image: Dr. Paul Donlin-Asp of the University of Edinburgh
Credit: Dr. Paul Donlin-Asp and CURE SYNGAP1
Mill Valley, CA – February 3, 2026 – CURE SYNGAP1 (fka SynGAP Research Fund), a 501(c)(3) organization, announces a $130,000 grant to Dr. Paul Donlin-Asp, PhD, of the Simons Initiative for the Developing Brain (SIDB) at The University of Edinburgh. The grant supports his work in investigating the molecular functions and regulation of local SYNGAP1 protein synthesis, with the goal of advancing therapies for SYNGAP1-Related Disorders (SRD).
Why We Supported This Project
Dr. Donlin-Asp’s research focuses on a critical aspect of SRD: the production of SYNGAP1 protein at
synapses, which plays a key role in regulating synaptic strength and plasticity—both essential for learning and memory. Disruptions in SYNGAP1 protein levels lead to the cognitive and developmental challenges observed in individuals with SRD.
This project seeks to map the mechanisms that control the regulation of SYNGAP1 protein in neurons, including the synthesis, location and duration of SYNGAP1 protein. By understanding these molecular processes, Dr. Donlin-Asp aims to identify specific therapeutic targets that could increase SYNGAP1 protein production from existing mRNA, potentially boosting protein levels in affected individuals. This research could lay the foundation for therapeutic strategies aimed at mitigating the symptoms of SRD by restoring proper synaptic function.
While this project focuses on uncovering the fundamental mechanisms that govern SYNGAP1 protein synthesis, its long-term potential is clear: to provide a pathway for developing targeted therapies that address SYNGAP1 protein deficiencies at their source. In doing so, this research brings us closer to CURE SYNGAP1’s ultimate goal—finding effective treatments, and eventually, a cure for SYNGAP1-Related Disorders.
Building on Groundbreaking SYNGAP1 Research
Dr. Paul Donlin-Asp is a prominent researcher at the Simons Initiative for the Developing Brain at the University of Edinburgh. His prior work has explored the regulation of protein synthesis in neurons, with a particular focus on SynGAP and its critical role in synaptic function. Similar work funded by CURE SYNGAP1 has also aimed to dissect the complex mechanisms underlying SRD, positioning this project as a continuation of CURE SYNGAP1’s commitment to supporting groundbreaking research in this field.
This project is the latest effort in CURE SYNGAP1’s mission to fund cutting-edge research, building on past initiatives that have provided valuable insights into the role of SYNGAP1 in neurodevelopmental disorders.
Perspectives from the CURE SYNGAP1 Community and Collaborators
Lindsay Wieczorek, PhD, CURE SYNGAP1 Board member and former Scientific Director, says, “Understanding the mechanisms behind SYNGAP1 protein synthesis and regulation in the brain is essential for unlocking potential therapies for SYNGAP1-Related Disorders. Dr. Donlin-Asp’s research explores how localized protein production at neural connections supports memory and learning by fine-tuning brain connectivity. By unraveling the molecular processes governing SYNGAP1 levels, this work could be used to develop novel strategies that restore normal cognitive function, offering hope for transformative treatments in neurodevelopmental disorders.”
Kathryn Helde, Chief Scientific Officer, is excited to see the work that comes from this project. “Dr. Donlin-Asp has a fantastic track record of showing patient-relevant findings that give insights to disease mechanisms. His work is collaborative and aims to understand questions that affect patient health.”
“The University of Edinburgh continues to be a centre of excellence in the UK, and the work that they do continues to offer hope to the SYNGAP1 community in the UK and beyond,” shared Julie Pender, Chair of CURE SYNGAP1 UK. “CURE SYNGAP1’s support of this research makes a big difference to those of us navigating SYNGAP1 here in the UK, knowing that our fundraising efforts will help bring a more positive future for our Syngapians.”
“This funding from [CURE SYNGAP1] is pivotal in advancing our understanding of how SYNGAP1 protein synthesis is regulated at the molecular level,” says Dr. Donlin-Asp. “By unlocking these intricate mechanisms, we aim to uncover novel strategies to enhance SYNGAP1 protein production—laying the groundwork for potential therapeutic interventions that could transform the lives of individuals affected by SynGAP-related intellectual disability.”
Family Donations Make Progress Possible
“SYNGAP1 families and our dedicated donors aren’t just waiting for progress—they’re making it happen,” said CURE SYNGAP1 President and Chief Operating Officer, Virginie McNamar. “Their commitment to fundraising and supporting our mission allows us to invest in critical research that drives real change. This grant is a direct result of their efforts, and we are deeply grateful for their support in pushing SYNGAP1 science forward.”
