– Project ‘NADER’ is intended to span multiple phases, starting from mapping rare diseases in the
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 two entities 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.
Speaking on the partnership’s potential to transform the way rare diseases are identified, Mr.
Commenting on the project launch, Dr.
The first phase will aim to set the roadmap to providing novel treatment options for identified patients through clinical trials.
“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. The BridgeBio vision is to serve patients around the world, and this is an important step in that direction,” said
As the collaboration unfolds, both
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This press release contains forward-looking statements. Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “aim,” “may,” “plans,” “projects,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans, and prospects regarding BridgeBio’s collaboration with Burjeel and Project “NADER”; the intent and ability to identify 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 potentially others through this collaboration; the success of the current and future relationship between BridgeBio and Burjeel; each phase of implementation of Project “NADER”, including the expected actions to be taken by Burjeel and local partners to deploy and implement the project; the potentials benefits of Project “NADER”, including the expectation to transform the way rare diseases are identified, the expectation that this project brings together Burjeel’s expertise in healthcare delivery and outreach and BridgeBio’s vast expertise in delivering breakthrough therapies for rare diseases, the potential to enable early interventions whether in the form of established treatments or novel therapies, and the goal to link patients to evolving treatment options through clinical trials of small molecules and gene therapies, reflect our current views about our plans, intentions, expectations, strategies and prospects, and are based on the information currently available to us and on assumptions we have made and are not forecasts, promises nor guarantees. Although we believe that our plans, intentions, expectations, and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations, or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio’s and/or Burjeel’s ability to identify rare diseases described in this press release, the success of the relationship between BridgeBio and Burjeel, the ability of the collaboration to execute each phase of Project “NADER”, the uncertainty regarding any impacts due to COVID-19, the impacts of current macroeconomic and geopolitical events, as well as those risks set forth in the Risk Factors section of our Annual Report on Form 10-K for the year ended
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