A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.

Be Biopharma Inc. has announced the FDA’s clearance of its IND for BE-101, an autologous potentially first-in-class B-cell medicine in development for the treatment of hemophilia B.

The success of a vaccine, a gene editing design for an untreated disease, or achieving cell engraftment after several attempts, comes from years of accumulated basic science studies, thousands of experiments, and clinical trials. Innumerable steps precede hits in gene and cell therapies before a first-time revelation, and most of them are failures at the time. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in Baltimore last week, several groups of scientists presented achievements that years ago looked impossible.

Positive data from Novo Nordisk A/S’s pivotal phase IIIa study of once-weekly and once-monthly doses of its hemophilia treatment, Mim8, are prompting the company to say it will submit the first regulatory approval request toward the end of this year. It could challenge Roche Holding AG’s Hemlibra (emicizumab), a bispecific factor IXa- and factor X-directed antibody for hemophilia A, that was approved in 2017 by the U.S. FDA.

From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.

Chronic granulomatous disease (CGD) is an immunodeficient disorder that is caused by mutations in genes that encode proteins of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase enzyme complex.