Help us reach $50,000 by Giving Tuesday!
Over the past year, you've helped the Rare Bird Foundation move mountains for children with MEF2C Haploinsufficiency Syndrome, a profound neurodevelopmental disorder and rare disease. We've developed two therapeutics that we've shown to increase the level of MEF2C in mouse and human cells, and we've launched the first-ever longitudinal clinical readiness study to map our disease's trajectory as a baseline for clinical trial.
We've also organized our community's first formal engagement with the FDA, characterized three distinct MCHS mouse models to understand the nuances of various mutations of MEF2C, and in the next couple months we will deliver our community's first iPSC lines and toxicology tests to optimize our treatments. Your support has enabled this ground-breaking work, and we couldn't have done it without you. For the first time ever, families whose children have received an MCHS diagnosis have hope that a treatment is on the horizon. (Read our annual report here.)
Of course, we have much still to do. In keeping with our #strive4five mission, we are committed to seeing our therapeutic candidates through the next 12 months of critical work--ensuring their efficacy, safety, deliverability, and regulatory approval--so we can get from lab to clinic and ultimately to patients worldwide within five years (that's three years from now!).
We have accomplished so much with your help and there are so many exciting partnerships and developments in the works--from industry and academic partnerships to get us the manufacturing materials we need quickly and at no cost, to cooperative research agreements to get all the necessary expertise in drug development, to additional clinical readiness sites so our global population can take part, to new translational projects to address additional avenues of relief for our children.
Please continue to support our work. If we raise $50,000 by Giving Tuesday--$5,000 a day for the next ten days--we will receive a $25,000 from a matching donor!
Where does your money go?
Rare Bird is a tax-exempt, U.S.-based organization dedicated to creating the first-ever globally approved drug to tackle MEF2C deficiency within five years, our #strive4five mission. Your donations will fund the following work over the next year:
1) Therapeutic development
- Optimization of dosage testing and dose response
- Early toxicology study in rats
- Mouse characterization in three mouse models to understand impact of different types of mutations
- Testing in mutation-varied iPSC lines
- Testing with human grade virus and viral permutations
2) Clinical readiness
- 33 patients seen for first of four visits over two years
- First ever iPSC lines in progress for six mutations (to be delivered in two months)
- Research EEGs to develop biomarkers
- Launch of second global site with subsidized clinical visits, allowing parties across the world to easily participate, improve clinical expertise/diagnosis in MCHS worldwide
- Part 11 compliance for fda approval
Why donate to Rare Bird?
- We are the only treatment-focused MCHS organization, and we are laser focused on developing and delivering therapeutics to the MCHS community
- We are the only U.S.-based MCHS organization focused on global delivery, so no one gets left behind
- Our team is the largest fundraiser and contributor to MEF2C research over the past three years. We have raised and channeled funds to all partners, totaling about $1m so far
- Proven strategic focus: We are founded and led by visionaries who have worked in the highest-stakes settings, and we pride our ability to strategize and deliver with efficiency.
- Depth and breadth of partnerships: We partner strategically to make sure we are tackling every unknown both efficiently and expertly.
Thanks again for all your support! As always, feel free to reach out to us with any questions.
With gratitude,
The Rare Bird Team
Organizer
Rare Bird Foundation
Beneficiary