Ahmad Masri, MD, MS, on the Potential of Gene Therapy in ATTR Amyloidosis


In observance of World Amyloidosis Day, the cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University discussed the changing landscape of care in this field.

“I encourage everyone to continue to learn about amyloidosis: systemic amyloidosis, ATTR amyloidosis, light chain amyloidosis, as well as other types of amyloidosis. These are not ultra-rare diseases like people used to think. These are patients in our clinics not infrequently. I encourage everybody to continue to think about this, read about it, and use it as a case study for how things evolve over time... the whole landscape looks very different in 2023, compared to just 10 years ago.”

Transthyretin (ATTR) amyloidosis is a form of amyloidosis related to misfolding of the transthyretin (TTR) protein; the misfolded protein builds up in the body, causing damage to various organs, including the heart. Although there are several FDA-approved therapies for ATTR amyloidosis currently available, these treatments are mainly limited to slowing or stabilizing progression of the disease and require continuous dosing; as such, significant unmet need remains for this patient population.

One new potential option on the horizon for the treatment of ATTR amyloidosis is Intellia and Regeneron’s NTLA-2001, an investigational CRISPR/Cas9-based gene therapy, intended to deactivate the gene responsible for producing TTR in the liver, thus preventing or substantially reducing production of the misfolded protein with a single, one-time treatment. NTLA-2001 is currently being evaluated in a phase 1 clinical trial (NCT04601051).

For World Amyloidosis Day, observed annually on October 26 by the patient and clinician communities, CGTLive™ interviewed Ahmad Masri, MD, MS, a cardiologist at the Center for Hypertrophic Cardiomyopathy at Oregon Health & Science University, about the current landscape of care for ATTR amyloidosis and how NTLA-2001 could potentially transform this landscape. He went over some of the data generated by the gene therapy so far, noting that it has shown the ability to reduce production of TTR by more than 85% to 90% after a single dose. Masri concluded the interview by summing up his thoughts on World Amyloidosis Day and giving his main message regarding the disease for doctors and the broader healthcare community to keep in mind in 2023.

Intellia and Regeneron present updated interim data from phase 1 study of CRISPR-based NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis demonstrating that deep serum TTR reductions remained durable after a single dose. News release. Intellia Therapeutics, Inc. and Regeneron Pharmaceuticals, Inc. June 24, 2022. Accessed October 26, 2023. https://ir.intelliatx.com/news-releases/news-release-details/intellia-and-regeneron-present-updated-interim-data-phase-1

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