceptions.)

Judson adds that because different diseases have different genetic mechanisms and affect different types of tissue, each presents a new set of research problems to be solved almost from scratch. As the millions burned away, it became clear that even with success, the cost per patient cured would continue to be enormous. And success had shown itself to be always glimmering and shifting just beyond reach.

The advent of CRISPR, a powerful gene-editing technique, has aroused hopes that gene therapy might finally fulfillexpectations. Researchers recently reported that they employed CRISPR to counteract a mutation that causes heart disease. Potentially, The New York Times reported last month, the method could apply to any of more than 10,000 conditions caused by specific inherited mutations.

CRISPR has also renewed concerns about the ethics of producing designer babies with enhanced physical and mental traits. But as Science noted recently, CRISPR poses some of the same risks as gene therapies. CRISPR still has a long way to go before it can be used safely and effectively to repairnot just disruptgenes in people.

So to answer the question posed in the headline: No, the gene-therapy era has still not arrived.

See the original post here:
Has the Era of Gene Therapy Finally Arrived? - Scientific American (blog)

MINNEAPOLIS (WCCO) Colin Cooley of Burnsville beat lymphoma four years ago, but the lymphoma came back in a different spot two years later.

Chemo wasnt cutting it, Cooley said. It was keeping it in check, but it wasnt getting rid of it.

He decided to undergo a clinical trial at the University of Minnesota. He received a gene therapy called CAR-T and is now cancer-free.

The FDA approved CAR-T Wednesday as the first type of gene therapy in the United States.

The treatment has been called a breakthrough in the fight against cancer. It is only approved right now to treat children with acute lymphoblastic leukemia, but doctors are excited about its potential for other cancers and diseases.

Doctors at the University of Minnesotas Cancer Day at the Minnesota State Fair called the therapy a major leap.

(credit: CBS)

Were able to take a patients own cells and turn them into something that can actually attack their specific cancer, said Dr. Edward Greeno, medical director of the University of Minnesotas Masonic Cancer Clinic. Many people have referred to this as living cancer because were taking live cells and turning them into your treatment.

First, a patients blood is drawn and their T-cells, or immune cells, are separated out. Those T-cells are then sent to a laboratory to be genetically modified and reprogrammed to zero in on the cancer.

Those modified cells are then multiplied in the lab before being returned to the patient via blood. They are essentially revved-up cells that are missiles for the cancer.

In one significant study, 83 percent of the patients who received CAR-T went into remission.

This treatment is expected to be offered for lymphoma patients next year. Dr. Greeno says it could be decades, though, before its offered to patients with other types of cancer.

Right now, its expensive almost $500,000 and used mostly on patients when other methods of treatment, like chemotherapy, have failed.

Before I didnt know if Id be here in three or four or five years, I didnt know, Cooley said. Now I feel like I have a new lease, some minor issues, but a new lease on life, and thats pretty exciting.

Read more here:
How Does Gene Therapy Work? - CBS Minnesota / WCCO

HUMANKIND VIDEOS Quiet dad freaks out when baby #4 is born | 0:39

A normally quiet dad of three girls can't help but celebrate when he finds out his fourth child is a boy. Humankind

1 of 8

This police officer with the Austin Police Department in Minnesota spotted an elderly lady dancing in a parking lot alone. The officer pulled up, blasted the music, and joined her for a dance. Humankind

2 of 8

Wyatt constantly pushes himself in the gym, and in everyday life, even after a motorcycle accident left him paralyzed from the waist down. Humankind

3 of 8

Branson found his two-year-old twin cousins face down in the pool. He started CPR, and helped save their lives. Humankind

4 of 8

After being bullied for most of her life, Lindsay has found her strength and confidence through sports. Humankind

5 of 8

A mom-to-be wanted to surprise her mom with her pregnancy, so she put a shirt on her dog that says, I am going to be sister. Animalkind

6 of 8

Roxy can't stop eyeing her owner's Doritos, but when she's caught staring, she quickly looks away. Animalkind

7 of 8

These kids rapping about school will get you out of your seat. Humankind

8 of 8

Quiet dad freaks out when baby #4 is born

Police officer finds lady dancing in parking lot alone, joins in

Paralyzed teens pull-up video is incredible to watch

Six-year-old saves drowning babies with CPR

Runner with alopecia overcomes bullying, finds confidence

Grandma can't contain her excitement over baby announcement

Dog can't stop eyeing mama's snacks

Kids combine rap and college goals to create songs of success

Dr. Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at C.S. Mott Children's Hospital in Ann Arbor, works with Maryam Rasheed of Macomb Township. Maryam was part of a clinical trial using gene therapy to successfully treat her leukemia.(Photo: Sophie Masson/Michigan Medicine)

The U.S. Food and Drug Administrationapproved on Wednesdaythe first-ever gene therapytotreat children and young adults withleukemia.

Called Kymriah, but better known as CAR T-cell treatment, the therapy is being hailed by doctors as revolutionary. Itinvolves genetically modifyinga patient's own T-cells, which thencantarget and kill a form of acute lymphoblastic leukemiacells.

