Advanced therapy medicinal products for local treatment using new delivery methods

Area of Science:

• Biomedical Engineering
• Molecular Therapy
• Regenerative Medicine

Background:

• Advanced therapy medicinal products (ATMPs), including gene and cell therapies, are increasingly approved, driving innovation.
• ATMPs use vectors to deliver genetic material for treating diseases, often involving ex vivo modification of autologous cells.
• Systemic delivery can cause off-target effects and toxicity, necessitating exploration of alternative methods.

Purpose of the Study:

• To review current knowledge on delivery methods for ATMPs.
• To discuss the advantages and disadvantages of various delivery approaches.
• To summarize clinical studies and future perspectives on localized therapy.

Main Methods:

• Literature review of ATMP delivery systems and materials.
• Analysis of therapeutic applications, considering disease, target tissue, and substance properties.
• Examination of clinical data and future trends in localized ATMP delivery.

Main Results:

• Delivery method selection is critical and depends on disease, target, and substance characteristics.
• Localized delivery offers potential for enhanced specificity, reduced toxicity, and improved therapeutic outcomes compared to systemic approaches.
• Various materials and vectors are employed for ATMP delivery, each with unique benefits and limitations.

Conclusions:

• Localized delivery strategies are crucial for optimizing ATMP efficacy and safety.
• Continued research into novel delivery systems and localized approaches will advance ATMP applications.
• The choice of delivery method significantly impacts treatment success and patient safety in gene and cell therapies.