Public Release: 

Functional genomics enables easy target identification and drives drug discovery

Technical Insights: Drugs in the Pipeline - An R&D Analysis

Technical Insights

Palo Alto, Calif. - May 10, 2004 - Functional genomics and proteomics have been quite successful in identifying functions of potential therapeutic targets such as encoded proteins. In fact, the possibilities of identifying more than 10,000 novel target antigens in the human genome may accelerate the discovery of new drugs and therapeutic molecules.

"As opposed to conventional sequence homology, functional genomics adds structure-based predictions to locate gene sequences with assigned and confirmed functions," explains Frost & Sullivan Industry Analyst Rajaram Sankaran. "It simultaneously sifts through well established targets to detect critical therapeutic targets."

Such structural information results in enriched annotations that improve the identification process and also provide a clear understanding of interactions between specific molecules and target proteins.

Additionally, functional genomics opens up the possibilities of genetically demarcating patients and predicting individual responses to drugs. This permits customized medications and dosages that improve treatment safety and efficacy in areas such as neuropsychiatry, cardiovascular medicine, endocrinology, and oncology.

The success of functional genomics is magnified when used in conjunction with combinatorial chemistry (combichem) where a molecular compound is introduced into a compound library to chemically interconvert. It follows a target driven approach wherein, molecular building blocks are designed to react together selectively and covalently.

With an increasing number of new molecular entities (NMEs) entering clinical trials - by 2008, a 65 percent increase in NMEs entering the market is expected - innovative techniques for 'fast track' drug development and early screening of compounds are being devised.

One such technique gaining popularity is high-throughput screening (HTS) that detects and provides optimization guidelines for lead compounds and validates drug targets. HTS development provides numerous advantages such as lower attrition rates, reduced time-to-market, and accelerated drug screening process.

However, almost 30 percent of NMEs fail to clear Phase I clinical testing, despite speedy and extensive pre-clinical screening. This may be attributed to pre-clinical animal and 'ex vivo' models, which provide inaccurate human pharmacokinetic (PK) and absorption, distribution, metabolism, and excretion (ADME) data.

Human targets, on the other hand, offer a holistic view of expected drug performance and improve efficacy and safety of the final drug. Following this trend, accelerator mass spectrometry uses microdosing to provide faster human bioavailability data during pre-clinical screening.

In microdosing, human drug dosages, which are 100 times below the required level, are sufficient to screen numerous compounds and yield early PK and ADME data. This ensures that optimal drug candidates qualify for Phase I clinical development.

Human drug absorption is another approach to obtain accurate PK and ADME data. In this, engineered capsules are used for non-invasive, targeted drug delivery during early clinical development to provide direction for the development of selected NMEs.

"Pharmacokinetic data generated during these studies could provide a detailed understanding of the complex absorption process at distinct intestinal sites," says Sankaran. "This may be critical for quicker decision-making process and appropriate selection of drug development strategies."


Drugs in the Pipeline - An R&D Analysis is part of the Healthcare Vertical Subscription Service, and provides a comprehensive analysis of drugs in the research and development pipelines for prominent ailments in the cardiovascular, central nervous system, and cancer therapeutic areas. It analyzes key technical challenges and drivers influencing growth and provides in-depth examination of cutting-edge developments. The research service also discusses enabling technologies and approaches such as nanotechnology for the improvement of pharmacokinetics, microdosing and drug absorption studies that could be useful in accelerating early phase drug discovery efforts. Executive summaries and interviews are available to the press.

If you are interested in an analysis overview which provide manufacturers, end-users and other industry participants an overview, summary, challenges and latest coverage of Drugs in the Pipeline - An R&D Analysis - then send an email to Julia Paulson - North American Corporate Communications at with the following information: Full name, Company Name, Title, Contact Tel Number, Contact Fax Number, Email. Upon receipt of the above information, an overview will be emailed to you.

Technical Insights is an international technology analysis business that produces a variety of technical news alerts, newsletters, and research services.

