Brain Trust Bio Inc. (BTB) will soon begin phase I trials in Australia of its IT-Riluzole delivered to the brain via a continuous intrathecal drug delivery method in people with amyotrophic lateral sclerosis. The company’s concept is to take known drugs that work and make them better by delivering them exclusively to where patients need them most, BTB co-founder and CEO Chen Benkler told BioWorld.
Neurovalens Ltd recently received U.S. FDA clearance for its Modius Stress device, a neurostimulator that treats people suffering from generalized anxiety disorder (GAD). It also closed a £2.1 million (US$2.65 million) funding round and is planning to raise up to $50 million in a series B fundraising round to be launched later this year.
Proof-of-concept findings had shown that mRNA silencing of striatal Cav1.3 channels prevented and reversed established levodopa-induced dyskinesia in parkinsonian rats, with these effects being maintained in aged rats.
Skyhawk Therapeutics Inc. signed on to work with Ipsen SA in a neurological disease-focused deal worth potentially $1.8 billion to discover and develop novel small molecules that modulate RNA. The deal is an option agreement for exclusive global rights to two candidates. Once the parties have identified and validated those candidates, Paris-based Ipsen will take on all further development and commercialization.
Quest Diagnostics Inc. expanded its Alzheimer’s disease (AD) portfolio with a blood-based biomarker test for phosphorylated tau 217 (pTau217). The test joins the company’s plasma tests for p-tau181, beta-amyloid 42/40 ratio and ApoE genetic risk as well as several cerebrospinal fluid tests for Alzheimer’s.
Brain Trust Bio Inc. (BTB) will soon begin phase I trials in Australia of its IT-Riluzole delivered to the brain via a continuous intrathecal drug delivery method in people with amyotrophic lateral sclerosis. The company’s concept is to take known drugs that work and make them better by delivering them exclusively to where patients need them most, BTB co-founder and CEO Chen Benkler told BioWorld.
The U.S. FDA has cleared Centessa Pharmaceuticals plc’s IND to initiate a phase I first-in-human, clinical trial of ORX-750 for the treatment of narcolepsy.
The generation of pathogenic autoantibodies is a crucial event in the development of inflammation and complement activation, leading to immune cell responses.
Ipsen SA and Skyhawk Therapeutics Inc. have entered an exclusive worldwide collaboration to discover and develop small molecules that modulate RNA for rare neurological diseases. Skyhawk has a unique platform that accelerates building RNA-targeting small molecules across several therapeutic areas.
Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.