The funds will support the firm's lead programs, ABS-1230 for KCNT1-related epilepsy and ABS-0871 for TRPV4-positive Charcot-Marie-Tooth disease type 2C.

In the growing rare disease precision medicine market, drug companies must avoid funding genetic testing that the Justice ...

The partners have committed to awarding two grants, totaling around $2.3 million, to researchers studying this lung cancer subset.

Verve is developing drug candidates for heart disease, including the PCSK9-targeted VERVE-102, a base-editing therapy.

The project will leverage Noetik's AI model and multimodal spatial data to search for biomarkers of patient response to Agenus' immunotherapy regimen.

Studies doing genome-wide testing can easily ID patients with naturally low neutrophil counts, but nobody's looking at it, ...

The firm will assess irpagratinib plus best supportive care in patients with FGF19-overexpressing advanced liver cancer.

The firm is developing the PHOX2B-targeted cell therapy, which has shown promise in preclinical xenograft models, with CHOP researchers.

The firm will stop shipping the gene therapy for these patients until a new immunosuppressive regimen is approved for managing acute liver failure.

The agency approved a perioperative Keytruda regimen based on a 30 percent reduction in the risk of recurrence, progression, or death versus the control arm.

The drug is already approved for KMT2A-rearranged AML, and Syndax is hoping for approval in NPM1-mutated disease by the end of the year.

The firm expects to complete enrolling the REVEAL trial, in which patients will receive the ASO, dubbed ION582, in 2026.