January 29 2024, 14:00 GMT
Aparito is pleased to welcome SynGAP Research Fund and Dravet Syndrome Foundation Spain to the Patient Group Accelerator Programme to co-develop new digital biomarkers (BMKs) and electronic clinical outcome assessments (eCOAs) to accelerate drug development in Syngap1 and Dravet syndrome.
Aparito launched the Accelerator in 2020 to provide an initiative where a tech company and the patient community, with the support of patient advocacy groups, can collaborate as peers.
Under the Accelerator framework, Aparito and patient advocacy groups work in partnership to understand and ideally fulfil patients’ needs in terms of measuring what matters most to patients and co-develop novel digital biomarkers/eCOAs.
We will co-develop a prototype, using Aparito’s Atom5™, to test the usability of the digital technology platform and potential future utility to capture and analyse patient-relevant digital BMKs to truly embed the patients’ voice into drug development tools that could lead to the development of medicines targeting unmet patient needs.
We are very pleased to see the consolidation of the Accelerator programme with the addition of two organisations as committed and passionate as Dravet Syndrome Foundation Spain and Syngap Research Fund. We hope to further advance the field of paediatric rare epileptic encephalopathies by co-developing new digital outcome measures to be used in future clinical drug development programmes.
Dr Elisa Ferrer Mallol, Head of Patient-led eCOAs at Aparito
We are very excited to collaborate with Aparito on developing new digital biomarkers and electronic clinical outcome assessments. Therapeutic development efforts are ongoing in various biopharma and academic labs – we expect to see trial starting in the near future, and this work is critical to finding potentially new, non-seizure based, endpoints to improve overall clinical trial readiness and accelerate drug development efforts for SYNGAP1-related intellectual disability.
Michael Graglia, Managing Director at SynGAP Research Fund
Through this project, we seek not only to facilitate drug development but also to address the unmet needs of those affected by Dravet syndrome. This initiative is instrumental in capturing the multifaceted nature of Dravet syndrome. Specifically now, as we delve into the potential of disease-modifying therapies, it becomes imperative to broaden our scope beyond merely assessing seizures, and it is necessary to have tools to evaluate these other symptoms. Together, we aim to change the landscape of clinical trials, paving the way for more patient-centric approaches and ultimately improving the lives of individuals facing Dravet syndrome.
Simona Giorgi, Scientific Director at Dravet Syndrome Foundation Spain
About Aparito
Aparito’s mission is to digitise clinical trials and accelerate drug development for patients with life-limiting diseases by supporting patient-centric clinical trials with innovative treatments.
Clinical trials can be conducted within patients’ homes by leveraging our Atom5™ platform incorporating disease-specific smartphone apps, video assessments and wearable technology to digitise clinical trials using novel eCOA and generate continuous real-world data.
Aparito’s patient-generated data platform is disease-agnostic and scalable, ready for rapid deployment in global rare disease studies.
Discover more about how we digitise clinical trials on LinkedIn, Twitter and at aparito.com.
About SynGAP Research Fund
The mission of the SynGAP Research Fund (SRF) is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies and support systems.
SRF was founded in the US in 2018 as a 501(c)(3) US public charity, and families created sister organizations for SRF in the UK in 2020, in Europe (Netherlands) in 2022, and in Latin America (Colombia) in 2023.
Completely parent-led, SRF is the largest non-government funder of SynGAP research having committed over $5 million in grants. The founders cover operational costs, ensuring donations fund science & patient-related programs. SRF’s grant program awards one or two-year grants to investigators, physician residents, and clinicians interested in studying SYNGAP1. SRF grants are intended to help researchers explore novel ideas and answer open questions related to the clinical aspects of and therapies for SRID.
SRF is a member of FasterCures, COMBINEDbrain, Global Genes Foundation Alliance, Everylife Foundation Community Congress, Epilepsies Action Network, Personalized Medicine Coalition, Rare Epilepsy Network, and the Epilepsy Leadership Council.
For more on SRF, visit curesyngap1.org or follow @cureSYNGAP1 on LinkedIn, YouTube, Instagram, Facebook, TikTok, or X.
About Dravet Syndrome Foundation Spain
Dravet Syndrome Foundation Spain is a leading patient-driven non-profit organisation whose main objective is to promote research, raise awareness and support individuals and families with Dravet syndrome, a catastrophic form of epilepsy that begins in infancy and causes cognitive, speech, behavioural, motor and sleep disorders, among other neurodevelopmental disorders. Dravet Syndrome Foundation Spain leads, supports and funds research projects ultimately aimed at improving the quality of life of those living with Dravet syndrome.
It also provides resources and support to affected individuals and families, as well as educational materials and events for professionals and the general public. Throughout its history, Dravet Syndrome Foundation Spain has received numerous awards, including the Epilepsy Award from the Spanish Society of Neurology or the Rare Disease Awareness Award from AELMHU, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories.
For more information about Dravet Syndrome Foundation Spain, visit dravetfoundation.eu.