In some cases, drugmakers have accused the FDA of reversing course on earlier feedback. Based on engagements with the agency, ...

The FDA said it could not agree to an approval path for AMT-130 based on Phase I/II data and recommended UniQure run a randomized, controlled study.

The firm has four clinical-stage pipeline candidates using its conditionally active biologic antibody technology.

The therapy shrunk tumors in 46 percent of patients in a Phase I trial and showed strong PSA responses at the recommended dose.

The agency and the firm have agreed on certain risk mitigation measures for the MAGNITUDE and MAGNITUDE-2 trials of nexiguran ziclumeran.

The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...

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

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

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

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.

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