The company's platform relies on a method called RABID-seq that combines barcoded viral tracing with single-cell RNA-seq to study interactions between brain cells.

The approval is for DEB patients from birth and allows for flexible administration at home by patients or caregivers.

The application was based on results from a Phase I/II trial in which Hyrnuo demonstrated a 71 percent response rate.

Investigators will analyze CTCs from colon, gastrointestinal, and lung cancer patients to help understand how they respond to exercise regimens.

Investigators will analyze CTCs from colon, gastrointestinal, and lung cancer patients to help understand how they respond to exercise regimens.

The principal investigator reported at the ASGCT meeting that the study met its primary endpoint, though there were two treatment-related serious adverse events.

Even though Arvinas and Pfizer appear to have "written off" the drug, in one analyst's view, Rigel believes it can drive adoption among community-treated patients lacking SERD access.

Attendees at a fireside chat at the ASGCT annual meeting noted disconnects between the agency's stated goal of speeding gene therapies to market and its actions.

The latest decision makes these treatments routinely available after having been accessible only through a managed access scheme.

On CGTxchange, sponsors can list their shelved cell and gene therapies in the hopes that other investors, partners, and ...

The former commissioner pushed a regulatory pathway for rare disease drugs and launched a voucher program for expedited ...

At the ASCGT meeting, scientists reported that Carvykti-treated cells overexpressed the enzyme Granzyme K, which may be ...