Gene therapy, which involves treating diseases by deleting, silencing, or editing defective genes and delivering genetic material to produce therapeutic molecules, has great potential as a new approach to treating neuropsychiatric disorders. Ace Therapeutics possesses extensive knowledge of the latest and most crucial molecules and factors in gene therapy for neuropsychiatric disorders. We provide a comprehensive suite of services, including the provision of appropriate animal models, gene editing technologies, gene delivery methods, and psychiatric disease-related outcome measures, to assist clients in preclinical studies of genome editing for neuropsychiatric disorders and to accelerate the development of gene therapies for related disorders.

Introduction of Gene Therapy for Psychiatric Disorders

The molecular etiology of various neurological and neuropsychiatric disorders is mostly associated with changes in the genetic background, which can be inherited/triggered by other environmental factors. For example, depression, schizophrenia, autism spectrum disorders, and other psychiatric disorders are often associated with abnormalities or mutations in gene expression and are affected by the co-regulation of multiple genes. Therefore, gene therapy has important potential applications in the treatment of psychiatric disorders.

Gene therapy involves various methods of genetic manipulation, including the deletion, silencing, or editing of defective genes and the insertion of healthier genes. These approaches aim to maximize the fight against the onset and progression of mental disorders by inducing genetic alterations at endogenous gene loci to modulate neuronal function, adjust neurotransmitter levels, and influence the stability of neural circuits. Although gene therapy for psychiatric disorders is still in the research and experimental stage, its potential therapeutic effects have attracted widespread attention and are expected to become an important area for the treatment of psychiatric disorders in the future.

How We Can Help You Develop Gene Therapy for Psychiatric Disorders?

Ace Therapeutics is a preclinical contract research organization that specializes in psychiatric disorders. We offer comprehensive services and support to help clients explore this novel therapeutic area. Our team of experts has extensive knowledge in virology, neurology, neurosurgery, molecular genetics, and immunology. We offer personalized gene therapy development strategies, which include selecting gene editing technologies such as CRISPR/Cas9, establishing animal models, designing viral vectors, selecting intracerebral delivery strategies, and evaluating efficacy and safety. Our comprehensive service portfolio can assist clients in expediting their gene therapy research programs for psychiatric disorders.

• Target identification and validation

We use advanced bioinformatics tools and high-throughput screening techniques to identify and validate genes associated with specific psychiatric disorders. By understanding the functions, expression patterns, and regulatory mechanisms of these genes, we can identify potential gene therapy targets and achieve precision targeting by designing interventional drugs against these genes.

• Gene therapy vector design and optimization

We provide a one-stop service for gene therapy vector construction, modification, and optimization to ensure that the gene can be effectively delivered to the target cell and express the desired therapeutic protein or RNA.

• Delivery system development

We help our clients choose the right delivery system for optimal drug delivery through chemical modification and nanotechnology.

• Preclinical efficacy and safety assessment

We employ in vitro cellular models and in vivo animal models to comprehensively analyze and evaluate the efficacy, safety, and pharmacokinetics of potential antipsychotic drugs to identify the most promising candidates for your consideration.

Our Technologies and Capabilities

Our professional research team and state-of-the-art experimental platform allow us to provide high-quality R&D services and collaborate with you on the development of nucleic acid-based antipsychotic drugs.

• Behavioral and in vivo testing
• Biomarker development
• Electroencephalography
• Electrophysiological analysis
• Neuroimaging
• In vivo efficacy testing
• Molecular biology
• Immunological and histochemical analysis
• BSL2 Facility.
• Expertise in vector design, gene selection, and targeted delivery.

Ace Therapeutics will work closely with our clients to provide them with comprehensive solutions from early planning of gene therapy development to final data analysis and interpretation. Our goal is to help our clients explore this emerging therapeutic area and realize innovative breakthroughs in the treatment of mental illness. Please contact us for more details.

References

1. Iqubal, A.; et al. Gene therapy, a novel therapeutic tool for neurological disorders: current progress, challenges and future prospective. Current Gene Therapy. 20.3 (2020): 184-194.
2. Mani, S.; et al. Gene therapy, a potential therapeutic tool for neurological and neuropsychiatric disorders: applications, challenges and future perspective. Current Gene Therapy. 23.1 (2023): 20-40.