RTW Foundation backs eight rare disease groups with $573,000 in grants

By AI, Created 5:01 PM UTC, May 18, 2026, /AGP/ – RTW Foundation said on May 12, 2026, that it is giving $573,000 to eight rare disease foundations working on ultra-rare disorders. The funding and advisory support are aimed at speeding research, trial readiness and early therapeutic development for conditions with few treatment options.

Why it matters: - Rare disease foundations often move first on conditions that have small patient populations, limited funding and few therapeutic options. - The grants are designed to support work across the research pipeline, from lab studies to clinical trial preparation. - RTW Foundation is pairing funding with scientific and strategic advice through its Rare Disease Advisory Program.

What happened: - RTW Foundation announced $573,000 in grant funding for eight rare disease foundations. - The announcement came May 12, 2026, from New York City. - RTW Foundation is the philanthropic arm of biotech investment firm RTW Investments. - The funded groups are family-led and patient-led organizations focused on rare disease research and therapeutic development.

The details: - Birdie’s Flight for a KDM5C Cure is supporting n-Lorem Foundation’s work on ASOs aimed at regulatory elements in KDM5C transcripts to raise endogenous KDM5C protein levels. - CHAMP1 Research Foundation is developing an RNA-based target-site blocker approach to restore CHAMP1 protein levels in patient-derived neurons and preclinical mouse models. - Miracles for Mighty Milo Foundation is funding transfer of Milo-derived iPSC lines to Dr. Francesca Maltecca’s lab at IRCCS Ospedale San Raffaele in Milan and initial feasibility work on AAV-mediated gene addition in patient-derived neural cells. - The MYT1L Project Foundation is backing a multi-institution screen for lead ASO candidates intended to increase productive MYT1L expression, with validation in iPSC-derived neurons and patient-derived cells. - Shwachman-Diamond Syndrome Alliance is supporting development of a prime editing-based strategy to test whether precise correction of the underlying genetic defect could provide a durable treatment path. - Sophie’s Hope Foundation/CureGSD1b is funding a natural history study at UTHealth Houston on Glycogen Storage Disease Type Ib, including metabolic complications, neutropenia, enterocolitis, liver disease and response to treatments such as empagliflozin. - The Styrke Foundation is supporting the first externally led Patient-Focused Drug Development meeting with the FDA for Diamond-Blackfan anemia syndrome, plus a patient and caregiver survey, a Voice of the Patient report, Styrke Canada and a DBAS natural history study. - The TESS Research Foundation is funding novel drug-screening assays to identify small-molecule activators of SLC13A5, a loss-of-function epilepsy target where existing compounds are inhibitors. - RTW Foundation said the grants support work from early laboratory studies and animal models through safety, toxicology and clinical trial preparation. - RTW Foundation also said the Rare Disease Advisory Program provides free scientific and strategic advising to help foundations assess research priorities, therapeutic opportunities and translational next steps. - Nicole Litt, program manager at RDAP, said the foundation is grateful to support rare disease partners and stand alongside families pushing research and access. - RTW Foundation said its broader mission includes supporting medicines for neglected rare diseases, youth science education and health equity initiatives in New York City. - The Foundation was founded in 2018 as the philanthropic arm of RTW Investments, LP. - More information is available at RTW Foundation.

Between the lines: - The grant list shows a mix of therapeutic strategies, including ASOs, RNA blockers, prime editing, gene addition and drug-screening assays. - The portfolio also includes natural history studies and FDA-facing patient engagement work, which can help de-risk later-stage development. - The focus on patient-led foundations suggests RTW Foundation is trying to fund both science and the infrastructure needed to move rare disease programs forward.

What’s next: - Each foundation will use the grants to advance its next research milestone, whether that is assay development, feasibility testing, natural history data collection or regulatory engagement. - The RDAP advisory work will continue as groups evaluate research priorities and translational steps. - Several projects are positioned to generate data that could support follow-on funding and preclinical development.