This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patients own T-cells can be collected, genetically modified and redirected to kill a patients tumor," said Dr.Gregory Yanik, clinical director of the Pediatric Blood and Marrow Transplantation Program at the University of Michigan's C.S. Mott Children's Hospital. Mottwas one of a few hospitals nationally to take part inclinical trials of the treatment.

"This allows us to turn patients own cells into a powerful weapon to fight the disease a weapon that does not rely on chemotherapy but takes a whole new approach to attacking childhood leukemia, Yanik said.

The CAR T-cell treatmentoffers new hope for children like Maryam Rasheed, 10, of Macomb Township.

Maryam was diagnosed with B-cell acute lymphoblastic leukemia at age 4, when her family was seeking refuge from religious persecution in Turkey, said Maryam's mother, Asmaa Rasheed.

Maryam Rasheed (right) with her brother, Rashid, and sister Samantha. Maryam, 10 of Macomb Township, survived acute lymphoblastic leukemia.(Photo: Rasheed family photo)

"My country is Iraq," Asmaa Rasheedsaid. "It wasnt safe. We are Christian. It was so hard over there in Baghdad. We run away to Turkey.

"We take her to hospital the first timebecause ... she stopped eating, stopped walking, stopped talking. We bring her to emergency. The doctor decided to take her bone marrow to do tests. Then the results came back, and she have leukemia."

Maryam underwent her firstchemotherapy treatment in Turkey.

"Over there, it was so hard," Rasheed said. "The doctors dont speak English over there. We know English a little bit. We speak Arabic."

Maryam Rasheed of Macomb Township undergoes treatment for acute lymphoblastic leukemia. She is now in remission.(Photo: Rasheed family photo)

Rasheed stayed with her daughter for two months in the Turkish hospital. A few months later,the Rasheed family was able to immigrate to the U.S. and settled in Michigan.

But Maryam's cancer returned. She was treated at Children's Hospital of Michigan with more chemotherapy and radiation. In 2013,her younger brother, Rashid, proved to be a match for a bone marrow transplant.

Still, the cancer wouldn't relent.

The Rasheed family learned of a clinical trial for CAR T-cell therapy under way atMott. It was the family's last chance,Rasheed said.

Maryam Rasheed, 10, of Macomb Township holds up her arms joyfully. She's surrounded by her sister Samantha (left), brother, Rashid, and baby sister Annabell.(Photo: Rasheed family photo)

"There was nothing to do," her mother said."In Detroit, there was chemo, radiation, bone marrow transplant. It returned back three times. She lose her hair three times. It was so hard for her and my family."

She remembers the date Maryam started the clinical trial at Mott: Dec. 17, 2014. Maryam spent Christmas and her seventh birthday in the hospital.

"I think we waited like 100 days,I dont remember exactly, and they did a bone marrow test, and the medicine, it work!" Rasheed said.

"It was like a dream, you know, like light coming from far away when youre in the dark. Theres nothing else we could do. But the CART-cell was like a shining light from far away."

Maryam has been in remission two years, andis starting fourth grade next week at Shawnee Elementary School in Macomb Township.

"Now, shes start her life, and doing everything a little kid is doing," said Rasheed, who says she hopes the treatment helps other children, too.

So does Yanik.

"Acute lymphoblastic leukemia is the most common form of cancer in children, accounting for approximately25% of all childhood cancers," Yanik said. "This particular therapy utilizes a childs own immune system to target their leukemia."

Theclinical trials focused on the 15% to 20% ofchildren whoseB-cell acute lymphoblastic leukemia had either relapsed or who had residual leukemia cells in their bone marrow after treatment.

"Historically, such patients would have an estimated cure rate of approximately 10%," Yanik said. "The two trials were groundbreaking. In the most recent trial, 52 of 63 patients with childhood leukemia successfully entered complete remission with this therapy."

Novartis Pharmaceuticals Corp. got the FDA approval for the gene cell therapy, whichinvolves drawing blood from childrenwith B-cell acute lymphoblastic leukemia. The T-cellsin the child's blood are thenshipped to a lab where they are genetically engineered so theywillseek outa particular protein in the leukemia cells and attack. Patients are then infused with the modified blood, and the T-cells go to work to find and kill the leukemia.

The New York Times reported Wednesday that the therapy will cost $475,000 for the initial treatment, with additional treatments administered at no cost.

Although 83% of the children in the clinical trials for CAR T-cell therapy went into remission, Yaniksaid it's too early to tell howcurative treatmentswill prove in the long run. And, its use will be limited to only a few medical centers in the U.S.

"The University of Michigan is the only site in the state and within this region that is licensed to administer these cells for childhood leukemia," he said.

Offering the treatment at a large medical center like U-Mis essential, said Dr. Rajen Mody,a pediatric oncologist at Mott, because of the severity ofpotential side effects.

"It can cause serious side effects, especially within the first 21 days," said Mody, who is Mott's director of pediatric oncology. "Patients can have high fevers, bleeding complications, trouble breathing, infections. ... Thats why a hospital like the University of Michigan is the ideal place. ... Patients who undergo this treatment are usually so sick after an infusion of the CAR-T cells, that they can't be safely treated at smaller hospitals."

