Developing efficient gene therapies requires a deep understanding of the various options for
therapy
delivery. We have extensive experience with a variety of gene delivery systems, and we excel in
designing optimal gene therapy strategies across numerous therapeutic indications.
Adeno-Associated Virus (AAV)
Deliver genes to a wide range of tissues in vivo with high efficiency and low immunogenicity.
Lentivirus (LV)
Ideal for therapies where transgenes are integrated into the genome ex vivo.
Herpes Simplex Virus (HSV)
Commonly engineered into oncolytic viruses for treatment of cancer.
Adenovirus (AdV)
Preferred for delivery of large DNA fragments and vaccine development.
Lipid Nanoparticles (LNPs)
Effective in delivery of therapeutic RNA both in vivo and ex vivo.
In addition to our experience working with the best delivery systems for a chosen
therapy, we have developed several technologies and approaches to overcome common obstacles:
Our
megaAAV
system is capable of delivering multiple DNA fragments into the
same
cell, subsequently reconstructing them into a large fragment in DNA, mRNA, or protein form,
allowing
for the expression of large genes or complex multi-component systems. Our megaAAV system
demonstrates significantly greater efficacy and wider application than the commonly used hybrid
and
intein-based dual-AAV systems.
Our
AAV capsid engineering
platform creates new AAV vectors with enhanced
properties. Through iterative genetic diversification and screening, we can generate diverse
libraries and identify variants with improved transduction efficiency, tissue specificity, and
stability. This high-throughput approach accelerates AAV vector optimization for targeted gene
therapy.
Our
expression regulatory element (ERE) engineering
platform utilizes
cutting-edge technology to construct and optimize non-coding genomic elements crucial for
regulating
gene expression. Using high-throughput screening both in vitro and in vivo, our platform enables
the
identification and engineering of novel EREs with enhanced gene regulatory properties. This
approach
is vital for advancing gene therapies with optimized gene expression profiles and for developing
more effective gene delivery systems.
Our
hematopoietic stem cell (HSC) gene therapy
product enables the treatment
of
hereditary hematological diseases by efficiently transferring genes to HSCs. Using a lentiviral
vector, we engineer and reinfuse HSCs into patients. These modified cells engraft,
differentiate,
and express the target gene exclusively in the hematopoietic lineage, providing life-long
correction
of HSC-derived diseases such as beta-thalassemia.
At Lantu, we are committed to making patients’ lives better, and we believe effective, accessible gene
therapies can only happen with a focus on evolving technologies. Our expertise as developers allows us
to
use the best possible systems, and our expertise as scientists allows us to create optimized, effective
therapies for all.
