Biomarin Pharmaceutical Inc. has reported progress across its research and development portfolio, including early-stage product candidates.

“From one to many” is how Actio Biosciences Inc. describes its approach to drug development. The firm emerged with a $55 million series A financing and an eye for biological targets found in both rare and common diseases, starting with TRPV4, a target associated with Charcot-Marie-Tooth disease type 2C and other bone diseases.

Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.

Alzheimer’s disease (AD) has a new candidate for its treatment. Nasal anti-CD3 monoclonal antibody (MAb) reduced microglia activation in the brain of mice without its effect being dependent on the β-amyloid (Aβ) deposits characteristic of this neurodegenerative disorder. “We have done many basic studies in the laboratory on microglia. Microglia activation occurs in many neurologic diseases. One of them is multiple sclerosis (MS). And it also occurs in AD,” senior author Howard Weiner told BioWorld.

AI-Stroke SAS is developing an artificial intelligence (AI)-based application able to detect strokes in real time using a simple smartphone or a tablet. The med-tech firm has just won an award in the i-Lab 2023 innovation competition, supported by the French Ministry of Research and sovereign bank Bpifrance SA. “We use the latest AI and computer vision technology to replicate a neurologist’s expertise immediately anywhere, whereas non-specialists can make diagnostic errors up to 50% of the time,” Cédric Javault, CEO and co-founder of AI-Stroke SAS, told BioWorld.

Artelo Biosciences Inc. has completed a pre-IND meeting with the FDA regarding the manufacturing, preclinical and clinical development plan for ART-26.12.