Inhaled gene therapy is an innovative therapeutic approach that delivers gene drugs directly to the lungs through respiratory administration. CD Formulation has extensive research and service experience in the development of inhaled formulations for gene therapy, we have a strong understanding of the formulation process, quality control, and industrialization. We are familiar with the gene therapy formulation development process and have extensive experience in inhalation formulation development.

Advantages of Inhaled Gene Therapy Formulation

Inhaled gene therapy formulations are a method of treatment in which gene therapy drugs are delivered directly to the lungs by inhalation. This method has the following characteristics in gene therapy diseases.

• Direct drug delivery to the site of action. Inhaled gene therapy delivers the drug directly to the lungs, reducing off-target doses and systemic toxicity when treating lung diseases.
• Rapid absorption. Since the lungs have a large surface area and abundant blood flow, inhalation drug delivery can achieve rapid absorption, allowing the drug to enter the blood circulation quickly.
• Non-invasive route of administration. Inhalation administration is a non-invasive method that is safe and convenient for patients.
• Reduced side effects. By delivering the medication directly to the lungs, systemic side effects can be reduced, especially for diseases that primarily affect the lungs.

Our Services for Developing Inhaled Gene Therapy Formulation

• Target disease and gene selection

Identify the disease to be treated and the target gene. For example, targeting the CFTR gene for cystic fibrosis (CF).

• Gene vector design

Design of gene vectors suitable for inhalation drug delivery, e.g., using viral vectors or non-viral vectors (e.g., lipid nanoparticles LNP).

• In vitro experiments

Testing the efficacy and safety of gene vectors in cell culture to assess transfection efficiency and possible cytotoxicity.

• Animal model development

Development or selection of suitable animal models to mimic human diseases for initial in vivo testing.

• Preclinical research

Evaluate the safety, efficacy, dosage, and method of administration of gene therapy agents in animal models. This includes aerosolization, aerosol behavior in the respiratory tract, and post-deposition disposition processes.

• Formulation optimization

Optimization of gene therapy formulations for stability and transfection efficiency based on preclinical study results.

• Biodistribution and pharmacokinetic studies

In this segment, we study the distribution, metabolism and excretion characteristics of gene therapy formulations in vivo.

• Safety assessment

We will conduct a comprehensive toxicology study to evaluate the long-term safety and potential side effects of the gene therapy formulation.

Fig.1 Inhaled gene therapy formulation development process. (CD Formulation)

Applications of Inhaled Gene Therapy Formulation Development

• Rare respiratory diseases. Utilizing inhaled gene therapy has shown significant therapeutic efficacy in modifying rare respiratory diseases, and inhaled gene therapy formulations have broad applications in the treatment of respiratory diseases.
• Alpha-1 antitrypsin deficiency (AATD). Development of novel inhaled gene therapies aimed at promoting the local production of alpha-1 antitrypsin for the treatment of AATD patients.
• Congenital lung diseases. Examples include surface-active protein deficiency, cystic fibrosis, and alpha-1 antitrypsin deficiency. Inhaled mRNA delivery platforms can provide non-invasive, direct access to the lung epithelial cells and alveoli for RNA drugs, with the potential to treat these diseases.
• Pulmonary fibrosis. A novel inhaled nucleic acid nano-delivery carrier was developed to successfully deliver small interfering RNA (siRNA) to reach the lungs of fibrotic mice for the treatment of pulmonary fibrosis.

Our Technologies for Inhaled Gene Therapy Formulation Development

Highlights of Our Inhaled Gene Therapy Formulation Development

• Continuous innovation. We are always committed to the research of new gene therapy technologies and therapies to maintain our leading position in the field of inhaled gene therapy.
• Professional knowledge and technical expertise. We have a team of experts consisting of molecular biologists, pharmacologists, bioengineers, etc., focusing on the research and development of inhaled gene therapy formulations.
• R&D capabilities. We have strong R&D capabilities to design and develop novel inhaled gene carriers and delivery systems.
• Advanced technology platform. We have developed inhaled gene therapy formulations to provide more efficient and safer gene delivery technology support to our customers.

Published Data

CD Formulation is an industry leader in gene therapy research, and we provide technical services for the development of inhaled gene therapy formulations that rely on our advanced technology platform to effectively advance gene therapy research. If you are interested in us, please feel free to contact us.