Skip to main content
Premium Trial:

Request an Annual Quote

Takeda Pharmaceutical, Rady Children's Partner on Rare Genetic Disease Drug Development

NEW YORK — Rady Children's Institute for Genomic Medicine said on Thursday that it has partnered with Japanese drugmaker Takeda Pharmaceutical to discover and develop therapies for rare child-onset genetic diseases.

Through the collaboration, RCIGM will provide Takeda with aggregated, de-identified genomic and phenotypic data derived from whole-genome sequencing performed on patients in specific disease cohorts. Takeda will use the data to discover and develop potential targeted therapeutics.

Additional terms were not disclosed.

"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," Dan Curran, head of the rare genetics and hematology therapeutic area unit at Takeda, said in a statement. "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."

In June, RCIGM — which is housed within Encinitas, California-based Rady Children's Hospital and Health Center — began working with Pacific Biosciences to explore the use of whole-genome sequencing data to diagnose rare diseases.

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.