Genomics research has generated a wealth of raw data on DNA sequencing and functional and structural genomics. This information may be used in basic research to better understand the molecular function of an organism or the genetic basis of a disease, and it has also caught the eye of the pharmaceutical industry.
The concept of rational drug design, developed when research is done on a single gene or protein at a time, has suddenly acquired a vast new potential with the dramatic increase in starting data provided by the genomics industry.
Genomics information may be used for rational drug design in a variety of manners. For example, researchers may compare either information from a lengthy DNA sequence or a large volume of protein sequences taken from individuals with and without a particular disease in order to locate prospective genes that differ between them.
Amino acid- or protein-sequence data from animals may then be analyzed to locate good models that in turn may be used to learn about the proteins encoded by the prospective disease genes and drugs that may block them. Using this methodology, pharmaceutical companies may detect proteins involved in a disease state that would never be found using older methods. Furthermore, by looking for animals with genetic similarities as well as those with a known tendency to develop a disease companies are likely to find more accurate models.
The patent assignment
Of course, this process can be time consuming so it advisable to file patent applications for inventions made during the course of drug development. The raw data likely will not be patentable for reasons discussed in our previous columns.
However, once DNA or protein sequences have been assigned a putative function, an application may be filed. The processes used to compare human pools and to locate animal models may also be patentable, particularly if they involve specialized software.
Patent claims may also arise from a novel animal model, especially if the animal has genetic variations similar to those found in humans with a particular disease. These animals, usually mice, may be created by knock-out techniques. There are numerous mouse strains with variations that have proved useful as models for human genetic disease. Likewise, transgenic animals containing a human gene known to be associated with a disease state, such as CFTR, and manifesting a disease similar to humans are all very useful. Finally, claims to any novel treatment compounds or methods should be filed.
Genomics research has also lead to a number of processes for the discovery of SNPs or haplotypes that may correlate with a particular phenotype. The use of SNPs in rational drug design may lead to a great deal of patentable subject matter, including new methods for SNP detection, screening assays, therapeutic compositions, and methods of disease treatment and prevention.
SNPs are predicted to exist every 100-300 bases so another approach to rational drug discovery is to look for SNPs or haplotypes in individuals who either are affected by a disease or respond more favorably to a drug. This may be done on a genome-wide basis or after the range of potentially affected genes has been narrowed.
Currently, a consortium of large pharmaceutical companies, academic institutions, and non-profit organizations has been established to sequence the genomes of 24 ethnically diverse individuals and compile a database of an expected 300,000 random SNPs. Other publicly available databases currently contain more than 222,000 SNPs, and Estonia, a former republic of the Soviet Union, is looking to obtain genetic sampler from more than a million of its citizens to create a linkage disequilibrium map to correlate SNPs or haplotypes with particular phenotypes.
Rochelle K. Seide is a partner at the law firm of Baker Botts. She is experienced in biotechnology, intellectual property, and patent issues, and also has a Ph.D. in human genetics. Michelle LeCointe is an associate at Baker Botts and a registered patent agent. They can be reached at [email protected] and [email protected].
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