Gene Therapy Drug Formulation Development

BOC Sciences offers a comprehensive range of gene therapy formulation development services, from custom design to mass production to meet the specific needs of gene therapy. Each service is designed to provide customers with targeted, compliant and scalable solutions.

What is gene therapy?

Gene therapy agents can be defined as the development of delivery systems to ensure that genetic material reaches target cells efficiently and safely. Unlike traditional medicines, composed of small molecules or biologics, gene therapy deliberately introduces nucleic acids (including DNA, RNA, and oligonucleotides) into cells and has special formulation requirements.

Types of gene therapy

There are two major approaches in gene therapy: in vivo gene therapy and ex vivo gene therapy.

In vivo gene therapy is the direct administration of therapeutic genes or gene-editing tools, such as the CRISPR/Cas9 system, into the patient. In this case, a viral vector, such as an adeno-associated virus (AAV) or adenovirus, can be used to deliver a copy of the target gene to the cell type of interest. One of the main advantages of this method is that it is easy to operate and can be directly applied to the target tissue, especially in cases where samples are not available and traditional methods may not be applicable. Applications of in vivo gene therapy include visual organs, muscular dystrophy, and certain vascular diseases.

Ex vivo gene therapy involves removing cells from the patient, performing genetic manipulation in the lab, and then replanting the modified cells back into the patient. It is commonly used in blood diseases, some cancers and immune deficiency diseases. Genetic modification can be carried out in a highly controlled environment, reducing untargeted effects and improving the accuracy of treatment. At the same time, ex vivo culture helps to expand the number of cells, thus improving the efficiency of treatment.

Schematic of different gene therapy approach. (Henderson, M. L., 2024)

Our gene therapy drug formulation services

Custom formulation design

BOC Sciences specializes in custom formulation design to provide personalized solutions to the unique needs of gene therapy. Whether the target is rare genetic diseases, oncology, or regenerative medicine, our team works closely with our customers to develop agents that optimize treatment outcomes. Through collaborative research, we design formulations based on nucleic acid type, disease indication and required delivery mechanisms to ensure precise targeting and therapeutic effectiveness. FDA approved gene therapy products include injections, oral solutions, topical gels, and we offer a variety of dosage form development services.

• Injectable liquid formulations development
• Oral liquid dosage form development
• Semi-solid dosage form development
• Solution Formulation Development Services
• Gel Development Services
• Nanogel Drug Delivery System Development

Excipients and additives to enhance delivery

The efficacy of gene therapy preparations often depends on the scientific combination of excipients and additives. BOC Sciences offers a selection of excipients designed to improve drug delivery, absorption and shelf life. For example:

Stabilizers are used to prevent nucleic acid degradation and extend the life of the preparation.

Osmotic enhancers facilitate cellular uptake of genetic material.

The protective agent resists environmental degradation and ensures that the preparation remains effective during use.

Analytical characterization

Accurate analytical characterization is critical for safe, high-quality gene therapy development. BOC Sciences uses a variety of advanced analytical methods to assess purity, stability and potency, including:

High performance liquid chromatography (HPLC) is Used to assess purity and consistency.

Enzyme-linked immunosorbent assay (ELISA) quantifies proteins and detects potential contaminants.

Polymerase chain reaction (PCR) technology is used for gene sequence verification and stability analysis.

Stability and compatibility testing

BOC Sciences performs rigorous stability and compatibility tests to verify the long-term viability of gene therapy agents. These tests assess the interaction of the formulation with container materials and delivery systems to ensure compatibility and reduce the risk of degradation. Long-term stability studies also assess the impact of factors such as temperature and humidity to provide customers with insights into optimal storage conditions, thereby extending the shelf life of products.

Sterilization and aseptic processing

In gene therapy, maintaining sterility is crucial to prevent contamination and ensure product safety. BOC Sciences uses rigorous sterilization and aseptic handling techniques designed to protect sensitive genetic material while meeting safety standards. By optimizing the sterilization method, we effectively prevent microbial contamination and improve the overall quality of the product.

Process development and scale production

Transforming laboratory-scale preparations into commercially viable products requires efficient process development and large-scale production. BOC Sciences provides end-to-end support to help gene therapy preparations comply with good manufacturing practice (GMP) standards during scale-up. Our services cover process optimization, scale-up strategies and technology transfer, enabling customers to smoothly transition from research and development to mass production.

Regulatory support and document preparation

When it comes to regulatory compliance for gene therapy, comprehensive documentation and an in-depth understanding of international standards are essential. BOC Sciences provides extensive regulatory support to assist clients in preparing documentation for compliance with FDA and EMA guidelines. Our regulatory experts provide guidance on documentation requirements, quality control programs and compliance measures to help customers confidently bring their products to market.

Advanced technologies in gene therapy formulation

Nanotechnology for targeted delivery

Nanotechnology can improve cellular uptake and the precision of targeting. The encapsulation and delivery of genetic materials directly into cells are accomplished with a range of nanovehicles, including LNPs, polymeric carriers, and liposomes, by BOC Sciences. These Nano-sized carriers can provide great delivery to the cell with precise targeting, enabling highly efficient transportation of genetic materials into the cell with reduced off-target effects and immune responses.

• Nano formulation development service
• Lipid nanoparticle development services
• Polymer nanoparticles development services
• Nanoparticle drug delivery system modification
• Liposomes

CRISPR/Cas systems and gene-editing formulations

With the continuous improvement of techniques in gene editing, the CRISPR/Cas system has become another extremely important strategy in therapeutic development. BOC Sciences offers optimized formulations for CRISPR component delivery and stability that could enable precise gene editing for a wide range of applications. The integration of CRISPR with state-of-the-art delivery systems gives us the opportunity to perform targeted genetic modifications efficiently with minimal side effects.

High-throughput screening for optimal conditions

HTS of various conditions rapidly identifies the optimum environment for gene delivery. BOC Sciences applies HTS to optimize parameters such as concentration, temperature, and pH to ensure the efficacy and stability of the gene therapy formulations.

Why choose BOC Sciences?

BOC Sciences represents an unparalleled combination in scientific, technical, and regulatory support for the formulation development of gene therapy.

With decades of professional experience from our team, each formulation is tailored to meet the high demands placed on this application area in gene therapy.

From initial development through large-scale production, at BOC Sciences, clients are guaranteed reliable, high-quality, fully compliant gene therapy solutions.

FAQ

1. How does gene therapy work?

There are a few ways gene therapy may work: it can replace a mutated gene with a healthy copy, inactivate a gene if it is malfunctioning when mutated, or introduce a new gene to the body to help fight a disease.

2. What diseases does gene therapy treat?

Nowadays, gene therapy is applied to a wide range of diseases that include genetic disorders: cystic fibrosis, hemophilia, muscular dystrophy, and certain cancers.

3. What is the difference between formulations used in gene therapy and those of 'classical' drugs?

Gene therapy formulations contain nucleic acids, which are sensitive to environmental conditions and, moreover, require targeted delivery, while the 'classical' small molecule drugs do not have to penetrate cells nearly as precisely.

4. What role do excipients play in the formulation of gene therapy?

Excipients enhance stability, increase cellular uptake, and protect genetic material during storage and delivery, all important to maintaining therapeutic efficacy.