Brendan Smith

Laura Chico

Neena Bitritto-Garg

Yun Zhong

Danielle Brill

Omari Baruti

Whitney Ijem

Alex Nackenoff

Madhu Kumar

Tessa Romero

- **UniCure** announced **75% statistically significant slowing of disease progression** in Huntington's disease patients treated with **AMT-130** after three years of follow-up, with a **pre-BLA meeting expected in Q4 2025** and **BLA submission in Q1 2026**.  **[0001787297_2194117_1]** 
- **MIRA** detailed its Parkinson's program, which has demonstrated **three positive studies and two positive studies UPDRS against sham**, and is leveraging **AI technology** to show physiological brain changes and strengthen pivotal study endpoints.  **[0001787297_2194117_0]** **[0001787297_2194117_4]** 
- **Passage Bio** reported its lead clinical program for frontotemporal dementia (FTD) with granulin mutation shows **high durable levels of target engagement of CSF progranulin** and stabilization of plasma neurofilaments.  **[0001787297_2194117_2]** 
- All three companies highlighted the advantages of **local, targeted delivery** for neurodegenerative gene therapies, emphasizing **enhanced bioavailability** and **reduced systemic safety risks**.  **[0001787297_2194117_1]** **[0001787297_2194117_6]** 
- Panelists observed that regulatory bodies, including the FDA, are demonstrating **increased flexibility** for rare diseases with high unmet needs, supporting novel trial designs, biomarkers, and single-arm studies, as outlined in the FDA's **rare disease evidence principles**.  **[0001787297_2194117_5]** **[0001787297_2194117_7]**

UniCure, MIRA, and Passage Bio Discuss Progress and Challenges in Neurodegenerative Gene Therapies

- **uniQure** reported **remarkable findings** from its Huntington's disease program (AMT-130), including a **75% statistically significant slowing of disease progression** based on composite UHDRS, with a pre-BLA meeting expected in **Q4** and BLA submission in **Q1 2026**.  **[0001787297_2194095_1]** 
- **Mira** highlighted its Parkinson's program as the **only treatment to have three positive studies and two positive UPDRS studies against SHAM**, utilizing an AI partnership to demonstrate **physiological changes in the brain** and neuroprotective effects, attributing its **incredibly good safety** to local delivery and the AAV2 capsid.  **[0001787297_2194095_0]** **[0001787297_2194095_6]** 
- **Passage Bio** presented its lead clinical program for **frontotemporal dementia (FTD) with GRN mutation**, showing **high durable levels of target engagement of CSF progranulin** and stabilization of plasma neurofilaments, with more data anticipated next year.  **[0001787297_2194095_2]** 
- Panelists discussed key challenges in gene therapy for neurodegenerative diseases, such as **delivery to the brain** and the **slow progression and heterogeneity of diseases**, while also noting the **FDA's increased flexibility** for rare diseases with high unmet needs regarding trial designs and biomarkers.  **[0001787297_2194095_2]** **[0001787297_2194095_3]** **[0001787297_2194095_4]** **[0001787297_2194095_7]**

Genetic Medicines Conference Highlights Progress in Neurodegenerative Disease Gene Therapies

Passage BIO (PASG)