Genzyme targets less-exotic diseases

If the future of medicine is making carefully targeted therapies for a carefully screened group of patients, then the future looks a lot like Genzyme Corp.

The 25-year-old Cambridge company -- one of the world's largest and oldest biotechnology companies -- has long specialized in selling biologic drugs to patients with rare diseases.

In the past few years, it has begun diversifying into serving people with more widespread ailments, from leukemia to creaky knees.

Last year, Genzyme posted a 310.7 percent increase in profit margin over 2004, sending it to number three on the overall Globe 100 list. The prior year's results had been weighed down by $250 million in charges from its purchase of Ilex Oncology Inc.

But Genzyme also reported steady growth of its core products, including top-selling Cerezyme, a protein that treats the rare Gaucher disease, which causes a range of symptoms including bleeding, fatigue, and broken bones. And the recent federal approval of Myozyme, for Pompe disease, a rare, debilitating muscle disease that can kill newborns within one year, expands its line of human enzyme replacement drug products.

With a vast brick cathedral-like plant on the Charles River in Allston and large facilities in Cambridge and Framingham, Genzyme is the poster child for the local benefits of the biotechnology industry: It has expanded steadily over the years, and unlike some firms it manufactures its flagship drugs in Massachusetts.

The Allston plant houses six $25 million bioreactors, in which genetically altered hamster cells crank out Genzyme's unique protein therapies.

As it matures, Genzyme also is trying to take a leadership role in biotech corporate citizenship. Its new eco-friendly corporate headquarters in Cambridge -- with a self-regulating climate system and waterless toilets in the men's bathrooms -- is one of a handful of buildings in the world to win the top US green building honor. And a planned $2 million donation to the Museum of Science would be the largest in the museum's history.

''We've grown up, and our needs have grown in terms of how we want to interact with the community," said chief executive Henri A. Termeer in a recent interview with The Boston Globe.

To be sure, questions hang over Genzyme, including the possibility of competition for its cash-cow enzyme replacement drugs. Analysts also wonder how it will make money from newly approved Myozyme, which cost the company $500 million to develop but is extremely expensive to produce at an effective strength.

The company chronically faces potential public and political backlash over the prices of its drugs. Its cash cows are so-called ultra-orphan drugs for rare diseases, which cost $200,000-plus annually for each patient.

Patients have a love-hate relationship with Genzyme. Often, it is the only drug company developing or offering a therapy for their disease, but the price can leave them struggling to stay insured.

To reduce its dependence on so-called ''orphan diseases," Genzyme has been pushing into kidney disease and cancer therapies. A $1 billion 2004 investment in Ilex gave it two leukemia drugs, Campath and Clolar. Another recent acquisition gave it Renagel, a drug for kidney dialysis patients that could hit $500 million in annual sales this year.

Its Synvisc is the leading injectible treatment for arthritic knees, and it also has found intriguing data that Campath can help not only cancer patients but also people with multiple sclerosis. Although the trial was suspended last September after a patient died, Genzyme hopes to restart testing on the drug this year.

Stephen Heuser can be reached at sheuser@globe.com.