uniQure (QURE) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Advances in gene therapy platforms

• Focus shifting to CNS disorders with four clinical programs expected by next year, leveraging deep expertise in AAV5 and AAV9 capsids for targeted delivery and safety.

• Platform technologies like miRNA silencing are being optimized to minimize toxicity and off-target effects, with proof of concept shown in Huntington’s models.

• Emphasis on suppressing gain-of-function mutations in neurological diseases, using advanced delivery and silencing strategies.

Temporal lobe epilepsy program insights

• AMT-260 targets a novel neuroexcitatory pathway not addressed by current anti-seizure medications, offering a one-time, localized treatment.

• Preclinical data in gold-standard models show substantial seizure reduction and positive neuroexcitatory function outcomes.

• Early clinical signals will focus on measurable reduction in debilitating seizures within 4–12 months, with straightforward proof-of-concept pathways.

• Market opportunity is significant due to high unmet need and limitations of current standard of care.

Huntington’s disease program update

• Recent data show statistically significant slowing of disease progression over two years, with more than 20 patients followed for 24 months.

• First clinical demonstration of neurofilament light reduction in CSF, with an 11% decrease versus a 25% expected increase in untreated patients.

• Regulatory path includes RMAT designation, enabling accelerated approval discussions and frequent FDA engagement starting in H2 2024.

• Natural history data and digital twin comparators are being leveraged to expedite efficacy demonstration.