Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.
Remynd NV has turned in positive phase IIa data for a new target in Alzheimer’s disease, showing that restoring calcium homeostasis in neurons reduces levels of pathological tau proteins, increases dopamine levels and has a positive effect on cognition in symptomatic patients.
A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.
Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.
Nanonewron Inc. has been awarded a $2.5 million NIH STTR phase II grant to support development of its TNF-α inhibitor NN-840 program for Alzheimer’s disease and other neurodegenerative conditions. The company aims to submit an IND application next year.
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
LB Pharmaceuticals Inc. became the first traditional biopharma company to price an IPO in the U.S. since February, raising $285 million through the upsized sale of 19 million shares at $15 apiece. Shares, now listed on Nasdaq under the ticker symbol LBRX, rose 15% to close Sept. 11 at $17.30.
Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.
The U.S. FDA issued new guidance for the development of non-opioid analgesics for chronic pain indications, with specific details on trial design, patient populations and meaningful outcomes, including reducing the nation’s reliance on opioids.
Researchers from Cyclone Therapeutics Inc. and the Scripps Research Institute have disclosed tetracycline derivatives reported to be useful for the treatment of fragile X syndrome, rheumatoid arthritis and Alzheimer’s disease.
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