Tag: gene editing
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Mechanism-Encoded Prediction of Prime Editing: What OptiPrime’s Ablations Actually Prove, and What Remains Unconfirmed
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice. The Nature Biotechnology version of the paper discussed here is paywalled and was not accessed by this outlet. Everything below marked “confirmed” was verified directly against the bioRxiv preprint (submitted 2026-02-20), which the…
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In-vivo gene editing — big pharma bet on the delivery platform, not the editor, and the real bottleneck (hard outcomes) is untouched
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice, not medical advice. The 30-second version What. In 2025–26 big pharma bought its way into in-vivo gene editing and in-vivo CAR-T (Eli Lilly–Verve up to $1.3B; AbbVie–Capstan up to $2.1B). But the bet…
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The real clinical cost of in-vivo gene editing is not potency but irreversibility — reading the MAGNITUDE hepatotoxicity signal, the death, and asymmetric risk by evidence tier
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice, and not medical advice. The 30-second version What. Across Parts 0–3 this series showed that in-vivo editing has already matched siRNA/ASO on the headline metric — knockdown depth (PCSK9 −88%, TTR −87%, Z-AAT…
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In-vivo gene editing, Part 2 — delivery is the rate-limiting step, and why that confines the field to the liver
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice, not medical advice. Delivery-performance figures (editing efficiency, knockdown) are largely company, conference or preclinical (NHP/mouse) data and are attributed as such in the text; safety events are attributed to regulatory/source documents and unresolved…
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Editing modality — nuclease, base, prime — and why the most precise editor is the least clinically mature
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice, not medical advice. The 30-second version What. In-vivo gene editing is not one technology but a precision spectrum: nuclease (double-strand break, random indels — a “knockout” tool) → base editing (single-base substitution, no…
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The in-vivo gene-editing landscape — one-shot, lifelong lipid lowering has arrived at the headline, but delivery, safety and irreversibility are the real bottlenecks
Evidence-first notes on bioscience and deep tech, at the edge of the lab and the market. Information only — not investment advice, not medical advice. All reduction and editing-efficiency figures are attributed to the sponsoring trial or company; many are company press releases or conference presentations rather than peer-reviewed data (noted inline). Safety events are…
