Chicago, Illinois – The University of Chicago Medicine Comer Children’s Hospital will be among the first in the country to offer gene therapy for sickle cell disease in patients 12 years and older, after federal regulators approved two new treatments in December 2023. Thousands of patients with sickle cell disease...
treatment News
LOS ANGELES, Calif. – Researchers at UCLA have developed a nonviral gene-editing strategy that could help pave the way for one-time treatments for people with cystic fibrosis (CF). The experimental therapy uses lipid nanoparticles — tiny fatty molecules — to insert a full, healthy copy of the CFTR gene into human airway cells,...
FAIRFAX, Va. — A minimally invasive treatment using MRI and transurethral ultrasound instead of surgery or radiation is effective in treating prostate cancer, according to new research to be presented at the Society of Interventional Radiology Annual Scientific Meeting in Salt Lake City. The traditional treatment options of radiation or...
Los Angeles, Calif. – A study led by UCLA Health Jonsson Comprehensive Cancer Center researchers found that using a combination of experimental immunotherapy drugs with chemotherapy significantly improves progression-free survival and overall survival for patients with metastatic colorectal cancer who have previously undergone standard chemotherapy treatment when compared to those who received the targeted...
San Francisco, Calif. – UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The trial is the first in the U.S. to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease....
PARIS – GenSight Biologics (Paris:SIGHT) (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that its gene therapy LUMEVOQ® has been granted Promising Innovative Medicine (PIM) designation by the UK’s Medicines and...
Newark — Ultima Genomics, Inc. and Genome Insight, Inc. have signed an agreement to collaborate to bring affordable whole genome sequencing solutions to cancer patients. As part of the agreement, Genome Insight will join the early access program for Ultima’s high-throughput NGS instrument platform, the UG 100™, and develop an optimized...
Ultimovacs has announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) for the company’s universal cancer vaccine UV1 for the treatment of stage IIB – IV melanoma. UV1, as an add-on therapy to checkpoint inhibitors ipilimumab and nivolumab, is currently being studied as a first-line...
Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+). If authorised, Ultomiris would be the first and only approved long-acting C5 complement inhibitor for the treatment of...
WILMINGTON, Del. — ULTOMIRIS® (ravulizumab-cwvz) has been approved in the United States as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval by the US Food and Drug Administration (FDA) was based on...