Gene Therapy for Genetic Disease

Approximately 350 million people worldwide suffer from genetic diseases, i.e. diseases caused by genetic abnormalities. Traditional therapies can only treat the symptoms but not the root cause. Advances in the field of gene therapy research have led to breakthroughs in the treatment of genetic diseases. CD Formulation, as a leading developer of gene therapy formulations, is committed to providing comprehensive technical support and solutions from target discovery to preclinical research for single-gene and multi-gene genetic diseases by focusing on the optimization of gene delivery systems and precision editing technologies.

Advantages of Gene Therapy for Genetic Disease

• A wider range of applications. Gene therapy applies to a wide range of genetic diseases, including single-gene diseases and polygenic diseases.
• Good long-term effects. With a single treatment, gene therapy often delivers lasting effects that last for a long time.
• Targeting the root cause. Directly repairing or replacing abnormal genes addresses the core of the genetic disease at its root, rather than just controlling symptoms.

Main Applications of Gene Therapy for Genetic Disease

Single-gene genetic diseases

Single-gene disorders (e.g., thalassemia, Duchenne muscular dystrophy) are caused by mutations in a single gene with a well-defined mechanism and are among the first breakthrough areas for gene therapy.

Advances in single-gene gene therapy research include gene replacement studies and gene editing. (Gene replacement is the delivery of normal genes via AAV viral vectors, e.g., for inherited retinopathies). Gene editing, i.e., direct correction of mutated sites using tools such as CRISPR/Cas9, has been studied for the treatment of β-thalassemia treatment has been achieved to take patients out of transfusion dependence.

In the development of gene therapy agents for monogenic genetic diseases, we can provide the following services.

• High precision vector design. Our self-developed AAV coat library can target specific tissues (e.g., liver, retina) to enhance efficacy and safety.
• cGMP production system. We provide scale-up production of viral vectors in compliance with GMP standards.

Polygenic diseases

Polygenic diseases (e.g. diabetes, hypertension) are affected by multiple genes and the environment, and the mechanism is complex, but gene therapy still shows potential. Current research progress includes intervening in the disease process by editing key pathway genes or epigenetic modifications, such as PCSK9 gene editing to lower cholesterol. As well as the use of engineered cell therapy such as CAR-T combined with gene editing for tumors or autoimmune diseases.

In the development of gene therapy formulations for polygenic genetic diseases, we can provide the following services.

• Artificial intelligence target prediction. Targeting core disease-causing gene networks based on multi-omics data analysis.
• Delivery system customization. Development of lipid nanoparticles (LNP) and novel viral vectors for multi-gene co-regulation.

Our Capabilities of Gene Therapy for Genetic Disease

• We have a team of experts and technologists with extensive expertise and years of research experience in gene therapy formulation development for genetic diseases.
• We can customize our gene therapy agent development services according to the characteristics of our clients' projects as well as their needs to ensure that each client's needs are fully met.
• We have strict quality control standards that cover all aspects of gene therapy formulation development.
• We have established a full chain of technology platforms covering vector design (AAV, lentivirus), gene editing tools (CRISPR, TALEN), scale-up production, and preclinical evaluation.

Explore Our Gene Therapy for Genetic Disease

CD Formulation provides specialized technical support and reliable solutions for the development of gene therapy formulations for genetic diseases. We look forward to working with you. If you are interested in us, please feel free to contact us.