toptech in health and wetllness-2012_ti.pdf

Top Technology Trends in Health and

Wellness (Technical Insights)

June 2012

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Technologies for the betterment of the healthcare system

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Contents

Section Slide Numbers

Executive Summary 3

3D Cell Culture Systems--Mimic In vivo Conditions 6

Nanoparticle-based Therapies--Cancer Therapeutics 11

Recombinant Expression Systems—Plant-based Transgenics for Protein Therapeutics 16

Biosensing--Nanobiomedicine Approach 21

Next Generation Prebiotics--Targeted and Second-degree Prebiotics 26

Genetically Engineered Foods--The 2nd Agricultural Revolution 31

Nutrient Encapsulation--Ensuring Nutrient Delivery 36

Nanofluidics and BioNEMs--Intelligent Miniaturized Systems 41

Next Gen Sequencing--Thousand Dollar Genome 46

Adult Stem Cell Therapies--The Commercial Cell of Origin 52

The Frost & Sullivan Story 59

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Executive Summary

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Research Scope and Index

Technical Insights division of Frost & Sullivan

evaluated technology trends in the Health and

Wellness sector and has identified 10 technology

trends that are likely to have an impact in the year

2012.

The slides provide information on the following

lines on a best effort basis, as applicable to the

technology under consideration. They are as

follows:

• Brief Snapshot of the Technology

• Recent Developments

• Key Stakeholders and their Solutions

• Future Scenario

To

p T

ec

hn

olo

gy T

ren

ds

3D Cell Culture System

Nanoparticle-based Therapies

Recombinant Expression Systems

Biosensing

Next-Generation Prebiotics

Genetically Engineered Foods

Nutrient Encapsulation

Nanofluidics and BioNEMS

Next-Gen Sequencing

Adult Stem Cell Therapies

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Executive Summary--Research Process and Methodology

• Technology Journals • Periodicals • Market Research Studies • Technology Policy Information Sites • Internal Databases • Thought Leader Briefings

Secondary Research

Primary Research

• Engineers • CTOs/CEOs/CIOs • Technical Architects • Research Heads • Strategic Decision Makers • Technology Policy Heads

2. Interview Participants

Stakeholder Insights,

Perspectives and Strategies

Innovators and Innovations

1. Patent Review

3. Assess Innovations

Technology Capabilities and Stakeholder Initiatives

Technology/Business Challenges

Application Market Potential and Needs

Technology/Business Drivers

OUTCOME--Forecast Future of Technology,

Market Adoption, and Potential Application Sectors

Research Methodology Research Process

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3D Cell Culture Systems--Mimic In vivo

Conditions

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3D Cell Culture systems are ex-vivo cellular arrangements, generated in a scaffolds that intend to

simulate the extracellular matrix (ECM) microenvironment to control culture conditions that propel

cell proliferation. One of the main goals of these systems is the replication of in vivo conditions of

organotypic cultured systems to perform in vitro diagnostic or in vitro metabolic studies. In

particular, the spatial and temporal distribution of these signals is tightly controlled and unique to

each organ.

• In the human body, all cells exist in a complex

three-dimensional (3D) network mesh and this has

motivated scientists to replicate the structure and

composition of natural cellular environments.

• The architecture of such a culture system enables

close interaction between cells, growth factors and

extra-cellular matrix.

• Products from 3D Biotek, Invitrogen, Millipore are already in

the market. Increasing awareness should enhance adoption

rates and newer application areas like 3D microspheres

which would impact the market by 2013.

• 3D cell culture systems can reduce animal testing, improve

efficiency and reduce cost and time of testing procedures.

Technology Snapshot

Importance Year of Impact

Overview

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Global Footprint

USA/ Canada

Extensive research in the areas of 3D

microplates, coverglass, scaffolds,

matrices, assay wells, organotypic

microtissue models,. Greater adoption

has been noted in the area of cell culture

and by 2015 there would be transition

from 2D to 3D cell culture.

Japan

Research and adoption in the areas of

cell culture applied to stem cells and

various other kinds of cells or models

to develop drugs and therapeutics

Europe

The main application areas include cell-

cell and cell-matrix interactions. Adoption

is noted in the areas of 3D cell culture.

Australia/New Zealand

3D cell cultures

3D scaffolds

3D organotyping

India

3D bioreactors, 3D scaffolds are

available through distributors of

top firms like Invitrogen, Sigma-

Aldrich . Basic research in the

area of 3Dscaffolds and matrices

is observed.

Source: Frost & Sullivan.

Intensity of Technology

Development

Very High

High

Medium

Low

Very Low

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Stakeholders and Developments

Key Challenge: Petri dishes, tissue culture flasks, and micro-well plates are commonly used systems for cell

culture owing to simplicity, convenience, and high-cell viability. These conventional flat systems or two-

dimensional (2D) mechanisms to culture cells played a vital role in understanding cell biology, but are

insufficient for the new challenges of cellular biology or pharmaceutical assays.

Company Product /

Technology Details

Life

Technologies

Corporation

Gibco Products

Invitrogen (renamed as Life Technologies) provides extracellular matrices,

scaffolds and proteins under the Gibco umbrella to support stem cell and other cell

cultures.

Hamilton and

Global Cell

Solutions,

Inc.

BioLevitator

BioLevitator a benchtop incubator and bioreactor that uses GEM (Global

Eukaryotic Microcarrier) technology that allows GEMs to be kept in suspension

with the ability to automatically culture them, and minimizes the need for manual

handling.

Sigma-Aldrich

Co. LLC 3D Matrices

The 3D matrices include HydroMatrix synthetic peptide, MaxGel human ECM, and

Mouse ECM. These products provide three-dimensional environments in which

cells are better able to mimic their in vivo counterparts.

3D Biotek,

LLC 3D Insert

3D Biotek is developing 3-D cell culture devices for stem cell/tissue engineering

and drug discovery applications. Product 3D Insert--PCL has been chosen by the

NIST as the reference 3D tissue engineering scaffold.

Synthecon

Inc.

BIOFELT, Rotary Cell

Culture Systems,

Biomerix

Synthecon allows users to grow dynamic cultures in which the matrix, suspended

in a fluid bath, and promotes 3-D cell growth

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Impact on Application Areas

• Organ Engineering

• Target validation

• Secondary screening/

Lead optimization

• Safety assessment/

Toxicology

• DMPK

• Primary screening

• Stem cell culture

• Cell biology model systems

• Developmental biology

• Cell supply and

cryopreservation

• 3D matrices and scaffolds

• Culturing tumor cells

• Drug testing

• Cell culture methodology

2012 2014

Tissue

Engineering

Drug Discovery

Basic Research

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Nanoparticle-based Therapies--Cancer

Therapeutics

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• Nanoparticles are used for a wide variety of biomedical applications to aid in laboratory

diagnostics and in medical drug targeting. In targeted cancer therapies they are used as

conjugates with cancer biotherapeutics such as peptides, proteins or antibodies.

