6 / 16
Page 27
conferenceseries
.com
Volume 04
Journal of Clinical Infectious Diseases & Practice
ISSN: 2476-213X
Rare Diseases Congress 2019
June 17-18, 2019
June 17-18, 2019 | Berlin, Germany
9
th
World Congress on
Rare Diseases and Orphan Drugs
Red blood cell-encapsulated enzymes: An innovative therapeutic approach to overcome challenges of
enzyme replacement therapies for rare diseases
Emmanuelle Cecile Dufour
Erytech Pharma, USA
M
any inborn errors of metabolism (IEM) disorders are due to defects in single genes encoding key metabolic
enzymes. In most cases, clinical manifestations of these disorders are driven by the over-abundance of a
metabolite or the scarcity of an essential metabolite. Though rare, IEM disorders can have devastating consequences
for patients and their families. While some Enzyme Replacement Therapies are commercially available for a few IEM
disorders, the clinical benefits of these approaches are often outweighed by the emergence of hypersensitivity and
the rapid clearance of enzymes. Therefore, there is a high need for better tolerated and longer-acting replacement
enzymatic activity to alleviate the burden of IEM disorders. RBCs are the most abundant cell type in the human
body and their biology is characterized by a long lifespan and access to all tissues and organs. Thanks to their
biocompatibility and shielding properties, they can serve as a circulating bioreactor when loaded with enzymes.
ERYTECH is a leader in RBC therapeutics. Its ERYCAPS® platform enables the encapsulation, at industrial scale,
of active drug substances inside RBCs using hypotonic loading, which has been shown to maintain all the RBC
functionalities. ERYTECH has demonstrated that RBC-encapsulated enzymes exhibit substantially improved
in
vivo
performance vs. non-encapsulated enzymes, including extended enzymatic activity. Results from two early
programs using enzyme-loaded RBCs in
in vivo
models for Arginase-1 Deficiency and Classical Homocystinuria
will be presented. These promising results combined with ERYTECH’s extensive clinical experience with RBC
therapeutics, support the possibility that RBC-loaded enzymes may provide superior safety and efficacy as compared
with traditional ERT approaches for the treatment of IEM disorders.
Recent Publications
1. Gay F, et al. (2017) Methionine tumor starvation by erythrocyte-encapsulated methionine gamma-lyase activity
controlled with per os vitamin B6. Cancer Med. 6(6):1437-1452.
2. Bourgeaux V, et al. (2016) Drug-loaded erythrocytes: on the road toward marketing approval. Drug Des Devel
Ther. 10:665-76.
3. Thomas X and Le Jeune C (2016) Erythrocyte encapsulated l-asparaginase (GRASPA) in acute leukemia. Int J
Hematol Oncol. 5(1):11-25.
Emmanuelle Cecile Dufour, J Clin Infect Dis Pract 2019, Volume 04