[Pharm-policy] patent on crucial technology used by dozens of companies for gene therapy experiments

[love@cptech.org]

http://www.wired.com/news/technology/0,1282,42069,00.html

Gene Patent Healthy for Introgen 
by Kristen Philipkoski 

2:00 a.m. Feb. 28, 2001 PST  
      
 A small Texas company has been granted a patent for a crucial
technology used by dozens of companies for gene therapy experiments. 

The U.S. patent could mean that companies such as Pfizer (PFE) and
Schering Plough (SGP) will find themselves paying royalties to Introgen
(INGN) of Austin, Texas. 

Introgen's stock rose 13 percent following the news on Tuesday. 

The patent is related to commercial purification and production of the
so-called adenovirus, which is a vehicle researchers use to carry
healthy genes into patients in gene therapy trials. Introgen's vice
president of intellectual property said the company will look for
potential licensing opportunities. 

"We expect that companies will have an interest in talking to us once
they have picked themselves up off the floor. They know our phone
number," said David Parker, vice president of intellectual property at
Introgen. 

One analyst said landing the patent looks good for Introgen. The
adenovirus vector is used in about 25 percent of gene therapy studies,
second only to the retrovirus which is used in 38 percent of trials. 

The payment of licensing fees could become a major issue for companies
such as Onyx Pharmaceuticals (ONXX), which has been using the vector for
years and is now in the final stages of FDA clinical trials with their
gene therapy for head and neck cancer. An Onyx spokeswoman said lawyers
at the company had not yet reviewed the patent. 

Other companies that use the adenovirus vector include Cell Genesys
(CEGE) and Genzyme (GZMO). 

Cell Genesys spokeswoman Jennifer Williams said the company uses four
types of vectors for their gene therapy research, and the adenovirus is
only third on the list in order of importance. The company's most
important vector, she said, is a so-called adeno-associated virus (AAV). 

Williams also said that Cell Genesys has the largest patent portfolio in
the gene therapy industry. 

Researchers at Schering-Plough are using the adenovirus in phase 2
trials on the treatment of ovarian cancer, but a company spokesman said
Schering-Plough has its own patents on its gene therapy technique. 

"There are a number of gene therapy products that have been developed
using adenovirus vectors and they're all different. There are numbers of
ways of altering the adenovirus to serve as your vector," said Bob
Consalvo, a spokesman for Schering-Plough. 

The adenovirus is popular with gene therapy researchers for several
reasons, Parker said. First, it can be made in reasonably large
quantities for a small amount of money. It also has shown few side
effects in the approximately 500 patients Introgen has treated using the
adenovirus. 

The adenovirus was singled out as the cause in the first death to result
from gene therapy in September 1999. However, the method used to deliver
the treatment was considered very risky. Instead of delivering the gene
therapy into a tumor like Introgen and most other gene therapy
researchers, University of Pennsylvania investigators injected the
adenovirus directly into 18-year-old Jesse Gelsinger's liver. He died
four days later. 

The FDA has since banned Penn's Institute for Human Gene Therapy from
doing experiments on humans, and in December the agency threatened to
ban the researcher in charge of the institute from further human
clinical trials. 

Many researchers say Gelsinger's death was tragic but an isolated
incident. About 3,000 patients total have been treated to date using the
adenovirus vector without the complications seen in Gelsinger's case. 

"I think the fact that (there was) one questionable outcome in an
academic environment that was kind of out there on the fringes is a
pretty good record overall," Parker said. 

Parker touted Introgen's own gene therapy method, saying the company has
been held up as a "poster child" by the FDA as an example of how gene
therapy should be done. 

Introgen was the first company to bring a gene therapy for cancer to
phase 3 clinical trials in head and neck cancer, which is the final
stage of research in FDA testing. Onyx and Vical (VICL) have since also
entered into phase 3 trials. 

Parker wouldn't comment on preliminary phase 3 results but said he would
be "very pleased if the results came back the same as in phase 2."