Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
One of the major obstacles to treating neurological disorders such as Alzheimer’s disease is the blood-brain barrier: drugs that are injected into the circulation usually do not enter the brain effectively. Researchers at Denali Therapeutics Inc., Biogen Inc. and the University of Minnesota have devised a vehicle for transporting antibodies against amyloid-β that can bypass the blood-brain barrier by binding to the transferrin receptor, which is expressed much more abundantly in capillaries than in arteries.
Maplight Therapeutics Inc. has synthesized G protein-coupled receptor GPR6 inverse agonists reported to be useful for the treatment of anxiety, bipolar disorder, eating disorders, depression, schizophrenia, Huntington’s disease, Parkinson’s disease and Alzheimer’s disease, among others.
Siteone Therapeutics Inc. has disclosed sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
Microglia are the brain’s innate immune cells that maintain brain health and play a key role in Alzheimer's disease (AD). Triggering receptor expressed on myeloid cells 2 (TREM2) acts as a regulator of microglial cells and restores neuronal functioning.
The Jackson Laboratory has disclosed short transient receptor potential channel 3 (TRPC3) and short transient receptor potential channel 6 (TRPC6) antagonists reported to be useful for the treatment of Alzheimer’s disease.
Childhood-onset neurodegeneration with cerebellar atrophy (CONDCA) is an autosomal recessive disorder that causes progressive motor and cognitive impairment in children. The disease arises as a result of inactivating mutations in cytosolic carboxypeptidase 1 (CCP1), leading to excessive polyglutamylation of tubulin in the brain. Researchers at Shimane University have shown in a mouse model that delivering a truncated form of CCP1 into the brain can substantially mitigate Purkinje cell degeneration and improve motor function.
Seal Rock Therapeutics Inc. has joined The Michael J. Fox Foundation for Parkinson’s Research (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE) program. The program supports the development of new therapies targeting LRRK2 for the treatment of Parkinson’s disease patients and fosters international collaboration across more than 30 academic and clinical centers and more than a dozen companies.
In Parkinson’s disease, α-synuclein accumulates in neurons and may thereby contribute to their degeneration. Reducing expression of α-synuclein may be an effective therapy, but delivering short interfering RNA (siRNA) to the brain noninvasively is notoriously ineffective, in part because siRNA does not pass the blood-brain barrier efficiently.
A little-known tissue composed of a cluster of immune cells could offer novel insights into the development of neurological disorders. Meninges' immune system changes with age and neurodegeneration. Are they protecting the brain or fueling disease? Mapping and analyzing the so-called ectopic lymphoid structures (ELSs) in the meninges at different ages in preclinical models of neurodegenerative diseases such as Alzheimer's may help clarify whether they are good, bad, or ugly, as in the iconic film by Sergio Leone.
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