Enzo Biochem Granted European Patent For Displacer Technology To Treat Gene Abnormalities

Methodology Could Make Possible Modification or Corrections of Cell Mutations

15-Jun-2004

Farmingdale. Enzo Biochem, Inc. , announced that the European Patent Office has granted it a patent that describes, in part, a method of performing targeted delivery and incorporation of genetic material into chromosomal DNA without using a viral vector, thus making possible the editing and correcting of certain abnormalities in genes.

The patent covers, among other things, constructs and methods that will allow a single stranded nucleic acid molecule to displace one of the strands of a double helical molecule. Among its many possible useful applications is one that can use what is called displacer technology to correct point mutations or effect small deletions or additions, even inside of living cells and tissue. This type of correction could permit individual alleles or genes to be modified, enabling mutations in cells to be corrected, resulting in corrections to the protein encoded by the genes, such that the protein would function more effectively.

"This technology holds promise of being one of the gentlest and most effective means of achieving gene therapy, or of achieving the addition, deletion or improvement of gene functions to improve quality of life for individuals," said Dean Engelhardt, Ph.D., Executive Vice President of Enzo. "One target of this new displacer technology would be to correct the genetic defect leading to sickle cell anemia."

Enzo Biochem holds a complementary U.S. Patent, No. 5,958,681, issued in March 2000, which covers nucleic acid compositions and methods for editing and correcting certain abnormalities in genes. "The essence of both of these patents," commented Dr. Engelhardt, "is to provide the ability to correct inherent errors in the genes of a subject, and of certain other medically important genetic material, where alterations may be indicated."

The newly awarded European patent, EP 0 450 370 B1, in addition to the performance of targeted delivery of genetic material, also provides for simple methods for preparing stable branch migration structures; procedures of simultaneous labeling and identification of specific DNA fragments that are significantly simpler, faster and cheaper than blot analyses; and labeling procedures that permit sequence enrichment or purification by affinity chromatography and selective cloning.

Enzo said it is currently engaged in pre-clinical studies incorporating gene-altering technology to achieve specific therapeutic benefits. "The European patent grant represents further acknowledgement of Enzo's novel approach for treating genetic defects and viral diseases using our Company's pioneering science," said Dr. Engelhardt.

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