The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.
Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.
In addition to isolating the first protein-coding gene from a eukaryote, Kedes furthered scientists’ understanding of actin genes and also laid the foundations for modern DNA databases such as GenBank.
Clinical trials were halted after the treatment’s vector that ferries in the healthy genetic sequence was identified in the genome of a patient’s cancer cells.
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.