Ceregene, a small San Diego-based biotechnology company, recently published new findings in the medical journal Nature Medicine suggesting that its experimental gene therapy could slow the devastating mental decline in Alzheimer’s patients.
In six patients, the rate of mental decline was cut by 36 percent to 51 percent after 22 months of being injected with critical genes directly into the brain.
South San Francisco-based Cell Genesys created Ceregene in January 2001 with $10 million in funding and intellectual property, and gene-therapy programs from Neurologic Gene Therapeutics, a virtual San Diego-based company founded by UC San Diego scientists.
“If validated in further clinical trials, this would represent a substantially more effective therapy than current treatments for Alzheimer’s disease,” said Dr. Mark Tuszynski, who is the scientific co-founder of Ceregene, and a neurologist at the VA San Diego Healthcare System.
“This would also represent the first therapy for a human neurological disease that acts by preventing cell death,” he said.
In this first ever gene therapy study for Alzheimer’s disease, doctors removed skin cells from each patient and genetically modified them so they would secrete a protective substance called nerve growth factor, which are then injected into the brain.
Initially, eight patients received the therapy. Two patients moved as the cells were being injected during surgery, resulting in bleeding in the brain. One patient died five weeks later.
As a result, the protocol was redesigned to perform the surgery under general anesthesia.
There have been no further complications since, according to the company.
Meanwhile, the autopsy of the patient who died revealed that dying brain cells had started to respond to the treatment.
Based on the findings of this Phase I study, Ceregene has started a new Phase I/II study using gene therapy at the Rush University Medical Center in Chicago.
Results of the Phase I study should be available this year or in early 2006 with data from the Phase II study, which will comprise 40 to 60 patients, expected in 2007, Tuszynski said.
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San Diego-based biotechnology firm MacroPore Biosurgery Inc. announced it has signed a definitive agreement to raise $11 million from the sale of 1.1 million common shares at $10 per share.
The unidentified investor has also been given an option to buy 2.2 million more common shares at $10 a share by Dec. 31, 2006, and been offered a voting seat on MacroPore’s board of directors.
The company refused to give more details following the May 2 announcement.
“We haven’t disclosed any background of the investment or identity of the investor, but we will do it as soon as we can,” said Stefanie Bacher, a MacroPore spokeswoman.
MacroPore said it will use the money to support research of its regenerative cell technology, still in preclinical development.
The deal is expected to be completed by May 31.
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Wider Net Loss:
Favrille Inc., a San Diego-based biopharmaceutical company, reported a net loss of $8.4 million for the first three months ended March 11 compared with $5.2 million for the same period last year, the company said late last month.
“The first quarter of 2005 was marked by the completion of our initial public offering, which yielded proceeds, net of underwriters’ discounts and commissions, but before expenses, of $40.9 million, which was a major milestone in our ability to continue development of our lead product candidate FavId,” said John P. Longenecker, the president and chief executive officer of Favrille.
Favrille plans to test the safety and efficacy of the cancer drug FavId in 342 patients with non-Hodgkin’s lymphoma who have received previous treatment, according to the company’s Web site.
As of March 31, Favrille had $58.9 million in cash.
The company expects total operating expenses for 2005 to range between $36 million and $38 million, including an estimated $3 million in amortization of non-cash stock-based compensation.
By year-end, Favrille expects to have cash left between $30 million and $33 million.
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