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LIPOSOME-BASED IMMUNOTHERAPY
FOR TYPE 1 DIABETES
TECHNOLOGY APPROACH
The invention uses autoantigen-encapsulating liposomes for the prevention
or treatment of autoimmune disorders, preferably type 1 diabetes (T1D),
in preference during the pre-clinical state.
BACKGROUND
T1D is one of the most common metabolic diseases in childhood and its
incidence is growing in developing countries. In addition, T1D has been
increasing throughout the world at 3% per year in recent decades. The
peak age for diagnosis of T1D is 12-14 years, but the average age of T1D
onset has decreased in the past two decades and a second peak at 4-6
years has been observed. T1D can also be diagnosed in adults. Currently,
there is no treatment to restore endogenous insulin secretion in patients
with T1D and lifelong insulin therapy is required. Additional, health
problems often develop later in life. Because of this and its chronic
nature T1D is a costly disease, not only for the affected individual and
his/her family, but also for the health authorities. Consequently, the
development of new therapies to induce long-term tolerance has been an
important medical health challenge.
OUR RESULTS TO DATE
The invention is based on a previous strategy of the group, consisting on a
cell immunotherapy based on autologous dendritic cells (DCs) loaded with
apoptotic β-cells for the treatment of autoimmunity in experimental T1D.
To overcome the difficulties of sourcing apoptotic β-cells, a synthetic
therapy based on liposomes rich in PS (phosphatidylserine) loaded with
insulin peptides (Figure 1) has been developed.
The researchers demonstrated that the present liposomes are effectively
engulfed by DCs, thus inducing tolerance to the antigen and down-modulating
the autoimmune cascade. This process is able to decrease the incidence and
delay the onset of experimental T1D (Figure 2).
Apoptotic mimicry provided by liposomes can offer a solution to the
complexity of cell-based therapies with many benefits, as they are lowcost, easy to produce and standardize, to generate on a large-scale and
customize.
Complete results upon CDA signature.
Figure 1. Cryogenic transmission
electron
microscopy
image
of
liposomes loaded with insulin
peptides (PSAB-lipo).
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Figure 2. Percentage of spontaneous T1D in Non Obese
Diabetic (NOD) mice treated with liposomes loaded
with insulin peptides (PSAB-lipo), empty liposomes
(PS-lipo), and sham group. Significant differences
between treated group and controls (*).
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LIPOSOME-BASED IMMUNOTHERAPY
FOR TYPE 1 DIABETES
ADVANTAGES
• The
antigen-specificity
of
treatment
would
not
require
immunosuppressants
• The potential for personalizing the treatment makes it a promising
strategy for the clinical use
• The facility of manufacture, standardization and relative low-cost of
production are key characteristics for the development of the therapy
• Other lipid formulations have been clinically approved for the treatment
of cancer or infections
INTELLECTUAL PROPERTY STATUS
PCT
LOOKING FOR
PRODUCT PROFILE
• Licensing Out
• Investment
• Co-development
Category
Target Product Profile
Clinical Indication
Prevention or treatment of autoimmune disorders,
preferentially type 1 diabetes (T1D)
Definition
Autoantigen-encapsulating liposomes
Efficacy
Prevents T1D and decreases insulitis progression
in non obese diabetic (NOD) mice
Mechanism of action
Impairs autoreactive T cell proliferation through
PGE2 production
INVENTORS
Department of Immunology, IGTP:
• Dra. Marta Vives-Pi
• Dr. Joan Verdaguer
• Dra. Irma Pujol-Autonell
ICN2:
• Prof. Daniel Maspoch
• Dra. Mary Cano
CLAIMS AND EVIDENCE
Dendritic cells pulsed with antigen-specific apoptotic bodies prevent
experimental type 1 diabetes. Marín-Gallén S, Clemente-Casares X, Planas R,
Carrascal J, Pujol-Autonell I, Carrillo J, Ampudia R, Pujol-Borrell R, Verdaguer J, Borràs
FE, Vives-Pi M. Clin exp Immunol 160:207- 214, 2010. PMID: 20030670.
Efferocytosis promotes suppressive effects in dendritic cells through
prostaglandin E2 production in the context of autoimmunity. Pujol-Autonell I,
Planas R, Ampudia R, Marín-Gallén S, Sanchez A, Carrascal J, Marin A, Puig-Domingo M,
Pujol-Borrell R, Verdaguer J, Vives-Pi M. PLOS ONE 8:e63296, 2013. PMID: 23691013.
Use
of
autoantigen-loaded
phosphatidylserine-liposomes
to
arrest
autoimmunity in type 1 diabetes. Pujol-Autonell I, Serracant-Prat A, Cano-Sarabia M,
Ampudia RM, Rodriguez-Fernandez S, Sanchez A, Izquierdo C, Stratmann T, Puig-Domingo M,
Maspoch D, Verdaguer J, Vives-Pi M. In press(PLOS ONE), 2015.
Núria Martí and Maria Ortega
Knowledge and Technology Transfer Office
[email protected]|[email protected]|T.+34 934 978 664
Institut d’Investigació en Ciències de la Salut Germans Trias i Pujol
Ctra. de Can Ruti. Camí de les Escoles, s/n, 08916 Badalona, Spain
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