Hereditary Transthyretin Amyloidosis Market: Intellia/Regeneron’s CRISPR Gene Editor Nexiguran ziclumeran Receives FDA RMAT Designation

September 6, 2026

GlobeNewswire USA

Overview

This article summarizes a market research report by DelveInsight on the hereditary transthyretin amyloidosis (hATTR) market. The report highlights Nexiguran ziclumeran (nex-z), a CRISPR-Cas9-based gene-editing therapy co-developed by Intellia Therapeutics and Regeneron, as a key asset. Designed as a single-dose treatment to permanently inactivate the TTR gene, nex-z has received Orphan Drug and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, along with Orphan Drug status from the European Commission, signaling high expectations for this innovative therapy.

In Depth

This article provides an overview of a market research report titled “Hereditary Transthyretin Amyloidosis Market Insight, Epidemiology, and Market Forecast” published by DelveInsight.

Report Overview

This report offers a comprehensive analysis of the global market for hereditary transthyretin amyloidosis (hATTR) projected through 2030, covering epidemiology, current treatment paradigms, and future market trends. hATTR is a progressive, fatal disease caused by the abnormal accumulation of transthyretin (TTR) protein, leading to systemic organ damage, including the heart, nerves, and gastrointestinal tract. The report focuses on unmet needs, the pipeline of novel therapies, key treatments, and emerging companies poised to enter the market.

Key Research Findings

Within the hereditary transthyretin amyloidosis (hATTR) treatment market, significant attention is being drawn to Nexiguran ziclumeran (nex-z), a CRISPR-Cas9-based gene-editing therapy co-developed by Intellia Therapeutics and Regeneron. Nex-z is designed as a single-dose treatment intended to permanently inactivate the expression of the disease-causing TTR gene in the liver. This gene-editing approach aims to fundamentally halt the production of abnormal TTR protein, thereby arresting disease progression. Nex-z has received both Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA (U.S. Food and Drug Administration), in addition to Orphan Drug Designation from the European Commission. These designations clearly indicate that nex-z is garnering high expectations and expedited development support from regulatory authorities as an innovative therapeutic for severe rare diseases like hATTR. The report suggests that gene-editing therapies such as nex-z may offer superior long-term efficacy and patient convenience compared to existing TTR stabilizers and TTR-silencing drugs, potentially transforming the treatment landscape for hATTR.

About the Publisher

DelveInsight is a market research and consulting firm specializing in the life sciences sector. It provides detailed market analysis, pipeline evaluations, and competitive intelligence across a broad spectrum of the healthcare industry, including pharmaceuticals, biotechnology, and medical devices. The company’s reports aim to offer deep insights and data to assist pharmaceutical companies, investors, and research institutions in making strategic decisions.

Source: https://vertexaisearch.cloud.google.com/grounding-api-redirect/AUZIYQEZzIJpV6MkLEBvr95emz3DS7xkcmEBuEmIW3-Dd3pcvMwgstLVDiuOZeaHm0jiSl4fXzTzDpkYA1BnTpCMsde3dik0yWQL13LRe6c3Xu7VHTnvzHMPXWF58TDf7r84gn3Wi-1BtJ0-YVx6m4oAqcLWyLml_DBpe6CQAHvvVQzt0h6VwdfRkT_dOrOj9kAL1UnVWnp1WsQj9kU7mxE01ua1XeK3xtaox9c9w0oqcdrriAAIg545sLQ1S5Rk0su_-TAZcj1nTrkTslDiKomIsI0PMofT0UNJUGRm11eKHKIf0A34MQa9ooEyGM5OnAwopv_OrnVseHYwgVGGdBZ10egnzZBrcCTEyC_nOr3cSeorNDZUub6qpY-YsTqi0ZCHNCVteJZIuDsn6TCyWCWxrpVZ3yU5VvIqZrhYiWM

Get our weekly technology intelligence — free

Receive an infographic that lets you judge at a glance whether each field’s analysis report is worth reading.

Subscribe Free — Weekly Tech Intelligence

By subscribing, you’ll receive Troy-Technical’s weekly technology intelligence newsletter.