Monday, February 10, 2025

KosBio is a pioneering biotechnology startup: to develop an innovative treatment for Friedreich’s Ataxia (FRDA)

KosBio is a pioneering biotechnology startup, born out of a deeply personal and transformative mission: to develop an innovative treatment for Friedreich’s Ataxia (FRDA), a rare and incurable neurodegenerative disease. Our founders, intimately connected to FRDA through their own family experiences, bring a unique level of commitment and dedication to this mission, distinguishing KosBio in the competitive biotech landscape. 

 Leading our therapeutic pipeline is KB-001(TM), a therapy based on repurposing an FDA-approved drug to activate a developmental signaling pathway relevant to FRDA. This strategic approach not only expedites the path to treatment but also reduces potential risks by leveraging existing approved drugs. 

In addition, KosBio is advancing the development of a series of novel, patented drugs (KB002-KB021). These drugs are projected to have significantly greater potency than KB-001(TM), marking a substantial advancement in our therapeutic arsenal.

Functional Characterization of Parallel Fiber-Purkinje Cell Synapses in Two Friedreich's Ataxia Mouse Models

Joseph DJ, Mercado-Ayon E, Flatley L, Viaene AN, Hordeaux J, Marsh ED, Lynch DR. Functional Characterization of Parallel Fiber-Purkinje Cell Synapses in Two Friedreich's Ataxia Mouse Models. Cerebellum. 2025 Feb 5;24(2):42. doi: 10.1007/s12311-025-01796-0. PMID: 39907933; PMCID: PMC11799031.

To investigate the neural circuit basis of this dysfunction, we employed field recordings to measure Purkinje cell (PC) function and synaptic properties along with western blotting and immunohistochemistry to determine their density and structure in two established FRDA mouse models, the shRNA-frataxin (FRDAkd) and the frataxin knock in-knockout (KIKO) mice. Western blotting demonstrated subtle changes in mitochondrial proteins and only a modest reduction in the density of calbindin positive cells PCs in the cerebellar cortex of the FRDAkd mice, with no change in the density of PCs in the KIKO mice. Though PC density differed slightly in the two models, field recordings of parallel fiber-PC synapses in the molecular layer demonstrated concordant hypo-excitability of basal synaptic transmission and impairments of long-term plasticity using induction protocols associated with both potentiation and depression of synaptic strength. These results indicate that synaptic instability might be a common feature in FRDA mouse models.

Heart Gene Therapy Astellas Withdrawing

Astellas Pharma Inc., February 4, 2025Q3 YTD/FY2024 Financial Results (pg 13 and 33)