Targeted drug delivery systems aim to increase the concentration of drugs in specific tissues while reducing side effects. The document discusses various drug delivery carrier technologies including lipid-based carriers like liposomes, polymer-based carriers, inorganic nanoparticles, magnetic particles, nucleic acid/peptide carriers, and cell-based delivery systems. It also covers the technology value chain and key innovations in targeted delivery systems for diseases like cancer and neurological disorders. While targeted delivery offers advantages, challenges remain around costs, long-term effects, and developing multi-pronged targeting approaches.

1. Targeted Drug Delivery--Technologies
and Applications
Novel Drug Delivery Systems and Clinical Trial
Management China 2013
Aiswariya Chidambaram
28th November 2013

2. Focus Points
Importance of Targeted Drug Delivery
Segmentation of Targeted Drug Delivery
Technology Capability – Drug Delivery Carriers
Technology Value Chain
Targeted Drug Delivery – Key Innovations
Targeted Drug Delivery – Drivers and Challenges
Focus Points
Key Technology Developments – Drug Delivery Carriers
Life Cycle Analysis of Drug Delivery Systems
Demand Side Analysis
Potential Applications by Therapeutic Area
Patent Distribution and Publishing Trends
About Frost & Sullivan
2

3. Importance of Targeted Drug Delivery
Targeted drug delivery is a system of drug delivery which increases the concentration of the drug in specific organs and tissues
relative to the others. This helps improve the efficacy of the drug while reducing side effects. Cancer, autoimmune diseases,
neurological disorders, pulmonary diseases, cardiovascular diseases and most other conditions that require effective, safe ,
specific targeting of certain receptors or direct delivery into the organ are attractive targets for targeted drug delivery.
Criticality of Targeted Drug Delivery
Physiological and Biological Barriers to Drug Delivery
Mucous
Barrier
(lungs, nose,
and cancer
cells)
Maximize
precision
Blood Brain
Barrier
(several
factors lead to
ineffective
delivery to
brain)
Subcellular
Targeting
(cytosol, ER,
nucleus
targeting)
Minimize
toxicity
Moderate
release
Size
Exclusion
(size barrier
of different
membranes)
Ineffective
Ligand
Targeting
(disease
specific
ligands on
cell surface)
Other
Barriers
(pH,
osmotic
potential,
electric
charge)
Source: Frost & Sullivan
3

4. Segmentation of Targeted Drug Delivery
• Oncology and neurology are the two most widely
researched diseases for targeted drug delivery.
• Delivery to the lungs, eyes, and nose are other areas of
interest as targeting these organs is relatively difficult.
Lipid-based
Polymer-based
Targeted Drug
Delivery
Carriers
Inorganic Nanoparticles
Magnetic Particles
Nucleic Acid/ Peptidebased
Cell-based
• Liposomes
nanoliposomes,
stimuli
responsive
liposomes, and conjugated liposomes with other functional
attributes are gaining attention from researchers.
• Peptides and nucleic acids -Chimeric peptides are formed
when a drug that is normally not transported through the
BBB is conjugated to a brain drug-targeting vector.
• Engineered polymers - PEG, polymeric nanomicelles and
other co-polymers; polymers designed to respond to
specific biological changes (such as pH, temperature,
chemicals and so on) so that the drug load is released
only upon stimulation.
• Conjugates - antibody conjugated liposomal carriers,
multifunctional nanocarriers, and other particles that form
conjugates with the drug.
Source: Frost & Sullivan
D4C1-TI
4

