1. Gene Therapy

Category Archives: Gene Therapy

Spark Therapeutics adds regulatory insider to support gene therapy for retinal degeneration

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A gene therapy spinout from Childrens Hospital of Philadelphia has added a head of regulatory affairs as it continues Phase 3 clinical trials for its treatment to reverse the effects of inherited retinal degeneration caused by a mutation in the RPE65 gene. It is the latest in a series of additions to the company following a $72.8 million Series B fundraise earlier this year.

Daniel Takefman previously worked for eight years as chief of the gene therapy branch at the Center for Biologics Evaluation and Research at the U.S. Food and Drug Administration, according to a company statement.

Spark Therapeutics, headed by CEO Jeffrey Marrazzo, has been bulking up its management muscle this year. Dr. Katherine High, the former founding director of the Center for Cellular and Molecular Therapeutics at CHOP and a pediatrician at the Perelman School of Medicine, became president and Chief Scientific Officer and has a board seat. It added Stephen Webster as CFO from Optimer Pharmaceuticals and Adolor Corp, whic Cubist Pharmaceuticals acquired in 2011. Steven Gelone joined as head of clinical research and development. Gelone had previously worked for ViroPharma in a similar role Shire acquired the company last year. Diane Blumenthal, another industry veteran, became head of technical operations. She had worked for Eli Lilly for 14 years.

Romuald Corbau, former research project leader for Pfizers gene therapy program for the hepatitis C virus, joined as Sparks translational research lead to help support the companys pipeline of gene therapies.

Earlier this month Spark received breakthrough therapy designation for its SPK-RPE65 treatment. The status speeds up the development and review of investigational therapies for serious or life-threatening conditions, particularly when theres an unmet need.

Marrazzo said earlier this year that the company expects to have data from the Phase 3 trial available in the first half of 2015. In addition to its lead indication, Spark is also working on a Hemophilia B gene therapy treatment to reduce the doses of clotting factor that hemophiliacs need to take to avoid excessive bleeding. The condition is estimated to affect 20,000 people, according to the Centers for Disease Control.

[Photo credit: BigStock Photos]

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Spark Therapeutics adds regulatory insider to support gene therapy for retinal degeneration

World's most expensive medicine Glybera goes on sale with $1m price tag

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The western worlds first gene therapy drug is expected to go on sale in Germany next year. Photograph: Eliseo Fernandez/Reuters

The western worlds first gene therapy drug is set to go on sale in Germany, with a price tag that could amount to an 870,000 cost to treat a single patient.

Glybera, a treatment for the rare genetic condition lipoprotein lipase deficiency (LPLD), which clogs the blood with fat, has been developed by Dutch biotech firm UniQure and Italian marketing marketing partner Chiesi. It is undergoing an assessment of benefits by Germanys federal joint committee, which will report by April 2015.

But the company is seeking a retail price of 53,000 (42,000) per phial, which equates to 1.1m (870,000) for a course of treatment for a typical LPLD patient. This price will be subject to a discount under Germanys drug pricing system.

A Chiesi spokeswoman confirmed the launch price and added that a final figure would be set after the German authorities gave their verdict and negotiations are held with health insurance funds. First commercial treatments are expected in the first half of 2015, she said.

UniQure, which will get a net royalty of between 23% and 30% on sales, said EU pricing was a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York next month.

With only 150 to 200 patients likely to be eligible for Glybera across Europe, the impact on healthcare budgets will be small, even at a very high price but this case will be watched closely as a benchmark for future gene therapies.

UniQure also has plans to seek approval for Glybera in the United States, which it hopes to get in 2018.

Although there is already a gene therapy for cancer on the market in China, that has not been rolled out to other countries, making Glybera a first for the west.

Proponents of the gene-fixing technology insist it stacks up as a cost-effective treatment, despite the high cost, as it could permanently cure many patients.

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World's most expensive medicine Glybera goes on sale with $1m price tag

Fin24.com | First gene therapy drug sets price record

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London - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1m ($1.4m) price tag, a new record for a medicine to treat a rare disease.

The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.

After a quarter century of experiments and several setbacks, gene therapy is finally throwing a life-line to patients by inserting corrective genes into malfunctioning cells - but paying for it poses a challenge.

