Solid Biosciences’ SWOT analysis: gene therapy stock poised for growth amid challenges

Investing.com. Published 07/29/2025 

SGT-212 for Friedreich’s Ataxia: Recently received FDA Investigational New Drug (IND) clearance Unique dual-route administration approach targeting both neurologic and cardiac manifestations Phase 1b dose-finding study set to begin in the second half of 2025

RWD132 Onset of Cardiomyopathy and Cardiovascular Disease-Related Burden in Friedreich Ataxia: Real-World Data From Medical Claims

Sheng-Han Kuo, Boyang Bian, Sarah M. England, Daniel R.J. Gomes, Jim McKay, Tony Wang, Robin L. Avila, Susan Perlman, RWD132 Onset of Cardiomyopathy and Cardiovascular Disease-Related Burden in Friedreich Ataxia: Real-World Data From Medical Claims, Value in Health, Volume 28, Issue 6, Supplement 1, 2025, Page S386, ISSN 1098-3015, doi:10.1016/j.jval.2025.04.1715. 

These data show the natural progression of CV events in FA. Our findings demonstrate that patient burden related to progressive CM begins at an early age and that other cardiac disease, particularly arrhythmias and rhythm and heart valve disorders, may begin 2-11 years prior to CM, with CV onset as early as age 7 in the youngest patients. These data have limitations in older populations and those in Medicare but provide previously unavailable data on the onset of CM and CV disease in patients.

False Beliefs, True Deficits: Investigating Social Cognition in Friedreich Ataxia

Heleven E, Vyhnalek M, Karamazovová S, Van Overwalle F, Naeije G. False Beliefs, True Deficits: Investigating Social Cognition in Friedreich Ataxia. Cerebellum. 2025 Jul 11;24(5):128. doi: 10.1007/s12311-025-01886-z. PMID: 40643773. 

 Friedreich ataxia (FA) is a cerebellar neurodegenerative disease primarily known for its motor symptoms, but emerging evidence suggests it also affects higher-order cognitive functions, including Theory of Mind (ToM). This study aimed to assess ToM in individuals with FA using a Picture Sequencing Task (PST) that distinguishes between mechanical, social script, true belief, and false belief scenarios, with a focus on the latter as key marker of mentalizing in cerebellar diseases. 

Our results reveal a selective impairment in false belief reasoning in FA, consistent with ToM deficits observed in other cerebellar and neurodevelopmental disorders.

Unveiling the Idiographic Portrait of Friedreich's Ataxia in an Omani Patient: A Multidisciplinary Case Stud

Al-Adawi, Samir and Al Busaidi, Saoud Jaber and Al Jahwari, Nasra Ali and Rajeev, Neeraja and Alriyami, Maha and ALBusaidi, Alya and Al Kindi, Farah Ahmed and Otaify, Ghada Ahmed and Ambusaidi, Aamal and Al Azri, Faisal Hamad and Gujjar, Arunodaya R. and Bolourkesh, Helia, Unveiling the Idiographic Portrait of Friedreich's Ataxia in an Omani Patient: A Multidisciplinary Case Study. doi:10.2139/ssrn.5344239

Friedreich’s ataxia (FA), a rare inherited disorder that mainly affects Caucasian populations, presents significant diagnostic challenges in regions with diverse genetic backgrounds. This case report details the diagnostic journey of a 22-year-old Omani male with undiagnosed FA, highlighting the value of the idiographic approach within a biopsychosocial framework. Initially presenting with psychotic symptoms, the patient underwent a comprehensive interdisciplinary evaluation, revealing neurological and psychiatric manifestations.

