Friedreich's Ataxia Research Alliance (FARA)

The FARA Grant Program is proud to award a postdoctoral research grant to Xiaonan Guan, PhD, at the New York Genome Center, to understand what causes frataxin gene silencing and how to reverse it. Learn more about this grant and other FARA-funded research at https://ow.ly/tTrc50VrHgE.

Don't miss the April 1 application deadline for research funding! FARA is looking to fund innovative, high-risk, high-gain, and potentially ground-breaking concepts in #FriedreichAtaxia research through our Award for Innovative Mindset (AIM grant). NOTE: While the presentation of preliminary and/or published data is encouraged, it is not required. Successful applicants will foster new directions, bring new perspectives to the field, and address neglected issues in FA research. Learn more about our research priorities and application process: https://ow.ly/RTrw50V8N8L

Don't miss the April 1 application deadline for research funding! FARA is looking to fund innovative, high-risk, high-gain, and potentially ground-breaking concepts in #FriedreichAtaxia research through our Award for Innovative Mindset (AIM grant). NOTE: While the presentation of preliminary and/or published data is encouraged, it is not required. Successful applicants will foster new directions, bring new perspectives to the field, and address neglected issues in FA research. Learn more about our research priorities and application process: https://ow.ly/RTrw50V8N8L

Today, RDI Strategic Engagement Manager Debra Bellon and Global Policy Coordinator Alanna Miller participated in a panel discussion led by Labrini Papageorgiou, Health Policy Director of RARE DISEASES GREECE and including the participation of Myriam RAI of the Friedreich's Ataxia Research Alliance (FARA) and Fernando Arnaiz of Roche, as part of the 5th International Conference of Rare Diseases. Debra and Alanna discussed the grassroots effort to make the WHA Resolution on Rare Diseases a reality and how we hope it will bring about real and sustainable improvements in the lives of #PLWRD around the world. The panel illustrated the multistakeholder readiness to make the #Resolution4Rare a reality and to implement the Global Action Plan on Rare Diseases around the world in order to ensure that it drives meaningful outcomes for our community. Learn more about the Resolution on our website: https://lnkd.in/dPkjSDJk

The FARA Grant Program is proud to award a general research grant to Alessandro Quattrone, PhD, at the Università di Trento, to use CRISPR editing to modify frataxin expression. This grant is co-sponsored by FARA Ireland. Learn more about this grant and other FARA-funded research at https://ow.ly/WS5G50Vp7Hi.

Join Solid Biosciences for an Informational Webinar on Tuesday, April 15 at Noon ET. Matthew Harmelink, MD, Therapeutic Head of Neuromuscular at Solid Biosciences, and child neurologist with expertise in neuromuscular and neurogenetic disorders, will talk about Friedreich’s ataxia (FA), the science behind gene therapy and the FALCON study, Solid’s unique gene therapy program for FA. Click here for more information and to register: https://ow.ly/B3IF50Vp97E

In a press release today, March 24, 2025, Lexeo Therapeutics reported their Fourth Quarter and Full Year 2024 Financial Results and Operational Highlights. Regarding their gene therapy program, LX2006 for the treatment of FA cardiomyopathy, the company reported further alignment on their development pathway following a meeting with the U.S. Food and Drug Administration (FDA). A pivotal study is planned, including adult and pediatric cohorts (both adolescents and children), and the use of prospective natural history data as external control. The company also plans to provide a mid-year clinical update with safety and tolerability data, pre- and post-treatment cardiac frataxin protein expression, and clinical biomarker data from the ongoing Phase I SUNRISE-FA and Weill Cornell clinical trials. Click here to read the full press release: https://ow.ly/2b7E50Vni0n

In a press release today, March 24, 2025, Larimar Therapeutics shared several clinical and regulatory updates on their nomlabofusp program in FA. Larimar reported that the Open Label Extension Study in adults continues to enroll, and that the adolescent cohort of the Pediatric PK run-in study is anticipated to complete dosing at the end of this month. Larimar also obtained feedback from both the FDA and EMA on the global Phase 3 study protocol and reported that they are on track to initiate this study by mid-2025, with potential sites in the U.S., Europe, U.K., Canada, and Australia. As part of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, Larimar shared that they have had more frequent interactions with the FDA, which have been informative as the company plans its Biologics License Application (BLA) submission. To read more about these updates, click here: https://ow.ly/Kek850VngCk

Researchers: Need funding for an innovative idea? Apply for an AIM grant from FARA! *No prior work in #FriedreichAtaxia required.* Applications due April 1. Learn more about the grant program's priorities on our website. We look forward to your high-risk, high-gain, groundbreaking ideas, as, together, we work to #CureFA!

The FARA Grant Program is proud to award the Award for Innovative Mindset (AIM) to Daniel Hart, PHD, at Epicrispr Biotechnologies, Inc., to develop a new type of gene editing to treat FA. Learn more about this grant and other FARA-funded research at https://lnkd.in/eyWqTM6h