The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits.

Now Chiesi has filed a pricing dossier with Germany's Federal Joint Committee, or G-BA, which will issue an assessment of the drug's benefits by the end of April 2015. The company is seeking a retail price of 53 000 per vial, or 43 870 ex-factory.

That equates to 1.11m for an typical LPLD patient, averaging 62.5 kg in clinical trials, who will need 42 injections from 21 vials. This price will be subject to a standard 7% discount under Germany's drug pricing system.

Under German rules, the launch price for a new drug is valid for the first 12 months.

A Chiesi spokeswoman confirmed the launch price, in response to inquiries from Reuters, prompted by information from health insurance sources. She added that a final figure would be set after the G-BA gives its verdict and negotiations are held with statutory health insurance funds.

"First commercial treatments are expected in the first half 2015," she said.

UniQure, which will get a net royalty of between 23 and 30% on sales, said EU pricing was a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York on December 1.

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Fin24.com | First gene therapy drug sets price record

117.36 /$ (1 p.m.)

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FRANKFURT/LONDON The Western worlds first gene-therapy drug is set to go on sale in Germany with a price of 1.1 million ($1.4 million), a new record for a medicine to treat a rare disease.

The sky-high cost of Glybera, from the Dutch biotechnology firm UniQure and its unlisted Italian marketing partner, Chiesi, shows how targeted therapies to fix faulty genes may upend the conventional pharmaceutical business model.

After a quarter of a century of experiments and several setbacks, gene therapy is finally throwing a lifeline to patients by inserting corrective genes into malfunctioning cells but paying for it poses a challenge.

The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD), which clogs the blood with fat. The medicine was approved in Europe two years ago, but its launch was delayed to allow for the collection of six-year follow-up data on its benefits.

Now Chiesi has filed a pricing dossier with Germanys Federal Joint Committee (G-BA), which will issue an assessment of the drugs benefits by the end of April 2015. The company is seeking a retail price of 53,000 (66,000) per vial, or 43,870 ($54,800) ex-factory.

That equates to 1.11 million for a typical LPLD patient, who will need 42 injections from 21 vials. This price will be subject to a standard 7 percent discount under Germanys drug pricing system.

Under German rules, the launch price for a new drug is valid for the first 12 months.

A Chiesi spokeswoman confirmed the launch price. She added that a final figure would be set after the G-BA gives its verdict and negotiations are held with statutory health insurance funds.

First commercial treatments are expected in the first half 2015, she said.

UniQure, which will get a net royalty of between 23 and 30 percent on sales, said EU pricing is a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York on Dec. 1.

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117.36 /$ (1 p.m.)

Gene Therapy – Treating the Disease Instead of the Symptoms | Tomorrow Today – Video

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Gene Therapy - Treating the Disease Instead of the Symptoms | Tomorrow Today
Children found to be particularly susceptible to illness could be suffering from a genetic - and potentially fatal - immune disorder. This year #39;s prestigious Robert Koch Prize has been awarded...

By: DW (English)

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Gene Therapy - Treating the Disease Instead of the Symptoms | Tomorrow Today - Video

First gene therapy drug sets million-euro price record

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FRANKFURT/LONDON - The Western world's first gene therapy drug is set to go on sale in Germany with a 1.1 million euro ($1.4 million) price tag, a new record for a medicine to treat a rare disease.

The sky-high cost of Glybera, from Dutch biotech firm UniQure and its unlisted Italian marketing partner Chiesi, shows how single curative therapies to fix faulty genes may upend the conventional pharmaceutical business model.

After a quarter century of experiments and several setbacks, gene therapy is finally throwing a life-line to patients by inserting corrective genes into malfunctioning cells - but paying for it poses a challenge.

The new drug fights an ultra-rare genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. The medicine was approved in Europe two years ago but its launch was delayed to allow for the collection of six-year follow-up data on its benefits.

Now Chiesi has filed a pricing dossier with Germany's Federal Joint Committee, or G-BA, which will issue an assessment of the drug's benefits by the end of April 2015. The company is seeking a retail price of 53,000 euros per vial, or 43,870 euros ex-factory.