5. Technology Capability–Lipid-Based Carriers
Liposomes are widely used for insoluble drugs and advances in conjugation technologies is enabling them to be used as
targeted delivery systems. For example, the biocompatibility and possible diversity with structures and compositions make them
suitable for a number of targeted delivery applications.
Conventional
Liposomes
Stimuli
Responsive
Liposomes
Stealth
Liposomes
Targeted
Liposomes
Polymer
Composite
Liposomes
Advantages
Enables passive/active
targeting
Easy and rapid
internalization
Low immunogenicity
Drawbacks
Rapid degradation (uptake
by RES(reticuloendothelial
system)
Poor scale up/need for
extensive modifications
Short shelf life
Improves solubility/
bioavailability
Drug protection/
Biocompatibility
Liposomes will find more adoption with the development of nanoscale liposomes embedded with drug depot and
polymer depots. Targeted liposomes and environment sensitive liposomes are the ones with maximum potential for
tumors and neurodegenerative disorders, and a number of existing chemotherapeutics are being encapsulated in
stimuli responsive liposomes.
Source: Frost & Sullivan
5

6. Technology Capability–Polymer-Based Carriers
Definition: Polymers have been widely used for sustained release of drugs, and adding functional targeting groups and other
moeties has enabled them to be used as targeted delivery vehicles. A number of natural and synthetic polymers
(degradable/nondegradable, hydrophilic/hydrophobic) with multifunctionalities are being produced for delivering drugs, while
polymer composites with lipids and inorganic nanoparticles are also being developed.
Stimuli
Responsive
Polymers
Polymer
Composites
Multifunctional
Polymers/
Co-Polymers
Advantages
Disadvantages
Hypersensitivity (certain
polymers)
Wide range of drugs can
be loaded
Artificial Cells
Cell mimicking properties
(certain polymers)
Biocompatibility Issues
(synthetic)
Easy Functionalization
(surface modification)
Controlled Release
Kinetics
Low Cost/Scalable
Polymeric templates have been recently used to develop artificial cells, such as platelets and biomimetic vesicles for
targeted delivery. Use of more natural polymers and polymer conjugated with other delivery methods will witness
increased attention from drug delivery companies and pharmaceutical companies in the next 2 to 3 years.
Source: Frost & Sullivan
6

7. Technology Capability–Inorganic Nanoparticles
Definition: Inorganic nanoparticles comprise nanoscale particles made from silica. metals, metal hydroxides, carbon and so
on. A number of multifunctional, inorganic nanoparticles are being developed for targeted drug delivery and imaging
applications. Hybrid drug carriers combining stimuli sensitive hydrogels and inorganic nanoparticles, and conjugation of
biomolecules to nanoparticles are areas of interest.
Carbon Based
Particles
Gold Based
(AuNPs)
Advantages
Optical Properties
Silica/Alumina
Nanoparticles
Quantum
Dots
Metals/
Oxides/
Sulfides
High Stability (over wide
temperature and pH
range)
Drawbacks
Toxicity Issues (LongTerm Safety )
Non Biodegradable
(accumulation)
Highly Tunable
Evade RES Clearance
Inorganic nanoparticles with multiple functionalities will prompt further research for development of effective cellular
delivery systems. A few gold-based colloids and nanoshells are in clinical trials for cancer applications.
Source: Frost & Sullivan
7

8. Technology Capability–Cell-Based Systems
Definition: Cells have been found to act as potential drug delivery agents and they can be used to directly encapsulate the
drug or used with nanoparticles for better pharmacokinetic properties, biocompatibility and higher drug loading capacity. The
figure indicates some of the major types of cell-based delivery platforms in development.
Dendritic Cells/
Tumor cells
Engineered
RBCs
Advantages
High Drug Loading
Capacity
Adjuvant Properties
Microbial Ghosts
and Viral
Particles
Genetically
Engineered Stem
Cells
Sustained Release
Biocompatible (except
microbial ghosts)
Scalable/Cost Effective
Drawbacks
Potential Immunogenicity
(viral, bacterial particles)
for non vaccine delivery
Storage and Formulation
Issues
Maintaining integrity
(RBCs, stem cells,
macrophages)
While viruses and virus-like particles (VLPs) have been widely used for vaccine delivery, the use of engineered
bacterial ghosts (BG), engineered RBCs and stem cells is slowly moving to the clinic. Many of these cells and cell
derived-particles are conjugated with other delivery strategies to improve the efficacy of treatment. Stem cell-based
therapies provide a promising approach to the treatment of several diseases in humans, and extensive research on
MSCs for targeted delivery is underway.
Source: Frost & Sullivan
8