Dr. Rajen Mody, a pediatric oncologist at the University of Michigan's C.S. Mott Children's Hospital.(Photo: University of Michigan)

Yanik is hopeful that successful treatment with CAR T-cell therapy in children with leukemia will open the door for similar therapies targeting other cancers.

"Aseparate CAR T-cell trial targeting diffuse large-cell lymphoma was recently completed with the results in that clinical trial now under review at the FDA," he said. That trial alsoincluded adult patientsat the University of Michigan.

Mody called the gene therapy revolutionary.

"This is clearly a life-saving and potentially curative therapy," he said."Its being tested in other types of leukemia and solid tumors. Its too early to say whether its going to work as well for other cancers.... We are not there yet."

Still, he said, it's made all the difference for Maryam and her family.

"She was one of the lucky ones coming from Iraq, and with all the things she has survived. And then coming here and surviving this,... she clearly has some goodluck.

"I think she should do very well. Patients who actually survive the first six months and still have CAR T-cells detected in their systems tend todo very, very well."

Contact Kristen Jordan Shamus: 313-222-5997 or kshamus@freepress.com. Follow her on Twitter @kristenshamus.

Read or Share this story: http://on.freep.com/2wV39dg

Read the original here:
First gene therapy to treat cancer gets FDA approval; UM only Michigan hospital to use it - Detroit Free Press

Nightstar Therapeutics has filed to raise up to $86 million in a Nasdaq IPO. The money will equip Nightstar to complete a phase 3 trial of its choroideremia gene therapy and advance two other eye disease candidates through early-stage clinical studies.

London-based Nightstar, also known as NightstaRx, is set to move the choroideremia asset into phase 3 in the first half of next year. The therapy, NSR-REP1, is advancing into the 140-patient trial on the strength of data on 32 subjects treated in investigator-sponsored studies. Those trials found 90% of patients either maintained or improved their visual acuity in the year after receiving the gene therapy.

Given choroideremia causes currently-untreatable progressive vision loss, Nightstar sees the data as supporting further development. The asset is moving forward with a fairly clean safety profile in the 50 people treated to date. Investigators have seen one adverse eventtransient intraocular inflammationthat may have stemmed from treatment with NSR-REP1.

Challenges await Nightstar as it scales up for the phase 3 trial and potentially commercial sales, though. The biotech acknowledges the administration of NSR-REP1 requires significant skill and training, potentially creating a bottleneck to use of the gene therapy. And as a small, unpartnered player in a new field, any number of events could knock it off course.

What Nightstar does have is a head start. Spark Therapeutics has the most advanced challenger to NSR-REP1 but its program is yet to move past phase 1/2. The field is similarly clear for Nightstars follow-up candidate NSR-RPGR, which moved into the clinic just ahead of MeiraGTxs rival X-linked retinitis pigmentosa gene therapy. AGTCs Biogen-partnered candidate is close behind.

Nightstar has reached this point using money from a succession of private rounds, starting with the 12 million Syncona invested when the biotech spun out of the University of Oxford in 2014. The biotech pulled in a further 5 million when it named former Johnson & Johnson VP David Fellows as CEO early in 2015. A $35 million series B round followed late in 2015. And Nightstar broadened its investor base and raised a further $45 million in a series C round a few months ago.

Along the way, Nightstar has built out a team in preparation for the broadening of its clinical trial program and life on public markets. Last month, Ex-Pfizer clinical lead Tuyen Ong, M.D., left PTC Therapeutics to serve as chief development officer. And in April, Nightstar hinted at its IPO plans by recruiting the man who led Intercept Pharmaceuticals repeated public raises, Senthil Sundaram.

The question now is how receptive public investors are to gene therapy biotechs. The companies in the sector to go public to date have delivered mixed returns, with the successes of bluebird bio and Spark offset by the steady decline of uniQure and implosion of Dimension Therapeutics.

See the rest here:
Nightstar files for $86M IPO to fund gene therapy trials - FierceBiotech

Erlanger hopes to bring the treatment to the patients who need it most within the next few years . (Coutesy: WTVC)

Wednesday, the Food and Drug Administration cleared the way for a ground breaking cancer treatment in the United States.

It's a gene therapy treatment named CAR-T therapy.

Dr. Meghann McManus is a Pediatric Hematologist Oncologist at Erlanger in Chattanooga.

On Thursday, Dr. McManus described the treatment as the "first gene therapy treatment for any type of cancer in the pediatric world or adult world."

In layman's terms, Dr. McManus said the treatment allows doctors to remove a patients "T-cells, which are a type of white blood cell," send them to a lab "where their genetically modified to fight their certain type of leukemia."

"It uses the patients own immune system, their own cells from their own body, to fight their leukemia. Which is not something that we do with any other disease," Dr. McManus said.

At this time, Dr. McManus said CAR-T therapy is approved for "children that have relapsed or with refractory disease."

"We may not be able to stop cancers from happening... but if we could treat it in a way to get rid of the cancer without big side effects or tolls on patients, it would change the way we do our job," Dr. McManus said.

Read more from the original source:
Local pediatric oncologist hopeful new gene therapy will save lives - WTVC

Please enable Javascript to watch this video

KANSAS CITY, Mo. -- Lucas Novick, 27, has been in a battle with leukemia since his freshman year of college.

