RARE Daily

Burjeel to Launch Rare Disease Research & Development Project with BridgeBio

July 13, 2023

Rare Daily Staff

Burjeel Holdings, one of the largest healthcare providers in the MENA region, and BridgeBio Pharma announced a new project to transform the field of early diagnosis and treatment of rare diseases or disorders in the UAE and the region.

The two entities signed a preliminary, non-binding collaboration agreement establishing a mutual intention to work together on project NADER (Needs Assessment and Therapeutics Development for Rare Diseases – ‘nader’ meaning ‘rare’ in Arabic). Genetic in origin, these often life-threatening or chronically debilitating diseases affect a small percentage of the population and are present throughout the person’s entire life, even if symptoms do not immediately appear. The partnership will launch operations in Abu Dhabi to conduct clinical trials and research, leveraging the Emirate’s advanced infrastructure for innovation and life science.

Project NADER aims to revolutionize the field of early diagnosis and treatment in order to potentially improve patient outcomes. The entities intend to collaborate on identifying patients with several rare diseases, including achondroplasia, hypochondroplasia, FGFR-driven craniosynostoses, autosomal dominant hypocalcemia type 1, limb girdle muscular dystrophy type 2i, congenital adrenal hyperplasia, Canavan disease, propionic acidemia, methylmalonic acidemia, pantothenate kinase-associated neurodegeneration, and others as mutually agreed.

Even though these rare diseases affect a small percentage of the population and can present with nonspecific signs and symptoms, they are often misdiagnosed or only diagnosed later in the disease course after severe complications manifest. Accurate and early diagnosis are crucial to starting intervention promptly and avoiding disease progression. The project will also allow identification of patients who have already been diagnosed but do not currently have access to cutting edge therapies or clinical trial options.

In the first phase of project NADER, the partners intend to utilize 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 of the project include analyses of vast amounts of data to identify patterns and markers associated with the diseases and generate personalized risk scores for patients, followed by genetic testing to confirm the diagnosis of identified potential patients. The project also aims to run awareness campaigns and education activities about such rare diseases, to further support the mapping activity and encourage local collaborations from other healthcare providers.

“Many rare diseases are highly clustered in our region due to our unique genetic pool. These diseases are often ‘lost in the system’ or have very limited treatment options,” said Khaled Musallam, group chief research officer of Burjeel. “Our ultimate goal from project NADER 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.”

Burjeel Holdings will deploy the project across its widespread hospitals and medical centers across 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.

“Project NADER marks a significant milestone in our commitment to delivering the best possible care to our patients,” said John Sunil, CEO of Burjeel. “It 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 identification of rare diseases, which often go undiagnosed until symptoms become severe. The project will enable early interventions whether in the form of established treatments or novel therapies.”

Photo: John Sunil, CEO of Burjeel

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