9. Technology Capability–Magnetic Particles
Definition: These are micro-and nano-scale particles loaded or conjugated with drugs that get activated when exposed to an
active magnetic field and release drug cargo at the target site. It is a highly controllable and effective form of drug targeting. In
addition to delivery, these particles are apt for safe image guided drug delivery.
Advantages
Evade RES clearance
Image guided delivery
with MRI
Biological
Non-Biological
Controlled Drug
Release
Highly targeted
Organic
Inorganic
Drawbacks
•Gradient loss for deep
seated tissues
•Accumulation of
magnetic material at
target site
•Requirement for
specialized
manufacturing and QC
system
Organic magnetic carriers include magnetoliposomes (ferrofluids entrapped in the liposome core) and polymer
magnetic particles. Iron oxide particles used directly with the drugs are inorganic magnetic particles. Incorporation of
magnetically responsive materials into microspheres makes them susceptible to applied magnetic field, so that they
are concentrated to the target site by the application of a magnetic field externally to that site. Ferriliposomes are
other magnetic particles that could be used in combination with magnetic resonance imaging (MRI) for targeted drug
delivery and also as theranostics. To improve biocompatibility and safety, biological magnetic particles, such as
magnetobiosomes and engineered erythrocytes are being designed, and these are highly promising platforms.
Source: Frost & Sullivan
9

10. Technology Capability–Nucleic Acid/Peptide Carriers
Definition: In addition to peptides and nucleic acids being used as effective drugs, they are also being explored for targeting
drugs. Cell penetrating peptides, polypeptide membranes, and DNA Nanorobots are some of the technologies with immense
opportunities. Delivery of siRNA and other nucleic acid drugs, which is still a major challenge can be overcome with the use of
peptide and aptamer-based targeting.
DNA Origami(DNA
Nanorobots)
Peptide
Conjugates
Advantages
Drawbacks
Highly Targeted
Mode of administration
Biocompatible
Aptamer Instability
Biodegradable
DNA/RNA
Aptamers
Cell Penetrating
Peptides
RNA/Peptide dual aptamer systems, polypeptide membranes are being developed for highly targeted delivery and being
extensively researched. DNA Origami is a promising technology area which will enable ‘smart delivery’ to become a near-term
reality. Overcoming aptamer instability via modifications is improving the clinical value of these systems.
Source: Frost & Sullivan
10

11. Technology Value Chain
Clinical Trials
Conceptualiza
tion of Drug
Delivery
Carriers
Development
of Drug
Delivery
System
Scale Up and
Large-Scale
Manufacture
(GMP)
Regulatory
Approval and
Marketing
Preformulation
and
Formulation
Source: Frost & Sullivan
11

12. Key Innovations–Targeted Drug Delivery
Multistage Nanocarriers (Leonardo Biosystems, TX, USA)
 Multi-stage nanocarrier-based delivery platform
 Customized mesoporous silica nanoparticles are used
 Functions better than single-stage systems
 For delivering siRNA, small molecule and imaging agents to cancer cells.
Trojan Horse CNS Targeting Technology for Stroke (ArmaGen
Technologies, CA, USA)
 The TNF-alpha decoy receptor is engineered as a fusion protein with a BBB
molecular Trojan horse (MTH).
 The MTH is an engineered monoclonal antibody (MAb) against the BBB
specific transferrin receptor (TfR).
 Facilitates receptor mediated transport of the drug across the BBB.
 Ensures effective and safe delivery of drugs to the brain.
Thermally Responsive Liposomes, Lysolipid Thermally Sensitive Liposomes
(LTSL) (Celsion Corporation, NJ, USA)
 A patented liposomal tumor targeting delivery system
 Delivers high concentrations of drug to target site (tumors) on exposure to local
hypothermia.
 Currently carrying out Phase 3 clinical trials (HEAT Study) for its lead candidate
ThermoDox®, a formulation of doxorubicin for targeting HCC (hepatocellular
carcinoma).
 Expanded applications to develop formulations for other chemotherapeutics,
such as docetaxel and carboplatin.
Blood
Brain
Barrier
Hyperthermia
Drug release
into tumor
Source: Frost & Sullivan
12

