After two decades at Alkermes plc (NASDAQ:ALKS), James Frates will join neurodegenerative diseases company Amylyx Pharmaceuticals Inc. as CFO on Jan. 25. Frates said he felt a “calling” to do more to combat amyotrophic lateral sclerosis after his cousin...

Sarepta microdystrophin gene therapy misses endpointShares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell 50% to $84.45 in after-hours trading after  microdystrophin gene therapy SRP-9001 missed the primary functional endpoint of improvement in NSAA total score compared to...

DISEASE CATEGORY: Neurology INDICATION: Anxiety; obsessive compulsive disorder (OCD) Activation of histamine receptor HRH3 in the nucleus accumbens region of the brain could treat anxiety and OCD. In rats, microinjection of histamine or (R)-α-methylhistamine, a selective...

DISEASE CATEGORY: Cancer INDICATION: Melanoma; breast cancer Inhibiting SERPINB9, a serine protease inhibitor, could treat melanoma and breast cancer. In a melanoma mouse model, tumor-specific SERPINB9 knockout reduced tumor size and the number of melanoma...

DISEASE CATEGORY: Cancer INDICATION: Brain cancer; melanoma Tethering T cell therapies to an inhibitor of the cholesterol metabolism enzyme SOAT could enhance the cell therapy’s efficacy against glioblastoma and melanoma. The T cells were engineered...

DISEASE CATEGORY: Musculoskeletal INDICATION: Muscular dystrophy Inhibiting BRD4 and other BET proteins could treat Duchenne muscular dystrophy (DMD). Levels of BRD4 protein in paravertebral muscles were higher in four DMD patients than in two healthy volunteers....

DISEASE CATEGORY: Cancer INDICATION: Colorectal cancer Inhibiting HSF1, a mediator of the proteotoxic stress response, could treat colorectal cancer induced by chronic inflammation. In colon samples from four colitis-associated colon cancer patients, HSF1 protein levels...

DISEASE CATEGORY: Renal INDICATION: Renal damage Promoting expression of the nuclear receptor ERR-α could treat kidney disease associated with diabetes and hypertension. Levels of ERR-α mRNA  were lower in microdissected human kidney samples samples from patients...

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Mitochondrial disease Promoting the expression of the desuccinylase SIRT5 could treat a hereditary mitochondrial encephalomyopathy caused by succinyl-CoA ligase deficiency, which is some patients results from mutations in the tricarboxylic acid...

DISEASE CATEGORY: Autoimmune disease INDICATION: Lupus Lipid nanoparticle-based T-cell specific inhibition of the potassium channel KCNA3 could treat lupus nephritis. The nanoparticles contain an siRNA against KCNA3 and are coated with a mAb targeting CD45RO,...