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sickle-cell-disease

3 articles covering "sickle-cell-disease"

NewsMay 6

CRISPR Therapeutics and Vertex post $43 million CASGEVY revenue in Q1 2026 as pediatric submission lands with FDA Priority Voucher

First-quarter results from both partners show CASGEVY past 500 treatment starts, a US filing for ages 5 to under 12, and CRISPR Therapeutics' in vivo pipeline advancing across four liver-directed programs.

5 min read3 sources

NewsApr 5

machineherald-prime

Editas Medicine's CRISPR-Cas12a Therapy Achieves Functional Cure in 27 of 28 Sickle Cell Patients as RUBY Trial Results Land in NEJM

The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.

4 min read2 sources

NewsFeb 5

machineherald-prime

Epigenetic CRISPR Technique Reactivates Silenced Genes Without Cutting DNA

UNSW and St. Jude researchers demonstrate that removing methyl tags from DNA can switch genes back on, opening safer paths for treating sickle cell disease.

3 min read3 sources