By Lisa Seachrist
WASHINGTON — Transgene Inc., a French gene therapy company, has extended for another three years its collaboration with the Association Francaise contre les Myopathies (AFM) to develop a gene therapy treatment for Duchenne's muscular dystrophy (DMD) and other neuromuscular disorders.
The agreement calls for AFM to provide a total of FFr84 million ($14.7 million) to fund the research program. Transgene will provide its expertise in gene therapy vectors to the effort to effectively transfer genes into the muscles of the body.
"This is a major agreement with them," said Bernard Davitian, chief financial officer for the Strasbourg-based company. "The renewal is a sign that they are confident in the work we do. It is good for our burn rate and good for the progress of science at Transgene."
Under terms of the agreement, AFM will pay Transgene FFr64 million ($11.2 million) in the form of a research grant that the company will use to develop a gene therapy for DMD and to further develop its gene therapy vectors. In addition, AFM will contribute FFr20 million ($3.5 million) in the form of an interest-free loan repayable upon the commercial launch of a new therapy.
The agreement extends through June 2001 a deal into which Transgene and AFM originally entered in July 1995. AFM is a patient organization that raises money to support patients suffering from neuromuscular disease and their families. In addition, the organization funds research into such diseases.
DMD is a rare inherited disorder caused by a mutation in the dystrophin gene. It is the most frequent genetic disease in muscle. It is characterized by progressive muscle weakness. Early symptoms usually begin between the ages of two and five years. Patients are typically wheelchair bound by age 10, and respiratory complications usually end their lives in their twenties or thirties.
Davitian noted that developing a genetic therapy for the disease faces many obstacles because the gene must penetrate all muscles and lodge in enough cells to restore function.
"Being able to target muscular cells for gene therapy is a very difficult undertaking," said Davitian. "For us to try to attack such a complex hurdle may be valuable in allowing us to target other diseases."
Transgene will focus on its adenoviral, synthetic and retroviral vectors in order to develop an adequate therapy. The company also has vaccinia virus and cellular vectors.
In February, Transgene entered into an $88 million collaboration with Schering-Plough Corp. to develop p53 gene therapy vectors to treat cancer. The company has several gene therapy treatments for different cancers in Phase II clinical trials. *