Quarterly Update
September 2026
It’s been a busy summer for the AUTS2 Research Collaborative. Over the past few months, our community has rallied behind an ambitious fundraising goal, researchers from around the world have come together around new scientific priorities and we’ve continued building the team, tools and partnerships needed to accelerate AUTS2 research.
Here’s a look at how far we’ve come — and what’s ahead.
$316,985 Raised
580
Donors
joined together to support AUTS2 research
80
Attended
The Future of AUTS2 Research Webinar
15
Researchers
came together to help shape our mouse model strategy
3
Team
members have joined ARC
since July 1 through AUTS2gether, surpassing our $300,000 goal
selected to advance to full review, representing $450,000 in proposed research
5
Applications
AUTS2gether: We Did It!
This summer, our community united to raise over $300,000 to accelerate AUTS2 research, surpassing our goal through fundraisers, donations, and spreading awareness. These funds will support up to five research projects tackling critical questions in AUTS2 biology and exploring potential paths toward future treatments.
Our Research Grants Move into Review
We've hit a major milestone with five full applications totaling $450,000 in proposed AUTS2 research, covering gene activation, DNA regulation, brain cell mapping, biomarker discovery, and stem cell models. These projects are now under scientific review, with funding decisions expected by year-end.
The Future of AUTS2 Research Webinar
Eighty participants joined our July webinar, "The Future of AUTS2 Research," featuring leading AUTS2 researchers discussing the current science and path toward treatments. The key takeaway: advancing therapies requires building a foundation of knowledge and research tools.
Building the Research Tools to Move AUTS2 Forward
We’re investing in the tools scientists need to better understand AUTS2 and advance potential treatments. We’ve funded three patient-derived stem cell (iPSC) lines and convened our AUTS2 Research Working Group to evaluate existing mouse models and identify gaps. We’re also exploring engineered cell lines to study specific AUTS2 mutations, building a shared research toolbox that can accelerate discovery across the field.
In the News
Awareness of AUTS2 is growing—and this quarter, stories from our community reached audiences in the United States and around the world.
Bulgaria
The Pochaleeva family's story was featured by NewsPoint.bg, highlighting their family's efforts to raise awareness and support AUTS2 research..
New York Daily News
ARC Founder Eliza Goldwasser called for earlier, more accessible genetic testing for children with unexplained developmental delays, highlighting the impact of receiving an earlier diagnosis.
CNBC
CNBC shared how an AUTS2 diagnosis led to the creation of ARC and its work to bring together families and researchers to accelerate progress toward treatments.
Spain
The Plana Usay family shared their daughter Beth's story and their efforts to raise funds for AUTS2 research, helping introduce AUTS2 to a broader audience in Spain.
Welcome to ARC
Kathryn Wagner MD, PhD
Medical Advisor, AUTS2 Research Collaborative
Kathryn is a physician-scientist and drug development leader with decades of experience in rare neurological disorders, including leadership roles at Johns Hopkins, Roche and Novartis. At ARC, she will help shape our clinical research strategy, including the development of our patient registry and natural history efforts to better understand AUTS2 syndrome and prepare for future clinical trials.
Interested in sharing your family's experience with Kathryn?
Reach out to info@auts2research.org
Cooley
We’re grateful to welcome Cooley as pro bono legal counsel to ARC.
As our research portfolio grows, so does the behind-the-scenes infrastructure required to move projects forward responsibly and efficiently.
Cooley is supporting ARC with agreements related to our research grants and scientific tools and resources, helping us build a strong foundation for continued growth.
What's Next
Reaching our $300,000 AUTS2gether goal is an incredible milestone. But it isn't the finish line.
Over the coming months, we're focused on three major priorities: awarding ARC's first research grants, advancing a new mouse model project and launching our revamped patient registry.
There is much more work ahead, and continued support will allow us to pursue promising opportunities as they emerge.
SAVE THE SEASON
Our first AUTS2 Family & Researcher Summit is coming in Summer 2027. We’re looking forward to bringing families, researchers and clinicians together in person to learn, connect and look ahead.