That equates to 1.11 million euros for an typical LPLD patient, averaging 62.5 kg in clinical trials, who will need 42 injections from 21 vials. This price will be subject to a standard 7 percent discount under Germany's drug pricing system.

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First gene therapy drug sets million-euro price record

Health Beat: Gene therapy: From bench to bedside: Blindness

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PHILADELPHIA -

In bright daylight, 10-year-old Mark DeVoe has no trouble seeing his friends, but inside, or even in the shade, Mark's eyes sometimes don't work.

"I have trouble seeing like, trees, when the road ends, and when there's like a drop there," Mark said.

At age six, Mark's doctors diagnosed him with the genetic condition choroideremia, which causes people to progressively lose vision until they are completely blind.

"I don't know what it's like to live in darkness, but I've seen it," said Susan DeVoe, Mark's mother.

Susan is a carrier of the blindness gene. Mark's grandfather has the condition.

"Watching my father go blind was devastating. I was a little girl. You know, you count on daddy to do things, and daddy couldn't do them," she recalled.

Dr. Jean Bennett is one of two U.S. researchers preparing to test a gene therapy for choroideremia in humans.

"I think gene therapy holds a huge promise for developing treatments for blinding diseases," said Bennett, ophthalmologist and molecular geneticist at the University of Pennsylvania.

Researchers will use a virus, carrying a normal choroideremia gene and inject the virus just under the retina. The gene should begin to work in a few weeks.

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Health Beat: Gene therapy: From bench to bedside: Blindness

UCLA Stem Cell Researchers Announce Gene Therapy Cure for 18 Bubble Baby Patients – Video

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UCLA Stem Cell Researchers Announce Gene Therapy Cure for 18 Bubble Baby Patients
Researchers at UCLA announced today that they had cured 18 children who were born with the so-called Bubble Baby disease, a genetic disorder that leaves the young sufferers without a working...

By: UCLA Eli Edythe Broad Center of Regenerative Medicine and Stem Cell Research

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UCLA Stem Cell Researchers Announce Gene Therapy Cure for 18 Bubble Baby Patients - Video

The promise and potential dangers of gene therapy | Tomorrow Today – Interview – Video

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The promise and potential dangers of gene therapy | Tomorrow Today - Interview
What role is gene therapy likely to play in the future? Professor Wolfgang Uckert, the president of the German Society for Gene Therapy, talks about the potential of the treatment. More tomorrow-...

By: DW (English)

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The promise and potential dangers of gene therapy | Tomorrow Today - Interview - Video

New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

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Chuck Bednar for redOrbit.com Your Universe Online

A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease.

The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition.

Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems, Dr. Kohn, who is also a professor of pediatrics and of microbiology, immunology and molecular genetics, said in a recent statement.

Children born with SCID have to be isolated in a controlled environment for their own safety, because without an immune system, they are extremely vulnerable to illnesses and infections that could be deadly. While there are other treatments for ADA-deficient SCID, Dr. Kohn noted that they are not always optimal or feasible for many children. The new technique, however, provides them with a cure, and the chance to live a full healthy life.

SCID is an inherited immunodeficiency that is typically diagnosed about six months after birth, the researchers said, and children with the condition are so vulnerable to infectious diseases that even the common cold could prove fatal to them. This particular form of the condition causes cells to not create ADA, an enzyme essential for the production of the white blood cells which are a vital component of a healthy, normally-functioning immune system.

Approximately 15 percent of all SCID patients are ADA-deficient, according to the university, and these youngsters are typically treated by being injected twice per week with the required enzyme. This is a process that must continue throughout a patients entire life, and even then it doesnt always work to bring their immune systems to optimal levels. Alternately, they could undergo bone marrow transplants from matched siblings, but those matches are rare and the transplanted cells themselves are often rejected by the childs body.

Dr. Kohn and his colleagues tested two therapy regimens on 18 ADA-deficient SCID over the course of two multi-year clinical trials starting in 2009. During the trials, the blood stem cells of the patients were removed from their bone marrow and genetically modified in order to correct the defect. All 18 of the patients were cured.

The technique used a virus delivery system first developed in Dr. Kohns laboratory in the 1990s a technique which inserts the corrected gene that produces the ADA into the blood forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells will then produce the T-cells required to combat infections.

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New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease


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