The gene therapy team at Great Ormond Street Hospital was highly critical of the study.
Glaxo will also work with the Italian researchers to develop the gene therapy for six other rare diseases.
The Ziopharm-Intrexon deal gives Kirk an opportunity for a payday no matter what happens with the gene therapy.
The gene therapy program is spread across three of Genzyme's four divisions, each of which has its own tracking stock.
The gene therapy experiment was largely funded by Project ALS, an innovative charity founded by ALS-sufferer Jenifer Estess and two of her sisters.
Its technique involves extracting a patient's own bone-marrow cells, isolating certain stem cells, and delivering the gene therapy before returning the cells to the body.
Sharpening its efficacy, the gene therapy is not activated until a patient takes a pill, controlling how much of the protein is released at what time.
She said they were currently funding research into drugs that mimic the action of nerve growth factor (NGF), the name given to the gene therapy involving stimulating cell function.
It turned out that the virus used to deliver the gene therapy was also inserting itself into cancer-causing areas of the genome, and not just where it was needed to cure the kids.
The Project ALS success could also give a big boost to the gene therapy field, which has fallen on hard times after numerous gene therapy treatments proved ineffective or caused serious side effects.
To move the gene therapy work forward, Kirk inked a deal in January for a 12% stake of Ziopharm Oncology, a biotech company led by former Memorial Sloan-Kettering Cancer Center surgeon Jonathan Lewis.
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The gene therapy involved changing the genetic makeup of a virus, then passing it into the lungs to "infect" the cancer cells, and pass on their genetic changes into the cancer cells to sending their reproductive capability haywire.
Besides Genzyme General, the gene therapy program is involved in Genzyme Molecular Oncology gzmo (nasdaq: gzmo - news - people), which focuses on cancer vaccines, and Genzyme Surgical Products gzsp (nasdaq: gzsp - news - people).
In the most notorious gene therapy disaster, teenager Jesse Gelsinger died after being injected with a gene therapy treatment at the University of Pennsylvania in 1999.
But scientists still face the challenge of developing the necessary gene therapy and targeting tumours with it.
The Glaxo gene therapy program will focus on a severe immune deficiency called ADA-SCID, in which kids cannot make a crucial enzyme called adenosine deaminase that is needed to create immune system cells.
In tests on 135 mice genetically engineered to develop ALS symptoms, mice that got the new gene therapy treatment lived 20% to 30% longer than mice that were injected with a control virus.
The new gene therapy treatment, dreamed up by Salk Institute brain scientist Dr. Fred "Rusty" Gage, avoids this problem by using a harmless virus, called AAV, to deliver genes for the neurotrophic factor IGF-1 directly to the motor nerves.
"This approval unlocks the potential of gene therapy because it is a first at either the EMA or FDA (U.S. Food and Drug Administration) for gene therapy, " uniQure's chief executive Jorn Aldag said in an interview.
Last month, the European Union approved Glybera for treatment of a rare genetic disease, making it the first gene-therapy medicine approved in the Western world.
The patients received gene therapy through a small incision made in the chest.
In the early 1990s, gene therapy was hyped by researchers and the media as the next big thing in medicine.
Despite the slow progress, Dr High says she is optimistic about the future of gene therapy.
ECONOMIST: The prospects for using genes as a therapy may be improving
In the area of gene therapy, progress has been slow, but in some cases remarkable.
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And with its approval, the floodgates for gene therapy could be opened.
FORBES: Europe Takes The Lead Toward Approval Of First Gene Therapy Drug
This month, a committee from the European Medicines Agency recommended the approval of a gene therapy drug, named Glybera (alipogene tiparvovec), for the treatment of a rare inherited genetic disorder.
FORBES: Europe Takes The Lead Toward Approval Of First Gene Therapy Drug
The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world's first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).
It is the first time that gene therapy has been used successfully to treat cancer.
There are other new techniques that may also act to spur the stalled field of gene therapy.
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