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RCIGM Announces Research Collaboration with Takeda to Advance the Development of Novel Rare Genetic Disease Therapies

A new research collaboration between Rady Children’s Institute for Genomic Medicine (RCIGM) and Takeda Pharmaceutical Company Limited (“Takeda”) aims to accelerate the discovery and development of new treatments for rare childhood-onset diseases by analyzing the natural progression of selected genetic disorders from genome-phenome datasets.

The project will examine genomic and phenotypic data from de-identified cohorts of pediatric patients to better understand the evolution of rare diseases that typically manifest in childhood. Ultimately, the goal is to improve clinical outcomes for patients by providing precise diagnoses and targeted treatments as early as possible to avoid morbidity or mortality.

Rady Children’s Institute for Genomic Medicine has pioneered rapid and ultra-rapid diagnoses of rare genetic disorders for critically ill newborns and children via whole genome sequencing. In medically urgent cases, involving children in intensive care settings, preliminary results are returned in less than three days.

“The results of the first 2,000 children to receive rapid whole genome sequencing at the Genomics Institute have confirmed our understanding that most childhood genetic diseases currently lack effective treatments,” said Stephen Kingsmore, MD, DSc, President & CEO of RCIGM. “We are striving for a future where we can not only diagnose a child’s rare disease but also provide disease-specific treatment. Our collaboration with Takeda is a crucial step toward this goal.”

RCIGM is embedded within Rady Children’s Hospital and Health Center, a comprehensive pediatric health system which serves San Diego, southern Riverside and Imperial Counties caring for more than 250,000 children annually. The Institute provides whole genome sequencing services and clinical decision support to a growing network of more than 60 hospital systems nationwide.

“Rady Children’s Institute for Genomic Medicine has led the way in using whole genome sequencing to better understand the genetic drivers of rare diseases in children,” said Dan Curran, M.D., Head, Rare Genetics & Hematology Therapeutic Area Unit at Takeda. “It’s through these efforts that we hope to derive new insights on how childhood-onset diseases manifest, enabling us to more quickly develop transformative therapies for the families who desperately need them.”

Under the terms of the agreement, RCIGM will provide aggregated, de-identified genomic and phenotypic data derived from WGS cases for specific disease cohorts. Takeda will oversee the drug discovery and development of potential targeted therapeutics resulting from the program. Additional terms were not disclosed.

“Rady Children’s is excited to improve pediatric healthcare by joining Takeda in targeting therapeutics for rare disease,” said Patrick Frias, MD, President & CEO of Rady Children’s Hospital & Health Center. “As the leading pediatric care provider for a multi-county region in Southern California, we serve a broad and diverse population. Our ability to care for children from birth through age 18 positions us to identify rare diseases early and track the natural history of childhood diseases with the goal of short-circuiting the therapeutic odyssey which now occurs in many children whose disease is not identified by current methods.”