To address the need for novel therapeutic candidates against Duchenne muscular dystrophy (DMD), investigators at Mitorx Therapeutics Ltd. developed a library of novel small-molecule mitochondriotropic agents.
Galectin-3 (Gal-3) is used as a marker of activated microglia, and thus a protein that drives inflammation. Patients with Alzheimer’s disease (AD) have shown increased levels of Gal-3 in their cerebrospinal fluid (CSF) compared to healthy controls, as well as increased expression in their brain tissue.
Vaccines that target α-synuclein have a lot of potential for the treatment of Parkinson’s disease (PD) and other synucleinopathies. Tridem Bioscience GmbH & Co. KG has developed the proprietary WISIT vaccine technology.
Leucine-rich repeat kinase 2 (LRRK2), a key drug target, plays a major role in Parkinson’s disease (PD). Mutations in LRRK2, specifically LRRK2 G2019S which increases LRRK2 kinase activity, have been observed in a majority of PD patients.
Ono Pharmaceutical Co. Ltd. has entered into a drug discovery collaboration agreement with the University of Oxford to verify drug discovery seeds and obtain screening compounds for the creation of new medicines.
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which there is a 10% rate of familial cases, with the rest being sporadic cases. Both genetic and environmental factors contribute to the etiology of ALS, and more than 120 genes have been reported to be tied to the disease, but few with strong association. Thus, identifying additional genes contributing to ALS will help shed light on the disease and its related therapies.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria. If approved, Emblaveo would be among the first beta-lactam/beta-lactamase inhibitor combos cleared for use in Europe.
Kazia Therapeutics Ltd. has out-licensed paxalisib as a potential treatment for intractable epilepsy in focal cortical dysplasia type 2 and tuberous sclerosis complex disease in a carve-out deal with Sovargen Co. Ltd. for $20.5 million plus sales royalties.
Graymatters Health Ltd. seeks patent protection for its non-invasive, self-neuromodulation approach for treating post-traumatic stress disorder. In what represents the company’s second PCT filing, GMH’s co-founders, Oded Kraft, Rani Cohen and Shai Attia, describe a method for achieving a reduction of at least six points in a Clinician-Administered PTSD Scale for DSM-5, following neurofeedback treatment delivered for eight to 12 consecutive weeks.
After many years of testing different monoclonal antibodies against amyloid-β protein, the results obtained are far from being outstanding, and the control of the progression and symptoms of Alzheimer’s disease (AD) remains elusive. At the recent AD/PD 2024 conference held in Lisbon, new non-anti-amyloidogenic strategies in the starting line against AD were discussed. Professor Einar Sigurdsson from New York University gave a presentation entitled, “Single domain antibodies for therapy and diagnosis of synucleinopathies and tauopathies.”