"I was having headaches that were so bad that they were causing vomiting pretty regularly and I couldn`t see straight well enough that I felt safe driving myself to school," Novick said.

Since 2009, Novick has endured a number of treatments including chemotherapy and a bone marrow transplant. The treatments have taken a physical and mental toll on Novick's body.

"The transplant that was supposed to save my life also nearly took it from me," Novick said. "The damage chemotherapy did to my body when I was first treated in 2009 and 2010 was such that I was walking with a cane after my 21st birthday. It did so much damage to my hip joints that they were replaced in 2011."

But after Novick's leukemia returned for a second time, he went to Children's Mercy Hospital where doctors were performing an experimental treatment.

"The approval of the CTL019 product for pediatric patients with relapsed refractory acute lymphoblastic leukemia is really exciting for us," Doctor Doug Myers, of Children's Mercy Hospital, said. "We`ve spent a lot of time working on ways to get the immune system into the fight against cancer because we think it can decrease toxicity, decrease the amount of chemotherapy and radiation that we use for these cancers."

Dr. Myers said the treatment helped Novick, a musician, back onto the stage and has held his leukemia awayfor two years.

"Those are really special rewards for us in this field that have seen so many failures of this type of therapy in the past. To see this go forward, move forward, do well enough for a pharmaceutical company will pick this up and take it the rest of the way, that`s a really special time for us," Dr. Myers said.

While doctors believe it's too early to call the new treatment a cure, many agree this is the first step to a new generation of cancer treatment.

"I know at the end of the day that this is the future of medicine," Novick said.

Read more from the original source:
Man describes new FDA-approved gene therapy for leukemia that ... - fox4kc.com

Gene therapy is the use of DNA as a potential therapy to treat a disease.In many disorders, particularly genetic diseases caused by a single genetic defect, gene therapy aims to treat a disease by delivering the correct copy of DNA into a patients cells.The healthy, functional copy of the therapeutic gene then helps the cell function correctly.

In gene therapy, DNA that encodes a therapeutic protein is packaged within a vector, often a naked virus, which is used to transfer the DNA to the inside of cells within the body. Gene therapy can be delivered by a direct injection, either intravenously (IV) or directly into a specific tissue in the body, where it is taken up by individual cells. Once inside cells, the correct DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patients disease and can provide long-term benefit.

Abeona is developing next generation adeno-associated virus (AAV) gene therapies. Viruses such as AAV are utilized because they have evolved a way of encapsulating and delivering one or more genes of the size needed for clinical application, and can be purified in large quantities at high concentration. Unlike AAV vectors found in nature, the AAV vectors used by Abeona have been genetically-modified such that they do not replicate. Although the preclinical studies in animal models of disease demonstrate the promising impact of AAV-mediated gene expression to affected tissues such as the heart, liver and muscle, our programs use a specific virus that is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS), making them attractive for addressing lysosomal storage diseases which have severe CNS manifestations of the disease.

Lysosomal storage diseases (LSD) are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Multiple tissues ranging from musculoskeletal and visceral to tissues of the central nervous system are typically involved in disease pathology.

Since the advent of enzyme replacement therapy (ERT) to manage some LSDs, general clinical outcomes have significantly improved; however, treatment with infused protein is lifelong and continued disease progression is still evident in patients. Thus, viral gene therapy may provide a viable alternative or adjunctive therapy to current management strategies for LSDs.

Our initial programs are focused on LSDs such as Mucopolysaccharidosis (MPS) IIIA and IIIB, also known as Sanfilippo syndromes type A and type B. MPS III is a progressive neuromuscular disease with profound CNS involvement. Our lead product candidates, ABO-101 and ABO-102, have been developed to replace the damaged, malfunctioning enzymes within target cells with the normal, functioning version.

Delivered via a single injection, the drug is only given once.

Here is the original post:
Gene Therapy - Abeona Therapeutics

Personalized cancer treatments known as CAR-T cells (chimeric antigen receptor T cells) have dominated the headlines lately, thanks to Novartis tisagenlecleucel, which won an early approval from the FDA for the treatment of leukemia on Aug. 30. But CAR-T treatments are labor-intensive and expensive to make, and they can attack healthy tissues in the body, leading to dangerous side effects.

Scientists at the Fred Hutchinson Cancer Research Center have developed a tool that they believe could address both those shortcomings of CAR-T and other forms of cell engineering. They have invented nanoparticles that deliver proteins to cells, which in turn edit those cells genes temporarily. Lead author and bioengineer Matthias Stephan describes it as hit-and-run gene therapy, and he believes the technique will streamline the manufacturing of cell-based therapies.

Heres how it works: The nanoparticles home in on specific cells, such as the T cells in the immune system. They then deposit messenger RNA (mRNA) to those cells, which triggers short-term changes in the proteins the genes produce. The technology does not permanently change the DNA, but it makes enough of an impact on it to produce a therapeutic outcome.

RELATED: Can CAR-T cancer treatments be fine-tuned to avoid toxic side effects?

Whats more, the nanoparticles can be freeze-dried and then activated with a small amount of water. They really let you fulfill all your wishes as a genetic engineer because you can pack in all your different [gene-therapy] components and further improve the therapeutic potential of your cell product without additional manufacturing steps, Stephan said in an article posted on Fred Hutchs website.

