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in vivo gene therapy

PharmaDeviceNews in vivo gene therapy
Representative image: Ensoma’s EN-374 clinical safety data highlight the growing promise of in vivo hematopoietic stem cell engineering for X-linked chronic granulomatous disease, as gene therapy developers test whether one-time treatments can move beyond complex ex vivo manufacturing and into scalable clinical use.
Pharma & Biotech

Ensoma’s EN-374 safety data open a new test for in vivo HSC engineering

In vivo gene therapy has reached human HSC engineering. Ensoma’s EN-374 safety data raise a bigger question: can durability follow?

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Pharma & Biotech

Scribe Therapeutics deepens in vivo CRISPR validation with second Eli Lilly collaboration milestone

Scribe Therapeutics hits a second in vivo CRISPR milestone with Eli Lilly. Explore what this changes for gene editing platforms and future therapies.

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Representative image. A laboratory research setting illustrating early-stage genome‑editing development, reflecting the FDA clearance allowing GenEditBio to begin its first clinical trial of GEB‑101 for TGFBI corneal dystrophy.
Pharma & Biotech

FDA gives go-ahead to GenEditBio’s first genome-editing trial for TGFBI corneal dystrophy

GenEditBio Limited, a Hong Kong–headquartered clinical-stage biotechnology firm focused on in vivo genome-editing therapies, announced that the United States Food and Drug Administration has cleared its Investigational New Drug application for GEB‑101, a CRISPR-based candidate for TGFBI corneal dystrophy. This regulatory milestone authorizes the launch of the Phase 1/2 CLARITY trial in the United States,

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