amyotrophic lateral sclerosis

[Scientific Reports] The authors investigated whether bone marrow-derived cells (BMDCs) could be applied as gene carriers for cell-based gene therapy by employing the accumulation of BMDCs.

[Cell Death & Differentiation] The authors discuss the interplay between distinct cell death signaling cascades and disease pathogenesis and describe pharmacological agents targeting key players in the cell death signaling pathways that have progressed through to clinical trials.

[Apic Bio, Inc.] Apic Bio, Inc. announced that the U.S FDA has cleared its Investigational New Drug (IND) application for APB-102, the Company’s lead gene therapy candidate designed to treat SOD1 amyotrophic lateral sclerosis (ALS) – a common cause of familial ALS.

[Nature Reviews Neurology] Scientists provide an overview of the diverse roles of non-neuronal cells in relation to the pathogenesis of ALS and the emerging potential of non-neuronal cell biomarkers to advance therapeutic development.

[Retrotope] Retrotope announced that enrollment has been completed for its multicenter Phase II clinical trial evaluating RT001, the company’s lead development candidate, in patients with ALS or Lou Gehrig’s disease.

[Nature Medicine] Investigators report that patients with sporadic myotrophic lateral sclerosis (ALS) present cell activity patterns consistent with two mouse models in which enrichments of vascular cell genes preceded microglial response.