Pancreatic cancer (PC) has become one of the cancers with the highest mortality rate due to its complex biology (e.g., high heterogeneity, inefficient drug penetration, and proliferative tumor microenvironment). Alfa Cytology aims to develop effective therapies for pancreatic cancer by creating targeted gene therapies to correct genetic defects and modify disease-causing and drug-resistant genes.
Overview of Gene Therapy
Gene therapy involves the introduction, removal, or alteration of genetic material within a patient's cells to treat or prevent disease. It can be used to replace a faulty gene with a functional one, inactivate a malfunctioning gene, or introduce a new gene to help fight a disease. This therapeutic approach utilizes vectors, often viruses modified to be safe, to deliver the genetic material into the cells. By targeting the genetic root of diseases, gene therapy offers the possibility of long-lasting and potentially curative treatments. Its applications extend to various conditions, including inherited disorders, some types of cancer, and certain viral infections.
Fig. 1 Schematic summary of the strategies of gene therapy for the pancreas. (Kamimura K, et al, 2018)
Approved Gene Therapies for Pancreatic Cancer
Although the number of approved treatments remains limited, significant progress has been made in gene therapy for pancreatic cancer. By focusing on specific gene mutations and pathways associated with pancreatic cancer, these treatments offer a customized approach that can improve efficacy and reduce side effects. Here are some of the approved gene therapies for pancreatic cancer:
| Gene Therapy Types | Description | Year Approved | Target |
|---|---|---|---|
| Oncorine (H101) | An oncolytic adenovirus-based gene therapy that selectively replicates within and lyses cancer cells, enhancing the immune response against tumors. | 2006 | Retinoblastoma pathway |
| Advexin (Gendicine) | A therapy that delivers a wild-type p53 gene to restore normal p53 function, promoting apoptosis in cancer cells with defective p53. | 2005 | p53 tumor suppressor gene |
| CovaMax | A drug designed to interfere with the expression of specific genes involved in stress responses, thereby reducing cancer cell survival under adverse conditions. | 2014 | Heat shock protein 27 (Hsp27) gene |
Our Services
Alfa Cytology provides a comprehensive gene therapy development service for pancreatic cancer, encompassing every stage from initial design to preclinical testing. This service includes vector design, therapeutic gene selection, gene editing techniques, and thorough preclinical evaluations to ensure the development of effective and safe gene therapies for pancreatic cancer.
Gene Therapy Types We Can Develop
DNA-based therapy utilizes viral vectors such as adenoviruses and lentiviruses to deliver therapeutic genes into the target cells. Technologies like CRISPR/Cas9 for precise gene editing and electroporation for enhanced DNA uptake are employed. This method ensures sustained gene expression and the ability to correct or silence defective genes.
RNA-based therapy involves the delivery of therapeutic RNA molecules directly into cells, avoiding genome integration. Technologies such as lipid nanoparticles for RNA delivery and synthetic mRNA for efficient protein translation are utilized. This approach provides temporary gene expression, reduces long-term risks, and allows for rapid development and production.
Nucleic Acid Drug Types We Offer
| Category | Structure | Target | Mechanism |
|---|---|---|---|
| Antisense oligonucleotides (ASO) | Single-strand DNA/RNA | -mRNA -Pre-mRNA -miRNA |
-Cleavage mRNA (RNase-H) -Exon skipping -Cleavage miRNA |
| Small interfering RNA (siRNA) | Double-strand RNA (hair-pine type is single-strand) | mRNA | Cleavage mRNA (RNA interference) |
| Circular RNA (circRNA) | Covalently closed single-stranded RNA (without free 5' and 3' ends) | -miRNA -Protein |
-miRNA sponge -Protein sponge -Translation template |
| MicroRNA (miR, miRNA) | Double-strand RNA (Pre-miRNA is single-strand) | mRNA | miRNA replacement (RNA interference) |
| Aptamer | Single-strand DNA/RNA | Protein (Outside of cells) | Inhibits the physiological effect |
Gene therapy offers a transformative approach to treating pancreatic cancer by addressing the genetic causes of the disease. Alfa Cytology's gene therapy development services are designed to advance this innovative field, offering targeted and effective treatment solutions. or more information and to explore collaboration opportunities, please do not hesitate to contact us.
Reference
- Kamimura K, et al. Gene Therapy for Pancreatic Diseases: Current Status. International Journal of Molecular Sciences. 2018, 19(11):3415.
