Nexell Buoyed by Link to Gene Therapy Success
Nexell Therapeutics shares soared 55% Friday on news that the Irvine-based biotech company’s product was one of the key components in a groundbreaking gene therapy trial reported by researchers last week.
French scientists reported using gene therapy to treat three babies with “bubble boy disease,” a rare inherited disorder that keeps newborns from developing a complete immune system. Ten months after treatment, the children exhibited seemingly normal immune systems--a result widely touted as the first time gene therapy has been used to successfully cure a disease.
Nexell said that cells from the patients, whose DNA was altered in the experiments, had been housed, cultured and treated in a special type of cell culture bag that it makes, called Lifecell X-fold bags. The cells were then injected back into the sick patients.
Nexell issued a news release Thursday afternoon discussing the Lifecell bag employed in the French trial. And investors Friday bid Nexell’s shares up $1.66 to close at $4.69 on volume of 10.7 million shares, more than double its daily average volume. Nexell was the leading percentage gainer on the Nasdaq biotech index of 203 issues.
The Nexell release quoted a member of the French team as saying that the use of Nexell’s bags, coated with an agent known as RetroNectin that allowed the missing gene to better enter the cells, was important to the experiment’s success.
“We have found that the Nexell cell culture containers, when coated with [RetroNectin], greatly facilitate [the insertion of the gene],” said Dr. Alain Fischer, head of the pediatric immunology and hematology unit at Hospital Necker in Paris.
The Lifecell bag has been used successfully at other gene therapy labs.
A leading gene therapy researcher said bags such as Nexell’s could become a standard component in the protocols used by scientists in future gene therapy trials.
Harry L. Malech of the National Institutes of Health said in an interview Friday that he is performing a gene therapy trial for a rare inherited disorder and using the RetroNectin-coated bags to grow and treat patients’ cells.
Malech said that there are other alternatives to bags for holding patient cells, such as plastic dishes or containers, but that the bags are easier to handle and have several advantages over other containers.
The technology to make the bags--which look like flatter, smaller versions of those used to store plasma and blood for transfusions--is patented, said Tad Heitman, Nexell’s spokesman. They are made of a special plastic that is charged and have small pores that allow exchange of gases, an important process when culturing cells.
Sales of Lifecell X-fold bags represent only a small percentage of the company’s revenue, Heitman said.
Nexell Therapeutics was created in May 1999. It had been a subsidiary of Delaware’s VIMRx Pharmaceuticals Inc. and Baxter Healthcare Corp.
Nexell, which develops cell therapies to treat cancer, this week posted a first-quarter loss of $9.3 million, compared with a year-earlier loss of $7.2 million. Revenue declined 13% to $4.8 million.
Despite the run-up in its stock Friday, Nexell’s shares are still well off their 52-week high of $16.05, reached in the midst of a biotech investor frenzy in March.
