The FDA cleared a second arm for enrollment in the trial after one NSCLC patient demonstrated a complete response to the non-boosted therapy.

The recommendation is based on results from 77 patients with pediatric low-grade glioma harboring a BRAF fusion or rearrangement or BRAF V600 mutation.

The trials in breast, lung, and colorectal cancer, supported by ARPA-H, will focus on guiding decisions across different lines of therapy using serial biopsies and tumor profiling.

GSK will pay the Chinese company $40 million upfront and up to $963 million in payments linked to development, regulatory, and commercial milestones.

Metabolon helped Kite study the link between metabolic signatures and the severity of neurological evens in samples from ...

Beam also unveiled a new platform-based approach to phenylketonuria treatments and a $500 million senior secured credit facility.

Hernexeos underwent an ultra-rapid 44-day regulatory review due to its inclusion in the FDA Commissioner's National Priority Voucher pilot program.

The start of the Phase I trial for IDE034 triggers a milestone payment to Biocytogen, from which Ideaya acquired the program in 2024.

The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...

Researchers are evaluating the P-CARE model, which integrates results from a blended genome-exome sequencing assay and family ...

In a study with three UK academic institutions, the platform agreed with MDx results 94 percent of the time in detecting ...

Sarepta posted $442.9 million in revenue for Q4 2025, down from $658.4 million in the year-ago quarter but beating the consensus Wall Street estimate.