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

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

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

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

The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...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

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

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.

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

The gene therapy, which was acquired by Novartis in 2023, is also being tested in a separate Phase I/II trial of pediatric ...