December 23, 2025, New York, NY – RTW Foundation, the philanthropic arm of biotech investment firm RTW Investments, is proud to announce $521,000 in grant funding for six rare disease foundations advancing critical rare disease research, each led by families working at the front lines of discovery, patient engagement, and therapeutic development.
Rare disease foundations play an essential role in accelerating research by mobilizing patient communities, supporting studies, facilitating trial readiness, and bridging the gap between families, researchers, and the pharmaceutical industry. Together, these organizations are addressing urgent unmet needs across the rare disease landscape, where complexities in treatment options, small patient populations, and limited funding often slow progress toward treatment.
RTW Foundation’s rare disease grants support essential work across the full research and development pipeline—from early laboratory studies and animal models to safety and toxicology assessments and preparation for clinical trials. Funding enables the development and manufacturing of new therapeutic candidates, as well as the experiments needed to demonstrate whether these therapies work in relevant disease models. As programs move into the clinic, grant support helps determine safe dosing, measure therapeutic benefit, and monitor patient safety over time. Together, this support helps families and foundations move research forward more efficiently and bring potentially life-changing gene therapies closer to the patients who need them most.
Beyond grant funding, RTW Foundation works closely with each organization through its Rare Disease Advisory Program (RDAP), providing free, expert guidance and operational support at every stage of development. This hands-on partnership helps shape research strategy, inform key decisions, and clearly communicate impact. By pairing tailored advisory support with targeted funding, the Foundation empowers patient-led organizations to make progress in conditions that are often overlooked and bring renewed hope to affected families.
“Our rare disease partners are driving progress against extraordinary odds,” said Joe Katakowski, Director of Research at RTW Foundation. “By combining funding with deep advisory support, we aim to be a true partner—helping organizations move faster, make informed decisions, and build momentum toward treatments families urgently need.”
Finding Hope for FRRS1L
Finding Hope for FRRS1L is a family-led foundation advancing gene therapy for children with FRRS1L. Founder, Chrissy Green, joined RTW Foundation’s Rare Disease Advisory Program (RDAP) in May 2025 to support the evaluation and development of AAV gene therapy-based treatment strategies. Through RDAP, RTWF helped clarify development options, connect the foundation with key partners, and strengthen a path forward—bringing the program closer to delivering meaningful hope for families.
TBRS Community (Tatton-Brown–Rahman Syndrome)
The TBRS Foundation is dedicated to advancing research and treatments for Tatton-Brown–Rahman Syndrome. Since joining RDAP in March 2024, RTW Foundation has supported the organization in navigating the complexities of targeting DNMT3A by reviewing potential therapeutic approaches and strengthening scientific partnerships. With a highly engaged patient community and leadership within the Overgrowth Syndromes Alliance, TBRS is moving forward with a clearer strategy to advance research and support the broader rare disease field.
Chelsea’s Hope
Chelsea’s Hope is a family-led organization advancing treatment development for Lafora disease and joined RDAP in May 2025. Its primary focus is completing a 10-patient Safety Study—an essential step toward clinical progress—supported by a globally engaged patient community across the U.S., Europe, and South America. Through RDAP, RTW Foundation provides strategic guidance to help close development gaps, strengthen partnerships, and keep the program on track toward the clinic.
RARE Hope
RARE Hope supports a diverse portfolio of rare disease research initiatives and participates in RDAP to accelerate therapeutic development across its foundations. Nina Frost leads research efforts for a rare disease called Alternating hemiplegia of childhood (AHC), RTW Foundation works closely with RARE Hope by sharing cross-program insights and highlighting opportunities that may strengthen and accelerate their ongoing collaborations with The Jackson Laboratory, Boston Children’s Hospital, and the Broad Institute. This coordinated approach enables foundations to leverage shared insights and advance research more efficiently.
Malan Syndrome Foundation
The Malan Syndrome Foundation is a family-led organization committed to advancing research and improving care for individuals with Malan syndrome. Through participation in RDAP, RTW Foundation supports the organization in strengthening its research strategy, identifying promising scientific pathways, and preparing for future therapeutic development. Together, the partnership is focused on building a sustainable foundation for long-term progress, helping to build the tools and models that will be needed for future therapeutic development efforts.
For Henry AHC
For Henry AHC is a parent-led foundation working to advance treatments for alternating hemiplegia of childhood (AHC). Through RDAP, RTW Foundation provides strategic and scientific guidance to support the organization’s research efforts and help navigate the complexities of therapeutic development. This partnership aims to accelerate progress related to promising ASO therapeutics for AHC patients, while keeping families and patients at the center of discovery.
Lachie’s Wish 4 CS
Lachie’s Wish 4 CS is a family-led organization dedicated to advancing research for Christianson syndrome. Through engagement with RDAP, RTW Foundation supports the foundation in refining its research strategy, exploring viable development pathways, and strengthening readiness for future studies. Together, the partnership works to move promising science, related to gene therapies or potential other treatment approaches, closer to meaningful outcomes for affected families.
About RTW Foundation
Founded in 2018 as the philanthropic arm of RTW Investments, LP, RTW Foundation works to power community initiatives and provide rare disease advising to improve the health of underserved populations. The Foundation supports the development of medicines for neglected rare diseases, empowers youth to explore careers in science, biotechnology, and medicine, and builds partnerships with local organizations to advance health equity in New York City. To learn more, visit www.rtwf.org