Tag: transthyretin
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ATTR-CM Part 3 — gene editing (nex-z): a single, permanent TTR knockout buys durability by genome permanence, but the real bottleneck is the asymmetric safety risk that irreversibility creates
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 knockdown figures are attributed to the sponsoring trial or company; nex-z Phase 1 numbers are Intellia investor/conference data (AHA 2025-11), company presentations rather than peer-reviewed data. Safety…
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The ATTR-CM commercial map — revenue and narrative are decoupled: stabilizers own the market, siRNA becomes a blockbuster, and the real bottlenecks are the add-on outcome threshold and the price-value gap
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 revenue, prescription, pricing and reduction figures are attributed to the company’s own results (press release / SEC), to the ICER report, or to market-analysis reporting. The acoramidis-versus-tafamidis real-world comparison…
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Hard outcomes in ATTR-CM — mortality and CV events are settled within each trial, but “which modality is better” is undeterminable because there is not a single head-to-head RCT
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. Every hard-outcome figure is attributed to its own trial’s primary paper or press release; the eplontersen miss and monotherapy signal are attributed to the AstraZeneca/Ionis press release of 2026-07-09 (press-release…
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The TTR silencers — siRNA vutrisiran succeeded, ASO eplontersen missed, but the deciding variable was the trial population, not the modality
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 knockdown, hazard-ratio and subgroup figures are attributed to the sponsoring trial, journal or company; several eplontersen numbers are a company press release rather than peer-reviewed data (noted inline), with…
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Stabilizers in ATTR-CM — tafamidis and acoramidis built the first hard-outcome standard of care, but the “next-generation” increment is not proven mortality superiority
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 efficacy figures are attributed to the sponsoring trial or company (ATTR-ACT to Pfizer/NEJM 2018; ATTRibute-CM to BridgeBio/NEJM 2024). Cross-trial comparisons are not head-to-head. The “near-complete (≥90%) stabilization” claim is…
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The ATTR cardiomyopathy landscape — four modalities target one protein, but the siRNA-success/ASO-miss split is cross-trial, not head-to-head
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 outcome figures are attributed to the sponsoring trial or company; some are company press releases rather than peer-reviewed data (noted inline). The eplontersen result below is a…
Uthando Nethemba 