Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).
The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease.
Theranocure Co. Ltd. has prepared and tested new vanillic acid derivatives targeting the dopamine D1 receptor (D1R) reported to be useful for the treatment of neuroinflammation and Alzheimer’s disease.
Work at Satellos Bioscience Inc. has led to the identification of new AP2-associated protein kinase 1 (AAK1) inhibitors reported to be useful for the treatment of muscular dystrophy.
Researchers from Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and China Pharmaceutical University presented the discovery of novel orally bioavailable inhibitors of transient receptor potential canonical channels 4 and 5 (TRPC4/5), being developed for the treatment of depression and anxiety.
In a study published in Nature Neuroscience, researchers from Tiziana Life Sciences Ltd. and collaborators investigated the potential of nasal delivery of the CD3 foralumab to reduce neuroinflammation and enhance microglial phagocytosis in a traumatic brain injury (TBI) model.
Eight months after announcing the $18.5 million first tranche of its series A, Augustine Therapeutics has closed the oversubscribed round at $85 million and is now ready to begin clinical development of its novel histone deacetylase-6 (HDAC6) inhibitors.
Sineugene Therapeutics Co. Ltd. has obtained IND clearance from the FDA for SNUG-01, a first-in-class tripartite motif protein 72 (TRIM72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). A phase I/IIa trial will evaluate SNUG-01 in adults with ALS.
Neuronos Ltd., a subsidiary of Beyond Air Inc.’s, has secured an initial $2 million in equity financing from private investors as part of a larger funding round. This investment will accelerate the preclinical development of Neuronos’s small-molecule drug for autism that leverages the regulation of nitric oxide (NO) levels in the brain. The company’s small-molecule therapy is based on research from Hebrew University.