Pelacarsen, a highly anticipated drug targeting Lp(a) to lower heart risk, failed to show cardiovascular benefit in a Phase 3 trial, raising questions about an

The FDA approved Etcamah (camizestrant), an oral SERD, in combination with a CDK4/6 inhibitor for first-line treatment of ESR1-mutated HR-positive advanced brea

The FDA approved Ionis Pharmaceuticals' zilganersen for the ultra-rare genetic disorder Alexander disease, with the company pricing the treatment at $285,000 pe

Novartis paused its autoimmune CAR-T cell therapy trials following three patient deaths, putting China's booming CAR-T industry under scrutiny.

A patient survived for nine months after receiving a kidney transplant from a genetically modified pig, marking a milestone in xenotransplantation.

Ultragenyx's gene therapy GTX-102 failed in a clinical trial for Angelman syndrome, sending shares down over 40% and prompting pipeline and expense reviews.

Intellia Therapeutics locked in a $400 million loan from OrbiMed linked to milestones for its hereditary angioedema (HAE) gene-editing therapy.

The FDA approved Guardant360, Guardant Health's liquid biopsy test, for use in advanced breast cancer, a landmark for circulating tumor DNA testing.

AbbVie gained FDA approval for a new drug targeting multiple myeloma, catching up to rivals in the blood cancer space.

A series of reports and studies highlight the growing promise of personalized mRNA cancer vaccines, with the field recovering from earlier setbacks.

The FDA approved Revolution Medicines' oral therapy for metastatic pancreatic cancer, while a separate study showed the drug class also shrinks tumors in lung c

Daraxonrasib, a drug originally developed for pancreatic cancer, showed early efficacy in shrinking tumors in a lung cancer clinical trial.

RiboX received FDA clearance to begin clinical trials for the world's first circular RNA-based in vivo CAR-T therapy.

uniQure submitted a Biologics License Application (BLA) to the FDA for its Huntington's disease gene therapy, sending shares up 20% on analyst upgrades.

Compass Pathways' stock rose after-hours following strong Phase 3 trial data for its psychedelic depression drug, with analysts projecting FDA approval by early

Former President Trump directed the Department of Veterans Affairs to launch a clinical trial testing psychedelic treatments for veterans with PTSD and other co

The failure of pelacarsen in a large cardiovascular outcomes trial raises doubts about the entire class of Lp(a)-lowering drugs.

Intellia Therapeutics shares surged as retail investors piled in ahead of the first-ever Phase 3 data readout for a CRISPR-based gene-editing therapy.