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STAT·3h ago·1 min read
save

Sickle cell gene-editing therapy shows signs of revival as developers revisit trial after earlier safety halt

Nov 5, 2026·read at STAT ↗Neutral

A recent STAT+ article highlights that a sickle cell gene‑editing therapy, whose trial was stopped three years ago after a serious adverse event in the first patient, may be making a comeback. The piece notes that the developers are re‑examining the approach in hopes of overcoming the earlier safety concerns.

The original study was halted when the inaugural participant experienced a severe reaction, prompting the sponsor to pause the program. No new data or timelines were provided, but the renewed interest suggests the company believes the issues can be addressed.

In the same briefing, the report points to new Chinese government restrictions that are slowing investigator‑initiated trials, a development that could impact U.S. biotech firms that have relied on China’s fast, low‑cost testing environment.

The article also mentions RevMed’s recent launch of a cancer drug, underscoring the mix of setbacks and advances shaping the biotech landscape.

source

This writeup was produced by pharmadog from original reporting by STAT.

Original headline: “STAT+: A possible comeback for a failed sickle cell gene therapy”

read at STAT ↗
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companies & drugs in this story

companies
Ultragenyx Pharmaceutical Inc.3ProMIS Neurosciences Inc.
drugs
Rare Disease2
topics
Gene Therapy (AAV)3Oncology3Rare Disease3CRISPR/Cas9Cardiology

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