• Among the next generation of nanoparticles that are being used for this purpose are

superparamagnetic nanoparticles, gold nanoparticles, interactive nanoparticles composed of

conjoined gold nanorods and iron oxide nanocrystals.

• Conventional cancer therapies such as

chemotherapy using small molecules or even

targeted therapy using antibodies do not target

cancer cells with a high degree of precision.

Nanoparticles can identify, target and kill specific

cancer cells while leaving healthy cells

undamaged.

• Nanoparticle conjugates of targeted cancer drugs

show superior therapeutic efficacy, specificity and

selectivity compared to conventional targeted

therapies.

• Several nanoparticle-based conjugates are the products of

research labs. Examples include the interactive nanoparticles

and the RNA nanospheres (RNA encapsulated in lipid-based

cationic nanoparticles), both from the Massachusetts Institute

of Technology.

• Companies such as Cytimmune and NanoSpectra

Biotherapies have already demonstrated heat-based

nanoparticle treatment. The year of impact can be expected

to be 2016 as several such conjugates have completed

Phase I clinical trials.

Technology Snapshot


Overview

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Global Footprint

North America

• USA has been active in this sector in both basic and

applied research. Dr. Sangeetha Bhatia’s group at the

Massachusetts Institute of Technology and the

California Institute of Technology have pioneered the

use of several types of nanoparticles from lipid-based

cationic particles to gold nanorods.

• Companies such as CytImmune, Tempo

Pharmaceuticals and Carigent Therapeutics are

already developing nanoscale systems for the delivery

of cancer drugs. Ortho Biotech, a subsidiary of

Johnson and Johnson has already launched such a

system using liposome nanoparticles (Doxil).

Europe

• The Turku Center for Biotechnology , the University of

London and the Karolinska Institut have been leading

research efforts for the use of gold, porous silica and

carbon-based nanoparticles in cancer therapy for

prostate, pancreatic and non-small cell lung tumors.

• Nanobiotix and Ensemble Therapeutics based out of

France and Magnamedics based out of Germany are

notable companies developing nanaoparticle-based

therapies for cancer.

• The European Commission has formulated a roadmap

till 2020 to incorporate milestone-based developments

for nanoparticle-based drug delivery.

Japan, Taiwan and Korea

• Japan has been exploring the use of platinum and

silica nanoparticles for targeted cancer therapeutics

through the Tokyo Institute of Medicine and the Riken

Institute.

• Korea Institute of Science and Technology has

pioneered the development of chitosan-based

nanoparticles fro targeting tumors.

• The National Health Research Institute in Taiwan has

optimized the use of nanoparticles in cancer by

combining a diagnostic contrast agent with an

anticancer drug.



Development

Very High

High

Medium

Low

Very Low

Australia

• Work in this sector is limited to basic

research. The Cancer Council SA has

funded researchers from both the Hanson

Institute at the Royal Adelaide Hospital and

the Wark Institute at the University of South

Australia to develop such nanoparticles.

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Funding Trends

• Nanomedicine has been stated as a key developmental area on the innovation agenda for a number of

countries including the United States, South Korea, Taiwan and Japan. The European Commission has

been maintaining nanomedicine as a key research focus since the 6th Framework Plan and has so far

already committed $367 million USD to the field since 2004.

• Among the applications of nanomedicine, drug delivery which includes targeted cancer therapy is

indicated to have the highest precedence with 59% funding being allocated to it in Europe and USA.

• NIH includes nanomedicine as a part of its Common Fund Program that is aimed at providing targeted

funding and training programs for ten key research areas. Nanomedicine has been on the Common

Fund since 2006.

Source: NIH, EC, KIST, ITRI Source: Journal of Nanomedicine, IEEE EMBS

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Impact of Technologies

Lipid-

based/Liposomal

nanoparticles

Carbon

nanotube-based

particles

Composite

nanoparticles

Silica-based

porous

nanoparticles

Gold

Nanoparticles

Co-development and licensing

projects with both government and

pharma companies such as Merck,

AstraZeneca and Novartis.

Applied research will be conducted

in these fields with funding from

NIAID, DARPA and the Medical

Research Council.

These nanoparticles will be exploited

to carry and dispense drug payloads

to breast and lung tumors in

preclinical studies.

These nanoparticles will be tested in

preclinical studies by UCLA, NIST

and NIH. Photoluminescent particles

particularly useful for dermal drugs

The efficacy of these particles is

being tested with different

biotherapeutics in vitro. Start-ups

like Pilot Therapeutics are also

involved.

Companies like Tekmira

Pharmaceuticals and Encapsula

NanoSciences are already

developing lipid nanoparticles for

pharmaceutical use.

McGill, University of Lancashire,

University of South Florida and

Stanford University have

identified carbon nanotubes as

conducive for drug delivery.

MIT has demonstrated the use of

interactive nanoparticles—

composites using gold nanorods

and iron oxide particles.

Silica-based porous nanoparticles

have been proposed for

photodynamic therapy by Nancy

University. Cornell University is

also a major participant.

Gold nanoparticles have been

used in conjugation with anti-

cancer drugs by researchers at

MIT—nanorods and colloidal

particles.

Pictures

2012 2015

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Recombinant Expression Systems—

Plant-based Transgenics for Protein

Therapeutics

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• Plant-based transgenics involve the use of plant-based material such as cash crops,

vegetables or moss for the extraction of recombinant proteins for therapeutics and research.

Human protein forms from antibodies to enzymes can all be isolated from plants.

• The most commonly used materials for such transgenics are tobacco, carrots and moss and

the process sometimes involves genetic engineering or cross-transfections.

• Plant-based transgenics do not have standardized regulatory protocols.

• Conventional recombinant expression systems

such as yeast and bacterial systems require

expensive infrastructure and production equipment

in the form of fermenters.

• The yield percentage using yeast or bacterial

system is usually around 35%-50%, much lower

than the required rate for therapeutics dosage.

• Plant-based transgenics are cost-effective in terms

of culture and production, can be used to derive

naturally glycosylated proteins and require

inexpensive raw materials.

• Plant-based transgenics are in demand as glycosylated

proteins can be naturally derived from them. In addition,

isolation and purification is cost-effective making it a

conducive choice for the manufacture of biosimilars and bio-

betters.