13. Key Innovations–Targeted Drug Delivery (continued)
IVECT Method , (Intezyne, Inc, FL, USA)
 A multidisciplinary approach of drug delivery that has the potential to revolutionize cancer
treatment.
 Developed proprietary polymeric micelles that deliver drugs to tumors using a triggered
release mechanism.
 It is a highly tunable and cost-effective platform that can incorporate different targeting
ligands.
 Currently has four cancer chemotherapeutics in its pipeline: two lead programs in final
preparation for IND submission and two others in earlier stages of preclinical development.
Resealed Erythrocytes for targeted and Controlled Dryg delivery, Erydex (Erydel Spa,
Italy)
 An erythrocyte based drug delivery system for sustained release of drugs
 Encapsulaties autologous red blood cells with glucocorticoid analoguedexamethasone
sodium phosphate (Dex 21-P).
 Used for treatment of chronic disorders such as cystic fibrosis, Ataxia Telangiectasia.
 Ventured in to diagnostics and targeted drug delivery (EryTargeting) aimed at delivery of drug
cargo only to targeted macrophages.
Resealed RBCs for Targeted Drug Delivery to Tumors , GRASPA (ERYTech Pharma,
France)
 A tumor targeting therapeutic utilizing erythrocyte encapsulation technology.
 Encapsulates a wide array of molecules such as peptides, proteins and small molecules for
delivery.
 Targets the tumor microenvironment using the encapsulated L-asparginase which depletes
the circulating asparagine, a tumor growth factor thereby starving the tumor cells to death.
 Currently tested in Acute Lumphoblastic Leukemia, Pancreatic cancer and Acute Myelod
Leukemia.
Source: Frost & Sullivan
13

14. Targeted Drug Delivery Systems–Drivers and Challenges
•
•
•
•
•
•
•
•
•
•
•
•
Need for effective delivery of biologics
Patenting opportunities for targeted therapies
Quicker time to market/ease of approval
Discovery of disease biomarkers
Development of spatially/temporally controlled systems
Advances in Nanotechnology
Improve patient compliance
Competition from medical devices for targeting
High costs of several types of systems
Potential long-term effects of nano particles
Lack of multipronged/ combinatorial targeting approaches
Poor funding scenario
Source: Frost & Sullivan
14

15. Technology Development— Lipid Based Carriers
2
1
Below listed are some of the areas being developed by universities and research institutions
• Dual response sensitive liposomes
Company
5
0
Technology Development
• Zwitterionic oligopeptide liposomes targeting mitochondria
• Targeted liposomes for intracellular delivery
4
4.0
• Self assembling nanoemulsions
• Liposomes fused on inorganic nanoparticles
3
Key
0-2
 Less than 10 company
developments
2 - 3.5  Between 10 and 20 company
developments
3.5 - 5  More than 20 company
developments
Key Developments
Silence Therapeutics (UK)
Silence's siRNA delivery platform is based on the proprietary lipid moeities that
embed siRNA into lipid-bi-layer particles. The siRNA is combined with Silence's
developed lipid moieties containing cationic lipids, co-lipids and PEGylated lipids to
form nanoscale structures.
Celsion Corporation(NJ, USA)
ThermoDox, the patented heat sensitive liposomal formulation of doxorubicin is in
clinical trials for liver cancer and also being tested for a number of oncology
indications.
LiPlasome Pharma ApS (Denmark)
The tumor targeting drug loaded lipid nanocarriers are designed to be susceptible to
degradation by phospholipase A2 (PLA2), which is high in the cancer environment.
The prodrug lipids are degraded by PLA2 and get converted to
active drugs such as anticancer lysolipids and/or fatty acid drug derivatives. The
degraded entities also enhance the permeability of the drugs across cancer cell
membranes to deliver high doses of drug to the target site.
Source: Frost & Sullivan
15

