Scientists’ recent experiments on gene therapy are promising, especially since this has been in the works for over 20 years. These new studies are receiving positive feedback, and interest. One of the world’s leading drug companies, Pfizer, is teeming up with the Philadelphia based Spark Therapeutics, a privately owned U.S. biotech firm. The two are setting up SPK-FIX—a program that studies potential treatments for patients with hemophilia.
Hemophilia is a genetic anomaly that stops a person’s blood from coagulating (clotting) after a cut or wound. When blood does not clot, the wound cannot heal normally, and the individual is at risk of other illnesses associated with heavy loss of blood. The SPK-FIX focuses on hemophilia B with the use of adeno-associated virus (AAV) to manipulate the corrupted gene that causes bleeding.
Pfizer is well known for its long-term investment in fighting against hemophilia. For over 17 years, Pfizer has been providing hemophilia products to patients. Even further, Pfizer sports a global portfolio of 22 approved medicines that treat rare diseases in hematology, neuroscience, pulmonology, metabolic disorders, and oncology. This new research program is the next step in its commitment to improving the lives of blood illness victims.
During the first half of the new year, the initial stages of the program will begin. Spark Therapeutics will manage the first part of the program, phases I/II clinical trials; and Pfizer will take over later studies. On the forefront of the program is Michael Linden, a professor at King’s College, and director of the University College London Gene Therapy Consortium. Linden, who has wide-ranging research experience in AAV gene technology, has agreed to stay with the Pfizer for a two-year secondment to lead rare disease gene therapy.
Linden’s knowledge is critical in Pfizer’s program, because rare diseases are illnesses that affect millions of patients worldwide. Gene therapy, previously a long and disappointing study, has only recently reached gene-altering advancements that can be sold. A new gene therapy drug is scheduled to go on sale in Germany for $1.4 Million in 2015. The new drug battles a genetic disease called lipoprotein lipase deficiency (LPLD) that clogs the blood with fat. These advancements give encouragement to the SPK-FIX program.
Though the new developments in gene therapy are promising, the large price tag attached to them is very disheartening. The SPK-FIX program has the potential to completely alter defected genes in hemophilia patients, but its affordability is the next issue.