FAQ

Aligning Research to Impact Autism (ARIA) is an initiative to accelerate scientific discovery and create more therapeutic options/opportunities for people with profound autism and people on the spectrum who seek additional support to improve daily life. ARIA connects emerging research, insights, and promising technologies from across scientific fields to build an integrated ecosystem that bridges clinical and translational research.

ARIA’s key programs include our Research Hubs, which support bold, cross-disciplinary research. The IMPACT Network is part of these Research Hubs and is designed to build clinical trial readiness. We also work closely with community collaborators who represent organizations, patient‑centered groups, and networks to ensure that the voices and lived experiences of individuals and families meaningfully inform ARIA’s research and clinical efforts.

We convened multidisciplinary working groups consisting of more than 150 leading scientists, researchers, and clinicians from across diverse research and clinical fields and individuals with lived experience to identify the most significant challenges hindering progress and promising scientific areas that can advance related research.

We are committed to discovering and advancing development of new and useful treatment options for people across the autism spectrum who are seeking care. We recognize there is also a pressing need to support people with profound autism and co-occurring or related neurodevelopmental conditions. For example, between 20-30% of autistic individuals require lifelong, 24/7 care. We have an opportunity to deploy translational research strategies, establish biomarkers, and develop new, effective therapies for autistic people who do not rely on speech alone to communicate and have high support needs.

We recognize and respect the power of language and the different preferences within the community of people living with, treating, studying, and researching autism. ARIA is using language and terminology that most clearly describe our mission and scientific goals.

ARIA is part of the CNS Quest, an initiative to accelerate discoveries, advance treatments and care, and improve the daily lives of people impacted by autism, Parkinson’s disease, bipolar disorder, and other CNS conditions—shortening the journey from lab to living well. The CNS Quest is a program of the Sergey Brin Family Foundation and Catalyst4.

Learn more about the CNS Quest.

Multiple programs will be implemented through the ARIA initiative, and they will roll out in stages. In 2026, ARIA will launch projects within all of the research hubs to initiate cutting-edge research that address high-priority research questions. In addition, sites will join ARIA’s IMPACT Network, a multisite clinical trial infrastructure.

The IMPACT Network is an international network of research and clinical sites working together to accelerate clinical trial readiness and develop new therapeutic opportunities for autistic individuals. Sites within the network will bring together expertise across disciplines to develop new outcome measures, clinical endpoints, and biomarkers that have the potential to transform clinical care.

Sites within the IMPACT Network are conducting a short-term natural history and clinical endpoint study, called the ARIA IMPACT-Ready Study, which aims to enable better understanding of how autism and related neurodevelopmental conditions develop and change over time. The study will encompass two cohorts.

• Cohort 1

  This group focuses on children and young adults (birth to 18 years) with genetic changes known to cause or contribute to autism who will be followed over time. This initially includes children and young adults with genetic variants (classified as pathogenic or likely pathogenic) in one of six genes strongly linked to autism (ARID1B, CHD2, GRIN2B, RNU4-2, SCN2A, and SLC6A1). Importantly, children qualify for this cohort based purely on their genetic variant; a formal diagnosis of profound autism is not required to participate.

• Cohort 2

  This group focuses on autistic children and young adults (ages 2 to 18 years) who have likely profound or profound autism, as defined by using few or no words to communicate, needing substantial and consistent support (often 24/7), and who do not have a definitive genetic cause for their profound autism at the time of enrollment. This cohort was created specifically to include autistic children and young adults with significant communication challenges and who have intensive support needs—individuals who have too often been left out of research. Whole-genome sequencing will be performed as part of the study, and if a definite genetic cause for autism cannot be identified, this cohort will be followed over time (Cohort 2a).

  After whole-genome sequencing, some children and young adults may be found to either have a genetic variant known to cause or contribute to autism that is not within the six genes of focus in Cohort 1, or they may not meet the study definition of profound autism. Meaningful genetic, clinical, and biomarker data will be collected from these participants at the baseline visit, but this cohort (Cohort 2b) will not be followed over time.

The ultimate goal of the ARIA initiative is to create more therapeutic options for people on the spectrum who seek additional support. To bring a novel therapeutic into the clinic, it must undergo rigorous testing through a clinical trial. Successfully executing a well-designed clinical trial that provides an answer to whether a drug or behavioral intervention should advance to clinical use requires many components, including validated outcome measures (tools to gather data on the population), biological markers (biomarkers), and effective ways to engage families and children.

