CER-1236 is designed to engineer T cells with phagocytic, or target-cell "eating," functions alongside traditional ...

The designation qualifies CERo and CER-1236 for certain incentives, such as FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the drug approval ...

The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend ...

Capricor Therapeutics (NASDAQ:CAPR) received the Orphan Drug designation from the U.S. FDA for its lead cell therapy candidate, Deramiocel, to treat becker muscular dystrophy. With its orphan drug ...

ABOUT ORPHAN DRUG DESIGNATION The FDA grants Orphan Drug Designation to investigational drugs intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S. Benefits ...

Discover six kidney disease companies actively working on innovative treatments that could make a difference to patients' lives.

Targeted Oncology connects oncology professionals with updates on immunotherapy, biomarkers, cancer pathways, and targeted ...

Researchers at Karolinska Institutet and Lund University in Sweden have identified a new treatment strategy for neuroblastoma ...

The FDA granted fast track designation to nuvisertib for those with intermediate or high-risk myelofibrosis, which ...

BIO International Convention (BIO2025), the world’s largest annual biotech event, will take place from June 16 to 19 (U.S.

Ocugen has announced the US Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to ...