A University of Michigan Rogel Cancer Center study found that online resources combined with phone-based coaching helped more eligible cancer patients complete genetic testing. About 10% of cancers are linked to inherited genetic variants, but as few as 1 in 15 eligible patients currently complete testing, with lack of knowledge being a key barrier.
UniQure reported its gene therapy for Huntington's disease showed diminished benefits at four years compared to three years in a small trial, with shares plunging 48% as the results cast doubt on the treatment's long-term efficacy.
PTC Therapeutics won the bankruptcy auction for Sangamo Therapeutics' gene therapy assets with a $211 million bid. Sangamo filed for Chapter 11 bankruptcy in June after failing to find strategic alternatives.
Catherine L'Estrange from North Acton became the first patient in the UK and second in the world to receive groundbreaking gene therapy for Bardet-Biedl syndrome, a rare condition that progressively causes blindness in children. The treatment, performed at St Helier Hospital in March, involves injecting healthy copies of a gene directly into the eye.
New research demonstrates that RNA-based testing can improve detection of MET exon 14 skipping mutations in lung cancer patients, while circulating tumor DNA (ctDNA) testing is increasingly being adopted for early cancer detection and treatment monitoring across oncology.
Three major research institutions announced the creation of the Center for Therapeutic Genetics, a nonprofit collaboration aimed at developing standardized genetic medicines, including base and prime editing, for rare and ultra-rare diseases that currently lack treatments.
Two patients with SCN2A-related developmental epileptic encephalopathy experienced reductions in seizure frequency after receiving individualized allele-selective antisense oligonucleotides. The treatments were well tolerated, with no ASO-related serious adverse events reported in two parallel single-patient studies published in Nature Medicine.
New clinical trial results presented at the American Society of Clinical Oncology show that Moderna's experimental personalized mRNA vaccine, combined with immunotherapy, significantly reduced the risk of deadly melanoma returning after five years. The vaccine is custom-made for each patient based on their specific tumor.
Researchers at Duke University are developing a gene therapy treatment designed to help repair heart damage following heart attacks, potentially changing how doctors treat cardiac injury from the leading cause of death in the U.S.
International research led by RIKEN Center for Integrative Medical Sciences discovered new associations between pathogenic BRCA1 and BRCA2 gene variants and four additional cancer types beyond the previously known breast and ovarian cancers.