In the field of autism, meaningful and measurable outcomes and endpoints are limited. A natural history study is an important first step toward novel therapeutics, providing researchers with outcome measures and endpoints needed for future clinical trials.

A short-term natural history and clinical endpoint study that collects data on the same individuals over time provides researchers, clinicians, and patient populations with a picture of baseline condition and natural change or progression, without interventions. These baseline measurements and the natural trajectories are crucial for studying and testing novel therapeutics.

We will begin our natural history studies by focusing on pathogenic variants in six genes strongly associated with autism, with plans to expand to additional genetically defined causes of autism and related neurodevelopmental conditions. Our initial focus will be on genes for which natural history data are currently limited, with the long-term goal of incorporating other genetic conditions as therapeutic targets emerge.

The six genes included in Cohort 1 were selected as a starting point, with the eventual goal to broaden inclusion over time as the IMPACT Network evolves. These genes were selected based on a framework that prioritized genes with strong evidence of association with autism, an understood genotype–phenotype relationship, and features that could be measured in clinical trials.

Genes were also selected to ensure sufficient patient numbers with established patient advocacy groups and to address a need for “trial-ready” multidimensional longitudinal natural history data that bridges caregiver reports with in-clinic assessments, quantitative phenotyping, and biomarker analyses. By focusing on genetic conditions that need these “trial-ready” natural history data, the IMPACT-Ready Study can establish the baseline infrastructure needed to scale research for these underserved populations, creating a foundation that can be extended to many more genetic conditions over time.

• Natural History: Our study protocol will comprehensively evaluate participants across a variety of developmental domains in order to understand variability in skills and abilities, as well as how these domains evolve as children age. These domains are of importance to the autism and neurodevelopmental disability communities and include Cognition, Language and Communication, Autism Symptoms, Motor Skills, Adaptive Behavior, Motor Skills, Behavioral Challenges, and Medical Concerns (including GI, epilepsy, and sleep). An important study takeaway will be insights into how these domains might evolve as children age.
• Outcome Validation: Currently, no validated outcome measures are available for the domains included in the study. The study protocol will allow us to assess what can be reliably validated.

A rising tide lifts all boats. Every family who participates—in any cohort—is contributing to something larger than any single condition. By deeply studying the initial genes in Cohort 1, we are uncovering shared biological pathways and perfecting measurement tools that can be applied across the entire autism spectrum. A discovery for one condition creates a wave of progress that accelerates answers, clinical readiness, and targeted support for everyone.

Participation also helps researchers gather information needed to understand how autism develops over time. This knowledge is essential for identifying the outcomes measures and end points that will help inform clinical trials that study new therapies and supports.

No, the IMPACT-Ready study will not include treatments. However, what we learn will help inform the development of future interventions, services, and improvements to clinical care.

The study will operate for 5 years, and individual participants will complete 1-5 in-clinic visits (up to ~2 years).

Enrollment within the study will occur in multiple waves starting in Fall 2026. Additional information will be made available at that time.

Yes, participation in online registries and most other research studies can happen at the same time as enrolling in the IMPACT-Ready study. Some research studies that involve therapeutic intervention may make participation in the IMPACT-Ready study more challenging. Study site staff will be best able to evaluate any family’s eligibility.

Every research study and registry is unique in the populations it studies, the data it collects, and the opportunities it offers families. Each provides its own value to the scientific, clinical, and patient communities. ARIA aspires to build on the learnings from these registries and initiatives to develop opportunities for clinical trial readiness and, in the longer term, interventional clinical trials through the ARIA IMPACT Network. These efforts, together, support a shared goal of advancing clinical trial readiness across communities.

ARIA has conducted landscaping discussions with many patient advocacy groups, community collaborators, and patient leaders in the profound autism and related neurodevelopmental condition space for several years. ARIA has also engaged a team of clinical and scientific experts, who have worked with patient communities for decades, to lead ARIA efforts. ARIA has also assembled a Community Advisory Committee. These insights informed the development of the IMPACT Network, IMPACT-Ready study, and the ARIA Research Hubs. ARIA will continue to develop long-term opportunities to collaborate with patient advocacy groups to ensure perspectives and feedback are heard. Visit the ARIA website to join our mailing list and receive updates.

The Aligning Research to Impact Autism (ARIA) initiative is committed to fostering an efficient and trustworthy research ecosystem that accelerates autism research. ARIA promotes open science practices to maximize research impact and reusability. The ARIA Open Science Policy ensures that ARIA-funded research outputs are shared throughout the research lifecycle, thoroughly documented, and made as accessible as possible while respecting ethical considerations and data protection requirements.