since starting FORGE
We know that a cure is possible and can be achieved with your support. This is our pathway to a cure.
Trustee report 2025
FORGE Projects initiated during 2025
In 2025, FORGE launched a series of strategic projects aimed at improving outcomes for individuals and families affected by SAVI (STING‑associated vasculopathy with onset in infancy). Because SAVI is an ultra‑rare condition, the global patient community is small, making it possible for FORGE to reach and support nearly every affected family worldwide.
The flagship initiative, FORGE CONNECT, focuses on building meaningful connections between patients, carers, clinicians, researchers, and industry partners to accelerate progress in treatment, research, and advocacy.
A central element of FORGE CONNECT is the creation of a dynamic Patient Registry, designed to engage families globally, gather essential data, and empower patients—especially children—to participate actively in their care. The registry will also strengthen relationships with research organisations and provide lay patient representation on FORGE’s Scientific Advisory Board. Through these connections, FORGE aims to widen access to emerging therapies and supportive technologies.
FORGE is also working with Genomics England’s Newborn Genomes Programme to advocate for the inclusion of SAVI in newborn screening. Although SAVI was not initially included due to the lack of a licensed newborn treatment, FORGE is collaborating with Fusion Pharma and Eli Lilly to explore pathways that could meet the programme’s criteria.
In parallel, the organisation is mapping global newborn screening frameworks.
Additional collaborations include a potential Imperial College London project analysing SAVI‑associated mutations in global genomic datasets, and strong partnerships with Ilya Pharma and Alnylam, both developing innovative therapies targeting SAVI’s underlying mechanisms.
Finally, FORGE has joined the Translational Autoinflammatory Research Network (TARN) and will represent the SAVI community at the 2026 NIH Symposium, strengthening its global advocacy and research presence.
Projects during 2026
On 3rd February 2026, FORGE met with the senior team from Sulis Therapeutics to hear about their latest research targeting the STING pathway, which plays an important role in autoimmune and inflammatory diseases including SAVI.
The Head of R&D at Sulis Tx shared promising preclinical results with us including research on cells taken from SAVI patients. Their approach, which is highly original, aims to remove STING-associated inflammatory change whilst preserving normal immune function. In animal models, the specific STING-targeting treatment they have devised, impressively reduced inflammation and organ damage resulting in improved survival. The therapy was also well tolerated.
The company is now seeking funding to take this research forward into phase 1 human trials and expressed interest in working closely with FORGE and the patient community. Further discussions are planned to explore future collaboration..
How we help patients and their families
FORGE supports patients and their families by sharing up-to-date, accurate information and providing emotional and peer support through our Community Support Hub.
We are fierce advocates for patients’ rights and actively influence policy change while raising awareness of rare genetic diseases. We fund research projects to identify new treatments and cures and work hard to increase collaboration between patients, researchers, and clinicians.
Through this, we hope to enhance the lives of patients and their families while advancing the understanding and treatment of rare genetic diseases.
How we help researchers and clinicians to achieve goals
FORGE plays a vital role in supporting clinicians and researchers committed to helping those affected by rare diseases. We provide crucial funding for research projects, allowing scientists to investigate new treatments and potential cures.
Our grants and financial support help bridge the gap between initial research and clinical trials, speeding up the development of innovative therapies. Furthermore, we act as a conduit, facilitating much-needed collaboration among researchers, clinicians and patients.
We hold impactful networking events, creating an environment where professionals can share knowledge, exchange ideas and build relationships. This connection is essential in advancing the understanding of rare diseases and enhancing patient care.
Our impact
We are a respected global resource that supports patients and their families, clinicians, and researchers. FORGE aims to significantly impact the understanding and treatment of rare genetic diseases.
Where we need you
Every donation helps us create a future where rare genetic disorders are understood and fully treatable.
Every gift of time supports us along our pathway to a cure.