Stephans team proved out their concept by testing the nanoparticles in three different cell-engineering applications, one of which was CAR-T. Currently, CAR-T treatments are made by giving T cellsgenes that teach them to destroy cancer cells. The Fred Hutch scientists used their nanoparticles to remove a different gene from T cellsone that normally prompts them to attack healthy tissue.

Then they tried enhancing the CAR-T cells in a different manner. They temporarily gave them genes that have the potential to make central memory T cells, which are able to survive over the long term, remembering their cancerous targets and attacking them should they ever resurface.

The scientists tested their engineered CAR-T cells in mouse models of leukemia and found that the animals that received them lived twice as long as mice that got conventional CAR-T cells. They also tested the nanoparticles in two other cancer-related applications of gene therapy.

Despite all the excitement over CAR-T, concerns about side effects continue to dog the field. A dangerous immune reaction known as a cytokine storm has been seen in trials of both Novartis treatment and Axi-Cel, a CAR-T from Kite Pharma, which is being acquired by Gilead. The third player in the CAR-T field, Juno Therapeutics, saw its late-stage trials delayed when some patients died of neurological side effects.

Fred Hutch scientists have been working on other techniques for improving CAR-T. In December, a set of researchers there who receive funding from Juno announced positive results from a trial of a fine-tuned CAR-T treatment in patients with chronic lymphocytic leukemia (CLL). Instead of using just one type of CAR-T, the team combined two specially selected cell subtypes into one treatment. They also announced they had identified biomarkers that they believe can be used to predict which patients are likely to have severe reactions to the treatment.

Stephans team is now collaborating with several companies to fine-tune CAR-T treatments for cancer, according to Fred Hutch. And they believe their freeze-dried nanoparticles may prove useful in developing treatments for a range of other diseases, too, including HIV and blood disorders caused by defective hemoglobin.

Read the original:
'Hit-and-run' gene therapy nanoparticles could enhance CAR-T ... - FierceBiotech

(KMSP) - Charlotte Gifford is almost 5 years old with dreams of looking like her favorite Disney princess.

Rapunzel, because she has the long hair, says Charlotte.

Charlotte lost her own long locks after being diagnosed last March with b-cell acute lymphoblastic leukemia, the most common form of leukemia to develop in children and young adults.

Once we got the word it was leukemia. Our world came down it crumbled, says Erica Gifford, Charlottes mom.

Erica and Adam Gifford are thrilled the Food and Drug Administration announced approval of the first gene therapy in the United States. Car t-cell gene therapy can be used for Charlottes type of leukemia, affecting anyone under 25 years old.

Its a new treatment with an entirely different flavor, says Dr. Daniel Weisdorf, Professor of Medicine and Chief Division of Hematology, Oncology and Transplantation. Its not a drug, but a way to take the patients own cells, their normal cells, and engineer the cells so they will specifically and uniquely attack the leukemia cells.

Weisdorf witnessed success from this treatment, as some patients from University of Minnesota Health and Masonic Cancer Center were part of the clinical trials.

The treatments are remarkably effective but complicated to administer because the patients get very sick, says Weisdorf.

The Leukemia and Lymphoma Society has helped fund research for decades, and today, Executive Director Teri Cannon is celebrating a major medical milestone.

Nowadays 90 percent of young people diagnosed with leukemia survive, says Cannon, Those kids that relapse and standard therapy doesn't work for them, 83 percent of the kids who have used car t-therapy in the clinical trial have survived. So that's going to bring us a lot closer to that 100 percent that is going to make parents happy.

As for Charlotte, she's doing well and in remission. Hopefully she'll never need the this newly approved gene therapy reserved for patients whose cancer has returned. For the Giffords, this major medical advancement offers their family and others optionsand hope.

You dont know how important it is until your own child is diagnosed with cancer, says Gifford.

Charlotte continues steroid, chemo and physical therapy. Her parents have started a Go Fund Me Page.

View post:
FDA approves gene therapy that could fight type of leukemia - KMSP-TV

The US Food and Drug Administration (FDA) on Wednesday approved a "historic" treatment that genetically engineers a patient's own immune cells to fight childhood leukemia.

The procedure, known as CAR-T cell therapy, takes a patient's immune cells, or T-cells and white blood cells, and genetically modifies them to give a power boost to viciously target leukemia.

Once transfused back into the patient, the immune cells then attack leukemia cells for months or even years.

"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. "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."

Read more:Fighting cancer with methadone - making chemotherapy more powerful

Last resort

The treatment was developed by Novartis Pharmaceuticals and the drug is known as Kymriah (tisagenlecleucel).

The immune system-altering treatment was approved for patients aged3 to 25 with a blood cancer called acute lymphoblastic leukemia (ALL),the most common form of childhood cancer in the US.

About 3,100 people under the age of 20 are diagnosed with ALL each year in the United States.Around20 percent of those with ALL fail to respond to traditional drug, bone marrow transplants and chemotherapy treatments. Typically, young patients with ALL who fail chemotherapy have only a 30 percent chance of survival.

The breakthrough treatment would only be used if the disease failed to respond to standard treatment.