• These products not only comply with the required safety

standards but are also more humane and environment-

friendly as they do not use any animal-based raw materials.

• The year of impact for these systems is, therefore, likely to

be 2015-16.

Technology Snapshot


Overview

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Global Footprint

North America

• USA does not have a very strong presence in plant-based

transgenic systems in terms of commercialization. This can

be due to the lack of standardized FDA protocols for

approving them.

• Basic and applied research are done extensively at a

number of institutes including the Roswell Park Cancer

Clinic, Arizona State University, School of Medicine and

Cornell University.

• Companies such as Medicago and GreenVax have gone

into Phase I clinical trials with their vaccines from Proficia

and tobacco plants respectively. However, advancement to

later phases has been a hurdle.

Europe

• The most active countries in Europe pursuing plant-based

transgenics for recombinant protein therapeutics are

Israel and Germany. The former houses two major

companies, Collplant and Protalix Biotherapeutics. The

former develops recombinant virgin collagen while the

latter is developing taliglucerase alfa for Gaucher, Fabry

and autoimmune diseases

• Germany is home to Greenovation, the only company

commercializing a moss-based recombinant expression

platform. Icon Genetics is also a leading company there.

• The adoption footprint is Europe is considerably higher

due to the provision of a risk analysis procedure from the

European Commission.

Australia and Africa

• AzarGen is an early stage recombinant

expression platform developer in South

Africa. Their technology is still in the design

phase.

• CSL in Australia has a plant for the

manufacture of recombinant proteins from

plant-based products but acquires

technology platforms for the same through

in-licensing deals. Source: Frost & Sullivan analysis


Development

Very High

High

Medium

Low

Very Low

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Funding Trends

• Plant-based protein has recently come into focus due to its applicability in producing recombinant human

vaccines. Major government institutions such DARPA and NIAID have extended investments to aid the

development of plant-based systems for recombinant proteins.

• The most ambitious project for plant-based recombinant systems in USA is GreenVax, a Texas-based

initiative that is aimed at producing vaccines for infectious diseases using tobacco plants.

• Europe is already active in this space with the majority of funding being allocated to Italy and Germany.

The latter is home to 2 major companies in this sector, Icon Genetics and Greenovation.

Source: NIH, EC

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Research Area Stakeholders Technology Developments & Trends

Recombinant

collagen from

tobacco

• Development of virgin homogeneous human collagen from

recombinant expression in tobacco plants.

• Two flagship products—Vergenix for wound repair and the same as a

flowable gel. They are also branching into orthopaedics applications

with Pfizer (bone void filler).

Taliglucerase alfa

from carrot and

tobacco

• ProCellex platform for optimized genetic engineering and plant-based

recombinant expression.

• Taliglucerase alfa for Fabry, Gaucher, biodefense and other

autoimmune diseases.

• Ready for USA market—was granted PDUFA status after successful

Phase II trial, by FDA.

Multi-stage drug

delivery chips using

nanoporous silicon

particles

• Formerly subsidiary of Bayer with Nomad Biosciences, currently

independent. Plant-based recombinant expression tools—work for

any plant.

• Two major recombinant expression tools—nuclear transformation

and plastid transformation suite.

• Highest soluble protein percentage and conversion quotient in

industry. They are working on therapeutic products in collaboration

with Pfizer.

Stakeholders and Technology Developments

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Biosensing--Nanobiomedicine Approach

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Carbon Nanotubes (CNTs) and other nanostructures, including Peptide Nanotubes (PNTs) exhibit

ideal properties for use as a sensing material: high surface area, chemical and mechanical

stability, which has resulted in the increasing design of a variety of sensors including biosensors,

electrochemical sensors and gas sensors, to be applied in life sciences and biomedicine fields.

The ability to incorporate any antibody without destroying the recognition function allows the

development of new highly specific targeted therapies for a broad spectrum of diseases.

CNTs/PNTs are excellent platforms for developing sensors due to the high effective area, together

with excellent electrical, mechanical, thermal properties and chemical stability.

• PNTs are useful nanomaterial building blocks that

have been used to construct a variety of device

geometries, as their self-assembly is robust, and

locations for their immobilization on substrates can

be targeted by biomolecular recognition.

• The biological functionalization of CNTs, attaching

proteins and/or DNA or RNA, has received a great

deal of interest due to the potential biomedical

applications of the nanotubes in both diagnostics

and therapeutics.

• This technology is expected to be increasingly adopted by

2013.

• Assembling PNT sensors with a simple chip geometry

enables the electrical detection of viruses and other

pathogenic organisms with an extremely low detection limit.

Technology Snapshot


Overview

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Global Footprint

USA/Canada

Nanoimmunology

Microarrays and molecular diagnosis

Implantable biosensing devices

Implantable nano-tagreting devices

Drug discovery and development based

on nanobiosensing platforms

Japan

Microarrays and molecular diagnostics

Nanoimmunology

New biomarkers discovery and

development strategy

Drug discovery and development

based on nanobiosensing platforms

Europe


Point-of-care systems



Drug discovery and development based on

nanobiosensing platforms




based on nanobiosensing platforms Argentina/Brasil

Molecular diagnosis




Development

Very High

High

Medium

Low

Very Low

India

Microarrays

Molecular diagnostics



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Research Area Company Technology Developments

Pathogen detection

systems through nano-

scale biosensing devices

BioForce

Nanosciences

, Inc

BioForce Nanosciences developed a platform for fast, sensitive and non-destructive virus detection, the

ViriChip virus detection platform. The platform consists of a silicon chip functionalized with an ultramicro

array of antibodies using the Nano eNabler, and a detection system based on Atomic Force Microscopy.

Metal nanoparticle

sensors for hydrogen

detection

Applied

Nanotech

Holdings

Applied Nanotech has developed a nanotechnology based Metal Nanoparticle Sensor (MNPS) Platform,

whose first product is a palladium nanoparticle-based hydrogen sensor. Nanoparticles of palladium

provide high surface to bulk ratio compared to macro scale palladium thin films used for hydrogen sensing

in biotech.

Protein interaction

studies for quantitative

assays on a single

platform

Axela Inc

The dotLab® System uses dot technology to significantly shorten the transition from protein discovery to

application in both research and diagnostics. New panelPlus™ Sensors simplify multiplexing, and allow

the freedom to combine established markers with novel content.

Interventional cardiology

and critical care

procedures

Biosensors

International

Biosensors International offers abluminal drug-eluting stents with a biodegradable coated, limus-eluting

technology on its proven stent platforms, the S-Stent™ and Juno™. The company’s key R&D initiative in

the interventional cardiology field is a family of "polymer-free" drug-eluting stent systems coated with

proprietary BA9™ anti-restenosis drug.


