Seven years and one month after applying, geneticist LouisKunkel and Boston Children's Hospital on Tuesday wereawarded U.S. Patent No. 5,239,060, protecting Kunkel'sdiscovery of the gene for dystrophin, the protein missing inmuscular dystrophy (MD).

In announcing the issuance, Genica Pharmaceutical Corp. ofCambridge, Mass., which holds worldwide licensing rights to thepatent, said it would now aggressively seek a corporate partnerto find a cure for the disease. "By pursuing a coordinated,multidisciplinary approach, we believe that the development ofan effective therapy for MD is achievable within the next fiveyears," said Robert Flaherty, Genica's president and chiefexecutive officer.

"The partnership between Genica and Children's Hospital is animportant step in ensuring the continuation of the ongoingwork, which is moving us closer toward a treatment that willhalt this devastating disease," Kunkel added.

Research neurologist Nigel Fleming and others, includingKunkel, founded Genica in 1988 to develop commercialproducts and processes from the invention. So far, the companyhas put two MD diagnostics on the market, a DNA depletion testand a dystrophin-protein-based analysis. Fleming, who is nowGenica's vice president of business development, told BioWorldthat the two diagnostics marketed to neurologists have so farnetted Genica "somewhat less than $5 million.

With the just-issued patent protecting its intellectual propertyposition, the company is now intensifying its drive to sign upcorporate partners "with skills complementary to ours --groups that have expertise and scientific depth to be able totake on a project like this," Fleming said.

"They would have to be interested in a project of this size," headded. "Clearly it's not on the vast scale of Alzheimer's disease,but neither is it as modest as cystic fibrosis."

Genica's management is seeking candidate companies with twodiscrete "skill sets:

-- Ability to screen large numbers of drugs in cultured humanmuscle cells.

-- Possession of proprietary delivery systems for gene therapy.

Genica hopes to wrap up the first two alliances by Christmas."These are important strategic relations for us to advance thetechnology now. At some later point, we would like apharmaceutical partner to take on the expensive clinical trialwork," Fleming said.

He added that Genica has been in active discussions with avariety of groups for a number of months -- on the cell-screening side, three or four prospects; and for gene therapy,one or two.

During the early half of the 1980s, Kunkel was a post-doctoralfellow at the Muscular Dystrophy Association in Tucson, Ariz.,and MDA funded his research at Children's Hospital. DonaldWood, now a scientific adviser to the association, was then itsresearch director, and he played a role in Children's HospitalUsassigning the pending patent to Genica, Feming recalled.

Wood told BioWorld that MDA supported Kunkel under acontract with the hospital. As he remembers, the total tabamounted to "one-half to three-quarters of a million dollars."

MDA, a not-for-profit organization, has a standard patent policyenabling it to recoup some or all of the amounts it contributesto its research projects. "The institutions where we fundresearch agree that if there is any proprietary exploitation ofany invention funded in part or total by this association, thatthere would be some agreement where we would share in apercentage of any royalty distribution," said RonaldSchenkenberger, MDA's director of research and patientservices administration. "We're flexible in that regard."

When the pivotal Cohen-Boyer gene-cloning patents issued in1981, 72 companies worldwide promptly lined up to lay down$10,000 a year apiece, for licenses to practice the protectedintellectual property. Does Genica foresee such a tribute for theKunkel dystrophin patent? The company's patent attorney,John Freeman of Boston's Fish & Richardson, said, "Is anyonetrying to cure muscular dystrophy with dystrophin? There's nocommercial activity yet."

"It's a very, very broad patent," Fleming said. "It includesclaims on cell therapy, and as we were the first to file anythingin the field, I believe ours antedates anything else." He added,"I believe Peter Law (of the Cell Therapy Research Foundationin Memphis P- see BioWorld, Aug. 23) has one (patent)pending."

Fleming concurred with Freeman about the commercialactivity, saying, "I don't see any people who should be payingus fees now. I think some of the other groups thatcontemplated doing Duchenne Muscular Dystrophy therapyrealize that we hold the proprietary position." One or twosmaller biotechnology companies "that realize they'll have towork collaboratively with us" have approached Genica, he said.

As for enforcing the patent in the future, Fleming said,"Intellectual property on treating MD is our mission and ourlife blood, so we will both defend it and pursue it."

-- David N. Leff Science Editor

(c) 1997 American Health Consultants. All rights reserved.