Supported by the Department of Health – Abu Dhabi (DoH), Burjeel Holdings has partnered with US-based BridgeBio Pharma to launch a multi-phase project to identify rare diseases in the Mena region and provide patients with world-class healthcare options.

California-headquartered BridgeBio is a biopharmaceutical company focused on discovering, creating, testing and delivering transformative medicines to treat patients who suffer from genetic diseases and cancers. Both entities inked an agreement to collaborate on the project titled Needs Assessment and Therapeutics Development for Rare Diseases (‘Nader’ meaning rare in Arabic). Clinical trials and research for the project will be conducted in Abu Dhabi.

The entities intend to collaborate on identifying patients with several rare diseases and disorders, including achondroplasia, hypochondroplasia, FGFR-driven craniosynostosis, autosomal dominant hypocalcaemia type 1, congenital adrenal hyperplasia, Canavan disease, propionic acidaemia, pantothenate kinase-associated neurodegeneration, among others.

Accurate and early diagnosis is crucial to starting intervention promptly and avoiding the progression of these often life-threatening or chronically debilitating diseases. The project will allow the identification of patients who have already been diagnosed but do not currently have access to cutting-edge therapies or clinical trial options.

Dr Asma Ibrahim Al Mannaei, executive director of the Research and Innovation Centre at DoH, noted that the partnership is aimed at propelling transformative medicine to safeguard the health and well-being of community members.

“Reinforcing Abu Dhabi’s position as a leading destination for life science and innovation, we remain committed to empowering healthcare providers in the emirate with the means to lead research studies and conduct clinical trials to map out a path towards discovery and breakthrough healthcare in the emirate and beyond. Similar initiatives translate the Department’s vision in securing health for all while accelerating healthcare outcomes for the benefit of the global community.”

Multi-phase project

In the first phase of Nader, the entities intend to utilise innovative risk assessment algorithms that will be deployed through the healthcare provider’s secure internal data to identify patients at risk for specific rare diseases. Expectations for future phases include analyses of vast amounts of data to identify patterns and markers associated with the diseases and generate personalised risk scores for patients, followed by genetic testing to confirm the diagnosis of identified potential patients. The project aims to run awareness campaigns and education activities about such rare diseases, to further support the mapping activity and encourage local collaborations with other healthcare providers.

Burjeel Holdings will deploy the project across its hospitals and medical centres in the UAE as part of routine diagnostic care, with plans to expand the project through its growing healthcare infrastructure in the region. Local partnerships will also be sought to facilitate wider implementation.

John Sunil, CEO, Burjeel Holdings, noted that the project will enable early interventions whether in the form of established treatments or novel therapies.

“The project brings together Burjeel’s expertise in healthcare delivery and outreach, and BridgeBio’s vast expertise in delivering breakthrough therapies for rare diseases. By combining our strengths, we seek to address the challenges faced in the identification of rare diseases, which often go undiagnosed until symptoms become severe.”

The first phase will aim to set the roadmap to providing novel treatment options for identified patients through clinical trials.

Dr Khaled Musallam, group chief research officer of Burjeel Holdings and project lead said: “Our ultimate goal from the project is to link patients to evolving treatment options through clinical trials of small molecules and gene therapies. The first step to bringing such innovative solutions to our region is to identify disease burden and patients’ unmet needs.”

Neil Kumar, founder and CEO, BridgeBio, hoped the partnership will bring more patients access to novel treatments, clinical trials, and capabilities.

“We are grateful to be working with Burjeel to expand our efforts in the Mena region. Our vision is to serve patients around the world, and this is an important step in that direction.”

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