Frost & Sullivan, an international growth consultancy, has been supporting clients' expansion for more than four decades. Our market expertise covers a broad spectrum of industries, while our portfolio of advisory competencies includes custom strategic consulting, market intelligence, and management training. Our mission is to forge partnerships with our clients' management teams to deliver market insights and to create value and drive growth through innovative approaches. Frost & Sullivan's network of consultants, industry experts, corporate trainers, and support staff spans the globe with offices in every major country.

Drugs in the Pipeline - An R&D Analysis

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The following is the list of key industry participants: Worldwide; Aastrom Biosciences, Inc.; Abbott Laboratories; Abgenix, Inc.; ACADIA Pharmaceuticals; Acorda Therapeutics Inc.; ACTIVE PASS PHARMACEUTICALS; ADERIS Pharmaceuticals, Inc.; Aesgen, Inc.; Æterna Laboratories Inc.; Agensys, Inc.; Akzo Nobel nv; Alfacell Corp.; Allon Therapeutics, Inc.; Allos Therapeutics, Inc.; AltaRex; Amarin Pharmaceuticals, Inc.; American Cancer Society, Inc.; Amgen Inc.; Amrad Corp., Ltd.; Anadys Pharmaceuticals, Inc.; AnorMED Inc.; Antigenics Inc.; Aphton; ApoImmune, Inc.; Applied Molecular Evolution, Inc.; Archemix Corp.; Arena Pharmaceuticals, Inc.; ARIAD Pharmaceuticals, Inc.; ArQule, Inc.; Astra Zeneca; AtheroGenics, Inc.; AVANIR PHARMACEUTICALS; AVANT Immunotherapeutics, Inc.; Aventis; AVI BioPharma; Avigen; Axonyx USA; Bayer AG; BioCryst Pharmaceuticals, Inc.; Biogen Idec; BioMedicines, Inc.; BioSyntech Inc.; Boehringer Ingelheim GmbH; Boston Life Sciences; Bristol-Myers Squibb Co.; CancerVax; Case Western Reserve University; Celgene Corp.; Centocor, Inc.; Chiron Corp.; ConjuChem; Corixa Corp.; Cortex Pharmaceuticals, Inc.; CoTherix, Inc.; CuraGen Corp.; Curis Inc.; CV Therapeutics, Inc.; CYTOGEN Corp.; CytRx Corp.; Daiichi Pharmaceutical Corp.; Dendreon LLC; Dynavax Technologies Corp.; Elan Corporation, plc; Eli Lilly & Co.; Enanta Pharmaceuticals, Inc.; EntreMed, Inc.; EpiGenX Pharmaceuticals, Inc.; Epimmune, Inc.; Fujisawa Healthcare, Inc.; Galenica Pharmaceuticals, Inc.; Genaera Corp.; Genentech, Inc.; GlaxoSmithKline; Institute of Cancer Research; Introgen Therapeutics, Inc.; Johns Hopkins University; Merck & Co., Inc.; Merck KgaA; Merck Sdn Bhd; MGI PHARMA, INC.; Microbiotix, Inc.; MitoKor; National Heart, Lung, and Blood Institute (NHLBI); National Jewish Medical and Research Center; NIH Neurological Institute; NPS Pharmaceuticals; Pfizer, Inc.; PLIVA d.d; Quark Biotech, Inc.; Sanofi-Synthelabo Inc.; Solubest; Texas A&M University; The Salk Institute for Biological Studies; The University of Edinburgh; University Health Network; University of Pittsburgh; University of Rochester Medical Center; Xenova Group plc.

Keywords in this release: functional genomics, encoded proteins, therapeutic targets, sequence homology, neuropsychiatry, cardiovascular medicine, endocrinology, oncology, combinatorial chemistry, combichem, compound library, new molecular entities, NME, high-throughput screening, HTS, accelerator mass spectrometry, microdosing, animal/ex vivo models, human drug absorption, HDA, Enterion capsule, pharmacokinetic data, PK data, absorption, distribution, metabolism and excretion data, ADME data

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