An experimental gene therapy gives me hope for my children

Here in New Zealand, clinicians and advocates are pushing our country’s government to update its genetic editing and modification legislation.

Historically, New Zealand has maintained some of the strictest gene technology regulations globally. Currently, genetic modification is heavily regulated under the Hazardous Substances and New Organisms Act 1996. The legislation was intended, in part, to protect the environment and public health from genetically modified organisms, which the act classifies as new organisms. However, these laws are limiting research and medical advancements in the field of gene technology.

The Gene Technology Bill, introduced in 2024, aims to establish a more up-to-date and scalable regulatory framework for managing this key area. If passed, this bill would allow for greater use of gene technology, paving the way for significant advancements and greater access to gene-editing therapies in New Zealand.

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April 7, 2025 News by Margarida Maia, PhD

1st FAP patient dosed in Phase 3 trial of gene therapy nex-z

A big step forward in gene therapy

As I’m an advocate for people with hereditary ATTR  (hATTR) amyloidosis, I often reflect on the days when I was fortunate enough to help facilitate clinical trials of an experimental gene therapy called NTLA-2001 for people with hATTR amyloidosis with polyneuropathy here in Auckland. That happened back in 2020; at the time, many viewed gene editing in humans as far-fetched and even dangerous.

In September 2019, my late husband, Aubrey, and I attended an ATTR amyloidosis conference in Berlin, where representatives from Intellia Therapeutics — the pharmaceutical company developing NTLA-2001 — gave a presentation. I don’t believe many people believed, at that point, that the gene-editing therapy could work for patients. But Aubrey and I listened in awe and left the conference convinced we needed to find a way to bring trials of the therapy to our country — which we did.

Fast forward to 2025, and NTLA-2001 — now known as nexiguran ziclumeran (nex-z) — is in a Phase 3 trial called MAGNITUDE-2, with the first patient dosed earlier this month. As of this writing, the study has six global locations, one of which is here in Auckland.

In this trial, up to 50 adults diagnosed with hATTR amyloidosis with polyneuropathy will randomly receive either the one-time gene therapy or a placebo. That marks a significant step forward in our fight against hereditary diseases and in the search for a cure.

Although Aubrey passed away in 2022 from complications of his disease, I know he would’ve been happy to see this kind of progress. We share four children who are at risk of inheriting hATTR amyloidosis, and a gene-editing therapy like nex-z could potentially allow them to live and thrive if they’re diagnosed.

Before this possibility existed, Aubrey and I were adamant that they shouldn’t find out their status until they were well-established adults who could handle the results maturely. But with a gene therapy in a Phase 3 trial, they’re now taking steps to get genetically tested and find out if they have amyloidosis. The sooner they’re tested, the better they’ll be able to prepare, and the sooner they can — hopefully — access treatment if and when it becomes available.

Access to this kind of therapy would have a profound impact on families like ours, which is why the Gene Technology Bill is so important. If my children are diagnosed with hATTR amyloidosis, I want them to be able to receive a treatment that could slow or even stop their disease progression.

Note: FAP News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of FAP News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.