We are proud to announce the first three recipients of funding via our Developing Medicines through Open Science program. These grantees are working towards treatments and cures for three rare diseases: a genetic neuromuscular disorder, a pediatric brain cancer, and a liver disease.
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Estrid Jakobsen at [email protected]
Julia Smith at [email protected]
Conscience Announces Recipients of Inaugural Program to Foster Collaborative Drug Discovery and Development
The Developing Medicines through Open Science program supports preclinical drug development in areas of pharmaceutical market failure
Program announcement leads into Rare Disease Day on February 28
Toronto, ON, Canada, February 27, 2025 — Conscience, a non-profit focused on accelerating drug discovery in areas of unmet medical need using open science and radical collaboration, is pleased to announce three recipients and projects have been approved for its Developing Medicines through Open Science (DMOS) program. The DMOS program is designed to foster collaborative drug discovery and development in areas often overlooked by the pharmaceutical industry, such as rare and orphan diseases and antimicrobial resistance. The inaugural round of this program is offering a total of $5M in funding and partnership opportunities to support significant advancements toward drug candidates.
Rare Disease Day is recognized on the last day of February to increase awareness for rare diseases and improve access to treatment for those living with rare diseases and their families. The DMOS program grantees are working towards treatments and cures for three rare diseases: a genetic neuromuscular disorder, a pediatric brain cancer, and a liver disease.
“The DMOS program is underscored by our vision of a world where more drug therapies are developed, and these treatments are both accessible and affordable,” says Anne Fortier, Vice President (VP), Drug Discovery at Conscience. “We believe collaboration is the solution and proudly support projects that undertake preclinical research to establish proof-of-concept for an open science approach towards treatments and cures for life-threatening or severely debilitating diseases in areas of market failure. We can’t wait to work with and learn from each of the projects currently in our program.”
Additional Quotes
“Rare Disease Day offers a reminder that while rare diseases affect a limited number of people, their effects are devastating to those impacted and their loved ones. That is why our government supports Conscience’s expertise through the Strategic Innovation Fund (SIF) to tackle rare diseases to improve access to care and treatments for Canadians. This contribution will help develop drug discovery through open science, and I look forward to the results of these projects.”
– François-Philippe Champagne, Minister of Innovation, Science and Industry
“We are grateful for how the Conscience grant is supporting Primary Sclerosing Cholangitis (PSC) research. PSC is a rare and devastating liver disease with no treatments or cure. This grant enables us to build on work that maps PSC liver cells for pursuing preclinical drug development. With an unwavering commitment to a cure, we believe in the power of an open science approach. We envision a world where a PSC diagnosis comes with a safe and effective treatment, and Conscience’s Developing Medicines through Open Science program takes us one step closer”
– Mary Vyas, President, PSC Partners Seeking a Cure Canada
DMOS Project Details
The following projects listed below were selected in this round. Although submissions for the inaugural round are now closed, DMOS may accept proposals on a rolling basis. For applicants interested in submitting a proposal that adheres to the program’s scope and eligibility criteria, please visit the website to learn more.
