Skip to main content
Premium Trial:

Request an Annual Quote

Supporting Vote

An advisory committee to the US Food and Drug Administration has voted to support Bluebird Bio's gene therapy for cerebral adrenoleukodystrophy, according to Bloomberg.

Cerebral adrenoleukodystrophy (CALD) is a rare, X-linked genetic condition affecting mostly boys that leads to progressive and irreversible neurodegenerative disease. About half of individuals with CALD die within five years of the onset of symptoms. Bluebird Bio's eli-cel (elivaldogene autotemcel) introduces functional copies of the ABCD1 gene using a Lenti-D lentiviral vector.

Last year, the FDA placed the company's eli-cel trial on a clinical hold after patients who received treatment later developed myelodysplastic syndrome. Bloomberg notes that the trial is still on hold, "creating an unusual situation for a therapy that's under consideration for approval."

Still, the FDA advisory panel voted 15-0 in support of the treatment. According to Bloomberg, some panelists suggested offering the treatment to patients without other options and all underscored the need for close monitoring of patients receiving the treatment.

The Scan

ChatGPT Does As Well As Humans Answering Genetics Questions, Study Finds

Researchers in the European Journal of Human Genetics had ChatGPT answer genetics-related questions, finding it was about 68 percent accurate, but sometimes gave different answers to the same question.

Sequencing Analysis Examines Gene Regulatory Networks of Honeybee Soldier, Forager Brains

Researchers in Nature Ecology & Evolution find gene regulatory network differences between soldiers and foragers, suggesting bees can take on either role.

Analysis of Ashkenazi Jewish Cohort Uncovers New Genetic Loci Linked to Alzheimer's Disease

The study in Alzheimer's & Dementia highlighted known genes, but also novel ones with biological ties to Alzheimer's disease.

Tara Pacific Expedition Project Team Finds High Diversity Within Coral Reef Microbiome

In papers appearing in Nature Communications and elsewhere, the team reports on findings from the two-year excursion examining coral reefs.