About the Simons Initiative for the Developing Brain (SIDB)
SIDB is a research center based at the University of Edinburgh, bringing together around 40 principal investigators across six research sites to understand the fundamental basis of monogenic neurodevelopmental disorders characterized by autism, intellectual disability and epilepsy. SIDB was founded in 2017, following a generous donation from the Simons Foundation Autism Research Initiative (SFARI).
SIDB partners with the Patrick Wild Centre, founded in 2010, with the mission of understanding the neurological basis of and developing new therapeutic intervention for monogenic forms of autism, and intellectual disabilities. A key research and clinical focus of both SIDB and the PWC is SYNGAP1 haploinsufficiency.
About SYNGAP1-Related Disorders (SRD)
SYNGAP1-Related Disorders (ICD-10 F78.A1) are a rare genetic disorder caused by variants on the SYNGAP1 gene that reduce SYNGAP1 protein levels. CURE SYNGAP1 has identified over 1,707 SRD patients to date, and the number grows weekly. This protein acts as a regulator in the synapses (where neurons communicate with each other). When SYNGAP1 protein levels are too low, we see an increase in excitability in the synapses making it difficult for neurons to communicate effectively. This leads to many neurological issues seen in SRD patients.
Symptoms of SRD include primarily neurological issues including autism spectrum disorder (ASD), intellectual disability, epilepsy, hypotonia (low muscle tone), gross and fine motor delays, global developmental delay, and visual abnormalities such as strabismus (crossed eyes) as well as gastrointestinal challenges and disordered sleep.
About CURE SYNGAP1’s Seven Scientific Programs
CURE SYNGAP1 has seven scientific programs. These programs reflect their urgency to develop disease modifying treatments. These include the following:
- BTS – Basic and Translational Science
- Purpose – Drug Repurposing
- SMART – SYNGAP1 Missense Analysis, Research & Therapeutics
- SBOM – SYNGAP1 Biomarkers & Endpoints
- Facilitate – Develop and Share Patient-relevant Research Tools and Reagents
- SRDC – SYNGAP1-Related Disorders Characterization
- ProMMiS: Prospective Multidisciplinary, Multisite Study for Clinical Excellence – Natural History Study at Multidisciplinary Clinics
This grant falls into the BTS program. Basic Science reveals what goes wrong in tissues and cells when a SYNGAP1 gene is broken. Translational Science is the designing and testing of different types of medicines for SRD. CURE SYNGAP1 has funded over $3M in BTS grants.
About CURE SYNGAP1
CURE SYNGAP1 improves the quality of life for SYNGAP1 patients through the research and development of treatments, therapies, and support systems.
CURE SYNGAP1 was founded in the US in 2018 as a 501(c)(3) US public charity. There are sister organizations founded by local families in the UK in 2020, Europe (the Netherlands) in 2022, as well as both Australia & Latin America (Colombia) in 2023. Completely family-led, CURE SYNGAP1 is a leading funder of SYNGAP1 research having committed over $8 million in grants as of December 31, 2025.
CURE SYNGAP1’s grant program awards pilot, one-year and two-year grants to researchers and clinicians studying SYNGAP1. Their mission is to accelerate the availability of safe and effective treatments that meaningfully modify SRD to reduce suffering for patients and their families. Current funding priorities include essential milestones for clinical trial readiness. You can learn more about CURE SYNGAP1 and their accomplishments by reading their most recent Impact Report.
For more on CURE SYNGAP1, visit curesyngap1.org or follow @cureSYNGAP1 on LinkedIn, YouTube, Instagram, Facebook, TikTok, and X.
CURE SYNGAP1 (fka SynGAP Research Fund) is a member of FasterCures, COMBINEDBrain, Global Genes Foundation Alliance, Everylife Foundation Community Congress, Epilepsies Action Network, Personalized Medicine Coalition, Rare Epilepsy Network, Epilepsy Leadership Council, Alliance for Genetic Etiologies in Neurodevelopmental Disorders and Autism (AGENDA), California Action Link for Rare Diseases, American Brain Coalition, Genetic Alliance UK, Rare Disease UK, Syndromes Without a Name (SWAN UK), Jumpstart Program, Patient Worthy, Autism Brain Net, Innovation and Value Initiative, Rare Disease Diversity Coalition, Cambridge Rare Disease Network, Breaking Down Barriers, Rare-X, Mencap, IndoUSRare, The World Orphan Drug Congress, and Research America.