Intellia Therapeutics’ Nexiguran Ziclumeran Receives RMAT Designation from FDA for Treatment of Transthyretin Amyloidosis with Cardiomyopathy
Cambridge, Mass. – March 26, 2025 – Intellia Therapeutics, a pioneering clinical-stage gene editing company dedicated to transforming medicine with CRISPR-based therapies, recently announced a significant milestone in its quest to revolutionize the healthcare industry. The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Intellia’s nexiguran ziclumeran (nex-z), also known as NTLA-2001, for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM).
About Transthyretin Amyloidosis with Cardiomyopathy
Transthyretin amyloidosis, or ATTR, is a rare, progressive, and often fatal disease caused by the misfolding of the transthyretin protein. The protein, which is produced in the liver, normally functions as a carrier for thyroid hormones and retinol-binding protein. However, in individuals with ATTR, the misfolded protein forms amyloid fibrils that accumulate in various organs, including the heart, leading to cardiomyopathy, nerve damage, and other complications.
Intellia’s Nexiguran Ziclumeran: A Promising CRISPR-Based Therapy
Nexiguran ziclumeran is a CRISPR-based therapy designed to address the root cause of ATTR amyloidosis by reducing the production of the misfolded transthyretin protein. The therapy uses Intellia’s proprietary CRISPR-Cas9 technology to edit the TTR gene, which encodes the transthyretin protein, and introduces a specific modification that results in the production of a non-amyloidogenic form of the protein. This, in turn, is expected to prevent the formation of amyloid fibrils and halt the progression of the disease.
The Impact on Patients
For patients with ATTR-CM, the RMAT designation is a beacon of hope. The designation, which is granted to investigational therapies that have the potential to treat, modify, or reverse the course of serious or life-threatening diseases, accelerates the development and review process of nex-z. This means that patients may have access to this groundbreaking therapy sooner than they would otherwise.
- Accelerated development and review process
- Potential for earlier access to a life-changing therapy
- A step forward in the fight against a rare and often fatal disease
The Impact on the World
The granting of the RMAT designation to nex-z is not only a victory for Intellia Therapeutics and the patients with ATTR-CM, but also a significant step forward for the gene editing industry as a whole. It represents a validation of the potential of CRISPR-based therapies to transform medicine and address some of the most challenging diseases.
- A validation of the potential of CRISPR-based therapies
- A breakthrough in the treatment of a rare and often fatal disease
- A step towards a future where gene editing offers a solution to many unmet medical needs
Conclusion
The granting of the RMAT designation to Intellia Therapeutics’ nexiguran ziclumeran is a momentous occasion that marks a significant step forward in the fight against transthyretin amyloidosis with cardiomyopathy. It is a testament to the potential of CRISPR-based therapies to transform medicine and address some of the most challenging diseases. For patients with ATTR-CM, it offers the hope of earlier access to a life-changing therapy. For the world, it represents a validation of the potential of gene editing to revolutionize healthcare and offer solutions to many unmet medical needs.
As we continue to witness the rapid advancements in gene editing technology, it is exciting to imagine the possibilities that lie ahead. With companies like Intellia Therapeutics leading the charge, we can look forward to a future where gene editing offers a solution to many of the most challenging diseases.
