Industry celebrates two newly approved genetic medicines, but patients with these blood disorders may face a long wait, and many will never access them because of their high cost.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
Change history
01 December 2022
In the version of this article initially published, the blood cell image shown was an incorrect version and has been replaced, while previously (14 November 2022), there was a mistake in paragraph 8, where “Biomarin” first appeared as “Vertex”; the changes have been made in the HTML and PDF versions of the article.
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Sheridan, C. For hemophilia and thalassemia, a new era of ‘one-and-done’ gene therapies has arrived. Nat Biotechnol 40, 1531–1533 (2022). https://doi.org/10.1038/s41587-022-01555-0
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41587-022-01555-0
