Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of Friedreich's Ataxia

NEW YORK, Aug. 11, 2026 /PRNewswire/ -- Graviton BioScience Corporation, a privately held, clinical-stage biotechnology company developing a diverse pipeline of therapeutics, including selective ROCK2 inhibitors for metabolic, inflammatory, fibrotic, and central nervous system disorders, announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application for a novel capsule formulation of GV101, a proprietary oral ROCK2 inhibitor being developed for Friedreich's ataxia (FA). This new formulation of GV101 was specifically optimized for development in FA and other potential orphan indications. Preclinical and clinical studies have demonstrated that GV101 increases frataxin protein levels, which is a potentially disease modifying therapeutic approach that addresses the root cause of Friedreich's ataxia (FA). Following the FDA's 30-day review period, Graviton received IND clearance on August 7, 2026, enabling the initiation of a Phase 2 clinical study in individuals living with FA. 

 Graviton now plans to initiate a 12-week, randomized, placebo-controlled, dose-finding Phase 2 trial enrolling up to 48 participants at multiple sites in the United States and internationally. The study is designed to evaluate the efficacy, safety, and tolerability of a range of GV101 capsule doses in individuals living with Friedreich's ataxia. The primary endpoint is an increase from baseline in frataxin levels measured in patients' cells.

Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow

Smith BK, Coker MA, Liberati C, Meyer BP, Norman S, Ehrbar J, Leon-Astudillo C, Subramony S, Corti M. Assessing airway clearance dysfunction in Friedreich's ataxia: A focus on peak cough flow. J Neuromuscul Dis. 2026 Jun 8:22143602261452334. doi: 10.1177/22143602261452334. Epub ahead of print. PMID: 42253100; PMCID: PMC13437907. 

The study demonstrates respiratory dysfunction in patients with FRDA, and shows that disease severity and muscle weakness affect airway clearance. PCF is a more direct and clinically meaningful indicator of cough effectiveness than FVCpp. Comprehensive respiratory evaluations, including muscle-strength testing, can identify individuals who may benefit from targeted interventions to prevent complications.

Impaired Glur2 palmitoylation in cerebellar Purkinje cells of a Friedreich ataxia mouse model

Elizabeth Mercado-Ayón, Jennifer Coulman, Jia-Ying Lee, Eunjoo Lancaster, Elliot Goga, Mohammad Asad, Eric Witze, David R. Lynch, Impaired Glur2 palmitoylation in cerebellar Purkinje cells of a Friedreich ataxia mouse model, Experimental Neurology, 2026, 115955, ISSN 0014-4886, doi:10.1016/j.expneurol.2026.115955.

Mechanistically, reduced GluR2 palmitoylation associates with decreased expression and palmitoylation of the palmitoyl acyltransferase DHHC3, while levels of depalmitoylating enzymes remain unchanged. In vitro, DHHC3 enhances GluR2 palmitoylation, supporting a direct enzymatic relationship. Partial restoration of frataxin expression rescues GluR2 and DHHC3 protein levels and partially restores GluR2 palmitoylation. These findings identify impaired GluR2 palmitoylation as an early, selective synaptic alteration in FRDA and implicate dysregulated lipid-dependent post-translational modification as a mechanism linking frataxin deficiency to cerebellar synaptic vulnerability.

Partial bypass of frataxin deficiency by ISCU M141I restores cytosolic and nuclear Fe–S cluster assembly

Mosbach, V., Maio, N., Hermet, L. et al. Partial bypass of frataxin deficiency by ISCU M141I restores cytosolic and nuclear Fe–S cluster assembly. Cell Death Differ (2026). doi:10.1038/s41418-026-01832-4  

Results reveal a previously unrecognized compartment-specific rescue of Fe-S cluster dependent processes by the ISCU M141I variant in mammalian cells, raising for the first time the possibility of compartmental regulation of Fe-S cluster biogenesis.

Autophagy activation by urolithin-a derivative UA-36 mitigates Friedreich's ataxia pathologies induced by frataxin deficiency

Gong Q, Liu T, Han X, Zhang R, Liu X, Xiong B, Ali T, Huang J, Xie Y, Li S, Yang X. Autophagy activation by urolithin-a derivative UA-36 mitigates Friedreich's ataxia pathologies induced by frataxin deficiency. Mol Biomed. 2026 Jun 4;7(1):82. doi: 10.1186/s43556-026-00457-w. PMID: 42240896; PMCID: PMC13237351. 

 Here, we investigate the therapeutic efficacy and underlying mechanisms of UA-36, a novel water-soluble and bioavailable derivative of urolithin A, in cellular and animal models of FA. In Fxn-knockdown N2a cells, UA-36 significantly restored FXN protein levels, enhanced autophagic flux, improved mitochondrial function, and attenuated oxidative stress–induced damage. In vivo, oral administration of UA-36 for eight weeks in YG8R transgenic mice, a well-established FA model, markedly improved motor coordination, gait performance, and skeletal muscle strength. Histological and ultrastructural analyses revealed substantial protection against cerebellar Purkinje cell loss and iron deposition, cardiac hypertrophy, and the degree of skeletal muscle atrophy and fibrosis. Proteomic analysis of cerebellar tissue demonstrated that UA-36 robustly reprograms the FA-associated molecular landscape by upregulating pathways related to autophagy, mitochondrial biogenesis, oxidative phosphorylation, and redox homeostasis, while suppressing apoptosis and neuroinflammatory signaling. Together, these findings identify UA-36 as a promising lead compound and provide compelling evidence that therapeutic enhancement of autophagy and mitochondrial quality control represents a viable, mechanism-based strategy for the treatment of FA.

