#USA #Las Vegas #gene therapy #Amyloidosis #hATTR

Hereditary Transthyretin Amyloidosis Market Growth

The market for hereditary transthyretin amyloidosis (hATTR) is currently witnessing notable expansion, thanks to increased awareness of the disease, technological advancements in gene-silencing therapies, and the introduction of innovative treatment options. In recent years, the global landscape of this market has evolved dramatically, as several new therapies are expected to enter the market, significantly improving patient outcomes.

Current Market Insights

According to DelveInsight's latest report, the hereditary transthyretin amyloidosis market is projected to experience robust growth by 2034. Among the leading markets analyzed, the United States is forecasted to remain the largest contributor to the hATTR treatment sector, making up approximately 65% of diagnosed hATTR cases in the seven major markets (7MM), which include the US, the EU4, the UK, and Japan. The momentum gained is credited to several factors, including increased disease awareness and the development of targeted therapies from key pharmaceutical companies such as Intellia Therapeutics, Neurimmune, and Novo Nordisk.

Emerging Therapies and Innovations

New treatment modalities are at the forefront of this market's growth. Innovative therapies like Nexiguran Ziclumeran (developed by Intellia Therapeutics) and ALXN2220 (developed by Neurimmune/Alexion Pharmaceuticals) are currently undergoing clinical trials and hold the potential to radically change the treatment landscape for hATTR. These therapies utilize advanced techniques including CRISPR-Cas9 for gene editing, aiming directly at the root cause of the disease—mutations in the transthyretin (TTR) gene.

Furthermore, traditional treatment options are evolving. Gene-silencing agents such as patisiran (ONPATTRO) and inotersen (TEGSEDI) work by reducing the production of both normal and mutated TTR proteins, playing a critical role in slowing the progression of the disease. Besides, TTR stabilizers like tafamidis (VYNDAQEL/VYNDAMAX) are also being used to prevent amyloid fibril formation, showcasing the diverse approach being taken to manage hATTR effectively.

Key Drivers of Market Expansion

Several key factors are propelling the growth of the hereditary transthyretin amyloidosis market:

• - Improved Diagnostic Techniques: Advancements in genetic screening and testing allow for earlier detection of hATTR, leading to timely intervention and treatment.
• - CRISPR-Cas9 Innovations: The potential of gene-editing therapies like Nexiguran Ziclumeran to provide a one-time curative solution is driving interest and investments in the sector.
• - Promising Pipeline of Therapies: The anticipated approvals and eventual market introduction of therapies like Coramitug are expected to significantly impact market dynamics between 2025 and 2034.

Competitive Landscape

The competitive arena for hATTR therapies is gearing up for intense activity. Currently, companies are engaged in research and development for a range of promising candidates, setting the stage for one of the most exciting periods in amyloidosis treatment history.
Intellia Therapeutics stands out with its CRISPR-based approach, while Neurimmune's ALXN2220 promises versatility in targeting different forms of ATTR. In tandem, the focus on patient-centric solutions is becoming evident, as supportive care and symptom management improve the quality of life for patients suffering from complications associated with hATTR.

Clinical Trials and Recent Developments

Highlighted recent developments include:

• - August 2025: Prothena Corporation announced progress on Coramitug entering Phase III trials.
• - June 2025: Alnylam Pharmaceuticals received additional indication approval for AMVUTTRA in treating various forms of hATTR.
• - May 2025: Intellia Therapeutics reported promising follow-up data from its Phase I clinical trial for Nexiguran Ziclumeran.

These advancements reflect a persistent commitment to tackling hATTR through innovative solutions and therapies that offer newfound hope to patients.

Conclusion

The hereditary transthyretin amyloidosis market is positioned for substantial growth, driven by a confluence of improved awareness, revolutionary treatment options, and clinical advancements. Enhancements in both therapeutic strategies and diagnostic techniques are likely to yield improved patient outcomes and create a positive trajectory for the market leading into the next decade. As development continues, the industry will await regulatory approvals and real-world applications of these groundbreaking treatments that could very well redefine the management of hATTR for patients worldwide.