16. Technology Development—Polymer Based Carriers
Below listed are some of the areas being developed by universities and research institutions
•
•
•
•
•
•
Artificial Cells, Synthetic Platelets (Scripps Research Institute and Sanford-Burnham Institute)
Dual Stimuli Responsive „Smart‟ Capsules (University of Melbourne)
Protein Polymer Drug Conjugates
Polymer drug depot in liposomal nanoparticles
Multifunctional polymeric vesicles
Stealth particles coated polymers, Amphiphilic Biodegradable Dendrimer-Like Star Polymers
Company
3
2
1
4
4.5
5
0
Technology Development
Key Developments
PolyTherics Limited (UK)
Flexible polymer platform for targeted, sustained delivery of biopharmaceuticals.
GlycoPol TM is a targeting glycopolymer developed by attachment of saccharides a
poly(methacrylate) backbone. Drugs can be attached to the backbone for targeted
delivery.
Intezyne, Inc. (FL, USA)
IVECT Copolymer Micelles that are based on the IVECT Method offer a lost cost,
modular and highly targeted delivery platform. It is highly tunable and can be used to
deliver a number of drugs for varied indications.
The lead candidate IT-141, has demonstrated significant activity against a diverse
number of cancer cell lines. Another formulation IT-143, is the encapsulated
daunorubicin, is being assessed for treatment of lung cancer, osteosarcoma, and
ovarian cancer.
Arrowhead Research Corporation (CA, USA)
The company has a portfolio of in house developed and acquired targeted delivery
platforms, of which DPCs(Dynamic polyconjugates) and RONDEL are based on
polymer nanoparticles. Both are being explored for delivery of siRNA therapeutics.
Source: Frost & Sullivan
16

17. Technology Development— Inorganic Nanoparticles
2
The adoption of inorganic nanoparticles is on the rise with the development of gold and silica
nanoshells, nanowires, nanorods, and many more. They are emerging as an important and useful
class of targeted entities that can be functionalized for specific needs. These inorganic nanoparticles
can also be used for simultaneous imaging and delivery applications. Iron oxide, silica, gold,
fullerenes, and carbon are the most widely researched materials.
3
1
4
3.5
5
0
Technology Development
Below listed are some of the areas being developed by universities and research institutions
•
•
•
•
•
Mesoporous nanoparticles conjugated with peptides, antibodies, and other entities
Stimuli sensitive nanoparticles
Surfactant functionalized nanoparticles
Aptamer gated nanoparticles
Multistage nanoparticles
Company
Leonardo Biosystems (TX, USA)
CytImmune Sciences Inc.
Key Developments
Multistage mesoporous silica nanoparticle based platform for spatiotemporally controlled drug release is attracting a lot of interest from investors
and pharma companies. The system is undergoing optimization and
enhancements.
The technology is based on pegylated colloidal gold nanoparticles that can be
used directly as drugs via tumor targeting molecules or can be used as
carriers for cancer drugs.
Source: Frost & Sullivan
17

