Myrtelle Inc. and Raaven Therapeutics AB have partnered on the development of novel recombinant adeno-associated virus (rAAV) vectors to advance gene therapy treatments for diseases of the central nervous system (CNS) in which myelin is affected.

Entrada Therapeutics Inc. has selected a second clinical candidate within its Duchenne muscular dystrophy (DMD) franchise, ENTR-601-45, for the potential treatment of people living with DMD who are exon 45 skipping amenable. The company plans to submit an IND application in the second half of next year.

Athira Pharma Inc. is advancing ATH-1105 toward a planned IND filing this year for first-in-human studies for amyotrophic lateral sclerosis (ALS).

It has been shown that site-specific phosphorylation of tau at threonine 205 (T205) by the kinase p38γ disengages tau from toxic pathways, thus acting as a neuroprotective function in Alzheimer’s disease. Researchers in Australia have designed a novel AAV-based gene therapy approach targeting p38γ, DBA/2.p38γCA, in murine models of epilepsy in which tau is known to play a neurotoxic effect.