Innovations In Epigenetics: Advances in Technologies, Diagnostics Therapeutics

Date: November 22, 2009
Pages: 165
US$ 3,835.00
Publisher: Business Insights
Report type: Strategic Report
Delivery: E-mail Delivery (PDF)
ID: I356ED2039FEN

Download PDF Leaflet

Innovations In Epigenetics: Advances in Technologies, Diagnostics  Therapeutics
The study of epigenetics - changes in gene expression that occur without a change in a cell’s DNA code – is becoming increasingly important as scientists explore how internal and external factors trigger cellular dysfunction and influence disease progression. Three main mechanisms - DNA methylation, histone modifications and changes in non-coding RNA - have been elucidated in epigenetics. Each in its own way may provide the industry with a greater understanding into the underlying mechanism of diseases, act as a potential source for biomarkers of disease and provide new targets for therapeutic intervention.

Besides everyday DNA analytical tools, epigenetic researchers have needed to adopt more complex technologies such as chromatin immunoprecipitation (ChIP) and DNA methylation methodologies as well as develop analytical tools to decipher the vast amount of epigenetic information. Advances in these technologies should enable epigenetic research to reduce cost and increase sample throughput making it more commercially attractive in the industry to discover biomarkers and validate novel epigenetic targets for diagnostic and pharmacological development.

Epigenetic medicine has arrived. The market is worth over $560 million derived from the sale of three anticancer products which target two epigenetic pathways - DNA methyltransferase (DNMT) and histone deacteylase (HDAC) – and around thirty epigenetic drugs are under development from more than a dozen biotechnology companies. These drugs focus mainly on the treatment of cancer, neurodegenerative and infectious diseases although research is underway to explore the role for epigenetics in cardiovascular, metabolic, ocular and other diseases. Whilst this market is still in its infancy KOLs believe it is on the cusp of a revolution, one which could change how patients are diagnosed and treated in the future.

Key Features of this report
  • Identify leading companies at the forefront of epigenetic research who will drive the market to develop a new generation of epigenetic therapies and diagnostics.
  • Provide case studies of these leading companies developing epigenetic therapies to treat cancer, neurodegenerative and infectious diseases.
  • Analyze the key trends and issues confronting the development of epigenetic therapies.
  • Review the alliances, mergers, acquisitions and business strategies adopted by biotechs and big pharma to capitalize on the latest epigenetic developments.

Scope of this report
  • Identify the companies leading the field in epigenetic research across a range of therapeutic areas
  • Review their business strategies and the development of their epigenetic programs
  • Learn more about the latest developments in epigenetic research and how non-coding RNAs may open up another Pandora's box for epigenetic research.
  • Identify innovative pharmaceutical companies and discover the strategies they are adopting to tap into the epigenetic potential both as biomarkers of disease and targets for therapeutic intervention

Key Market Issues
  • Declining industry productivity: It is a well documented fact that productivity in the pharma industry has declined during the past 15 years. The identification of new biomarkers of disease could aide the R&D process and support the preclinical and clinical development of small molecule therapeutics.
  • Innovative new drug classes: The identification of underlying mechanisms of diseases could identify new target for small molecule therapeutic intervention that may be used alone or in combination with traditional therapies to extend their usage.

Key findings from this report
  • New epigenetic therapies have entered the market. The first medicines were approved in 2006 for the treatment of cancer and a host of new small molecule therapies are now under development which are more selective and target different classes of chromatin modifying enzymes enabling a more targeted approach to treatment.
  • The ability of innovative companies to generate intellectual property is a key driver for the sector as the market become more competitive in this new area of science. The unraveling of the human epigenome will be the first step in identifying new epigenetic markers which may have potential as biomarkers an area of considerable interest for the pharmaceutical industry at the present time.

Key questions answered
  • Which companies are the market leaders in the epigenetics industry, identified through therapeutic areas (cancer, infectious diseases, neurodegenerative diseases, metabolic, cardiovascular, ophthalmic, other diseases) and recent alliances with the pharmaceutical companies?
  • What are the key products of these companies and how do they exemplify the future direction of the epigenetic therapies?
  • How is the pharmaceutical industry approaching the need for new targeted epigenetic therapies and what strategies are they taking?
Innovations in Epigenetics
Executive summary
Application in R&D & technological advances
Exploring new therapeutic targets
Epigenetic market, leading companies & pharmaceutical strategies
The future of epigenetics


What is epigenetics?
Epigenetics and phenotypes
Epigenetics a new biological paradigm
Epigenetics, genetics, genomics & pharmacogenomics
Molecular mechanisms associated with epigenetics
DNA methylation
Histone modifications
Nucleosome remodelling
Non-coding RNA
X chromosome inactivation
Gene imprinting
RNA interference
Epigenetic basis of disease
Epigenetic syndromes
Angelmann syndrome
Beckwith-Wiedemann syndrome
Prader-Willi syndrome
Why is epigenetics of interest to the pharmaceutical industry?
Biomarker discovery & diagnostics
Novel small molecule epigenetic therapeutics
Novel antisense therapeutics
Stem cells & regenerative medicines


The application of epigenetics in drug R&D
DNA methylation biomarkers
Histone modification biomarkers
Technological advances in epigenetics
Chromatin immunoprecipitation
DNA methylation technologies
Non-coding RNA technologies
Bioinformatic modeling