18. Technology Development— Cell Based Carriers
2
The adoption of cell-based drug carriers is relatively low despite research being carried out for more
than a decade. Modified RBCs form the majority of therapeutic carriers, while stem-cell based drug
targeting is showing promising results in clinical trials. Microbial cells have been used for targeting
vaccines and drugs for several years, and modified forms of these cells are now being developed for
more effective and safe targeting.
3
1
4
2.5
0
5
Technology Development
Below listed are some of the areas being developed by universities and research institutions
•
•
•
•
•
•
Resealed erythrocytes/ Engineered erythrocytes with viral fusion proteins (for example, Erythro-magneto-HA virosomes)
Engineered mesenchymal/ neural stem cells (for example, Silica nanorattle-drug anchored mesenchymal stem cells
Virosomes/Bacterial ghosts for DNA vaccine and subunit vaccines
iPSCs reprogrammed for organelle specific targeting
Magnetotactic bacteria
Prodrugs/Drugs bound to RBCs in circulation for long-term thrombosis treatment
Company
Key Developments
EryDel SpA (Italy), EryTech Pharma (France)
Erythrocyte loaded drugs for multiple disease areas currently in clinical
development. These are being used for controlled delivery as well as targeting.
Pevion Biotech AG (Germany)
Proprietary virosome-based platform for targeted drug delivery and adjuvant
activity for subunit vaccines. A number of vaccines are in advanced phases of
clinical trials using their VLP technology, and has also been outlicensed to
several pharma majors.
Engene IC (Australia)
EDV (Engene Delivery Vehicle) technology basically consists of inert bacterial
cell derived nanoscale minicells conjugated with bispecific antibodies for highly
targeted intracellular delivery of cancer drugs/siRNA. Immune stimulating
properties enhance performance and the drugs using this platform are currently
in clinical trials.
Source: Frost & Sullivan
18

19. Technology Development—Magnetic Particles
Below listed are some of the areas being developed by universities and research institutions
• Bacterial magnetosomes from magnetotactic bacteria for drug targeting that can be functionally superior
to artificial magnetic particles
• Ocular magnetic drug targeting
• Magnetic resealed erythrocytes
• Enzyme and temperature responsive magnetic nanoparticles
• Multifunctional nanoparticles for delivery and imaging (for example, multilayered nanorattles)
Company
2
3
1
4
1.5
0
5
Technology Development
Key Developments
Vascular Magnetics, Inc, (PA, USA)
Biodegradable, magnetic drug-loaded particles in combination with a magnetic
targeting catheter and a magnetic field generating device that guides the
particles to narrowed arteries in PAD (Peripheral Artery Disease).
Nanobiomagnetics Inc. (SW R and D) (OK,USA)
Magnetic vectored drug delivery using magnetically responsive therapeutic
constructs.
nanoTherics (UK)
Magnetic transfection method MagnetofectionTM using DNA, siRNA coupled
with magnetic nanoparticles to form a complex. Upon exposure to oscillating
magnetic arrays, cells show uptake of the complex via rapid endocytosis.
MagForce AG ( Berlin, Germany)
Iron oxide nanoparticles with aminosilane coating that safely delivers drugs to
tumors upon activation by their proprietary NanoActivator™ magnetic field.
Source: Frost & Sullivan
19

20. Technology Development— Aptamers/Peptides/Nucleic
Acids
2
3
Below listed are some of the areas being developed by universities and research institutions
1
• Peptide Dendrimer Conjugates
• Engineered Oleosin–Self assembled to form Biomimetic Vesicles (University of Pennsylania)
• Peptide and Aptamer Functionalized Nanoparticles; Aptamer nanoparticle Bioconjugates
(PANOPTES project --novel peptide-based nanomaterials for ocular delivery)
• Cell penetrating peptides (HIV TAT peptide, human calcitonin (hCT) hormone)
• Carbohydrate mimetic peptides (UC, Berkeley)
• Cell specific aptamers, peptide conjugated environment sensitive particles
Company
4
2.5
5
0
Technology Development
Key Developments
Savara Inc. (TX, USA)
Condensed nanoparticles of cell penetrating peptides and RNA
therapeutics for targeted intracellular delivery. This is known as
Nanonucleic technology used primarily for RNAi therapy.
Artificial Cell Technologies, Inc (CT, USA)
Layer by layer assembly of polypeptide artificial biofilm nanoparticles for
developing synthetic vaccines. The synthetic nanoparticles incorporate
immunogenic epitopes and give rise to “Artificial Virus” like structures
that can be used for vaccine delivery.
Aptagen, LLC (PA, USA)
Aptabodies™ developed by the company are proprietary, functionalized
aptamers that can be used for drug delivery or diagnostics. The
company is a good collaborative partner for pharma/biotech companies
for delivery of peptide therapeutics.
Source: Frost & Sullivan
20

