Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role in brain development and function.
Changchun Genescience Pharmaceuticals Co. Ltd. has discovered new ubiquitin carboxyl-terminal hydrolase 30 (USP30) inhibitors potentially useful for the treatment of mitochondrial and Parkinson’s disease.
Montara Therapeutics Inc. has been awarded a research grant of approximately $1 million from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to advance a Brainonly therapy targeting mTOR for Parkinson’s disease.
Janssen Pharmaceutica NV has identified new endosomal/lysosomal proton channel TMEM175 activators that are potentially useful for the treatment of Parkinson’s disease.
Lysoway Therapeutics Inc. has been awarded an additional research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support development of its TMEM175 agonist program for Parkinson’s disease.
Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease.
The FDA has accepted the IND application from Rest Therapeutics SAS for RST-101, the company’s lead investigational candidate for the early treatment of post-traumatic stress disorder (PTSD).
Recent findings are reshaping current understanding of the post-infection landscape of SARS-CoV-2. Although previous studies had already suggested that autoimmunity might underlie the persistent neurological symptoms seen in long COVID, researchers at Yale University and Mount Sinai now reinforce this hypothesis. SARS-CoV-2 infection appears to trigger an autoimmune mechanism that drives chronic pain, fatigue and cognitive impairment in some patients.
Cavalon Therapeutics Inc. and Northwestern University have disclosed new voltage-gated calcium channel Cav1.3 blockers potentially useful for the treatment of Parkinson’s disease and aldosteronism.