Friedreich's Ataxia Research Alliance (FARA)

The FARA Grant Program is proud to award a Postdoctoral Research Award to Chen Liang, PhD, at the University of Rochester to characterize muscle fatigue and calcium ion contribution to muscle dysfunction in FA. Read more about this grant and the research FARA funds at: https://lnkd.in/gGGJZ3Ua

The FARA Grant Program is proud to award a Postdoctoral Research Award to Sujyoti Chandra, PhD, at Stanford University School of Medicine to study new pathways affecting the mitochondria in #FriedreichAtaxia. Read more about this grant and the research FARA funds at: https://lnkd.in/gAR6HRs5

There are several upcoming opportunities to learn more about genetic medicine and stay informed about current and upcoming gene therapy clinical trials in Friedreich's ataxia. Click below for registration and additional details about these informational webinars. 1️⃣ FA Community Conversations Genetic Medicine hosted by FARA on Thursday, August 29 at noon ET FA Community Conversations Registration: https://lnkd.in/gqQG5BjN Join us for the next session of FA Community Conversations where we will discuss genetic medicine treatment approaches like gene therapy and gene editing. FA Community Conversations are intended to be interactive discussions with the FA community. This session will feature short presentations by FARA staff followed by Q&A. 2️⃣ An Informational Webinar hosted by Astellas Pharma on Tuesday, September 3 at noon ET Astellas Pharma Webinar Registration: https://lnkd.in/gb2J7zPK At this webinar, Bryan McGill, MD, Medical Lead of Cell and Gene Therapy at Astellas Pharma will be introducing the FA community to Astellas Pharma and sharing their plans for the phase 1 clinical trial of ASP2016, a gene therapy designed to target the heart. 3️⃣ An Informational Webinar hosted by Lexeo Therapeutics on Tuesday, September 24 at noon ET Lexeo Therapeutics Webinar Registration: https://lnkd.in/gFXQ6jHD Lexeo Therapeutics will be hosting this webinar to share information and preliminary data from their phase 1/2 trial of LX2006, a gene therapy that targets the heart. More details to follow. Have questions about genetic medicine? Click here to submit your questions ahead of time to be discussed during the webinars: https://lnkd.in/gxgT2Jbf

The FARA Grant Program is proud to award a Postdoctoral Research Fellowship to Carla Pernaci, PhD in 🧠, at UC San Diego to study the contribution of the resident immune cells of the brain to #FriedreichAtaxia pathology. Read more about this grant and the research FARA funds at: https://lnkd.in/gzdCBEBi

Join us for the next session of FA Community Conversations on Thursday, August 29, at 12 p.m. ET where we will discuss genetic medicine treatment approaches like gene therapy and gene editing. Learn more and register here: https://lnkd.in/gujKmnnm FA Community Conversations are intended to be interactive discussions with the FA community. This session will feature short presentations by FARA staff followed by a Q&A moderated by FA community members Becca Lange and Shandra Trantham. #GeneTherapy #GeneEditing #FriedreichAtaxia

The FARA Grant Program is proud to partner with fara Australia - Friedreich Ataxia Research Association to award a Postdoctoral Fellowship to David Rufino-Ramos, PhD at Harvard Medical School to study the application of several CRISPR-derived editing tools to contract the GAA repeats in Friedreich's ataxia (FA). Read more about this grant and the research FARA funds at: https://lnkd.in/gXSeJ2Wc

The FARA Grant Program is proud to award a General Research Grant to Brent Stockwell, PhD at Columbia University to develop drugs that target iron dysregulation in FA. Read more about this grant and the research FARA funds at: https://lnkd.in/gF6hUxGP

Larimar Therapeutics reported several updates on their FA program in their second quarter 2024 operating and financial results press release today, August 7, 2024. -Planning initiation of pharmacokinetic (PK) run-in study in adolescents and children with FA to begin by year-end 2024 -Planning initiation of global confirmatory study by mid-2025 with potential sites in the U.S., Europe, U.K., Canada, and Australia -Joined TRACK-FA Neuroimaging Consortium as an industry partner and more... Click here to read the full press release:https://buff.ly/3X4uJ2V

Are you a #raredisease patient or care partner? Interested in sharing your perspectives on safety considerations for approved #genetherapy treatments and long-term studies after gene therapy? Request to participate at our virtual listening meeting by 8/30: https://bit.ly/3XUEQrP

Today, the Pediatric Inclusion Roundtable: Effective Inclusion of Children Early in Clinical Trials meeting brought together stakeholders to discuss pediatric drug development for rare diseases like Friedreich’s ataxia. FARA extends sincere gratitude to all of the speakers, including representatives from FDA, The National Institutes of Health, industry sponsors, and patient advocacy groups. Special thanks to Ron Bartek for spearheading this effort along with Leavitt Partners and all of the speakers, moderators, and participants today.