The therapy would cost $475,000 (400,000 euros) per dose, but Novartis said anyone who didn't respond within a month would not have to pay. Typical treatments of ALL, such as bone marrow transplants, can run up to $800,000 in the first year.

Application to be filed in EU

According to studies, about 83 percent of patients responded to CAR-T cell therapy went into remission after three months.

The treatment does carry potential side effects, including an immune overreaction, fever, high blood pressure, neurological reactions, acute kidney injury and decreased oxygen.

Questions also remain about the drug's possible long-term side effects and ability to preventALL from coming back.

Due to their age, many of those suffering from the disease would be covered by their parent's health insurance or Medicare in the US.

The procedure is best known for treating a now 12-year-old girl named Emily Whitehead, who has been cancer-free for five years after being the first child to receive the experimental therapy.

An application for CAR-T is expected to be filed with the European Medicines Agency by the end of the year.

cw/cmk(AP, AFP)

Read the rest here:
US approves breakthrough gene therapy for childhood leukemia - Deutsche Welle

Hurricane Harvey Flooding Claims 6 Members Of Same Family

Members of the Saldivar family ranging from 6 to 84 perished inside a van swept by currents as they sought higher ground. (Patch)

U.S. OK's First Gene Therapy Treatment For Childhood Leukemia

The Food and Drug Administration on Wednesday signed off on the first gene therapy treatment in the U.S. that doctors say uses a patient's own cells to seek out and destroy deadly childhood leukemia. (Patch)

Legendary Basketball Coach Rollie Massimino Dead At 82

Legendary college basketball coach Rollie Massimino, who led the Villanova Wildcats to a storied NCAA Championship in 1985 against Georgetown University, has died, according to the chancellor of Keiser University, who expressed "heartfelt sadness" on behalf of the school. Massimino served as head coach of the Keiser men's basketball team for the past 12 years. He was 82. (Patch)

World's Worst Babysitters Leave 9-Month-Old At Long Island Casino

Three people, including two teens, were arrested after they left a 9-month-old baby at a Long Island casino Tuesday, police said. The trio was supposed to be babysitting the child, but a dispute with the baby's mother quickly sent things haywire. (Patch)

Indiana Couple Completes Decades-long Goal To Visit Every Cracker Barrel

An elderly couple from Goshen finished a decades-long mission over the weekend: to visit every Cracker Barrel location, WGN-TV reports. Ray and Wilma Yoder flew out to Tualatin, Oregon, so they could visit and eat at their 645th Cracker Barrel on Monday morning. (Patch)

Quick Hits

Trump Claims To Have Witnessed Harvey's 'Horror And Devastation' Firsthand - But Didn't (Patch)

Here's How To Throw A Killer Labor Day Party, According To Pinterest (Patch)

'Having A Baby Means Losing A Paycheck' For Pregnant Police Officer Forced On Unpaid Leave: ACLU (Patch)

Microsoft, Amazon Collaborate So Personal Assistants Can Chat (Patch)

Deer In The Headlights Detained On San Francisco-Oakland Bay Bridge (Patch)

Girl, 7, Halfway Through Hug-A-Cop Tour Across USA

Rosalyn Baldwin is on a mission. Her goal? To hug a law enforcement officer in every state of the USA. Spanning 21 states so far, the seven-year-olds journey has been shared via her website, which is maintained by her two sisters, OceanRose and Scholar24, as well as her Facebook page. (Patch)

Harvey Slams Southeast Texas: Helicopter Rescues; Heartbreaking Scene As Toddler Clings To Mother

People are being plucked from homes by helicopter, boats are going door to door and the governor is sending the National Guard to the area. And in Beaumont, one of the more heartbreaking scenes of the storm unfolded when rescuers saved a toddler who was clinging to the body of her mother, who had died. (Patch)

All Houston ISD Students To Receive 3 Free Meals Per Day This School Year

All Houston ISD students will eat for free during the 2017-18 school year thanks to a waiver of the required application process for the National School Lunch/Breakfast Program from the United States Department of Agriculture and the Texas Department of Agriculture. (Patch)

Hurricane Harvey Scam Alert: Beware Of ICE, HSI Impersonators

Houston Police Chief Art Acevedo said there are reports that people impersonating Homeland Security Investigations special agents. Acevedo said they are knocking on doors in the Houston area telling residents to evacuate in the aftermath of Hurricane Harvey presumably so these impostors can rob the empty homes, he said. (Patch)

Gas Prices Rising Thanks To Harvey, Says AAA

While Hurricane Harvey was bearing down on Texas and the Gulf Coast, drivers looking to fill up their tanks were greeted at the gas station with one of the largest one-week surges in gas prices this summer, according to AAA. Prices increased 4 cents to a nationwide average of $2.37 per gallon. (Patch)

Parade For Purple Heart Recipient Planned In Illinois

A United States Air Force veteran who was seriously wounded in Afghanistan a dozen years ago and given a small chance to live will be given a parade in the town where he attended high school. (Patch)

Hurricane Harvey: More Than 300 Gators East Of Houston Could Soon Escape Enclosure

As if things weren't bad enough in the Houston area amid Tropical Storm Harvey, up to 350 alligators at a sanctuary east of town could soon escape if water levels continue to rise, according to reports. (Patch)

Photo of Overjoyed Mom Becomes A Social Media Sensation

Her daughters, Hayley and Millaina, knew the photo of their mom jumping for joy with a glass of wine in her hand was being taken as a joke, but mom told Patch, "Those facial expressions are for real!!!" (Patch)

This Day In History

1422 King Henry V of England dies of dysentery while in France. His son, Henry VI becomes King of England at the age of 9 months.

