Rett Syndrome Research Trust Announces 2025 Research Awards to Advance Genetic Medicines

Trumbull, CT — The Rett Syndrome Research Trust (RSRT), the organization singularly focused on curing Rett syndrome, today announced 2025 research awards totaling $6.4 million, further advancing genetic medicine programs into the clinic. RSRT’s cumulative $92 million investment in Rett research since its founding in 2008 has fundamentally reshaped the Rett research landscape—transforming a historically underfunded rare disease into a high-profile focus of genetic medicine innovation. Through sustained, strategic funding, RSRT has attracted world-class academic laboratories, launched multi-institutional consortia, catalyzed biotechnology company engagement, and accelerated the translation of Rett discoveries from basic science to clinical development.

Rett syndrome is a rare and devastating neurodevelopmental disorder caused by mutations in the MECP2 gene. After a period of relatively normal early development, affected children lose speech, purposeful hand use, and motor skills, and often develop seizures, breathing abnormalities, and severe disability.

RSRT’s 2025 awards reflect a deliberate strategy to develop genetic medicine cargoes that target the root cause of the disorder while simultaneously investing in next-generation delivery platforms.

“Since 2008, RSRT has taken a disciplined approach to curing Rett syndrome—funding the science, building the infrastructure, partnering with biopharma, and pushing promising technologies toward clinical reality,” said Monica Coenraads, Chief Executive Officer of RSRT. “The 2025 awards reflect both how far the field has advanced and how urgently we must continue to innovate. Our goal is not incremental improvement, but transformative, durable benefit for every person living with Rett.”

Highlights of 2025 Research Awards Include:

• RNA Trans-Splicing Innovation
  RSRT awarded funding to multiple programs advancing RNA trans-splicing approaches, including an early proof-of-concept project with ReSplice Therapeutics and a larger milestone-driven $1 million collaboration with Tacit Therapeutics. These efforts aim to replace mutant MECP2 exons at the RNA level while preserving the gene’s natural regulatory control. Importantly, a single trans-splicing therapeutic would address 97% of all MECP2 mutations and a second would target the remaining 3%.
• Advancing DNA Base Editing Toward the Clinic
  RSRT is investing in several complementary efforts to prepare DNA base editing for clinical entry, including partnerships with Profluent to AI-design next-generation base editors with improved safety, size, and target coverage; Jackson Laboratory and Senti Biosciences to develop drug-inducible control circuits for regulated editing activity; and University of California Davis to evaluate long-term safety risks associated with sustained editor expression.
• Transforming Brain Delivery
  Recognizing delivery as a critical bottleneck, RSRT previously negotiated access to Apertura Gene Therapy’s blood-brain-barrier (BBB) penetrating capsid, CapX. In parallel, RSRT is investing in the development of non-viral delivery by funding a collaboration between Canadian biotech, QurCan Therapeutics, and University of Massachusetts Medical School to evaluate nanoparticle-based delivery as well as exploratory studies with United Kingdom based, Evox, using engineered exosomes. Importantly, all of these delivery approaches can be used to delivery base editing technologies.
• Manufacturing and process development for CapX
  RSRT is funding the essential work to transform a laboratory-produced genetic delivery vehicle into a high-quality, reproducible, and safe pharmaceutical product that can be manufactured at a large scale for clinical use.
• Revisiting Gene Replacement with Improved Delivery
  RSRT is supporting necessary gene replacement safety studies using engineered brain-penetrant capsids.
• Clinical Acceleration Through Biomarkers
  Building on prior success, RSRT awarded additional funding to Victor Faundez and Sasha Jacobs to extend validated cerebrospinal fluid biomarkers into blood-based assays. Identifying robust biomarkers from blood will allow for a transition to a faster and less invasive monitoring of treatment response in clinical trials.

RSRT’s ability to make these exciting and promising awards is thanks to the families affected by the disorder who fundraise for the research.  This is the results of their efforts and their commitment to changing the lives of their loved ones and all with Rett syndrome.

“RSRT is the largest global funder of Rett syndrome research, but we don’t merely fund research—we actively work to identify the genetic medicine approaches most relevant for Rett, we recruit thought leaders, both academic and industry, to work on these approaches, we derisk and we find biopharma partners to advance the work into clinical trials,” said Robert Deans, RSRT’s Chief Technology Officer, “And most importantly, all of our activities are done with a relentless focus on patients.”