21. Market Position
Life Cycle Analysis of Drug Delivery Systems
Conventional Liposomes
Stimuli sensitive
liposomes
Non Biodegradable Polymers
Stimuli responsive
polymers
Conjugates/Hybrid Carriers/
Composite particles
Engineered peptides/Aptamers
Magnetic nanoparticles
Functionalized Polymersomes
Multistage/Multifunctional
nanoparticles
Inorganic nanorods, nanoshells,
nanowires
Biological Magnetic
carriers
Stem cell carriers
Artificial cells/
Biomimetic particles
DNA Nanorobots
Development
Growth
Time
Maturity
Decline
Source: Frost & Sullivan
21

22. Demand Side Analysis
Applications
Versatility
‘Smart’
Delivery
Tunability
End-User
Requirements
Market
Exclusivity
Cost
Effectiveness
and Scalability
Biocompatibilit
y
Source: Frost & Sullivan
22

23. Potential Applications - Ocular
InSite Vision Inc.
Integral BioSystems
Novagali Pharma
Quark
Pharmaceuticals
Ocular Therapeutix
• Challenges with regard to topical administration include poor access to the posterior portion of the eye,
need for frequent doses, and poor controlled delivery.
• Ophthalmic inserts developed for sustained release suffer from limitations, such as high costs and difficulty
with insertion.
• Liposomes and polymers - most widely used vehicles for ophthalmologic delivery. Targeting ligands that
can cross the Blood Retinal Barrier(BRB) explored for a number of orphan retinal diseases.
• Identification of ocular transporters and development of transporter targeted drugs modified with targeting
ligands
• Other commonly deployed delivery systems - functionalized nanoparticles, in situ forming gels, and
colloidal dosage forms
Source: Frost & Sullivan
23

24. Potential Applications - CNS
Serina Therapeutics Inc.
to-BBB
ArmaGen Technologies
VECT-HORUS
Angiochem Inc.
Lauren Sciences LLC
Advectus Life
Sciences Inc.
• Systemically administered drugs have limited access across the BBB, while local administration is painful
and challenging.
• Most innovative drug developments for neurological disorders are biologicals, such as cell based therapy,
RNAi or gene therapy.
• Drug delivery systems that can cross the BBB (blood brain barriers) primarily include nanoparticles with
targeting ligands and CNS targeting vectors that can bind to receptors on the BBB and cross the barrier in
order to improve efficacy and increase safe therapeutic window.
• Most of the research is in the basic research or pre-clinical stage
• A number of partnerships and collaborations have been witnessed in the last 1 to 2 years. (For example,
Genzyme Pharmaceuticals and Pharmidex Pharmaceutical Services Ltd)
Source: Frost & Sullivan
24

25. Potential Applications - Oncology
Celsion Corp
BIND Biosciences
LiPlasome Pharma
Alchemia
Savara
Pharmaceuticals
Silence
Therapeutics
Intezyne
EnGene IC
ERYTECH Pharma
CytImmune
Sciences Inc.
Cerulean Pharma Inc.
MagForce AG
• Oncology is the most well penetrated area - more than 80% of targeted drug delivery activity is focused on
safe and effective delivery of chemotherapeutics to cancerous cells.
• Strategies based on differences in tumor cell metabolism, tumor microenvironment, and expression of
receptors on tumor cell surfaces are being developed.
• Most of them in clinical development are conjugates of polymers, liposomes, and inorganic nanoparticles,
while magnetic targeting and imaging is also being explored by a few Participants.
• Cell-based delivery systems that can trigger the immune system against cancer cells and multifunctional,
multiple ligand targeting strategies will take predominance.
Source: Frost & Sullivan
25