1864 During the American Civil War, Union forces led by General William T. Sherman launch an assault on Atlanta.

1897 Thomas Edison patents the Kinetoscope, the first movie projector.

1962 Trinidad and Tobago becomes independent.

1993 Russia completes removing its troops from Lithuania.

Famous Birthdays

AD 12 Caligula, Roman emperor

1928 James Coburn, American actor

1944 Roger Dean, English illustrator and publisher

1946 Tom Coughlin, American football player and coach

1970 Debbie Gibson, American singer-songwriter, producer, and actress

Photo: Former Villanova head coach Rollie Massimino looks on prior to the 2016 NCAA Men's Final Four National Championship game between the Villanova Wildcats and the North Carolina Tar Heels at NRG Stadium on April 4, 2016, in Houston. Credit: Streeter Lecka/ Staff/ Getty Images Sport/ Getty Images

Thanks for your feedback! Now share it with your friends!

Thanks for your feedback.

Originally published August 31, 2017.

Read more from the original source:
Harvey Haunts Family Leukemia Gene Therapy Massimino Dies: Patch Morning Briefing - Patch.com

FINDINGS

Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia. In a preclinical study, researchers found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells.

Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as bad cholesterol) and an increased risk of early heart disease.

The LeXis gene belongs to a unique group of genes that until recently were considered junk DNA because scientists believed they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes,now referred to as long noncoding ribonucleic acids, or lncRNAs, is a rapidly evolving area in biology.

Researchers wanted to test whether a single injection of LeXis could slow the development of heart disease. To do so, they gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.

In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.

Although previous research has shown that lncRNAs can be important, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. Junk genes could one day offer a framework for treating people with familial hypercholesterolemia and other conditions that are otherwise very difficult to treat.

The papers authors are Xiaohui Wu, Zhengyi Zhang and Dr. Tamer Sallam of UCLA; and Dr. Peter Tontonoz, Marius Jones and David Salisbury of the Howard Hughes Medical Institute.

The study waspublished onlinein the journal Circulation.

The research was supported by grants from the National Heart, Lung, and Blood Institute; the American College of Cardiology; and the Lauren B. Leichtman and Arthur E. Levine UCLA Cardiovascular Discovery Fund.

Learn more about the cardiovascular research theme at UCLA.

More here:
Gene therapy using 'junk DNA' could lower risk for heart disease - UCLA Newsroom

Three patients who received SPK-8011 a gene therapy developed by Spark Therapeutics for hemophilia A show increased production of clotting factor VIII without any safety issues reported, says the company.

The patients are the first to receive the treatment as part of a Phase 1/2 clinical trial, with early data showing they have not yet experienced spontaneous bleeding episodes.

The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease a significant achievement in the gene therapy field and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention, Katherine A. High, president and CEO of Spark Therapeutics, said inreleasingsecond-quarter 2017 financial results.

The study (NCT03003533) examines escalating doses of the treatment a one time infusion of a non-infectious virus that delivers a functional copy of the gene encoding clotting factor VIII.

The first two patients received the lowest dose and have now been followed for 23 weeks and 12 weeks. Measurements show their levels of the clotting factor steadily rising, reaching a stable level of 11 and 14 percent of normal values. Withthose results in hand, researchers decided to go for a higher dose in the third patient. So far, the increase of factor VIII in that third patient is higher than that observed in the other two, corresponding to the increased dose.

Researchers have not detected immune reactions to the therapy in any of the patients. This is important, as antibodies to SPK-8011 ould potentially render it ineffective.No serious adverse events have been reported and none of the patients required treatment with corticosteroids.

Spark, based in Philadelphia, has also reported on its second gene therapy, SPK-9001, now being developed to treat hemophilia B.

Researchers presented data from the Phase 1/2 study (NCT02484092) at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, showing that the 10 participants had lowered their annual bleeding rate by 96 percent. They also lowered their annualized infusion rate by 99 percent.

In addition, their levels of clotting factor IX, 12 weeks after the treatment, had stabilized at 33 percent. Four of the patients have been followed for more than a year after the treatment, and one for as long as 18 months.

None have developed immune reactions to the treatment and no bleeding episodes have been recorded. One patient with severe joint disease had precautionary factor infusions for persistent knee pain.

We are excited about the progress we are making to achieve our goals of our investigational hemophilia A and B programs: to safely achieve predictable, consistent and sustained activity levels that prevent spontaneous bleeding, said High.

Go here to read the rest:
Spark's Gene Therapy SPK-8011 to Treat Hemophilia A and B Advance in Clinical Trials - Hemophilia News Today

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales.

Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia.

The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn.

It would be the first gene therapy for cancer and if approved is expected to generate a spike in revenues for Oxford BioMedica, with the potential for further tie-ups on other cancer treatments.

