Gene Therapy
Conference Coverage
This test may guide AML therapy for Black pediatric patients
New research suggests that a polygenic score can presage how well Black pediatric patients with AML will respond to treatment.
News
FDA approves first 2 gene-editing therapies for sickle cell
These expensive “milestone treatments” greenlighted by the FDA are the first gene-based therapies for sickle cell disease.
Feature
DLBCL: Major new treatment breakthroughs
After decades of slow progress, new drugs and therapies are paving the way to more effective treatment of DLBCL.
From the Journals
Hemophilia A gene therapy under FDA review
Recently published clinical data showed that a costly new gene therapy for hemophilia A was found largely durable at 2 years.
News
Europe approves first gene therapy for hemophilia B
Exorbitantly priced gene therapy to treat hemophilia B has been approved for sale across the European Union and European Economic Area.
Feature
MD-researcher keeps her eyes on the prize
Researching CAR T-cell therapies for T-ALL, a physician-scientist works to saves children’s lives, as hers was saved.
Conference Coverage
Beta-thalassemia: Benefits of gene therapy outweigh costs
Following treatment with betibeglogene autotemcel, some patients went without transfusions for 8 years and counting.
Conference Coverage
Pricey gene therapy looks cost-effective for SCD
Researchers calculated that a costly new treatment being developed for sickle cell disease (SCD) promises to be worth the price.
News
FDA approves first gene therapy for hemophilia B
The gene therapy will cost $3.5 million, making it the most expensive treatment to date.
Conference Coverage
‘Superior’ CLL regimen cuts chemo in half
Where chemo is still used to treat CLL, this three-cycle regimen combined with ibrutinib and obinutuzumab shows progression-free and overall...
News
FDA approves liso-cel as second-line therapy for LBCL
Liso-cel could now become a “new standard of care for patients after failure of first-line therapy,” offering significantly improved outcomes for...