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

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

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The FDA released a much-anticipated draft guidance on how makers of rare disease genetic medicines can leverage a platform ...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

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

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.

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

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