We aim to create the first-ever globally approved drug to tackle MEF2C deficiency.
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The transcription factor Myocyte Enhancer Factor 2C (MEF2C) regulates which genes get turned on or off in our cells and is fundamental to our body’s development and function. The deficiency in MEF2C results in MEF2C Haploinsufficiency Syndrome (MCHS). Hundreds of children live with this ultra-rare disease, a complex form of Autism with severe implications on nearly every facet of daily life. But right now, there is no therapeutic that treats MCHS.
We seek to change that.
Working with our partners worldwide, Rare Bird aims to create the first-ever globally approved drug to tackle MEF2C deficiency within five years.
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Why Therapeutics?
MEF2C plays a crucial role in multiple organ systems, and guides the development, maturation, and differentiation of brain, immune, muscle, blood vessel, and other cells. MCHS with severe implications on nearly every facet of daily life. Patients experience complex seizures, severe hypotonia, visual impairment, gastrointestinal difficulties, absent speech, and a host of additional medical complications. We seek to develop targeted therapeutics that address the root cause of MCHS as well as the particular symptom profiles that we see in our population.
Who Benefits?
The vast majority of our population are children under five years old, whose brains are actively maturing. We fully expect that the therapeutics we develop will tangibly benefit our diverse patient population.
But the benefits of our work do not end here.
Our scientific research aims to generate treatment options for MCHS specifically, but our discoveries will also expand the scientific community’s understanding of critical neurological diseases—from Autism to Alzheimer's—and advance the cause of rare disease treatment more broadly.