Genetic Medicines
The Genetic Medicines Hub works to develop genetic medicine therapeutics for specific genetic causes of autism, exploring versatile treatment approaches such as DNA and RNA editing.
Why Genetic Medicines?
What is Known
Several genetic variants associated with autism are rare with a large effect size, positioning them as good candidates for DNA or RNA editing-based interventions. This Hub is also poised to integrate learnings from other CNS conditions that are achieving success bringing genetic medicines to the clinic.
Challenge
Two barriers that have prevented widespread use of genetic medicines for genetic variants associated with autism include:
- Rare genetic variants do not readily attract commercial investment.
- Clinical trials often are not designed to include the infrastructure that would allow for robust recruitment of communities with rare genetic variants.
Opportunity
The Genetic Medicines Hub is leveraging AI-informed design to drive high-throughput screening of novel antisense oligonucleotides (ASOs) targeting rare, high-burden genetic variants. An ASO is a targeted therapeutic approach that modulates the levels of specific proteins associated with genetic disorders. Learnings will be integrated into a knowledge-sharing platform to drive the rapid identification and development of ASOs for future discoveries.
Integration
ARIA has been designed to remove silos and foster connections across disciplines. Discoveries and tools derived from the Genetic Medicines Hub will feed into other ARIA Research Hubs, including:
- Protein-Protein Interactions Hub
- Human Developmental Neurobiology Hub
- IMPACT Network
Funded Projects
The Genetic Medicine Hub is one of six research hubs within ARIA’s ecosystem. Projects within the Genetic Medicine Hub are conducted by collaborative, multidisciplinary research teams. These efforts will foster cross-disciplinary connections, fuel discovery, and accelerate the development of personalized therapies for autistic individuals and people with related neurodevelopmental conditions.
More information on funded projects will be available soon.
