Autism Spectrum Disorder (ASD)
The central therapeutic challenge in autism spectrum disorder (ASD) lies in developing treatments that effectively address the core social communication deficits and restrictive behaviors. With our deep expertise in ASD therapy development, Protheragen is well positioned to provide tailored solutions and comprehensive support to facilitate your journey from ASD therapy research to commercialization.
Overview of Autism Spectrum Disorder (ASD)
Autism spectrum disorder (ASD) is a complex neurodevelopmental condition characterized by persistent deficits in social communication and interaction, alongside restricted, repetitive patterns of behavior, interests, or activities. Symptoms typically emerge in early childhood and result in significant functional impairment. The disorder exhibits high heritability, involving polygenic risk and rare pathogenic variants in genes affecting synaptic function. Prevalence is estimated at ~1 in 54 children, with a male predominance (4:1), and frequent comorbidities include intellectual disability, epilepsy, and anxiety.
Fig.1 Risk factors and phenotypes of autism spectrum disorder (ASD). (Zhuang H, et al., 2024)
Pathogenesis of Autism Spectrum Disorder (ASD)
The pathogenesis of autism spectrum disorder (ASD) is primarily driven by a complex interplay of highly heritable polygenic risk factors and rare deleterious mutations in neurodevelopmental genes (e.g., SHANK3, *NLGN3/4*, FMR1), which converge to disrupt synaptic formation, function, and plasticity, leading to altered brain connectivity and an imbalance between excitatory and inhibitory neurotransmission.
Fig.2 Potential influences of autism spectrum disorder (ASD). (Zhuang H, et al., 2024)
Therapeutic Development for Autism Spectrum Disorder (ASD)
| Drug Names | Mechanism of Action | Targets | Research Phase |
| Risperidone | Atypical antipsychotic; antagonizes dopamine D2 and serotonin 5-HT2A receptors | Dopamine D2 receptor, Serotonin 5-HT2A receptor | Approved |
| Aripiprazole | Atypical antipsychotic; functions as a partial agonist at dopamine D2 and serotonin 5-HT1A receptors | Dopamine D2 receptor, Serotonin 5-HT1A receptor | Approved |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
Specializing in comprehensive solutions for autism spectrum disorder (ASD), Protheragen integrates diagnostic development with cutting-edge in vitro diagnostic (IVD) kits for early disease identification. Our therapeutic development pipeline leverages exclusive disease models, such as physiologically representative blood-brain barrier (BBB) models, to expedite the exploration and authentication of central nervous system (CNS) targeted treatments.
Therapeutic Development Services
By Mechanism of Action
Disease Model Development Services
In Vitro Model Development
- Mecp2 Knockout Model
- Fmr1 Knockout Model
- Shank3 Knockout Model
- Tsc1 or Tsc2 Knockout Model
- UBE3A Overexpression Model
- Other Models
To advance the commercialization of novel therapies for autism spectrum disorder (ASD), Protheragen provides comprehensive preclinical research services, covering pharmacodynamics (PD), pharmacokinetics (PK), and toxicology studies. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Zhuang H, Liang Z, Ma G, et al. Autism spectrum disorder: pathogenesis, biomarker, and intervention therapy[J]. MedComm, 2024, 5(3): e497.