Aicuris Anti-infective Cures AG has announced its intention to focus on the development and commercialization of anti-infectives for immunocompromised individuals, and will discontinue research activities outside of this strategic scope.
Recent studies in chronic hepatitis B patients have shown that bepirovirsen, an antisense oligonucleotide (ASO), may be considered a suitable option for the treatment of hepatitis B virus (HBV) infection.
Vanda Pharmaceuticals Inc.'s antisense oligonucleotide (ASO) VCA-894A has been awarded orphan drug designation by the FDA for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2.
Stoke Therapeutics Inc. has received clinical trial application (CTA) clearance from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study (OSPREY) of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA).
Researchers from Biorchestra Ltd. presented preclinical data for BMD-001, a novel nanoparticle-formulated antisense oligonucleotide (ASO) being developed for the treatment of Parkinson’s disease (PD). miR-485-3p has been previously identified as a pathway and therapeutic target in neuroinflammatory disease, and the potential relevance of this target in PD was further confirmed through RNA-Seq and analysis of expression levels in animal models and patients with PD.
Quralis Inc. raised $88 million in series B round to fund clinical development of its two lead programs in amyotrophic lateral sclerosis (ALS) and to take forward earlier-stage pipeline projects in ALS and frontotemporal dementia.
Resalis Therapeutics Srl closed a €10 million (US$10.6 million) seed round to progress toward the clinic a micro-RNA (miR) inhibitor in development for metabolic disease indications. Riccardo Panella, chief scientific officer and founder, identified the potential of a particular miR species, miR-22, as a target for metabolic syndrome while conducting studies on its potential role in oncology while at Harvard Medical School and Beth Israel Deaconess Medical Center.
