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A Cell Break Masterminded By The Mapping Of The Human Genome

Managing Editor

The completion of the Human Genome Project (HGP) in 2003 has busted many technologies out of their cells, immediately commuting pipelines that seemed to be serving life sentences in clinical development, and confined by suspect technologies that were arresting corporate progress and detaining funding opportunities.

Three established markets in the RNA/DNA field that are poised to additionally grow on that success are RNAi, pharmacogenomics and DNA forensics, while others, such as antisense and ribozymes, stand to have their values affected with tangential growth, or even complete failure, attributable to the market dominance of one of the three aforementioned technologies. Still other disciplines, such as theranostics and molecular diagnostics, can capitalize on the afterglow of that success to energize their respective markets.

The United States Human Genome Project, begun in 1990 as a 15-year task and finished ahead of schedule in 2003, was coordinated by the U.S. Department of Energy and the National Institutes of Health. The stated goals of the project were to:

  • identify all the approximately 20,000-25,000 genes in human DNA,

  • determine the sequences of the 3 billion chemical base pairs comprising human DNA,

  • store this information in databases,

  • improve tools for data analysis,

  • transfer related technologies to the private sector, and

  • address the ethical, legal, and social issues (ELSI) that may arise from the project.

Thus, the goal of the government was not to engage in therapeutics development, but to deliver the decoded data necessary to boost research efforts in the corporate drug discovery milieu. This report examines the successfulness of that endeavor and, more thoroughly, the markets involved in DNA/RNA R&D activities which are most likely to have the biggest impact on the biotechnology industry.

An important feature of the HGP was the federal government's objective to give complete access of data and transfer of technology to the private sector upon completion of the project. By licensing technologies to private companies and awarding grants for innovative research, the project helped to strengthen the U.S. biotechnology industry and induced the development of many new therapeutic applications. Sequence and analysis of the human genome working draft were published in the February 2001 and April 2003 issues of Nature and Science.

There were also a number of private sector companies vying with the government to map the human genome in order to exploit any resultant marketing, capital or research value. Celera Genomics, as an example, was founded solely to sequence and assemble the human genome. The company completed its own human genome mapping project in close proximity to the HGP and now licenses its subscription database to a number of companies. Celera had the foresight to diversify into therapeutic drug discovery programs in which it could exploit its own genomic data and continue moving forward.

Many of the other competing companies were trumped by the HGP, lending more apprehension to the already-tentative market, as industry observers and investors were left wondering about the subsequent direction and response of those businesses.

At the turn of the century, there was a rush to jump aboard the genomics bandwagon while it was enjoying its advantageous status as the technology-of-the-moment. A few years later, sector uncertainty temporarily alarmed the genomics market and daunted venture capital interest, as news of the impending completion of the human genome mapping was circulated throughout the biotechnology industry.

Companies that were conducting similar genome-decoding research were forced to consider that they had expended years and money, only to lose the race to solve the long-standing challenge of deciphering the function of each human gene, leaving their R&D efforts and future in flux.

Other genomics companies, but especially industry investors, were unsure what impact the project's completion would have on the industry as a whole, so it became a cautious market until the post-HGP impact could be analyzed.

In the meantime, companies rushed to drop the word "Genomics" from their corporate names, indicating hesitance to be too closely associated with a discipline that many interpreted would peak with the culmination of the HGP, inasmuch as there was so much expectation associated with the achievement of the project.

Biotechnology companies began using new terms, as well as reverting to pre-genomics ones, in making concerted efforts to describe their areas of concentration. They were now practitioners of gene therapy, gene expression, cellular biology, RNA manipulation, biomarkers, target validation, DNA therapeutics, molecular diagnostics, genotyping, bioassay-anything except genomics.

The completion of the HGP eventually provided an answer to the question of what would come next as a result of the mapping. Derivative opportunities abounded with an increase in the number of emerging, as well as established, markets utilizing the knowledge gained from that project to increase opportunities in a wide range of applications, especially those concentrating on RNA or DNA function.

The genomics field is once again popular, after meandering through the slight identity crisis leading up to its highly anticipated and well-publicized HGP success, when biotechnology industry observers were uncertain what would be done with the results of the mapping of the human genome.

There are many RNA and DNA technologies being utilized in drug discovery efforts across the industry. The BioWorld RNA/DNA Therapeutics & Technologies Report 2006: Major Market Opportunities in RNAi, Pharmacogenomics and Forensics examines the status and prospects of the three major markets, with secondary attention dedicated to the sector's ancillary disciplines.

RNAi has been successful in outlasting the hype that gave the sector its wings, and is seeking to be regarded as a valid market, based on its initiation into the clinical trials stage with a number of candidates. That crucial step places the market at a decisive phase in which scrutiny and corroboration replace publicity and premise.

This segment is very dynamic with opportunity and challenge. Affirmative indicators are present, as partnering transactions are increasing, employment activity is vigorous, and investment attention is strong. There are also a number of dedicated RNAi therapeutics companies that are creating diversification models in their pipelines, hoping to bolster market share and create internal capital-generating mechanisms to support RNAi R&D. If these constructive factors are ultimately to define the market, then the present time would likely represent the most prudent opportunity to invest or otherwise participate in the RNAi sector.

