A Look at 2024: Our Year in Review
2024 was huge for RSRT. We built serious momentum in pursuing cures — kicking off a gene-editing dream team, awarding $9.3M+ for breakthrough research, and futhering genetic medicines. Your support made all these accomplishments possible. This is what progress looks like — let’s keep up the pace!
Roadmap to Cures 2.0
Rare diseases don’t cure themselves. Developing genetic medicines is tough, expensive, and takes teamwork. We’re the organization leading this global effort with our bold $40 million initiative, Roadmap to Cures. The mission is clear and urgent: advance three new genetic medicines for Rett into clinical trials over the next four years. In 2024 our community raised, donated, and made generous multi-year pledges to reach $10.4 Million towards this goal, getting us over 25% there.
RNA Editing Highlights
There are lots of reasons to be excited about RNA editing (the process of fixing “typos” in the instructions DNA gives our cells). This year saw the first success of this approach in human clinical trials by Wave Life Sciences. It’s a big deal breakthrough because RNA editing has the potential to treat about a third of the mutations that cause Rett syndrome. 2024 also ended on a high note as we broadened our partnership with ProQR, a biotech company in the Netherlands focused on RNA editing.
Fueling Collaboration
The MECP2 Editing Consortium Launches
The new Consortium is an integral component of our Roadmap to Cures initiative. Uniting top researchers to share data and expertise, it’s designed to speed progress and deliver the genetic medicines our children deserve.
“RSRT doesn’t just write checks. RSRT is the only Rett foundation in the world that is actually guiding the shape of research and potential cures.”
- Bob Deans, PhD | Chief Technology Officer & Head of Research
Gene Therapy Updates
The idea behind gene therapy is simple: deliver healthy genes to compensate for mutated ones. Bringing gene therapies to people is anything but simple. In 2024, we got positive initial reports on data from the first Rett gene therapy clinical trials at Taysha and Neurogene. These promising results were made possible by previous RSRT-funded projects. However, the journey has not been without heartbreak. Read our summary of these successes and challenges.
Monica Coenraads:
25 Years & Going Strong
An Unwavering Commitment to Cures
In 2024 we celebrated the remarkable role that our founder has played in advancing Rett syndrome research over the last 25 years. The progress we’ve made toward curing Rett is thanks to many people — but we can all agree that Monica has been the driving force! Watch this heartwarming video tribute.
New Awards
Jennifer Doudna
UC Berkeley/Innovative Genomics Institute
We've recently awarded funding to Kai Chen, a postdoc in the lab of Jennifer Doudna (yep, the same Jennifer Doudna who won the Nobel Prize for discovering CRISPR). We're excited to leverage the resources and creativity of her lab at UC Berkeley for Rett research. Dr. Chen will advance research on non-viral lipid nanoparticle (LNP) delivery to the brain, along with novel gene editors.
Jiangbing Zhou
& Guoping Feng
Yale & MIT
Choosing the right delivery method is critical to advancing genetic medicines. We’ve got to take as many shots on goal as possible and explore all the options. The non-viral delivery research we’re funding in Dr. Zhou's lab at Yale is one of the bold pathways we’re pursuing.
Jiangbing Zhou
& Shawn Liu
Yale & Columbia
We funded Dr. Liu’s research into reactivating the silent MECP2. After showing success in cell culture, his work is moving into preclinical proof of concept. Now, he’ll be testing the combo of new cargo and delivery platforms in mouse models.
Jackson Labs
A Priceless Resource
Through our partnership with Jackson Labs we continue to grow the mouse model resources needed by the scientific community to advance cures. We are also exploring novel ways to objectively measure symptoms in these animal models. Our efforts will ultimately save time and money and, most importantly, will bring more scientists and biotech companies into the fight to cure Rett syndrome.
More Award News Soon!
Stepping out of the Dark Ages and Into the Data
“You can't manage what you can't measure.”
- Peter Drucker, Mgmt. Education Pioneer
It’s clear that clinical development for neurological disorders like Rett syndrome is overdue for a transformation. Our VIBRANT program tackles this challenge head-on, setting a new standard for Rett clinical trials and outcome measures.
#ParentPowered
Imagine the power of Rett parents worldwide joining forces to concentrate their knowledge, share it with clinicians and biopharma, and, at the same time, improve their ability to care for their own children. We're not just imagining it — we’re making it happen with the Rett Syndrome Global Registry.
digital Natural History Study
We teamed up with Citizen Health to leverage data from medical records to easily and quickly build natural history studies. Sharing information about your child’s health is a simple way to empower Rett researchers with data that can benefit our entire community.
Industry Asked, We Delivered
RSRT’s biorepository of patient cell lines (all donated by Rett families) recently celebrated fulfilling over 100 sample requests to scientists worldwide. This milestone is a testament to the importance of this resource and is one of many examples of how RSRT identifies and fills gaps.
New Look, Same Drive
We got a new look and a revamped logo! This bold refresh pushes creative boundaries to reflect the cutting-edge nature of RSRT’s research program. The result is a modern and energized brand ready to lead us into the future.
RAREcast Podcast Episode
How One Patient Organization Catalyzed Drug Development
Don’t miss Monica on the RareCast podcast! In this episode, she shares what it means to push the envelope on developing genetic medicines. Spoiler alert: It takes a community like ours that’s tenacious about raising research funds to help our children.
A ⏰ with Monica
You know Monica’s always been passionate about connecting one-on-one with Rett families and giving her honest and informative take on all things Rett. Now, she can do it directly through your smartphone! Check out some 2024 highlights from the series.
Fundraising Milestone
$100M Raised since Inception
We’re happy to share that in 2024, we hit a major milestone: over $100M raised since RSRT launched in 2008. This achievement is powered by the passion and generosity of families, donors, and supporters just like you. Last year alone, you helped raise over $8M to accelerate cures, putting us at $106M in total contributions. Thank you — your support has done so much to push important research forward.
An Event-full Year
In 2024 we had a ton of events that brought us together and combined to raise $4.8 million for RSRT. Almost 5,000 people joined us at these events, both big and small, in cities and in small towns. There were galas, happy hours, outdoor festivals, and more — each helping us get closer to our goal.
To the families who take action and their networks who give generously, we’re so thankful and inspired. None of this is possible without your support.
Drawing a Crowd
Another way that families successfully raised funds for the research last year was by crowdfunding. In 2024 more than $500,000 was raised for RSRT on various crowdfunding platforms. We thank every family that took action, your impact is speeding us towards cures.
$106K in a Day!
#GivingTuesday on Givebutter
Donations kept rolling in the day after #GivingTuesday, and we were surprised and excited to exceed our fundraising goal of $100K! We appreciate every single one of our supporters — you're making a difference.
The Power of Matching
Thanks to our amazing community, we smashed our year-end matching campaign goal! Together, your donations totaled over $256,000, including the generous match from the Rossin Family and Magnolia's Hope. We're deeply grateful to every supporter. Take a few minutes to watch this video for a glimpse into Tim's life with his daughter, Eleanor. Hear what inspires him, and see just how vital your contributions are to our mission.
