Project ‘NADER’ is intended to span multiple phases, starting from mapping rare diseases in the UAE and the wider region through innovative risk assessment algorithms and moving towards clinical trials with transformative medicines

Abu Dhabi and Palo Alto, CA, 12 June 2023: Burjeel Holdings, one of the largest healthcare providers in the MENA region, and BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, have announced a new project to revolutionise 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 a 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 revolutionise the field of early diagnosis and treatment in order to improve patient outcomes. Both entities intend to collaborate on identifying patients with several rare diseases, including achondroplasia, hypochondroplasia, FGFR-driven craniosynostoses, autosomal dominant hypocalcaemia 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 two 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 of the project 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 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. Burjeel Holdings will deploy the project across its widespread hospitals and medical centres 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.

Commenting on the project launch, Dr. Asma Ibrahim Al Mannaei, Executive Director of the Research and Innovation Center at the Department of Health – Abu Dhabi (DoH), said: “As part of our ongoing efforts to advance early diagnosis and treatment, the Department of Health – Abu Dhabi is proud to witness yet another leading partnership 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.”

Speaking on the partnership’s potential totransform the way rare diseases are identified, Mr John Sunil, CEO of Burjeel Holdings, said: “Project ‘NADER’ marks a significant milestone in our commitment to delivering the best possible care to our patients. 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.”

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: “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. 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.”

“Building on our already established foundation of genetic disease patient care, we hope that BridgeBio can help bring more patients access to novel treatments, clinical trials, and capabilities alongside a strong partner like Burjeel, and so we are grateful to be working with them 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,” said Neil Kumar, Ph.D., BridgeBio founder and CEO.

As the collaboration unfolds, both Burjeel Holdings and BridgeBio anticipate significant progress in the field of rare diseases in the region. The project could also provide patients in the region with increased access to BridgeBio’s state-of-the-art pharmacologic and gene therapies that are currently in development.

About Burjeel Holdings

Founded in 2007, Burjeel Holdings is one of the leading private healthcare services providers in the MENA region. With a network of 62 assets, including 16 hospitals and 24 medical centres, as well as pharmacies and other allied services, the group provides the highest standard of patient care in the region. Burjeel Medical City, the flagship facility of Burjeel Holdings, is a 400-bed multi-specialty hospital and quaternary care centre located in Abu Dhabi, UAE. It offers high-quality specialised treatment and complex care in over 40 adult and paediatric specialties, aided by state-of-the-art medical technology and an international team of experts. Find more information at www.burjeelmedicalcity.com, www.burjeelholdings.com.

About BridgeBio Pharma, Inc.

BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.

UAE’s Burjeel Holdings to Launch Rare Disease Research & Development Project ‘NADER’ in Partnership with US-based BridgeBio Pharma