Potential new therapeutic targets
Epigenetics in cancer
DNA methyltransferase & DNMT inhibitors
Case study: Vidaza (Celgene Corporation) & Dacogen (Eisai /J&J)
Case study: Zebularine a research tool
Histone acetylase (HAT) and histone deacetylase (HDAC)
Case study: Zolinza (Merck & Co.)
Case study: MGCD0103 (MethylGene/Taiho Pharmaceutical)
Case study: PCI-24781 (Pharmacyclics/Servier)
Histone demethylases and histone methyltranserases
Case study: PG11144 & PG11047 (Progen Pharmaceuticals)
Potential epigenetic based diagnostics
Diagnostic DNA methylation cancer biomarkers
Case study: Epi proColon (Epigenomics AG)
The next generation of epigenetic cancer biomarkers
Epigenetics in neurological disorders
Case study: EVP-0334 (EnVivo Pharmaceuticals)
Epigenetics in infectious diseases
Case study: MGCD290 (MethylGene)
Case study: siRNA targeting HIV-1 (Kevin Morris, Scripps, La Jolla)
Epigenetics in metabolic disorders
Epigenetics in cardiovascular disease
Epigenetics in ocular disorders
Case study: Kinase inhibitors (Otsuka Pharmaceutical/MethylGene)
Case study: Kinase inhibitors/S-adenosyl methionine (SAM) (Epizyme).


Epigenetic market
Epigenetic therapeutic revenues: Now and the future
Leading epigenetic companies
4SC AG, Planegg-Martinsried, Germany
Celgene Corporation, Summit, New Jersey
Curis Inc, Cambridge, MA
Chroma Therapeutics Ltd, Oxon, UK
Constellation Pharmaceuticals, Cambridge, MA
EnVivo Pharmaceuticals, Watertown, MA
EpiTherapeutics Aps, Copenhagen, Denmark
Epizyme, Cambridge, MA
Gloucester Pharmaceuticals, Cambridge, MA
MethylGene, Inc. Montreal, Qu?bec
Pharmacyclics, Sunnyvale, CA
Progen Pharmaceuticals, Brisbane, Australia
Repligen Corporation, Waltham, MA
SuperGen, Dublin, CA
Syndax Pharmaceuticals, Waltham, MA
TopoTarget, Copenhagen, Denmark
Summary of epigenetic-based companies
Recent alliances, mergers & acquisitions in epigenetics
Pharmaceutical strategies in epigenetics
GlaxoSmithKline, Middlesex, UK
Novartis, Basel, Switzerland
Merck & Co., Whitehouse, NJ
Eisai Corporation of North America, NJ
Takeda, Osaka, Japan
Overall conclusions


The future of epigenetics
Epigenetic consortia; unraveling the human epigenome
NIH’s Roadmap Epigenomics Program initiative
European Epigenome Network of Excellence
Human Epigenome Consortium
KOLs in epigenetics
John Mattick, Institute for Molecular Bioscience, University of Queensland, Australia
Kevin Morris, Scripps Institue, La Jolla, CA
Monika Lachner, Max-Planck Institute of Immunobiology, Department of Epigenetics, Freiburg, Germany
Johnathan Whetstine, Department of Medicine, Massachusetts General Hospital Cancer Center
Peter Fraser, Head, Senior Fellow of the Medical Research Council, UK, The Babraham Institute, Cambridge
Summary of KOLs commentary
Fundamental research
Technological demands
Financial constraints
Intellectual property
Biomarker discovery & diagnostics
Therapeutic intervention
Regenerative medicines




Figure 1.1: Timeline of epigenetics study
Figure 1.2: Schematic of chromatin structure
Figure 1.3: Schematic of epigenetic mechanisms associated with health and disease
Figure 1.4: Epigenetic applications by the pharma industry
Figure 2.5: Epigenetics in the drug discovery & development process
Figure 2.6: Schematic of key technologies used in epigenetics
Figure 2.7: Schematic of ChIP technology
Figure 2.8: Epigenomics AG methylation specific PCR (MSP) methods
Figure 3.9: DNA methylation targets for epigenetic cancer therapies
Figure 3.10: Mechanism of action of HAT and HDAC
Figure 3.11: Epi proColon high throughput colorectal epigenetic test
Figure 3.12: Competitive pricing of Epi proColon
Figure 3.13: EnVivo’s HDAC project screening cascade
Figure 3.14: Agouti mice and epigenetic manipulation
Figure 3.15: Epizyme’s rational design of small molecule HMT inhibitors
Figure 4.16: Epigenetic therapies: product class and developmental phase
Figure 4.17: Epigenetic therapies: therapy target and developmental phase
Figure 4.18: CUDU-101 structure & design: combining multiple pharmacophores


Table 2.1: DNA methylation PCR methods
Table 2.2: Techniques to analyze DNA methylation
Table 3.3: Examples of DNMT inhibitors: potential anticancer agents targeting epigenetic pathways
Table 3.4: Examples of DNMT inhibitor research tools
Table 3.5: HDAC inhibitors: potential anticancer agents targeting epigenetic pathways
Table 3.6: Examples of HDAC inhibitor research tools
Table 3.7: Histone modification and their effect on gene expression
Table 3.8: Safety profiles of PCI-24781 rivals
Table 3.9: DNA methylation sites associated with cancers
Table 3.10: Validated and hypothetic miRNA targets for diabetes & obesity
Table 4.11: Epigenetic therapeutic revenues ($m), 2009-2020
Table 4.12: Preliminary clinical data for Pharmacyclics PCI-24781
Table 4.13: Recent alliances, acquisitions and mergers in the epigenetic arena
Table 5.14: NIH Epigenetic Roadmap – funded epigenetic projects
Skip to top

Ask Your Question

Innovations In Epigenetics: Advances in Technologies, Diagnostics Therapeutics
Company name*:
Contact person*:
Request invoice
Your enquiry:
Please click on a Check Box below to confirm you are not a robot: