The investigational gene therapy AMT-130 is slowing disease progression for people with early-stage Huntington’s disease, as well as lowering levels of neurofilament light chain (NfL), a marker of nerve cell damage. That’s according to two-year data from two Phase 1/2 clinical trials: one in the U.S. (NCT04120493)…

Neurologists in the U.S. are optimistic about the potential for a new treatment for Huntington’s disease, but emphasize the need for therapies that halt nerve degeneration before symptom onset, according to a report by Spherix Global Insights. Most of the specialist physicians surveyed hoped for a gene therapy…

With early data on AMT-130 for Huntington’s disease reportedly looking promising, uniQure said it’s planning to meet early next year with regulatory authorities to discuss the late-stage development of its gene therapy candidate. By that time, more data on the ongoing Phase 1/2 clinical trials testing the therapy…

Asklepios Biopharmaceutical (AskBio) has launched a first-in-human clinical trial testing AB-1001 — an experimental gene therapy designed to modulate cholesterol metabolism in the brain — in people with Huntington’s disease. The trial is currently recruiting participants at a site in Paris. It is open to adults, ages 18…

A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…

The first two dosing groups in a Phase 1/2 trial of AMT-130, uniQure’s investigational gene therapy for Huntington’s disease, are now fully enrolled. The trial (NCT04120493), being conducted across 12 U.S. locations, is assessing the safety and tolerability of a single AMT-130 infusion into the brains of…

The first two patients in Europe have been dosed in uniQure’s Phase 1/2 clinical trial investigating AMT-130, a one-time investigational gene therapy for Huntington’s disease. The trial is underway in Poland, where it is still open for enrollment, and is expected to expand to other sites in the…

AMT-130, an investigational gene therapy for the treatment of Huntington’s disease, was overall well-tolerated up to a year after treatment in the first patients to receive the therapy in a clinical trial. The new data, announced by AMT-130’s developer uniQure, cover the first four participants in a Phase…

A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according to a positive review by the study’s independent data safety monitoring board (DSMB). The DSMB reviewed safety data from four patients given the higher dose of the therapy, which…

NeuExcell Therapeutics has entered into a research deal with Spark Therapeutics to advance the development of a safe and effective neuroregenerative gene therapy for Huntington’s disease. Under the terms of the agreement, Spark, a member of the Roche Group, will have access to NeuExcell’s gene therapy platform…