AUTS2 Research Collaborative
Summer 2026 Quarterly Update
Launched
April 24, 2026
Convened 14 leading researchers
By the Numbers
Raised
$100,000+
Connected with
30+ families
Achieved 501(c)(3) Status
Funded 3 patient‑derived
iPSC lines
Celebrate AUTS2 Awareness Day
AUGUST 2, 2026
We're excited to celebrate our first‑ever AUTS2 Awareness Day this August. Throughout July, families and supporters around the world will come together to raise awareness, share their stories, and support research through our AUTS2gether fundraising campaign. Funds raised through AUTS2gether will directly determine how many research projects we can fund this year — more on that below.
Upcoming Events
As part of the AUTS2gether campaign, we'll also be hosting a series of virtual events to learn, connect, and engage with the AUTS2 community. Our first three events are outlined below.
NAVIGATING THE HEALTHCARE SYSTEM & SELF‑ADVOCACY
Tuesday, July 14 at 12:00 PM ET
Dr. Alice Veksler — a public health expert and AUTS2 mom — will share practical strategies for preparing for appointments, communicating with providers, navigating insurance, and advocating for your family. We'll be collecting questions in advance
EXPLORING THERAPEUTIC
PATHS FOR AUTS2
Date and Time TBD
Researchers will discuss:
• CRISPRa genetic medicine approaches to increase AUTS2 expression
• Drug repurposing strategies for AUTS2
BUILDING THE FOUNDATION FOR FUTURE AUTS2 TREATMENTS
Date and Time TBD
Researchers will discuss:
• Patient‑derived stem cell models
• Biomarkers to measure treatment response
• New insights into how AUTS2 functions across different brain cell types
What We’ve Been Up to Official Launch & 501(c)(3) Status
APRIL 24, ARC OFFICIALLY LAUNCHED
The response was immediate. In less than six weeks, this community raised $100,000. We're also thrilled to share that ARC has received official 501(c)(3) nonprofit status (EIN: 41‑5081067). While donations have always been tax‑deductible through our fiscal sponsor, Rare Village Foundation, ARC is now an independent nonprofit — meaning you can give directly to ARC, and Donor‑Advised Funds can now be directed to us as well.
What’s on the Horizon
FAMILY ADVISORY COUNCIL
We're launching a global Family Advisory Council. Local ambassadors will help rally AUTS2 families in their communities, ensure family perspectives shape our research priorities, and represent the interests of families in their regions. Interested in serving as a representative where you live? We'd love to hear from you.
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Since launch, ARC has convened two international Working Group meetings, bringing together 14 leading researchers and clinicians from institutions across the US, Europe, Israel, and China.
The first meeting focused on developing shared tools for the AUTS2 community. Researchers' input directly shaped ARC's decision to fund three patient‑derived iPSC lines through NeuCyte — license‑free and expected to be broadly available to investigators worldwide by the end of 2026.
The second meeting focused on drug repurposing, reviewing findings from an analysis of more than 40,000 compounds conducted through ARC's partnership with Unravel. These findings have helped identify promising next steps, and ARC is actively exploring opportunities to expand this work.
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On May 16th, we hosted our first Family Virtual Launch. Families tuned in from across the US, UK, Europe, Asia, and the Middle East — some in the middle of their night. The eagerness to connect, contribute, and be part of something larger than their own family's experience was palpable. We are so grateful to everyone who joined.
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Our inaugural grant cycle recently completed its LOI review, and we were energized by the quality and diversity of applications. Next month, we move to full applications — and how many grants we're ultimately able to fund will depend directly on what we raise through AUTS2gether this summer.
Proposed projects include research into potential AUTS2 therapies including CRISPRa, patient‑derived stem cell models, biomarkers for future clinical trials, and fundamental AUTS2 biology. ARC will continue accepting Letters of Intent on a rolling basis.
SAVE THE DATE: FIRST FAMILY MEETING — SUMMER 2027
ARC's first in‑person gathering will take place in Summer 2027, bringing together researchers, clinicians, and families. Hear the latest research, connect with other families, and celebrate how far we've come. Interested? Complete the interest form below to help us plan.
Community Announcements
Have EEG Results to Share
Researchers are exploring whether AUTS2 has a distinctive EEG signature that could serve as a valuable biomarker for future drug development and clinical trials. If your child has completed an EEG, we'd love to hear from you.
Interested in Volunteering
AUTS2 families bring an extraordinary range of skills and experience. Whether you have professional expertise, lived experience, language skills, or simply time and enthusiasm, there are meaningful ways to contribute. Reach out to us to get involved!
Thank You
Everything we've accomplished in these first two months has been made possible by this community. We're just getting started — and we couldn't be more grateful to be doing this work alongside you.
With gratitude,
Eliza and Ethan Goldwasser and the ARC Team