Open Science for Children’s Health
We are a virtual biotech company focused on transforming how new medicines for childhood diseases are developed.
A World Where Every Child Has Access to Treatment
Diffuse Intrinsic Pontine Glioma (DIPG) is a rare and aggressive pediatric cancer located in the pons region of the brainstem, classified as a broader class of H3 K27M mutant Diffuse Midline Gliomas (DMG). Focal radiation remains the standard of care; however, the median survival is only 9-12 months, with no effective chemotherapeutic options available. Traditional drug development models struggle to address rare diseases like DIPG due to small patient populations and high risks.
At M4K Pharma, we are proving there’s a better way.
We are revolutionizing the discovery and development of affordable medicines through open science. By openly sharing knowledge, aligning global research efforts, and breaking down barriers like patents, we are accelerating breakthroughs and ensuring that treatments are accessible to all.
Progress Towards a Cure
Pioneering a New Therapeutic Approach for DIPG
Our first program focuses on developing an ALK2 enzyme inhibitor, the first therapeutic designed specifically for DIPG. About 25% of DIPG cases are linked to mutations in the ALK2, an enzyme identified to play a contributing role in the pathogenesis of DIPG. We are leveraging highly potent, selective, and drug-like molecules targeting this protein to create a promising therapeutic option for children affected by this devastating disease.
We are committed to transparency. All research data is shared freely in the public domain. Stay connected to our mission, track our progress, and join the conversation through our regular online meetings.
Accelerating Progress with Trusted Partners
Supported by trusted partners, we are committed to ensuring that no child’s future is left to chance.