pharmaceuticals and biotechnology hirzun mohd yusof, phd manager sime darby technology centre

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Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

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Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre. Biotechnology Definition. OLD: Recombinant genetic engineering...using biological processes to develop products - PowerPoint PPT Presentation

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Page 1: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Pharmaceuticals and Biotechnology

Hirzun Mohd Yusof, PhDManager

Sime Darby Technology Centre

Page 2: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Biotechnology Definition

• OLD: Recombinant genetic engineering...using biological processes to develop products

• NEW: Use of biological processes to solve problems or make useful products. Life sciences…biology/chemistry technology affecting discovery and development of products for:

–Human healthcare (therapeutics, diagnostics, drug delivery, cell and gene therapy…even moving toward some devices and drug/device combinations)

–Wellness…not just sickness–Agriculture (food, feed, fibers, transgenics)–Environment (bio-remediation)–Bio-based industrial processes and efficiency–Bio-based energy–Supply (reagents, biologicals)

• All driven by a new set of enabling technology (genomics, combinatorial chemistry, SNPs, proteomics, etc.)

Source: Burrill and Company

Page 3: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Biotech industry?

Biotechnology

Genes and proteins

Human Agricultural

•TrangenicPlants •Animals

•Drug development•Gene surgery•Genetic testing

Microbes

•Alcohol•Mining•Waste management

Chemicals

•Extracts•Oils•Pharma-intermediaries•Nutraceuticals

“Spare parts”

•Tissue regeneration•Growing organs

Page 4: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Development is evolutionary…

1953: Watson and Crick DNA Structure1970: First enzyme discovered to cut DNA molecules at a

specific site1971: First complete synthesis of a gene1972: First time DNA fragments linked1976: First NIH research guidelines1980: Oil-eating microbes patented by Exxon1982: First recombinant DNA vaccine for livestock1983: First whole plant grown from biotechnology1986: First genetically engineered vaccine for humans: Hep

BFirst anticancer drug through biotech: interferon

1990: First food product from biotech approved: modified yeast

Page 5: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Development is evolutionary…

1994: First FDA approval for food product

1997: First weed & insect resistant crops developed

First cloned animal: Hello Dolly!

2000: First complete plant genome mapped

108.9 million acres of biotech crops grown in 13 countries

2004: First genetically modified pet: the GloFish

2004: About 25% of all prescription products on market since 2000 are from biotechnology

Page 6: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

The Human Genome & Biotechnology

• “A milestone in biology unlike any other.”• “The HGP could fundamentally restructure the nation’s

$1.3 trillion healthcare industry in the next 20-30 years: PwC/Russ Coile

• June 26, 2000; 5-years ahead of schedule• A short 50 years after the discovery of DNA by Watson

and Crick in 1953• 30,000-40,000 genes not the 100-120,000 thought earlier• Five times as many as in baker’s yeast• About twice as many as that needed to grow a worm or

fly!• Bananas share about ½ our genome while mice share

90%!• BUT, each single human gene can make 10 proteins vs. a

worm or fly’s genes making just one or two.

Page 7: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

PM’s Support of Biotechnology

“…. Biotechnology has great potential in Malaysia and it could be a catalyst for new growth areas in the country’s economy as well as a

source of new wealth and income for the people, Biotech is useful in many areas – agriculture, livestock farming, herbal industry and traditional and

modern medicine.”

YAB Prime Minister of Malaysia Dato’ Seri Abdullah Badawi

at the luncheon meeting with top CEO’s of US Biotechnology firms

during BIO 2004 Convention and Exhibition,

9 June 2004 in San Francisco, California

Page 8: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Malaysia Biotechnology Focus Area

HEALTHCARE INDUSTRIAL

AGROBIOTECHNOLOGY

Diagnostics

Vaccines

Drug Discovery

Wellness

Bio-generics

Alternative protein production

Enzyme & industrial chemical

Bio-energy

Plant

Natural Products

Marine

Animal

Source: CEO/President MIGHT’s presentation in Sarawak BioSymposium, March 2005

Page 9: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Nine Policy Thrusts

Thrust 1 : Agriculture biotechnology developmentTransform and enhance value creation of the agriculture sector through biotechnology.

Thrust 2 : Healthcare biotechnology developmentCapitalise on the strengths of biodiversity to commercialise discoveries in natural products as well as position Malaysia in bio-generics market.

Thrust 3 : Industrial biotechnology developmentEnsure growth oppurtunities in the application of advanced bio-processing and bio-manufacturing technologies.

Thrust 4 : R&D and technology acquisitionEstablish Centres of Excellence, in existing or new institutions, to bring together multidisciplinary research teams in co-ordinated research and commercialisation initiatives. Accelerate technology development via strategic acquisitions.

Thrust 5 : Human capital development Build the nation's biotech human resource capability in line with market needs through special schemes, programmes and training.

Page 10: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Nine Policy Thrusts…continue

Thrust 6 : Financial infrastucture developmentApply competitive "lab to market" funding and incentives to promote committed participation by academia, the private sector as well as government-linked companies. Implement sufficient exit mechanism for investments in biotech.

Thrust 7 : Legislative and regulatory framework developmentCreate an enabling environment through continuos reviews of the country's regulatory framework and procedures in line with global standards and best practices. Develop a strong intellectual property protection regime to support R&D and commercialisation efforts.

Thrust 8 : Strategic positioningEstablish a global marketing strategy to build brand recognition for Malaysian biotech and benchmark progress. Establish Malaysia as centre for Contract Research Organisations and Contract Manufacturing Organisations.

Thrust 9 : Government commitmentEstablish a dedicated and professional implementation agency overseeing the development of Malaysia's biotech industry, under the aegis of the Prime Minister and relevant government ministries.

Malaysia Biotechnology Corporation

Page 11: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

BIOPHARMACEUTICAL

Page 12: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Biological based therapeutics

• Biologically derived material that possess the ability to heal or to cure human illness

• Traditionally extracted from human cadaver or other sources

• Currently replaced by biotechnological methods through revolution in genetic engineering

Page 13: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Protein drugs have emerged from the shadows to become, in the last 20 years, significant products in the arsenal against illnesses.

While conventionally made proteins are considerable, proteins resulting from the biotechnology revolution drive future

growth of the protein drug industry.

The Biotech WaveThe Biotech Wave

Page 14: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

• In the last 5 years, 168 biopharmaceutical products obtained marketing approval in major markets. In the next 5 years, It is anticipated that an additional 82 to 137 new biopharmaceutical products will be marketed

• The aggregate sales of biopharmaceuticals amounted to US$23 billion in 2002 and US$38 billion in 2003. It is forecasted to reach US$100 billion by 2010

• The annual growth rate is established at 15% and some clusters such as monoclonal antibodies have an even higher growth rate (25%)

• Therapeutic proteins (e.g. growth factors, hormones, antibodies) represent the major part of biopharmaceutical sales

Market PotentialGrowth of the therapeutic protein market

Source: ADL Survey, 2004; market forecasts from Datamonitor, IMS, DSM and PhRMA , 2002 -2004

Page 15: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

• Therapeutic proteins are currently manufactured via one or two methods:

– Mammalian cell culture (CHO cells): >60% of marketed products

– Bacteria culture: >30% of marketed products

• The industry recognizes that there will be a shortfall risk in protein manufacturing capacities over the next 5 years:

– Over 350 biotech products are currently in late-stage development for 200 serious diseases

– A significant number of major biopharmaceutical products will be coming off patent

• It is estimated that up to 4 times the current protein production capacity will be needed to satisfy demand by 2010

– Manufacturing facilities using traditional technologies require significant upfront investments and long construction and validation times

Market potentialDemand for protein production capacity

Source: ADL Survey, 2004; market forecasts from Datamonitor, IMS, BSM and PhRMA , 2002 -2004

Page 16: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

• Demand for medium-sized production batches (up to 10 kilograms per year) will be among the fastest growing:

There is a great opportunity over the next 5 years for new entrants with competitive protein production technologies to

capture significant market share

Market potentialOpportunities for new entrants

Dr Ulrich Steiner

Page 17: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Worldwide Contract Manufacturing Market

US$ MM

Protein Drugs (Final products) 38,000

Annual growth 15%

Outsourcing Ratio 15%

Contract Manufacturing 900

2004 2014 (Forecasts)

US$ MM Protein Drugs (Final products) 175,000

Outsourcing Ratio 30%

Contract Manufacturing 10,500

Market potentialContract manufacturing market

Contract ManufacturingGeographical Breakdown

(2014 forecasts):

Europe 30%North America 40%Asia 30% (>US$ 3,100 MM)

Contract manufacturing of biopharmaceuticals will be one of the fastest growing industrial

sectors in the next 10 years:

– Significant market size and rapid growth

– Modest start-up investments

– Relatively low risk (cf. drug discovery)

Page 18: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Generic Biological Drugs

• Biological products are approaching the end of their market exclusivity with over $10 billion in 2000 sales coming off patent over the next five years

• Generic biologic products represent a significant opportunity and anticipate progress on this cutting edge of technology

-Coan & Ellis, Generic Biologics: The next Frontier, ABN Amro Report

Page 19: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

BRAND NAME

GENERIC NAME SOURCES 2001 GLOBAL SALES, US$ MILLION

U.S. PATENT EXPIRATION

Epogen or Procrit

Epoetin alfa Amgen, Johnson & Johnson, and Sankyo

$5,772 2004

Novolin Human insulin Novo Nordisk 1,829 2005

Neupogen Filgrastim Amgen and Roche 1,533 2006

Humulin Human insulin Eli Lilly 1,061 2001

Avonex Interferon beta-1a Biogen 972 2003

Intron A Interferon alpha-2b Schering-Plough 700 2002

Cerezyme or

Ceredase

Alglucerase Genzyme 570 2001

Humatrope Somatropin Eli Lilly 311 2003

Activase Alteplase Genentech, Boehringer Ingelheim,

Mitsubishi, and Kyowa Hakko Kogyo

276 2005

Nutropin Somatropin Genentech 250 2003

Protropin Somatrem Genentech 250 2005

TOTAL $13,524

Market potential

Generic biopharmaceuticals market

Page 20: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Generic Biotechnology Feasibility

Numerous arguments why generic biotechnology products would not be feasible.

interrelated concerns over safety and immunogenicity

science’s ability to manufacture and measure such products – process dependant

Since those early reservations, there has been a combination of technological advances, (e.g., in vitro/biochemical and analytical assays).

Examples ofsome new analytical methods that are assisting in the standardization ofbiological products are:• MALDI-TOF Spectroscopy• Reflectometric Interference Spectroscopy• Capillary electrochromatography• Signal Transduction Fingerprinting• Bioinformatics, including Microarray Technology and pharmacogenomics.

Page 21: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Company Marketed Products Products in development

Bharat Biotech

(Hyderabad, India)

Hepatitis B Streptokinase, VEGF

Dr Reddy’s Labs (Hyderabad, India)

G-CSF EPO, IFN-Beta, hGH, tPA, IFN-gamma

Dragon (Vancouver) EPO TPO, G-CSF, insulin, hepatits B

GeneMedix

(New Market, UK)

GM-CSF IFN-alpha-2b, EPO, insulin, IFN-gamma, IL-2

LG Chem

(Seoul, S. Korea)

EPO, hGH, IFN-alpha-2a, IFN –gamma, GM-CSF, hepatitis B

Rhein Biotech (now acquired by Berna)

Hepatits B, IFN-alpha, IL-2 EPO, G-CSF,

Shantha (Hyderabad, India)

Hepatitis B IFN-alpha, insulin, GM-CSF, G-CSF, streptokinase, tPA, EPO, hGH

Companies involve in generic biologics

Page 22: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Lyophilization

Bulk Filtration

Seed Fermentation

Drop Tank

Main Fermentation

Feed Media 2

Feed Media 1

Buffer ExchangeDiafiltration

3rdChromatography

Buffer ExchangeDiafiltration

UFConcentration

2ndChromatography

Buffer ExchangeDiafiltration (UF)

Buffer Exch.1stChromatography

Liquid VialProduct

LyophilizedProduct

Fill/Finish

MicrofiltrationCentrifugation

Transfer to Receive Tank

UFConcentration

Refolding

IB Dissolution

IB Wash

Centrifugation

IEX

Tank Fusion

UFClarif.

ColumnCleavage

DilutionDiafilt.

UF

Holding Tank

Homogenization

Dilution

Cell Paste

Typical Process Flow of Biopharmaceutical Production

Page 23: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Micro-organisms

(bacteria & yeasts systems) No No < 100 kg

Transgenic Plants Systems

(tobacco, corn, alfalfa, potatoes) Yes No/Yes > 100 kg

Cell Culture

(CHO cells) Yes Yes < 100 kg

Transgenic Animal Systems

(cattle, goat, rabbit milk,

chicken eggs, pig semen)

Yes Yes > 100 kg

Compatibility with Complex Proteins

Glycosylation Scale

COMPETITIVE ADVANTAGES

SUPERIORITY OVER ALTERNATIVE SYSTEMS

Page 24: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Companies active in Animal Transgenesis:

• Mammary gland – Milk

• GTC Biotherapeutics (recombinant form of human antithrombin-

Market Authorization Application EMEA) , BioProtein, Pharming

(recombinant human C1 inhibitor – Phase III) , Nexia

Biotechnologies

• Bone marrow –blood

• Hematech – Kirin (human polyclonal antibody)

• Chicken ovary – eggs

• Avigenics (lead product schedule for Phase II in 2005),

Tranxenogen, Vivalis + 10 others

• Seminal vesicle – semen

• TGN Biotech

Challenges for the future….drug from transgenic animal

Page 25: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

DRUG DISCOVERY

Page 26: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Drug Discovery

Diseases -defects in cellular communication & control

Mediated by post-translational modification e.g.phosphorylation, acetylation

Idea for targets

Drug Discovery Process

Viral infections

Osteoporosis

Neurological

Diabetes

Obesity

Cancer

Cardiovascular

Respiratory

Amplification &expansion - cascades of phosphorylation events: signal transduction pathways. Aberrations linked to disease

Page 27: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Company-financed R&D by Product Class, Estimated 2000 (US only)

Product Class 2000 Spend in $ billions

acting on central nervous system &sense organs

$6.0

affecting neoplasms, endocrinesystem & metabolic diseases

$5.0

acting on cardiovascular system $4.1

acting on infective & parasiticdiseases

$3.6

biologicals $2.4

acting on respiratory system $1.5

acting on digestive or genito-urinarysystem

$0.9

acting on the skin $0.2

diagnostic agents $0.2

vitamins and nutrients $0.1

other human use $1.7

Parexel’s Pharmaceutical Sourcebook2000

$25.7

Page 28: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Target Types Therapeutic area Example Targets

Kinase Cancer; cardiovascular;inflammation; CNS; anti-infectives etc.

MAPK eg Erk1/Erk2, JNK and p38 -mitogen induced cell cycle progressionthrough G1 phase, regulation ofembryonic development; cellmovement, apoptosis, cell & neuronaldifferentiation.

Phosphatase Cancer; cardiovascular;CNS; diabetes etc

Polymerase/Nuclease/Helicase

Cancer; anti-viral; anti-bacterial; immunology etc.

Protease Cardiovascualr; cancer;CNS; inflammation; anti-viral etc

Activation by cleavage of peptides andproteins involved in regulation of statusof cell, e.g metalloproteinase, tumornecrosis factor; caspases andapoptosis

Ion Channel CNS; inflammation;asthma; cancer;cardiovascular; depression

GPCR CNS; Cardiovascular;cancer; inflammation;asthma; metabolism

Activated G protein interacts withadenylate cyclase causing cAMPstimulation,cAMP binds to for e.g. PKAsubunits, these subunits phosphorylatetargets in cytoplasm & nucleus such asCREB to increase activation oftranscription

Nucleic acid/ProteinBinding

Cancer; hormone therapy;

Transporters/Nuclearreceptors/Protein binding

Cancer; antimicrobial;cardiovascular

Drug Targets

Page 29: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Human genome 22,300 genes

Addressable by protein therapeutics ~10,000 genes

Target for small molecular drugs ~10,000 genes

Disease modifying ~ 4,500 genes

Additional targets for antisense and siRNA therapies ~2,100 genes

Druggable ~3,000 genes

Additional targets for protein therapeutics ~1,800 genes

Target universe –post genomics

Source: Drug Discovery Today, Aug 2005

Potential target within human genome:addressable by small molecules (enzymes, GPCR, channels and NHRs)addressable by protein therapeutics (membrane proteins and soluble factors)

Page 30: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

What is screening?

A Drug Discovery technique where a number of chemical substances are tested for their ability to interact with specific proteins (targets) that are believed to be important in disease states

Source of compounds:

Chemical synthesisCombinatorial chemistryNatural compound (plant,microbial, marine

organisms)

Page 31: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

The phases of discovery & development of a new drug

Identify disease

area

Target Selection

Target Validation

Assay Development

Hit Identification

LeadOptimization

ProfilingScreens

ADME-TOX

ClinicalTrials

Market

Over the past ten years, expectation has been raised with high-throughput screening…results of high-throughput screening has been dissapointing….

…Steve Carney, Editor Drug Discovery Today, Aug 2005

Page 32: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Clinical trial to drug

Potential lead compound

Characterization and structure elucidation of active compound

Process flow of discovery of new compound from nature

Microbial culture collection

Bioassay screening for bioactive compound

Plant extracts

Page 33: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

List of companies involved in natural compound libraries

Company Location Library Type

 Albany Molecular  USNatural products libraries include over 100,000 samples of extracts derived from nature.

 Analyticon Discovery   Germany MEGAbolite® libraries consist of pure structurally elucidated natural compounds.

 Cerylid  Australia Natural products library of over 600,000 extracts from more than 60,000 biotic samples. available through partnerships only. 

 Interbioscreen  RussiaOver 25 000 unique diverse and rare natural & related compounds. 

  Microsource / MSDI  US30,000 extracts of more than 12,000 specimens from the Amazon. A 720 compound/ 9 plate  collection of pure natural products and derivatives

Moscow MedChem Labs  Russia Library of natural compound is now about 200 compounds 

 Sequoia Sciences  USNovel libraries of compounds isolated from plants for drug discovery 

 SPECS and Biospecs  Netherlands800+ isolated natural products or derivatives from plants, fungi, bacteria, sea organisms, etc. (purity >80%).

TimTec US

True natural product (240-compound) and natural product-derived(1600-compound) libraries;2500 plants available from stock for extraction, 9000 plants available for collection

Page 34: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Pharmacogenomics…in drug discovery

The use of genomics approaches to elucidate drug response:

• Via DNA: genetic approach (pharmacogenetics).

• Via RNA: expression profiling.• Via Protein: Proteomics.

Page 35: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Pharmacogenomics Market

World wide market size and growth

2003 : USD 670 million2008E: USD 1,655 milliom2002-2007: 20%

Three major areas:

SNP DiscoverySNP GenotypingDiagnostics

In 2003:

Page 36: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Human Genome contains many variations or polymorphisms

No two human genomes are identical

Page 37: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

SNP (Single Nucleotide Polymorphism)

Every individual has ~0.1% of the genome that is different. In average, every 1Kb has a SNP can be used as a marker on the genome.

Page 38: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Applications of the SNP Analysis

• Disease gene hunting

• Prognostics / diagnostics of genetic risks

• Pharmacogenomics and drug discovery

• Personalized medicine

Page 39: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

GENE X GENE Y

%

%

Normal Population

Patient Population

SNP & Disease Gene ID

%

Page 40: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Population A Population B

Most likelyDrug acts on therapy

Most likelyDrug has no efficacy

Drug treatment Choose other drug

SNP and Drug Response Evaluation

Page 41: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Genetic basis for individual differences in drug absorption and metabolism

For example:A drug is safe for 70% of people,ineffective for 25%,harmful for 5%

Genetic Variation

Polymorphisms vary

across populations

Page 42: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Target ID Drug screen

High throughputscreen

Lead compound

Clinical trial

Diagnosis/Therapy

Chemical

Natural

Combinatorial

Proteomics

Genomics

SNP applications> 7 years

SNP applications2-4 years

SNP and Drug Development

Page 43: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Advantages to the pharmaceutical industry:

• Increase efficiency of target and lead discovery• Reduce timelines and costs of clinical trials• Product differentiation in the market place

Trends in Biotechnology 19, 491-496 (2001).

Personalized medicine

Page 44: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Advantages to patients and clinicians:• Higher probability of desired outcome with a drug• Low probability of side effects• Preventive strategies• Focused therapies• Reduce costs• Better health and better healthcare

Trends in Biotechnology 19, 491-496 (2001).

Personalized medicine

Page 45: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

SNP or DNA/Protein marker discovery• Access to patient populations• Genotyping costs & Technology development• Computational methodologies

Marker utilization in practice• Assay platform development• Large scale data & knowledge management• Ethical, legal, & social considerations• Physicians & patients education Trends in Biotechnology 19, 491-496 (2001).

Challenges to Personalized Medicine

Page 46: Pharmaceuticals and Biotechnology Hirzun Mohd Yusof, PhD Manager Sime Darby Technology Centre

Conclusion: Malaysia’s aspiration in biotechnology vs regulatory Issues

Challenges:

• IP regulatory

• Generic/Biosimilar biopharmaceutical???

• New technologies (biopharmaceutical from transgenics)???

• cGTP - Good Tissue Practice (tissue replacement, tissue engineering, cell and tissue banking)???

• Pharmacogenomics (ethical issues, diagnostics, pre-disposition screening)