Galecto Inc.’s late-September news from its ongoing phase IIa trial with GB-2064 in myelofibrosis hiked shares of the Boston-based firm and piqued interest in the lysyl oxidase-like 2 (LOXL2) target, where such names as Gilead Sciences Inc. and Pharmaxis Ltd. also have been busy.

The U.S. FDA wants more data on PTC-518 before PTC Therapeutics Inc.'s phase II study of Huntington’s disease can continue enrollment. While stopped in the U.S., the study of the oral, small-molecule splicing modifier still is enrolling participants at sites in several European countries and in Australia.

Milestone Pharmaceuticals Inc. saw a premarket surge in its share price value after a phase III trial of the company’s self-administered, heart rhythm-restoring nasal spray, etripamil, hit its target in a phase III trial, setting up a potential filing with the U.S. FDA in mid-2023.

Human brain organoids transplanted into rats could be used as an in vivo model for the study of neuropsychiatric diseases. Researchers at Stanford University managed to mature human organoid neurons in the somatosensory cortex of the animal's brain and incorporate them into its neural circuitry.The integration improved the morphological and physiological properties of the transplanted neurons. Compared to those of organoids in a Petri dish, human cells preserved their own identity, and they modified the rat's learned behavior through stimulation and reward experiments.

Genetic testing has the potential to identify infants and children with autism potentially years earlier than clinical assessments, improving response to therapies and long-term outcomes. Sema4 Holdings Corp. presented results of a study Oct. 13 at the Child Neurology Society Annual Meeting that showed exome sequencing improved the diagnosis of autism spectrum disorders by up to 21% compared to current methods. At the same time, the research suggested that exome sequencing can identify children at risk of common comorbidities such as epilepsy, enabling more timely interventions.

GSK plc has announced results from its respiratory syncytial virus (RSV) vaccine in older people that look stronger than those from its rival, Pfizer Inc., as the race to develop a first ever vaccine against the common respiratory disease heats up.

Relmada Therapeutics Inc. is scratching its collective chin as it sifts through data from the failed phase III Reliance III study of REL-1017 (esmethadone) in treating major depressive disorder (MDD). A higher-than-expected placebo response, however, prompted the company to label the study’s results as “paradoxical.”