26. Potential Applications – Infectious Diseases
Artificial Cell
Technologies Inc.
EryDel
Xenetic Biosciences
Arrowhead Research
Corporation
Pevion Biotech AG
• High degree of noncompliance to therapy in a number of infectious diseases - led to development of
targeted drugs that can specifically target the pathogen and associated pathways.
• TB, HIV, and malaria are some of the highly researched areas.
• Virosomes and bacterial ghosts have been widely used for vaccine delivery.
• By developing different nanoparticle formulations with cell penetrating peptide conjugates, glycomimetic
peptides, and other moieties, intracellular delivery to pathogens is becoming possible.
Source: Frost & Sullivan
26

27. Potential Applications – Cardiovascular
Vascular Magnetics Inc.
Spheringenics Inc.
BIND Biosciences
• Eluting stents and cardiovascular implants – invasive drug delivery methods – strong need for non-invasive
drug delivery systems.
• Targeted immunoliposomes and biodegradable targeted polymeric particles are being developed for
cardiac delivery and imaging.
• Cell-selective targeted drug delivery - key research area for cardiovascular applications.
• In atherosclerosis and other ischemic conditions, it is necessary to develop systems that can release drugs
on sensing difference in shear stress or tissue injury.
Source: Frost & Sullivan
27

28. Potential Applications – Pulmonary
MannKind Corporation
Pulmatrix
BioParticle
Technologies
Liquidia Technologies
Insmed Inc.
• Most appropriate route for the treatment of asthma, cystic fibrosis and other pulmonary diseases such as
tuberculosis, COPD, and lung cancer; explored for systemic diseases like diabetes.
• Useful for developing inhalable vaccines, which hold huge market potential as a needle-free vaccination
strategy for a number of respiratory infections.
• Peptide and protein drugs are ideal for pulmonary delivery as they undergo degradation in the
gastrointestinal (GI) tract).
• Liquidia‟s PRINT (Particle Replication In Non-Wetting Templates) and MannKind‟s Technosphere have
demonstrated success for pulmonary delivery.
• Liposomes are the most extensively studied system for controlled drug delivery to the lungs.
• Emerging areas of interest are magnetic aerosol droplets, bioresponsive nanoparticles, and co-polymers
with better release profiles.
Source: Frost & Sullivan
28

29. Potential Applications–Mapping
Disease prevalence and
criticality of delivery challenges
Funding
Research Initiatives
Company
Developments
Cardiovascular
Medium
Medium
Medium
Low
Ocular
Medium
Medium
Low
Low
CNS
High
Medium
Medium
Medium
Oncology
High
High
High
High
Medium
Low
Low
Low
High
High
Medium
Medium
Infectious Diseases
(Targeting Pathogens)
Pulmonary
Criteria
Key - Funding
Low  Less than $100 million
federal and private funding
Medium  Between $100 - $200
million federal and private
funding
High  More than $200 million
federal and private funding
Key – Research Initiatives
Low  Less than 50 published
research projects by
academia
Medium  Between 50 and 100
published research projects
by academia
High  More than 100 published
research projects by
academia
Key – Company Developments
Low  Less than 10 companies
working on TDD
Medium  Between 10 and 30
companies working on TDD
3.5 - 5  More than 30 companies
working on TDD
Source: Frost & Sullivan
29

30. Assignee-wise Patent Distribution
Total Number of Patents/applications – 1080 (100 unique patent families).
Source: Frost & Sullivan
30

31. Top Countries for Publishing Patent Applications
US = United States
EP = European Union
WO = WIPO
AU = Australia
CA = Canada
JP = Japan
CN = China
AT = Austria
DE = Germany
ES = Spain
Most of the innovations in targeted drug delivery is happening in the US and European Union, which is clearly indicated by the number of patent
applications published from these regions.
Source: Frost & Sullivan
31

32. Patent Publishing Trends
This chart shows the patenting trends in the last 4 years and the patenting trend has been quite stable. On an average 250 patents were published
every year.
Source: Frost & Sullivan
32

33. Questions

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