Weight-loss drugs stole much of the spotlight in 2023, but these medical advances treating other conditions are also worthy of attention (SN: 12/13/23).
Green light for CRISPR gene editing
On December 8, the U.S. Food and Drug Administration approved the world’s first CRISPR/Cas9 gene-editing therapy (SN: 12/8/23). The treatment, called Casgevy, targets sickle cell disease by helping patients produce healthy hemoglobin. In people with the disease, hemoglobin is abnormal, causing red blood cells to become hard and crescent shaped, which can block blood flow. By March 2024, the FDA will decide whether the same therapy can be used to treat beta-thalassemia, a disorder that reduces hemoglobin production.
Slowing down Alzheimer’s
The Alzheimer’s drug lecanemab (brand name Leqembi) won full FDA approval in July. Like the drug aducanumab approved in 2021, lecanemab removes the amyloid plaques that build up in the brains of people with Alzheimer’s. The drug doesn’t stop the disease, but in a clinical trial, lecanemab slowed cognitive decline by about 30 percent over 18 months compared with a placebo (SN: 8/12/23, p. 9).
A gene therapy for muscular dystrophy
In June, the FDA approved the first gene therapy for children with Duchenne muscular dystrophy. Due to a faulty gene, people with this muscle-wasting disease don’t make the protein dystrophin, which helps keep muscle cells intact. The therapy helps the body produce a version of the missing protein (SN: 6/22/23).
Guarding against RSV
Several ways to protect against respiratory syncytial virus arrived this year. In May, the FDA approved the first RSV vaccine, called Arexvy, in the United States, for adults age 60 and older (SN: 6/17/23, p. 8), and then in August, a vaccine for pregnant people, called Abrysvo (SN: 8/25/23). A monoclonal antibody — a lab-made antibody that mimics immune system proteins — won approval in July to protect children 2 and younger from the virus, which…
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