After meeting with the agency, the firm is optimistic it can submit a BLA for isaralgagene civaparvovec in the second half of 2025 using an intermediate endpoint.

Broad Institute and Northwestern University researchers are encouraging others to examine noncoding genes, when investigating ...

The combined company, which will be called Opus Genetics, will focus on advancing a pipeline of gene therapies for inherited retinal diseases.

Through a new partnership, Caris will use its comprehensive genomic profiling tests to sequence more than 10,000 early-stage breast tumors collected in TAILORx.

The firm will use to funds to validate its CD123-targeted CAR T-cell therapies in acute myeloid leukemia in clinical studies.

About 500 children worldwide have received the gene therapy, including some families traveling abroad to gain access to it.

The firm will put the funds toward advancing its autologous CAR T-cell therapy MB-105 into Phase II clinical trials in CD5-positive lymphoma.

The strategic shift will allow the firm to focus on advancing a newly announced in vivo gene-editing therapy candidate that's in preclinical development.

NEW YORK – Cancer Research UK and Australian charity the Minderoo Foundation said on Wednesday they are collectively committing approximately £3.4 million (about $4.4 million) to fund an adaptive ...

Swedish regulators cleared a clinical trial application, allowing Karolinska University Hospital investigators to begin studying 177Lu-AKIR001.

The companies will study genetic associations linked to autoimmune diseases to identify target genes as well as patients most likely to respond to therapy.