Gene Therapy Development Services
Gene therapy applies gene editing technology to introduce precise genes into the patient's cells in order to tackle the cause of disease from its core, which emerged conceptionally in few decades ago. Our Company provides leading-edge research services and essential platforms to help you get ahead of the curve in the development of gene therapy for Autoimmune Diseases & Inflammation.
Gene Therapy in Autoimmune Diseases & Inflammation
The rise of gene therapy is not surprising, as its advantages in principle that a single treatment can achieve long-term treatment results. In autoimmune diseases, the objective of gene therapy is to deliver presupposed therapeutic genes to the areas where it is required to regulate the levels of pro-inflammatory cytokines and chemokines, as well as modulate the activity and function of immune cells locally, in order to achieve immune tolerance and balance.
Currently, three major advanced gene therapies are available for the delivery of therapeutic genetic material to radically replace, restrain or augment genes, all of which have achieved some degree of clinical and commercial development.
In vivo gene therapy
In vivo gene therapy is a broad term within the field of gene therapy that involves the direct transportation of genetic material to targeted locations within the body using viral vectors or non-viral substances.
In situ gene therapy
In situ gene therapy is a more targeted treatment approach, delivering genetic material directly to the diseased site rather than undergoing systemic treatment throughout the entire body.
Ex vivo gene therapy
Ex vivo gene therapy is another concept within cellular therapy, involving the genetically modification of target cells in vitro, which carry therapeutic characteristics and are reintroduced into the body.
Fig.1 An overview of the types of gene therapy techniques (Reghupaty S.C., et al., 2019)
Gene Therapy Delivery
Since gene editing technologies, including ZNFs, TALENs and CRISPR-Cas9 techniques, have become highly advanced in precision modification of disease-causing genes, the focus has shifted towards the development of safe and efficient gene delivery systems. On the basis of experience from multiple pre - and post-clinical trials, the delivery system must meet sufficient criteria to ensure that the efficacy of gene therapy is not compromised.
- The delivery system can be exempt from attacks by the immune system.
- The gene therapy drugs do not degrade in potency until they reach the target site.
- The delivery system is able to cross the cell membrane and enter the cell interior to function.
- The gene therapy drugs can be accurately expressed and sustained.
Prospectively, these challenges should be overcome in the development of gene therapy in order to improve the practical applicability of gene therapy for Autoimmune Diseases & Inflammation.
Our Services
Gene therapy has been proved to be highly effective and hundreds of drugs have entered clinical trials, but there is still a lack of development projects for Autoimmune Diseases & Inflammation. Our Company provides gene therapy development services with the support from multidisciplinary experts and utilization of various technological platforms, including but not limited as:
Gene Therapy Cargo Development
- DNA Therapies
- RNA Therapies
- Antisense Oligonucleotides
- CRISPR-Cas System
Gene Therapy Vector Development
- Viral Vectors
ADV, RV, AAV, LV - Non-Viral Vectors
Plasmids, Liposomes, Nanoparticles
Our Advantages
- Effective response to customer needs
- Strict execution and production standards
- Rapid trial turnaround time
- Professional and experienced technical team
Staffed by genetics experts and researchers from genetic engineering with solid foundation in developing gene therapies, our company is committed to providing you with professional services for the gene therapy development for Autoimmune Diseases & Inflammation. If you are interested in our services, please don't hesitate to contact us.
Reference
- Reghupaty, Saranya Chidambaranathan, and Devanand Sarkar. "Current Status of Gene Therapy in Hepatocellular Carcinoma." Cancers 11,9 (2019): 1265.