The interim Phase I trial data was only from 35 people, but results look good so far. An experimental gene-editing therapy that aims to lower bad cholesterol for the long-term after a single infusion is off to a positive start in an early clinical trial. Researchers running a Phase I safety trial for the drug,
A phase 2 study published in the Journal of Clinical Oncology shows that a stem cell product called dilanubicel safely enabled umbilical cord blood transplants in 28 patients A new way of using umbilical cord blood for treating blood diseases could make the treatment more accessible to patients who need a stem cell transplant. A
Results of a phase 1 clinical trial show that patients who undergo a blood stem cell transplant involving a donor have a lower risk of relapse and lower rates of graft-versus-host disease when they receive the targeted therapy VIC-1911 along with the standard-of-care regimen that includes post-transplant cyclophosphamide and sirolimus. Led by Shernan Holtan, MD, Chief of Blood
Cedars-Sinai investigators working to optimize a cell-based treatment for retinitis pigmentosa have uncovered how transplanted neural stem cells interact with host retinal cells to preserve vision. The findings, published in Nature Communications, may guide future research toward strategies to treat degenerative eye disease. “We used single-cell analysis to show that neural stem cells can protect vision in
A study published today in Stem Cell Reports demonstrates that genetically engineered human pluripotent stem cells (hPSCs) can overcome immune rejection in mice with humanized immune systems, surviving for five months in a stringent transplantation model. The findings provide proof-of-principle for the development of a potential universal donor hPSC line designed to resist immune attack. Led by Danny Chan,
Cell and gene therapies, or CGT, have come a long way since they were first introduced. In the last few decades, both cell therapy — the transplantation of living cells — and gene therapy — the use of genetic material to modify cell functions — have been increasingly incorporated into clinical practice. Various challenges and advances
“Charting the Translational Pathway: ISSCR Best Practices for the Development of PSC-Derived Therapies,” offers insights into the comprehensive, globally informed guide to navigating the complex journey from laboratory discovery to approved therapy. The International Society for Stem Cell Research (ISSCR) today announced the upcoming release of “Charting the Translational Pathway: ISSCR Best Practices for the
The U.S. Food and Drug Administration announced it has approved Omisirge (omidubicel-onlv), the first hematopoietic stem cell transplant (HSCT) therapy to treat patients with severe aplastic anemia (SAA). Omisirge is indicated for adults and pediatric patients 12 years and older with hematologic malignancies and now is approved for adults and pediatric patients six years and
Blood cancer patients who may have previously struggled to find a donor for transplantation now have more options. A new study shows that patients achieve good outcomes with an partial match drawn from the national public registry of donors when they are treated with the immune-suppressing drug cyclophosphamide. Survival rates at one year were on par
Damage to joint cartilage is painful and limits mobility. Researchers at the University of Basel and the University Hospital of Basel are therefore developing cartilage implants from cells from the nasal septum. A recent study shows that a longer maturation time for the cultured cartilage brings a significant improvement even in cases of complicated cartilage