On the other hand, there are unresolved and unfolding issues that may not affect this market in a positive manner. Decisions have begun to be rendered in major intellectual property disputes, big pharma and biotech are choosing and aligning with preferred partners through licensing and acquisition deals and VC patterns are being defined. Such dynamics can create an intensely competitive market, in which the standing of companies on marginally tenuous ground can devolve into untenable positions.

If scientific and corporate dilemmas such as delivery, stability and litigation cannot be effectively surmounted, the market could wind up in a pattern of no progress for years to come. There is also the equivalent of corporate "trash-talking," as contentious discussion implies that reputation and destiny may hinge on impending and developing court decisions, inasmuch as a patent infringement ruling may profoundly imprint a company's credibility at this pivotal time in this highly observed market.

This book explains what has transpired in the past year, as RNAi has retired from the glare of the Next-Big-Thing limelight and is settling into the more staid, but no less scrutinized, atmosphere of the clinical environment where hype must transform into empirical accomplishment.

This publication addresses the emergence of RNAi as a leading target validation, biomarker, and general reagent tool, and its effect on competitive technologies, such as antisense. The status of RNAi therapeutics in development and the current IP issues that involve many of the primary players in the market are also focused on as major issues which have the potential to profoundly affect investor interest and pipeline development.

Pharmacogenomics and forensics applications have been the benefactors of literally billions of bits of information that can potentially impact those technologies immediately and profoundly in the search for truly novel therapies. These technologies are curretly far more than hypotheses because the HGP has performed the equivalent of a bulk of the legwork, thrusting the laboratory and preclinical drug development process as much as years ahead.

The DNA-based drug market has been defined and is in motion, Biotechnology clinical trial pipelines are using pharmacogenomic data to advance products In March 2005, the FDA released guidelines (www.fda.gov/cder/Guidance/6400fnl.htm) for next-generation clinical research strategies titled "Guidance for Industry Pharmacogenomic Data Submissions." These steering principles describe the regulatory landscape and establish criteria for the facilitation of the incorporation of pharmacogenomic data into the drug discovery process on the path to the development of safer, efficacious personalized drugs. This guideline acknowledges the standing of pharmacogenomics as an emerging and valid field that is actively contributing in the therapeutic development process.

A potential downside that is addressed is the theory that the best scenario possible for pharmacogenomics and forensics may only offer niche markets that don't traditionally attract huge venture capital interest. There is debate over whether profit is marginalized too much to attract investor interest as a result of personalized drugs that have therapeutic benefit for as few as one person or a family or a small ethnic or geographic group.

DNA forensics is already a gainful market in certain biodefense, judicial and law enforcement, authentication and identification applications, but its largest application in the future may potentially be as a component in the drug development process.

This report addresses the likelihood of these DNA-specific technologies to become blockbuster models in spite of target applications that are apparently limited by some form of specific genetic makeup that, by design, would exclude the mass public.

The sequencing of the human genome was never meant to be an endpoint. The project itself was not intended to directly result in therapeutics product development; rather it was meant to provide a blueprint that would offer factual data therapeutic research could use to develop marketable technologies and drugs to treat unconquered diseases and other applications. The HGP was designed and undertaken to culminate with the goal of ushering the beginning of an era of novel drug discovery and to expedite the development of existing therapeutic endeavors. This report offers a professional evaluation of some primary RNA/DNA markets which stand poised to capitalize on that scientific achievement.

RNA/DNA technology has the ability to confirm the past, match up the present and predict the future through utilization of therapeutics, forensics and predictive technology administered in personalized and precise individual, group or mass applications. RNAi seeks to become the tool of choice for characterization of gene function and validation of drug targets in clinical trial advancement. Down-regulation of single or multiple genes in a pathway is a very powerful tool for understanding biological function and mapping regulatory, signaling and metabolic pathways. Pharmacogenomics look promising as a technology that presents an opportunity to create an entirely new class of drugs that are administered on a personal level with laser-like precision and reduced side effects. Forensics has shown its value in a number of security-related applications that address the issues that reflect the concerns of contemporary society.

This intention of this report is to recount, depict and assess these markets and presume their capabilities, attributable to the founding and developmental factors that distinguish each of them.

This publication assumes to report the market through the employment and application of the resources, knowledge and experience of Thomson BioWorld and its ancillary businesses, in order to impart information, identify trends and evaluate the designated markets based on historical factors, market data, authority interviews and probability forecasts.

This report addresses the major players, therapeutic applications, R&D status, competitive environment, industry events and market prognoses that comprise and distinguish the subject matter, while presuming an opportunity for the reader to assimilate the acquired business intelligence for personal use without legal liability toward the authors of this report or Thomson BioWorld.

Presumptions, forecasts, conclusions, observations, calculations and statistics are a compilation of the following sources: rudimentary and secondary investigation conducted by the BioWorld Publishing Group staff; attributable third-party research and published data; discussions, interviews or conference participation with industry executives and thought leaders.

Projections and analyses of the BioWorld editors are not meant to be used as a guarantee of market performance, rather they constitute a speculative and educated appraisal, which can be read for evaluation and utilized for purposes of informed consideration and contemplation.