Accelerating GBA1 Progress: Collaboration, Innovation, and Global Partnerships at the 2025 Montréal Meeting

Held in Montréal from June 5 to 7, the second GBA1 Meeting 2025 brought together leading scientists, industry partners, and patient advocates to accelerate discovery in GBA1-associated Parkinson’s and related diseases. Organized by McGill and supported by The Michael J. Fox Foundation and the Silverstein Foundation, the event highlighted rapid progress in understanding and treating GBA1 variants, with a clear message: collaboration and patient-centeredness are key. 

🧪 Day 1 focused on small molecule development, with updates from Vanqua Therapeutics, Gain, Congruence, and Bial. We heard from trainees during an engaging data blitz and were deeply moved by patient and caregiver advocates Pola and Rich Sussman, who urged researchers to share results in a clear, hopeful, and meaningful way. 

🌍 Day 2 showcased major cohort studies like GP2 and PPMI, discussed the clinical implications of GBA1 mutations, and emphasized the importance of harmonized data pipelines and cross-sector collaboration. 

🧬 Day 3 delved into gene and cell therapy approaches, with presentations from Sanofi, Prevail, Capsida, Aspen, and Spur Therapeutics. Each presentation pointed to an exciting horizon for targeted interventions. 

Across all three days, the message was consistent: scientific innovation is accelerating. But to truly move the needle, we must streamline data sharing, embrace diverse voices, and design with patients in mind. 

I was truly impressed by the great minds leading such important initiatives and had the opportunity to connect with global advocates from the UK, South Korea, and beyond. 

The GBA1 community is stronger than ever, and I’m excited to see what comes next. 

#ParkinsonsResearch #GBA1 #Neurogenetics #Neuroscience #Collaboration #GeneTherapy #PatientEngagement #OpenScience #MichaelJFoxFoundation #Montreal