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news.BioXcel has announced that its first-in-class cloud based Pharma big-data analytics platform, PharmGPS now has a dedicated Orphan Disease suite to aid in the discovery, development, licensing and commercialization of drugs for the approximately 7,000 Rare and Ultra Rare Diseases.
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Partners will benefit from real-time multi-factorial analytics to design target-indication product concepts for rare diseases based on severity, epidemiology, gene ontology, pathways, proteinopathy, standard of care, emerging innovation, enabling technologies and drug pipeline.
Rare diseases may affect less than a few hundred or up to 200,000 people and in the U.S. alone, 30 million or 1:10 Americans have a disease that is designated as being "rare".
Surprisingly, less than 5% of rare diseases currently have an effective treatment. Adding to the sheer complexity of such diseases and to the chasm of unmet medical needs, these diseases span multiple therapeutic areas including cardiology, dermatology, endocrinology, genetic disease, immunology, infectious disease, neurology, oncology, ophthalmology, and respiratory disease.
BioXcel’s PharmGPS Orphan Disease suite addresses this complexity in a highly systematic manner to explore innovative ideas and deliver business value.
Commenting on the current therapeutic landscape Dr Krishnan Nandabalan, President and CEO, said, "After the successful launch and commercialization of PharmGPS for major chronic diseases, we have built an Orphan Disease suite comprising distinct target-indication tiles for antibody, protein, RNA, small molecule and gene therapy modalities. The underlying metadata and analytics for 7,000 diseases, 3,000 associated genes and hundreds of disease pathways related to therapeutic modality tiles, facilitates the design of dynamic solutions for developing innovative treatments."
Dr Vimal Mehta, Chairman and Co-Founder further added, "We continue to innovate and expand the potential of PharmGPS™ to address the significant unmet medical needs of those with rare diseases. We are providing access to this suite through a partnership model to design and develop breakthrough treatments. In addition, partners will benefit from BioXcel’s cross-functional team’s domain expertise to generate unique product concepts with unparalleled speed."
One of the biggest challenges the industry faces today is making business critical decisions that will result in first-mover advantage and high productivity in a continuously evolving complex innovation and healthcare environment.
The platform’s unique system-based approach enables various stakeholders to make informed, unbiased decisions and realize value, impacting billions of dollars. The platform helps partners take primordial decisions for a disease on the ideal mode of pharmacotherapy, enabled by real-time analytics of all orphan diseases with parameters that encompass strategic, medical, scientific and commercial aspects of the diseases.