Larimar Therapeutics Publishes Nonclinical Data Supporting the Therapeutic Potential of Nomlabofusp in Patients with Friedreich’s Ataxia

BALA CYNWYD, Pa., July 08, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the publication of two peer-reviewed articles highlighting nonclinical data on the therapeutic potential, pharmacology, and mechanism of action of nomlabofusp as a novel frataxin (FXN) protein replacement therapy designed to address the underlying cause of Friedreich’s ataxia (FA). These data were included in the briefing package reviewed by the U.S. Food and Drug Administration (FDA) in support of potentially using skin FXN concentrations as a reasonably likely surrogate endpoint (RLSE) for Larimar’s registrational program seeking accelerated approval for nomlabofusp.

LEXEO THERAPEUTICS ANNOUNCES FDA BREAKTHROUGH THERAPY DESIGNATION FOR LX2006 IN FRIEDREICH ATAXIA

NEW YORK, July 07, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering novel treatments for cardiovascular diseases, today announced that the U.S. Food and Drug administration (FDA) has granted Breakthrough Therapy designation to LX2006 based on clinical evidence generated on both cardiac and neurologic measures of Friedreich ataxia (FA). LX2006 has also been selected to participate in the FDA Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, intended to enable earlier patient access to therapies with expedited clinical development timelines.

Lexeo Therapeutics Announces FDA Breakthrough Therapy Designation for LX2006 in Friedreich Ataxia

07/07/2025 ;Lexeo Therapeutics (Nasdaq: LXEO) has achieved a significant milestone as its drug candidate LX2006 received FDA Breakthrough Therapy designation for treating Friedreich ataxia (FA). The designation was based on promising interim clinical data from Phase I/II trials showing meaningful improvements in cardiac biomarkers and functional measures. The drug has demonstrated clinically significant improvements in cardiac and neurologic functional measures, with increased frataxin expression observed in all cardiac biopsy participants at three months post-treatment. To date, 17 participants have been treated across two trials. The company plans to initiate a registrational study by early 2026. Additionally, LX2006 has been selected for the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program, aimed at facilitating faster patient access to promising therapies.

Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation

Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation. Yameogo, Pouiré et al. Molecular Therapy Nucleic Acids, Volume 0, Issue 0, 102617. DOI:10.1016/j.omtn.2025.102617

This ASO strategy may be therapeutically feasible for FRDA patients with other point mutations that cause splicing defects. Success in developing treatments for disorders with only a few known cases will give hope to FRDA patients carrying these rare point mutations.

Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation

Biogen Launches Phase 3 Pediatric Trial Of Omaveloxolone For Friedreich Ataxia

Biogen Inc.June 23, 2025. It has begun dosing in the global Phase 3 BRAVE trial to test omaveloxolone (SKYCLARYS®) in children aged 2 to 16 with Friedreich ataxia (FA), a rare neurological illness. About 255 participants will be included in the randomized, double-blind experiment, which will have a 52-week treatment duration and an open-label extension. SKYCLARYS is now the only approved therapy for FA in people aged 16 and up, and it is available in more than 40 countries.

The primary endpoint of the BRAVE research is the Upright Stability Score, which is part of the modified FA rating scale and is used to assess progression in children. Global enrollment is still ongoing.

The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis

The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis, Perlman, Susan et al.. Journal of the Neurological Sciences, Volume 0, Issue 0, 123594. doi:10.1016/j.jns.2025.123594

 After matching, the study included 652 patients with FRDA and 3260 matched controls (mean age 33.2 years; 51.4 % females). During the follow-up period (median 26.2 months for cases and 28.3 months for controls), the incremental clinical burden in FRDA cases vs. matched controls was high: patients with FRDA had significantly higher odds of loss of ambulation (odds ratio: 158.0 [95 % confidence interval (CI): 112.4–222.3]), cardiomyopathy (59.2 [41.6–84.1]), scoliosis (49.0 [35.4–67.9]), falls (7.4 [5.9–9.2]), diabetes (2.5 [2.0–3.2]), head injury (2.4 [1.9–3.0]), and fracture (3.3 [2.6–4.2]) compared to controls (all p < 0.001). Patients with FRDA also experienced a higher risk of mortality than controls (hazard ratio: 3.9 [95 % CI: 2.4–6.4]).