Oxford BioMedica is also trialling its lentiviral vectors, which help manipulate genes, in potential treatments for Parkinsons Disease and an eye condition.

In half-year results today, Oxford BioMedica pared back losses to 2.2m, compared to 6.9m in the red the previous year. Revenues jumped 26pc to 15.7m.

See original here:
Gene therapy darling Oxford BioMedica pares losses - Telegraph.co.uk

Please enable Javascript to watch this video

GAINESVILLE, Florida Imagine only being able to see the things in front of you in soft focus, and just in black and white.

For people with the genetic eye condition achromatopsia, those are just some of the side effects.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

"I need to have very dark, very tinted sunglasses to feel comfortable outside, to see really well," said Tara.

Tara has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light.She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

"I cannot drive a car so I rely on public transportation and my bike to get around," she said.

"There are currently no approved or no effective treatments for achromatopsia," said Dr. Christine Kay, a surgical ophthalmologist at the University of Florida.

But she's working to change that.

She is one of a handful of experts testing a gene therapy.

"For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision. and those are cells at the very bottom layer of the retina," explained Dr. Kay.

Using a tiny cannula, doctors deliver a normal copy of one of two mutated genes, the CNGA-3 or CNGB-3 gene, directly into the eye to restore vision.

Tara`s myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial.

In the meantime, Tara says she's learned to adapt to achromatopsia and live without limitations.

"And I hope, ya know, all young achromats, ya know, learn the same thing."

Dr Kay says if the gene could eventually be delivered to the surface of the retina, additional patients, like Tara, could be treated. AGTC, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

NEW TECHNOLOGY: A new clinical trial is underway testing gene therapy for achromatopsia. The genes are responsible for releasing proteins essential for the function of all the cells. Researchers remove the virus from the host DNA so it does not have the capacity to make the patient sick, and then insert the gene of interest into the DNA. Surgically done by a vitro retinal surgeon, the gene is then directly delivered to the retinal tissue. Researchers try to avoid directly touching the retina, to avoid detaching it and keep the surgery less evasive. The CNGA3 or CNGB3 gene would help restore the patient`s vision. This can only be done on certain patients; those with extremely severe achromatopsia are not able to participate in this trial because of the risk of retinal rupturing.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

See the original post here:
YOUR HEALTH Gene therapy for better eyesight - WQAD.com

Investment Thesis

With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders.

Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS).

The company's strong balance sheet was further bolstered by a raise of circa $78.1 million through public offering at a price of $7.25 per share, with a revised analyst share price target estimates from Jefferies LLC of $17 per share (jefferies.com), as a "financial investment hedge against emerging gene-editing technology of CRISPR," the CRISPR/Cas9 system, has seen less successful testing, coupled with scientific criticism for its reported high incidence of nonspecific DNA cleavage, mosaicism (failure to replicate mutant alleles in only some of the cells) and inversely, and overproduction of mutations in a given organism.

SGMO's latest 10-Q (SEC.gov):

In the context of gene therapy, Sangamo ($707.16 million market cap) (capitaliq.com) was the only firm with innovative gene therapy drugs to advance in two clinical trials (namely the SB-728-T program in the Sangamo ZFP Therapeutic Pipeline targeting HIV/AIDS), and the first to conduct genome editing studies in clinical trials, as covered in the Financial Times. The stock's performance has been volatile, and investors were disinclined to back the drug lottery in gene therapy as biotech became heavily saturated in players, regulations and setbacks, stunting the prospect of any tangible advancement.

Sangamo has jumped on the "radar" to become a buy. The FDA has authorized Fast Track designation, the scientific backing is legitimate (and has received industry-wide appraisal), the longer-term targets look plausible and Sangamo's management and consultants are capable of executing given their credentials and two decades of development.

By all measures of valuation, Sangamo is undervalued, and price-action enthusiasts will recognize the surge in trading volumes and the breaking of the 50-day moving average. Early riders would have capitalized on the short-term price depression triggered by the publicizing of the mucopolysaccharidosis Type I (MPS I) and MPS II delay, an overreaction from the market that posed a great value opportunity at the beginning of this month, whose benefits were felt just last week when the stock surged on the impressive FIH results.

Gene therapy (and emicizumab) renders recombinant factor VIII/IX proteins redundant. This a slice of the industry that is represented by $8-10 billion annually (with 90%+ profit margins) shared by Shire (NASDAQ:SHPG), Bayer (OTCPK:BAYRY), Novo Nordisk (NYSE:NVO), Pfizer and Bioverativ (NASDAQ:BIVV), an overvalued stock whose current operations consist entirely of the moribund method of treating hemophilia - a potential sell. It recently acquired True North and thus entering into cold agglutinin disease market, a result of severe hemolytic anemia.

Sangamo now has the backing and leeway to make its drugs commercially viable, magnetizing further interest - 48 institutions have increased their respective positions in SGMO, with Wasatch and BlackRock leading the pack. The smart money and medical experts are backing Sangamo.

Bold tickers indicate potential buys should this thesis play out as predicted.

This is not investment advice, you are advised to carry out your own due diligence.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read the original here:
Sangamo Therapeutics: The Breakthrough In Gene Therapy - Seeking Alpha

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

Original post:
Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS