Cure Rare Disease (CRD) announced that the FDA has approved the first-in-human IND for CRD-TMH-001, its first therapeutic candidate using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy.
Syncthink Inc. is preparing to launch its neurological impairment and disease diagnostic in Europe, after securing CE marking for the ocular biomarker device. The company is raising a new round of funding to support commercialization of the Eye-Sync system and has two projects exemplifying applications of the device running with academic collaborators in the U.K.
Beckley Psytech has divulged ergoline analogues acting as 5-HT2A and/or 5-HT2B and/or 5-HT2C receptor agonists reported to be useful for the treatment of depression.
Central nervous system specialist Cerevance Inc. has announced a multiyear strategic research collaboration with Merck & Co. Inc. to identify novel targets for Alzheimer’s disease in a deal worth more than $1.1 billion, plus royalties. Boston-based Cerevance will receive $25 million up front with the $1.1 billion due when certain development and commercial milestones are achieved.
Following the readout of a phase II trial evaluating its COVID-19 oral antiviral Pentarlandir (SNB-01), Taiwan’s Syneurx International Corp. said it expects to launch a phase III test of the candidate in the next few months.
Data on C2N Diagnostic LLC’s new blood test combining a proprietary p-tau217 measurement with the amyloid beta (a-beta) 42/40 ratio, a component of C2N’s commercially available PrecivityAD blood test, could help to predict people at risk for Alzheimer’s disease (AD) – even at the earliest stages.
Shares in Karuna Therapeutics Inc. soared Aug. 8 as the biotech moved closer to a breakthrough for schizophrenia therapy after its potential first-in-class drug Karxt (xanomeline + trospium) met its goals in a closely watched phase III trial. Analysts said results from the Emergent-2 trial were at the top end of what was expected, lifting Karuna’s shares (NASDAQ:KRTX) 71.8% to a 52-week high of $241.19.
To explore the role of histone deacetylase 11 (HDAC11) in Alzheimer's disease, Massachusetts General Hospital researchers generated selective, brain-penetrating HDAC11 inhibitors.
Beta-site APP-cleaving enzyme 1 (BACE1) inhibitors have a long history of failure in patients with Alzheimer's disease (AD). Clinical development of verubecestat, elenbecestat, lanabecestat, umibecestat, atabecestat and LY-3202626 were all discontinued.
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