Visual Pathways Involvement in Friedreich’s Ataxia Patients Without Macular Impairment

Parisi, V.; Barbano, L.; Di Renzo, A.; Dell’Aquila, C.; D’Andrea, M.; Castelluzzo, A.M.; Colacino, G.; Gioiosa, V.; Coppola, G.; Casali, C.; et al. Visual Pathways Involvement in Friedreich’s Ataxia Patients Without Macular Impairment. J. Clin. Med. 2026, 15, 6097. doi:10.3390/jcm15156097 

 Fourteen FA patients (14 eyes; mean age 38.64 ± 8.21 years) without functional or structural macular abnormalities, confirmed by multifocal electroretinogram (mfERG) and spectral domain–optical coherence tomography (SD-OCT), and 20 age-matched healthy controls were enrolled. Patients were classified as late-onset (FA1, disease onset > 25 years) or early-onset (FA2, disease onset < 25 years). All participants underwent comprehensive ophthalmological and neurological evaluations. RGC function and visual pathway conduction were assessed through simultaneous pattern electroretinogram (PERG) and VEP recordings using 60′ and 15′ checkerboard stimuli. Retino-cortical time (RCT) was calculated as the difference between VEP P100 and PERG P50 implicit times. Statistical analyses included a general linear model. Results: 60′ PERG amplitude (A) was significantly (p < 0.01) reduced in the FA group compared to the Control group. FA patients showed significantly delayed 60′ and 15′ VEP ITs and significantly increased 60′ and 15′ RCT values, particularly in the FA1 group. In late-onset patients, no significant relationships were found between age at onset of disease and all electrophysiological parameters (60′ and 15′ VEP and PERG ITs and As and RCTs). Conclusions: In our selected FA, RCG function and neural conduction along the post-retinal visual pathways are impaired. The abnormal neuronal conduction is greater in late-onset FA patients and is not related to patients’ age and the age at the onset of the disease. The duration of the disease has a slight effect on the post-retinal neural conduction on small optic nerve fibers.

BRD4 recruitment into HP1 condensates desilences transcription without erasure of repressive chromatin

Brandon, C.J., Robinson-Thiewes, S., Kaulage, M. et al. BRD4 recruitment into HP1 condensates desilences transcription without erasure of repressive chromatin. Nat Cell Biol (2026). doi:10.1038/s41556-026-02044-1 

 How genes are desilenced without erasure of repressive chromatin is a poorly understood phenomenon. A dominant mode of repression occurs through methylation of lysine 9 of histone H3 (H3K9me3), a mark that engages heterochromatin protein 1 (HP1) to drive chromatin compaction and transcriptional silencing. The erasure and replacement of this repressive mark with acetyl/acyl groups recruits positive factors such as BRD4/BET to elicit gene transcription. Here we report that, in Friedreich’s ataxia, a synthetic gene regulator (SynGR1/SynTEF1) licenses transcription across repressive chromatin without removal or replacement of H3K9me3 or HP1. By recruiting BRD4/BET into repressive GAA repeats in frataxin (FXN), SynGR1 creates a paradoxical state wherein gene transcription and repressive chromatin coexist. Contrary to convention, we find that BRD4 partitions into phase separated HP1 condensates in vitro and into HP1 puncta in patient-derived cells, thus offering a mechanistic explanation for desilencing transcription without the dispersal of mesoscale repressive chromatin. More broadly, our study highlights the dynamic nature of repressive chromatin and the context dependence of epigenetic marks in regulating gene expression

Larimar Therapeutics Reports Second Quarter 2026 Financial and Business Update

BALA CYNWYD, Pa., Aug. 04, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar). “Open label (OL) study data announced in June further reinforce the disease-modifying potential of nomlabofusp, demonstrating continued directional improvements in key clinical endpoints over time alongside a well-characterized safety profile. Following receipt of minutes from a successful Type B multidisciplinary pre-BLA meeting with the Food and Drug Administration (FDA), the first module of our rolling Biologics License Application (BLA) has been submitted, with completion expected in the second half of 2026. We continue to see strong enthusiasm from patients and investigators as we advance the OL study with additional participants dosed in July and several adults and adolescents in screening. We are also on track to initiate dosing in our global confirmatory study this quarter. Looking ahead, we are focused on execution as we work to bring forward nomlabofusp as the first potential therapy to address the underlying cause of disease for pediatric and adult patients living with Friedreich’s ataxia (FA).”

Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression

A. Gitman, N. Bhandari, M. Castellaro, K. Schadt, M. Cancio, and D. R. Lynch, “ Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression,” Annals of Clinical and Translational Neurology (2026): 1–3, doi:10.1002/acn3.70455. 

After the transplant, her neurologic disease progressed similarly to other patients, but her blood frataxin levels returned to normal and cardiac hypertrophy decreased. This shows that FRDA patients can be treated with bone marrow transplantation, but such treatment alone has no direct effect on progression of neurologic disease.

Adjuvia Therapeutics Closes $8 Million Series Seed Financing to Advance Novel Mitochondrial Disease Therapy into the Clinic

Jul 29, 2026. SAN FRANCISCO--(BUSINESS WIRE)--Adjuvia Therapeutics, a biotechnology company developing therapeutics to treat mitochondrial dysfunction and disease, today announced the closing of an $8 million Series Seed financing. The financing will support the submission of an Investigational New Drug (IND) application for Adjuvia's lead candidate, ATI-105, later this summer, followed by the initiation of the Phase 1 clinical trial in healthy volunteers in Fall 2026. Adjuvia also plans to begin a Phase 1/2 study in patients with Friedreich's ataxia in early 2027.