NeuroSearch

(Atonomics

A/S)

The PoC system, Atolyzer® , aims to address a wide range of important clinical markets including

cardiology, maternal tests and prostate cancer.

Label free biomolecular

interaction measurement

for bioprocess

applications

SAW

Instruments

GmbH

The biosensor sam®5 enables the label free measurement of biomolecular interactions and their kinetics

in real time. Binding processes can be detected selectively and with highest sensitivity, and can further be

analyzed.


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Short term

2012-2013

Monitoring

Thera

peutics

Dia

gnostics

Medium term

2013-2015

Long term

2015-2017

Clinical monitoring

Point-of-care assessment

Implantable post-

surgical biosensing monitors

Nanotechnology has made it possible for a single chip to possess more than million features with supra-sensitive detection abilities than

that of conventional methods. Because of the nature of this industry, breakthroughs in applications and solutions can be expected in the

near future in the areas of sustainability, nanotechnology and biocybernetics.

With recent advances in micro, nano-

fabrication techniques and

multidisciplinary research studies

focusing on bridging sensing platforms

for medical applications, new applications

in clinical monitoring are arising.

High resolution and high throughput

screening capabilities, which enable a

wide variety of biological assays can be

significantly enhanced by using novel

biosensing platforms.

The use of nanoscale biosensors is

applied to therapeutic areas by

evaluating cellular systems response in

drug discovery process.


Nano-therapeutics,

nano-immunology assessment

Implantable targeting devices

Microarrays technology

Nano-beads for genetic diagnostics

Implantable biomarkers

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Next Generation Prebiotics--Targeted and

Second-degree Prebiotics

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• Prebiotics are slightly fermented or non-fermented, non-digestible ingredient that promote the

growth of beneficial microorganisms in the human intestine.

• Targeted prebiotics are unique ingredients from various carbohydrate and cellulose sources

that promote the growth of specific bacteria for a particular function.

• Second-degree prebiotics prevent the growth of a particular species of pathogenic bacteria

promoting immunity.

• Prebiotics help in growth of bacteria in the intestine

directly. Compared to probiotics, the survival rate of

prebiotics is higher as the action of prebiotics starts

inside the small intestine; whereas probiotic

bacteria are affected by acidic pH of the stomach.

Targeted and second degree prebiotics promote

the functionality of prebiotics.

• Prebiotics are easy to be integrated into solid foods

such as cereals. They can also be incorporated

into beverages and powders.

• The year of impact of targeted beyond 2015 as it is in applied

research stage.

• The year of impact of second-degree prebiotics is beyond

2013 as it is in clinical trials stage.

• Prebiotics’ ultimate aim is to be available as drug

replacements over the counter. This is expected to impact

beyond 2020

Technology Snapshot


Overview

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Global Footprint

Latin America

Native fruits and other

ingredients are exported to the

US and Europe to produce

prebiotic ingredients.

Japan

Japanese companies

are researching and

manufacturing prebiotic

ingredients. They are

focusing on prebiotics

as drug replacers.

Switzerland and

Netherlands

Leading companies such

as Nestle and Danone are

researching to add

second-degree prebiotic

ingredients in their food

products .

Australasia

Unique vegetation helps in providing

sources for next generation prebiotics.

United States of

America

Prebiotic companies are

researching the

possibility of utilizing

inulin as a targeted

prebiotic ingredient

since this has the

highest market share in

the world in the US.



Development

Very High

High

Medium

Low

Very Low

France

Leading cosmetic

companies such as Loreal

and Dedeor use prebiotics

in their cosmetics.

UK

Utilization of

prebiotics for feeds

of animals to

prevent specific

diseases.

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Funding Trends

• The highest amount of funding is provided to dairy foods integrated with prebiotic ingredients. Prebiotics are sometimes

mixed with probiotics to improve digestion. Hence the research is backed by both functional foods and dairy food

companies.

• To reduce recalls of meat due to bacterial outbreaks, second degree prebiotics are being researched. This aspect is being

aided by governments of various countries.

• Prebiotics in cosmetics is relatively new and is still in basic research stage. Leading cosmetic companies are funding the

same.

Funding sources

Dairy

Confectionery and processed foods

Animal Feed

Dairy products

40%

confectionery and processed

foods 30%

animal feed 15%

beverages 10%

cosmetics 5%

Beverages

Cosmetics Source: NIH, FDA

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Impact of Technologies

Prebiotic

blends

Prebiotics in

cosmeceuticals

Second

generation

prebiotics

Synbiotics

Encapsulated

prebiotics

Prebiotics as

drug

replacement

Prebiotic blend combinations of inulin and fructooligosaccharides (FOS) are likely to be launched and globally marketed.

Loreal and Dedeor are likely to introduce skin products containing prebiotics based on their patented technologies

Applied research is estimated to increase in the field of utilizing prebiotics to prevent cardiovascular diseases.

Synbiotic yoghurt technologies are likely to be patented. Products are expected to start launching by the end of the year.

There is an estimated increase in the amount of research for encapsulated prebiotics to prevent specific diseases that are not intestine related.

Along with Japan, the US and Europe, middle east and other Southeast Asian countries are likely to research in this area.

Prebiotic blends are combination of two or more prebiotic ingredients. Combinations of inulin were tested and launched.

Glasgow university researched the possibility of prebiotic ingredient glucomannan hydrolsylates to prevent acne

Researchers across the world are developing prebiotic ingredients that can prevent gastrointestinal diseases.

Blends of prebiotics and probiotics known as synbiotics are being clinically trialed by dairy companies to sample yoghurts.

Encapsulated prebiotics are being trialed for cosmeceuticals. They help in drug delivery apart from functioning as bacteria promoters.

Possibility of combining targeted, second-degree and encapsulated prebiotic technologies could lead to drug replacement prebiotics.

Pictures

2012 2015

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Genetically Engineered Foods--The 2nd

Agricultural Revolution

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Ever since advances in biological sciences have paved way to manipulate

organisms at the DNA level, modern biology has made huge strides in improving

life forms to suit humankind. This has extended to plants and animal food too.

Genetically engineered food also known as biotech food are a class of agricultural

(both animal and plant based) products that have been modified at the genetic

level. These types of food are specially modified with enhanced properties that

enable the food to either grow in a more sustained manner or to provide additional

nutritive value to the final product

The importance of genetically engineered food cannot

be understated. Many times, such food helps

overcome issues of poor consumption, in other

instances such food aids in providing additional

nutritional benefits to people who do no have easy

access nutritional supplements. It is even possible to

incorporate vaccines into genetically modified plants

so that when consumed the food will provide the

necessary immunity against dangerous diseases

• Products that are meant for agricultural users are already

available in the market with new technology incorporating

multi-stacked traits.

• New, state-of –the-art technology includes test tube food.

Such kind of foods are excepted to be commercialized in the

near future with the year of impact being 2014

Technology Snapshot


Overview

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Global Footprint


Adherence to CODEX

Standards

Very High

High

Medium

Low

Very Low

USA/Canada

GM food need not be labelled.

Does not follow CODEX

standards Europe

Very strict labeling laws.

Follows CODEX standards

India

Moderate laws regarding

GM crops. Strict when

implied in food.

Follows CODEX

standards

China

Pro GMOs especially

in case of GM Rice.

Not too strict in

following CODEX

standards

Australia & New Zealand

Strict Laws. Labelling laws

similar to E.U. Follows

CODEX standards.

Latin America

Pro certain GM crops such as biofuels

generating crops. Moderately strict in

terms of GM food Crops.

Follows CODEX standards in parts.

The Food and Agriculture Organization of the United Nations (FAO) and the World Health Organization (WHO) established the CODEX Alimentarius, sets

standards and guidelines to protect the health of consumers and ensure fair practices in the international food trade. In addition to standards for specific

foods, the Codex Alimentarius contains general standards covering matters such as food labeling, food hygiene, food additives, pesticides and safety.

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Key Challenge: Difficulty in obtaining biological sequences; long R&D timelines and nature overcoming target

mutations are the most significant technology related challenges. While high R&D costs, ill-perception of BT

products, patent infringement and enforcement and lack of uniform laws for international trade are some of the

most significant business challenges facing genetically engineered food as of today.

Company Product /

Technology Details

Okanagan

Specialty

Fruits Inc

Modified Apples

The Canadian based company has applied for permission to grow and market its ‘arctic

granny’ and ‘arctic golden’ apples. The genetically engineered fruit, was originally

developed in Australia and has the ability to keep their white flesh even when exposed

for several hours. The browning of cut apples usually leads to the perception that the

food has spoilt and increased wastage of good quality fruit. This innovation allows for

the fruit to be wasted less easily

AquaBounty

Technologies Modified Salmon

The US based company has produced its in-house developed AquAdvantage Salmon.

This is a genetically enhanced breed of salmon that grows faster than regular salmon.

The company is currently seeking federal approval for the salmon. If approved, the

salmon would be the first genetically engineered animal in the human food supply.

Considering the fact that salmon (and hence even caviar) are a rare commodity, this

should reduce strain on naturally occurring salmon population and preserve its diversity.

Agrargen Modified Camelina

The company plans to introduce its in-house developed modified Camelina that

contains increased oil content. This is said to enhance biofuel production by making the

process more economical.

35 D4A9-TI


2014 2016 2020

Crop protection and production are the most important traits

being imparted to crops currently. This trend will decrease in the

long term once all popular crops have been suitably genetically

modified.

Currently, there are very few commercial animal products that

have been genetically modified. With food security predicted to

be a major issue in the long term, such foods will be more

commonplace.

Currently, functional foods have little bearing in the mainstream

global market as they’re resigned for countries with poor

nutritional access. This trend is expected to be continued in the

long term.

Pharmaceuticals are probably the biggest impact in the long

term. This is due to long timelines of research and

commercialization.

Legend: Low High

Crop Production

Protection

Animal Husbandry

Functional Foods

Pharmaceuticals

Renewable Chemicals

Renewable chemicals will benefit from genetically engineered

plants. Varieties that allow for greater productions and easier

extraction of renewable chemical content will show greatest

promise.

36 D4A9-TI

Nutrient Encapsulation--Ensuring Nutrient

Delivery

37 D4A9-TI

Nutrient Encapsulation involves the incorporation (entrapping or enveloping) of core materials that

include food ingredients, enzymes, cells or other materials such as additives, nutrients, flavors,

colors, micro organisms, essential oils, vitamins, minerals, in an enclosing material (carrier, shell,

wall, capsule, or membrane).

The enclosing materials are designed to protect the core material from the damage and to prevent

unwanted reactions with the environment, moisture and heat. The coating materials are typically

made from sugars, proteins, gums, natural and modified polysaccharides, synthetic polymers, and

even fats.

• Nutrient Encapsulation helps in extended shelf life

of the product and improved performance

characteristics.

• It increases the bioavailability of the encapsulated

ingredient and helps in controlling their reactions

within a specific environment.

• It prevents loss of nutrients and prevents their

interactions with atmosphere and other

constituents during storage.

• While encapsulated probiotics, nutrients (amino acids,

vitamins and minerals) are available in market, controlled

release and multi-functional products are expected to be

widely adopted within early 2015.

• While early adoption of nanotechnology and

microencapsulation for increasing product performance is

perceived, they are expected to gain prominence by 2014.

Technology Snapshot


Overview

38 D4A9-TI

Global Footprint

USA/ Canada:

Extensive research to ensure stability

during processing and packaging,

reduction of capsule size and enhancing

bioavailability.

UK:

Research efforts are more focused on

novel encapsulation materials, controlled

release and increasing bioavailability


Research efforts mimicking global

trends. Current focus on novel

encapsulation technologies and multi

component encapsulation.



Development

Very High

High

Medium

Low

Very Low

Rest of Europe:

Focus on nanoencapsulation and

microencapsulation techniques, especially

in countries like Germany, The

Netherlands, Israel and Sweden..

39 D4A9-TI


Key Challenge: While many nutrients are encapsulated, product stability and release of active ingredient at

targeted site are the major challenges that need to be addressed for sustained growth of the industry.

Company Product /

Technology Details

Technion Israel

Institute of

Technology

Nano capsules to

protect hydrophobic

nutraceuticals

Nano capsules (conjugates) made of casein and maltodextrin are developed to

protect hydrophobic nutraceuticals such as vitamin D. The conjugates were used

for the enrichment of clear beverages . The development of nano capsules have

the additional benefit of masking off-flavors and off-odors.

Nanologica

Mesoporous Silica as

encapsulating

material

Mesoporous silica particles are developed as an encapsulation material using

nanotechnology. The material is perceived to be safe, stable, cost effective and is

capable of addressing the challenges such as off-taste masking, controlled release

and increasing nutrient bioavailability

Tropicana

Products

Fatty acid enriched

fruit juices

Fortified fruit juices with omega 3 essential fatty acids using double encapsulation

techniques to ensure nutrient bioavailability and to ensure increased performance

characteristics.

Biogaia AB ProTectis and

ProDentis

Encapsulated probiotic strain, Lactobacillus reuteri in chewable tablets, capsules

and other carriers to promote oral health, combat infant colic and digestive

aliments.

Lipofoods Lipophytol

Micorencapsulation techniques used for coating the microparticles of phytosterols,

thereby, providing a water-dispersible form to supplement phytosterols into the

daily diet.

40 D4A9-TI

Technology Roadmap

2010 2013 2015 2017 2019


Self

assembled

Nutrients

Microencapsul

ation

Powdered

Technology

Controlled

release

Mechanism

Targeted

Delivery

Droplet

technologies

Hybrid

Technologies

Nano capsules

Nutrient Encapsulation aid in incorporating reactive additives and other ingredients into stable ingredients to add value to the final product. The development of

encapsulation technologies has the ability to transform health and wellness industry and helps in expansion of application areas such as nutraceuticals, fortified

and other functional foods apart from conventional applications that include taste-, color- and odor- masking and flavor stabilization,

41 D4A9-TI

Nanofluidics and BioNEMs--Intelligent

Miniaturized Systems

42 D4A9-TI

Nanofluidics refers to the control and manipulation of excessively small fluids of dimension less

than 1nm. Fluids in the nano-scale range exhibit behavior normally not observed in the macro

scale. This industry is experiencing considerable growth in recent years. A benefit of nanofluidics

is that it offers the possibility of exploring fluid behaviour using controlled regular nanostructures.

This technology has had a great impact on drug delivery devices as the systems have the ability

to completely control drug release rate depending on the on-demand requirements for extended

periods of time.

The use of NEMS based technology in medical

applications is an area with immense potential.

Demand for improved healthcare at reduced cost,

along with point-of-care applications for the elderly

population, has made nano-scale biomedical

technologies an attractive opportunity for investors

and manufacturers..

Nanofluidic technology has been successfully implemented in

technologies involving analytical separations and the

manipulation of proteins, RNA and DNA. However most of the

applications are in combination with bioMEMS technology. The

number of application areas is emerging enabled by innovative

fabrication methods. An important advantage of nano-scaled

systems is the small amount of sample or reagent used for

analysis. This reduces the time required for sample processing

and further speeds up the processes thereby increasing

throughput of the analysis.

Technology Snapshot

Importance Strength

Overview

http://www.esm.psu.edu/wiki/research:juh17:acoustofluidics

43 D4A9-TI

Global Footprint

• Nanofluidics is a converging technology and OEMs are manufacturing NEMS based devices in combination with MEMS

based systems.

• BioMEMS technology has revolutionised drug discovery and molecular based diagnostics. It is expected that these two

applications will boost the nanofludics and bioNEMS technology in a similar fashion.

• In the western countries such as North America (NA) and European Union (EU), nanofludics technology is currently driven by

the government initiatives and funding undertaken for promoting nano-based technologies.

• The Chinese market is slowly catching up with the western and European countries in terms of the bioMEMS industry.

Chinese medical companies have traditionally distributed North American and European products on their home market. That

is starting to change slowly and China is emerging as a key participant in the bioMEMS field. Given the current technology

development trends taking place in China, it can be expected that China will soon emerge as a key global player in this field.

44 D4A9-TI

Company Product Description

BioNano Genomics NanoAnalyzer System

Minute samples are utilised by the nanoAnalyzer System to analyze several

gigabases per hour. Valuable information is obtained about the genome structure

as the sample is analysed without breaking up the molecules. The technology

enables high-throughput processing. The proprietary nanoAnalyzer technology is

capable of delivering precise genetic data in a fraction of the time and at a cost

which is a fraction of existing technologies.

RainDance Technologies RDT 1000

RainDance Technologies’ (RDT) is a company which develops a proprietary

microdroplet-based technology with special emphasis in the prediction and

prevention of human disease. Some of the proven application of this technology

are for assays such as PCR and cell sorting. The technology proposed by

RainDance produces picoliter-volume droplets at a rate of 10 million per hour,

avoiding complex automation solutions.

Vyteris Inc LidoSite Topical System

Topical local anesthetic delivery system indicated for use on normal intact skin to

provide local analgesia for superficial dermatological procedures such as

venipuncture, intravenous cannulation, and laser ablation of superficial skin lesions.

Boston Scientific TAXUS Express

The TAXUS stent uses Translute™ Polymer which is a proprietary polymer carrier

technology, to control drug release. The Translute Polymer protects the drug and

maintains coating integrity during preparation, delivery, and stent expansion.

Cytosurge

FluidFM

FluidFM technology utilises hollow connecting cantilevers with a microfluidic

system for fluid application. The technology is used for dispensing and stimulating

single cells under a physiological condition. Nanofluidic channels allow soluble

molecules to get dispensed through submicrometer aperture in the AFM tip.


The regulatory barrier is a huge challenge for companies in this field. Companies might have the expertise to manufacture

bioNEMS but they have modest experience in the rigorous regulatory process. Apart from the regulations that medical devices

must comply with, there are additional standards for bioMEMS devices, applicable for NEMS based devices as well which

manufacturers must comply with.

45 D4A9-TI

2010 2015

Application Roadmap

2020 2025 Beyond--


Lab Automation

µTAS

LOC

• Injectable BioMEMS

• Implantable LOC

Nanodrugs, Drug

Delivery, Diagnostics,

cancer Treatment

Nano Robotics, Nano

sensors

In Vitro Analysis Nano-In Vivo Analysis

BioNEMS

Benchtop

Devices

Portable

Devices

Wearable

Devices

Implantable

Devices

Nanomaterials and

Implants

Digestable sensors

46 D4A9-TI

Next Gen Sequencing--Thousand Dollar

Genome

47 D4A9-TI

• The large scale adoption of sequencing can be useful for

clinical diagnostics and drug discovery in addition to basic

biomedical research and genetics studies.

• Regular genetic testing can improve clinical management

of diseases based on patient stratification and personalized

therapy design.

• Regular use of sequencing can reduce healthcare costs,

bring down drug development costs and timelines and

improve therapeutic outcomes for many diseases.

• Predictive disease models can be developed with use of

valuable sequencing data.

• Several next gen sequencers are already in the market and these

include Pacific Biosciences’ SMRT sequencer, Ion Torrent’s Ion

Proton and Oxford Nanopore’s GridION and MinION. Solid state

nanopore based sequencers. should enter the market by 2013

• Regular application in clinical diagnostics and genetic screening

tests by 2013 or 2014.

• Microbiome research and research on human pathogen interactions

should find increased applications by 2015.

• Whole genome sequencing lower than $1000 combined with low

instrumentation costs is likely to be achieved by 2013.

Technology Snapshot


Overview

Next Gen Sequencing involves technologies that can significantly decrease the cost and time of

sequencing while improving accuracy and length of reads. Technologies, such as real-time single molecule

detection, nanopore sequencing, high-density semiconductors allow multiplexing, label-free detection,

increased read-lengths, and reduced overall costs and time. Use of novel materials and the integration of

biology, chemistry, electronics, and micro/ nanofluidics is playing a significant role in development of follow

on next gen sequencing technologies. Portable sequencers, (PoC) which are cost effective and can be

used for small size runs will find increased applications in diagnostics and regular point-of-care clinical

studies.

48 D4A9-TI

Global Footprint

USA/ Canada

Highest amount of research and funding for next gen sequencing

projects. Most of the Tier 1 companies as well as mid and small sized

innovation driven companies are based in USA and research on DNA

methylation , RNA profiling, epigenetics and so on.

Adoption of next gen sequencing in drug discovery, genetic tests is the

highest in US and several partnerships between sequencing

developers and research labs, allied tech developers is helping to

increase adoption and shorten time to market.

The government initiatives are the maximum in US and these include

$1000 genome project, Human Microbiome Project(HMP), 1000

genomes project- supported by NHGRI. China

BGI (Beijing Institute of Genomics), the

largest global genomics center is the pioneer

in sequencing research and has

partnerships with US companies and

institutions around the globe. However, the

developments in terms of next gen

sequencing technologies has been slow in

China and increased private and federal

investments combined with increased

collaborations will help develop better

analytical systems .

Europe

More than 220 high throughput next gen sequencing facilities in

focusing on different applications. The adoption of gene

screening tests for genetic diseases is relatively low in EU.

EU Epigenomics Consortium , E-Rare are some of the initiatives

focused of NGS technologies and applications. The

developments of NGS technologies is moderate except a few

countries such as UK and Germany that have developed

innovative sample preparation methods and sequencers,


The research is slow paced and the

number of sequencing providers are

also few in number.

India

Research is relatively low - mainly in the area of

bioinformatics for next gen sequencing. Adoption of

next gen sequencing is witnessed mainly in basic

research (plant genomics, cancer genomics) and

so on. Tier 1 company Life Technologies(Ion

Torrent) recently launched its Ion Proton to

increase adoption for drug discovery and

diagnostics applications. Currently there are about

15 sequencing centers in the country.



Development

Very High

High

Medium

Low

Very Low

49 D4A9-TI

Stakeholders and Technology Segments

Technology Segments Stakeholders Trends/ Developments

Sequencing by

Synthesis

Sequencing by

Ligation

Nanopore

Sequencing

Direct Imaging

by Advanced

Microscopy

• The development of single molecule analysis SBS methods such as Life

Technologies- Ion Torrent’s products has led to drastic improvements in

accuracy and read lengths while bringing down the costs of sequencing.

• Non optical detection techniques that detect based on changes in pH or

current will help reduce reagent costs. Use of non labeled nucleotides are

simplifying the sequencing process.

• Development of high density chips such as Ion Torrent’s Ion Proton that can

improve throughput and generate large amounts of data per run.

• Generally read lengths are short with SBL methods; new platforms are

aimed at improving the read lengths.

• Hairpin DNA system using magnetic beads being developed by the startup

Picoseq can be used for high throughput sequencing using hybridization.

• Highly scalable technology area that can revolutionize sequencing in terms

of costs, speed and throughput. This single molecule, label free method of

sequencing area is being actively explored by companies and academia,

which are working on different types of nanopores and different detection

methods.

• Oxford Nanopore has launched its first nanopore based sequencer recently-

both portable and desktop versions have been introduced. A few of the

nanopore sequencers are expected to enter the market in the next 2-3 years (

eg. Noblegen Biosciences, Stratos Genomics, Genia).

• Only a handful of companies are working on direct microscopy based

sequencing methods- still in research phase and far from commercialization

• The instrument is prohibitively expensive but the sequencing costs would drop

drastically.

• TEM(Transmission electron microscopy), SEM( Scanning electron microscopy)

and AFM (atomic force microscopy) are some of the microscopy based

sequencing platforms under development.

50 D4A9-TI

Recent Developments

Company Product /

Technology Details

Stratos

Genomics

Sequencing by

Expansion™ (SBX™)

The company is developing a nanopore sequencer based on DNA expansion

technology. The DNA is encoded into constructs called Xpandomers (reporters) .

These reporters have a high signal to noise ratio and are best adapted for

nanopore detection and Stratos is developing a sequencing instrument based on

this.

GnuBIO Inc Integrated GnuBIO

system

The desktop microfluidic sequencer to be launched later in 2012 is based on SBH

chemistry. The integrated sequencing platform is centered around picoinjector

technology that the company licensed exclusively from Harvard University. Sample

preparation, target enrichment, sequencing, and data analysis can be done in

microfluidic channels in a much simpler and rapid fashion when compared to

competing methods.

Oxford

Nanopore

Technologies

GridION and MinION

MinION ( disposable chip for PoC settings) and GridION (high throughput desktop

sequencer) launched in 2012 utilize biological nanopores for low cost, rapid and

high throughput sequencing. Illumina has made investments in ONT and the

company has a number of other collaborations with academia, working on

advanced technologies.

Noblegen

Biosciences Optipore

Optipore is an enzyme free, optical method that uses nanopores. It allows

analysis at single molecule and it is a highly multiplexed, rapid method of

sequencing in a highly cost effective manners. The system converts DNA into a

synthetic coded version that is then captured by a beacon as it passes through the

nanopores and the color codes correspond to the bases. The second prototype of

the sequencer is under development and the company expects to go commercial

in the next 1-2 years.

51 D4A9-TI

Industry Landscape--2015

Technology

Developments

Academic and

Corporate Research

Government

Initiatives

• Efficient massive parallelisation and automation of sample preparation, combined with improved sensitivities

will enable reduced sample preparation time, costs and IT burden.

• Portable sequencers that will be capable of sequencing entire genomes of bacteria, viruses and larger

genomes.

• Solid state nanopores and the use of semiconductors more regularly in sequencing applications

• Sequencing research is expected to reach significant height by the year 2015. With increase in federal and

venture funding for core and allied technologies, technology developers are poised to deliver a number of

products in the next couple of years. Many consortia and research groups are working on bioinformatics tools

and cloud based data management systems that can be effectively used for data analysis.

• Current initiatives such as the $1000 Genome, 1000 genome project, Human Microbiome Project in the USA

and few EU initiatives will eventually allow next gen sequencing to find increased utility in drug discovery ,

companion diagnostics and regular genetic screening and SNP testing. Regulatory policies that favor the

rational use of next gen sequencing for healthcare applications is likely to become more standardized.

52 D4A9-TI

Adult Stem Cell Therapies--The Commercial

Cell of Origin

53 D4A9-TI

• Adult stem cell therapies are more feasible lines of

treatment compared to embryonic stem cells as their use

is not impeded by ethical issues and the policies are

globally uniform.

• Adult stem cell therapies are also less time-consuming in

terms of developmental cycle and therefore, more

attractive for technology investors. In addition, as the cells

are extracted from the patients own marrow, they preclude

the possibilities of immunogenic reactions, making the

therapy viable with respect to regulatory standards.

• Adult stem cell therapeutics are still in their infancy in the United

States and Europe as no stem cell therapies have been approved

to date.

• The most advanced therapies such as those from Cytori

Therapeutics, Osiris Therapeutics and Aastrom are in early or late

Phase 3. These therapies are for autoimmune or cardiovascular

diseases. Other therapies for oncology, neurology and

dermatology are being developed and are in Phase I or II. The

year of impact is therefore, 2017-20.

Technology Snapshot


Overview

• Adult stem cells are undifferentiated cells found among differentiated cells in tissue/organ. They can

differentiate themselves to yield some or all of the major specialized cell types of that tissue/organ for

replacement of damaged or injured tissue. Adult stem cells can be derived from marrow (stromal cells) or

from placental cord blood. Therapies are being developed for a range of diseases from myocardial

infarction to liver disease and diabetes. Stromal cells are extracted from the patient and differentiated in

vitro.

• Globally, more than 200 companies are involved in developing stem cell products and the therapies are

likely to be disruptive treatments for medical sectors (For instance knee implants, bone marrow

transplant, heart muscle repair).

54 D4A9-TI

Global Footprint

North America

• USA has five states most active in the development

of adult stem cell therapeutics—New York, New

Jersey, Maryland, California and Massachusetts.

• Osiris Therapeutics and Aastrom have stem cell

therapies for cardiovascular diseases and

autoimmune diseases. In Phase III trials.

• All the above-mentioned states have state-centric

funding as well as regulatory guidance institutions to

accelerate the development process.

Europe

• UK, Sweden and Denmark are the countries in

Europe that have more permissible policies and

are concentrating on adult stem cell therapies,

particularly for autoimmune diseases and

oncology.

• ReNeuron, Capsant, NsGene A/S and the

Karolinska and Lund research centers are the

centers of development.

• Stem cell therapies are mostly in Phase I or II

and commercialization is slower.

Rest of APAC

• The biggest participants in the stem cell industry

are Singapore, Australia and Israel.

• Pluristem Therapeutics and Gamida Cell already

have products for autoimmune diseases and cell

expansion (inflammatory diseases) in Phase II/III.

• Mesoblast in Australia is both developing and

promoting APSCs for cardiovascular, diabetes,

oncology, eye disease, and orthopedic therapies.

All products are in Phase I/II.

•

China

• The Ministry of Science and Technology has

made available up to $293 million for stem cell

research/spinoffs from 2006-2010.

• The Chinese Academy of Science provides an

additional $20 million as well as set-up

assistance for new start-ups in this field.

• There is also a clear focus of resources on the

applications of stem cell research, such as

therapeutic uses and drug testing.



Development

Very High

High

Medium

Low

Very Low

55 D4A9-TI

Funding Trends

• NIH is the primary institute for stem cell funding, particularly for start-ups engaged in the development of

adult stem cell therapies. It has shown a steadily increasing trend and the investment was close to $350

million in 2010, excluding ARRA funding.

• The Small Business Innovation Research grant has also been a good source of funding for early-stage

startups but has shown a decline since 2010. It has however, increased till 2011, with the allocated

budget for 2012 being $3.1 million.

0

50

100

150

200

250

300

350

400

2002 2003 2004 2005 2006 2007 2008 2009 2010

Fu

nd

s (

$ m

illio

n)

Year

NIH Stem Cell Funding 2002-2010

Embryonic

Non-embryonic

ARRA 0

1

2

3

2006 2007 2008 2009 2010

Fu

nd

ing

($ m

illio

n)

Year

SBIR Funding 2006-2010

Source: NIH, EC, MRC

56 D4A9-TI

Technology Benchmarking

Therapeutic Efficacy Development (Stage and

Progress) Disease Market

ReNeuron Low High 1 5 2 3 4

Low High 1 5 2 3 4

Low High 11.9

Osiris

Therapeutics Low High

1 5 2 3 4

Low High

1 5 2 3 4

Low High 13.4

Aastrom Low High

1 5 2 3 4

Low High

1 5 2 3 4

Low High 12.9

Gamida Cell Low High 1 5 2 3 4

Low High 1 5 2 3 4

Low High 13.1

Mesoblast Low High 1 5 2 3 4

Low High

1 5 2 3 4

Low High 13.5

Pluristem

Therapeutics Low High

1 5 2 3 4 Low High

1 5 2 3 4 Low High

13.0

Total

15 Points

1 5 2 3 4

1 5 2 3 4

1 5 2 3 4

1 5 2 3 4

1 5 2 3 4

1 5 2 3 4

Dashboard

Summary

• The companies have been rated on the basis of the stage and progress of development of the therapy, the market for

the diseases at which the therapies are targeted and the therapeutic efficacy of the treatment (as indicated by results of

clinical trials).

• Aastrom and Osiris Therapeutics have both scored the highest with respect to the development stage and progress of

their therapies. They are both US companies, implying that easy access to the streamlined regulatory process provided

by the FDA for APSC therapies.

• Mesoblast has a leading position in terms of disease market as it is currently running clinical trials for the top 3 diseases

identified by WHO and NIH for highest healthcare burden and mortality rates, i.e., diabetes, cardiovascular diseases and

oncology. The only other company that has a disease portfolio close to that of Mesoblast is Pluristem Therapeutics.

• ReNeuron and Gamida Cell have scored the highest in terms of therapeutic efficacy of their treatments. Both companies

have reported successful Phase IIa trials with over 70% efficacy quotients. Although the other companies have also

advanced to Phase III trials, the efficacy quotient has been less and the pharmaco-toxicity profile is not at the same level

as the treatments from these two companies.

57 D4A9-TI

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In addition to the above, our robust in-house forecast and benchmarking models, along with

the Frost & Sullivan Decision Support Databases, have been instrumental in the completion

and publishing of this report.

We also certify that no part of our analyst compensation was, is or will be, directly or

indirectly, related to the specific recommendations or views expressed in this service.

58 D4A9-TI

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59 D4A9-TI

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60 D4A9-TI

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