| Project 1 |
| Focus: Small Molecule PRMT6 Inhibitors for the Treatment of Spinal Bulbar Muscular Atrophy Description: Spinal Bulbar Muscular Atrophy (SBMA) or Kennedy’s disease, is a rare, inherited neuromuscular disorder that leads to progressive muscle degeneration, weakness, and twitching. It affects approximately one in 100,000 individuals worldwide, with a significantly higher prevalence in Canada’s Indigenous populations, particularly in Alberta and Saskatchewan, due to genetic factors. There are no approved treatments for SBMA.Recipients: SME: Agora Open Science Trust Representative – Dr. Peter Sampson, VP of Drug Discovery and Development Principal Investigator: Dr. Rachel Harding, Assistant Professor at the Leslie Dan Faculty of Pharmacy and Principal Investigator at the Structural Genomics Consortium, University of Toronto Co-investigator: Dr. Mark Reed, Scientist at the Krembil Brain Institute, University Health Network Scientific Approach: The project focuses on developing a novel therapeutic approach targeting PRMT6, a co-activator of the Androgen Receptor (AR) implicated in SBMA progression. Building on a novel and highly potent lead compound, they will undertake a highly collaborative, drug discovery approach with a team of experts in computer and AI-driven drug design, chemistry and molecular biology to create a drug for the treatment of SBMA. Conscience Funding: $386,220 CAN Term: 2 years |
| Project 2 |
| Focus: Pre-Clinical Development of an ALK2 Inhibitor for the Treatment of Diffuse Intrinsic Pontine Glioma Description: Diffuse Intrinsic Pontine Glioma (DIPG) is a rare, inoperable and fatal pediatric brain cancer, with most children surviving only nine to twelve months after diagnosis. Radiation therapy is the only current treatment option offering only temporary relief. New treatment options are challenging as many drugs fail to reach the tumour due to the brain’s protective barrier. Recipients: SME: M4K Pharma Inc (owned by Agora Open Science Trust) Representative and Principal Investigator – Dr. Peter Sampson, VP of Drug Discovery and Development Co-investigators: Dr. David Uehling, Director, Medicinal Chemistry, Drug Discovery Program, Ontario Institute for Cancer Research (OICR)Dr. Neil Vasdev, Scientific Director and Chief Radiochemist of the Brain Health Imaging Centre and Director of the Azrieli Centre for Neuro-Radiochemistry, Centre for Addiction and Mental Health (CAMH) Scientific Approach: Around 30 per cent of DIPG tumors have mutations in a protein called ALK2. Research has shown that blocking the activity of ALK2 impacts DIPG cell growth, making ALK2 a promising target for drug development. The team has demonstrated that their potent, selective ALK2 inhibitors are brain penetrant, slow DIPG cancer cell growth, and extend the survival of mice with DIPG. Conscience Funding: $1,599,329 CAN Term: 2 years |
| Project 3 |
| Focus: Open Drug Discovery to Treat Primary Sclerosing Cholangitis Description: Primary Sclerosing Cholangitis (PSC) is a rare liver disease with no approved treatments and a significant unmet medical need. The limited patient population makes investment in drug development for PSC challenging. This project seeks to address this by taking a collaborative open science approach to advance new treatments for this rare disease, which currently has only a limited number of active pharmaceutical programs. Recipients: SME: Agora Open Science TrustRepresentative: Dr. Peter Sampson, VP of Drug Discovery and Development Principal Investigator: Dr. Sonya MacParland, Senior Scientist in the Ajmera Transplant Centre, University Health Network Co-Investigators: Dr. Ian McGilvray, Professor of Surgery, University Health Network Dr. Gary Bader, Professor, University of Toronto Scientific Approach: The investigators have developed the first transcriptomic map of the PSC liver, providing an unprecedented view of the disease mechanisms at the cellular level. This work has enabled the identification of known targets and their corresponding drugs, highlighting SYK inhibition as an avenue to target immune-mediated fibrotic pathways within the PSC liver. This project will test the ability of SYK inhibitors to modulate fibrogenic processes in human explanted PSC tissue in comparison to healthy human liver. Conscience Funding: $461,241 CAN Term: 2 years |
DMOS offers a unique opportunity to contribute to the advancement of drug discovery and development through open science. In 2024, proposals were submitted to Conscience for consideration, each requiring a comprehensive preclinical research plan targeting an area of pharmaceutical market failure, with research conducted at Technology Readiness Levels (TRLs) 2-6. Additionally, each proposal needed to involve at least one Canadian SME, commit to Conscience’s Open Science Policy, and provide proof of matching funds to cover the remaining cost, as Conscience currently funds a maximum of 33 per cent to academics and 50 per cent to SMEs.
About Conscience
Conscience is a nonprofit biotech focused on transforming the drug development landscape by enabling the discovery of potential treatments for diseases that have received limited attention from the pharmaceutical industry. Conscience seeks to use collaborative approaches, open science, and artificial intelligence to break down barriers of profit-driven models. Powered by a network that includes academics, industry, technologists, policy experts, and public support, Conscience aims to drive innovation in drug discovery and development, and treatment accessibility. Through key initiatives, such as its DMOS (Developing Medicines through Open Science) program and CACHE (Critical Assessment of Computation Hit-finding Experiments) Challenges, Conscience is accelerating the path to treatments for those who need them most to ensure no one is left behind. For more information, visit www.conscience.ca.
