Toronto, ON, Canada — Agora Open Science Trust, in collaboration with the Structural Genomics Consortium (SGC) and an international team of academic researchers, is pleased to announce the launch of a new one-year research project funded by Kennedy’s Disease Association (KDA). This new grant seeks to accelerate the development of targeted therapeutic strategies for Spinal Bulbar Muscular Atrophy (SBMA), a rare neuromuscular disorder with no drugs to slow or halt disease.

This award is part of KDA’s 2025 funding program, in which the organization announced more than $900,000 in grants and fellowships, the highest number of awards in its history. KDA received an unprecedented number of research proposals this year, reflecting growing momentum and global interest in advancing research for Kennedy’s disease and related neuromuscular disorders.

SBMA, also known as Kennedy’s disease, is caused by a toxic gain-of-function mutation in the androgen receptor, leading to progressive muscle weakness and motor neuron degeneration. Despite significant advances in understanding disease mechanisms, no disease-modifying therapies are currently available.

The newly funded project will focus on Protein Arginine Methyltransferase 6 (PRMT6), a transcriptional co-regulator of the androgen receptor that has emerged as a promising therapeutic target in SBMA. Building on the SGC’s development of SGC6870, the first highly selective allosteric PRMT6 chemical probe, the team will combine AI-guided molecular design, medicinal chemistry, and disease-relevant cellular models to advance optimized PRMT6 inhibitors with therapeutic potential.

“This project brings together complementary expertise across chemical biology, artificial intelligence, and neuromuscular disease biology to try to address a critical unmet need for people with SBMA,” said Dr. Rachel Harding, Assistant Professor at the University of Toronto and Principal Investigator at the SGC-Toronto. “By leveraging open science tools and patient-derived models, we aim to generate both optimized chemical matter and rigorous biological validation for PRMT6 as a therapeutic strategy in SBMA.”

Unlike the majority of drug discovery research, this project will operate entirely in the open, with no patents filed at any stage. All data, protocols, and research outputs generated through the project will be shared openly, in keeping with the open science principles of Agora Open Science Trust and the SGC. Chemical structures and datasets will be made publicly available to enable reuse and follow-on research by the global SBMA and rare disease communities. “We believe this open model gives exceptional return on investment for the SBMA community - a collaborative approach where every discovery is laid out for others to build on, expert contributors can add their insight in real time, and research progress is accelerated for the benefit of the entire community.” added Dr. Harding.

The project is structured around two integrated aims:

• AI-driven design and experimental validation of improved PRMT6 inhibitors using biochemical, structural, and cellular assays; and
• Evaluation of lead compounds in SBMA patient-derived motor neuron and muscle cell models to assess efficacy, safety, and modulation of androgen receptor activity.

"KDA is excited to support Professor Harding's collaborative and innovative project.  Her team of international experts and use of AI to examine the potential of PRMT6 as a therapeutic pathway represent a new and important milestone in Kennedy's disease research", said Terry Thompson, President of Kennedy's Disease Association.

The international collaboration includes investigators based in Canada, the United Kingdom, and Italy, spanning expertise in chemical biology, artificial intelligence, medicinal chemistry, and neuromuscular disease biology. In addition to leading academic groups, the project also includes participation from industry, with Variational AI contributing advanced AI-driven molecular design capabilities. Their involvement underscores the growing interest from industry partners in engaging with open science–led research models that enable transparent collaboration while accelerating discovery for rare and underserved diseases.

Leadership contributions come from Dr. Rachel Harding (University of Toronto, SGC-Toronto), Dr. Matthieu Schapira (University of Toronto, SGC-Toronto), Dr. Peter Sampson (Agora Open Science Trust), Dr. Carlo Rinaldi (University of Oxford), and Dr. Emanuela Zuccaro (University of Padova).

“This award from KDA enables a highly collaborative, transparent approach to rare disease drug discovery,” said Dr. Peter Sampson, Vice President of Drug Discovery and Development at Agora Open Science Trust. “It exemplifies how open, cross-border collaboration can advance therapeutic discovery for diseases that remain underserved by traditional drug development models, while laying a foundation for future translational efforts in SBMA”.

About Agora Open Science Trust

Agora Open Science Trust is a Canadian charity whose mission is to accelerate the discovery and development of affordable new medicines through open science. Agora’s first initiative – M4K Pharma (‘Medicines for Kids’) – is using open science to drive preclinical and clinical development of a novel ALK2 inhibitor for the treatment of Diffuse Intrinsic Pontine Glioma (DIPG), a rare pediatric brain cancer. Agora’s pipeline of collaborative open science drug discovery programs has recently expanded to include programs for Spinal Bulbar Muscular Atrophy (SBMA), a rare genetic neuromuscular disorder, and Primary Sclerosing Cholangitis (PSC), a rare liver disease — both of which currently have no approved treatment.

Agora continues to welcome collaborative funding partners whose support will help advance M4K2009 into clinical evaluation and sustain its mission to develop affordable medicines through open science.

You can support its mission at https://www.agoraopensciencetrust.org/donate-to-our-mission. 

About the Structural Genomics Consortium (SGC)

The Structural Genomics Consortium is a global public-private partnership that seeks to accelerate drug discovery by fostering collaboration among a large network of scientists in academia and industry and making all research outputs openly available to the scientific community. The current SGC research sites are located at Goethe University in Frankfurt, Karolinska Institute, McGill University, UCL, the University of North Carolina, Chapel Hill, the University Health Network (UHN) and Unicamp.

More information about SGC: https://www.thesgc.org/