PTC Therapeutics Inc. said a “highly competitive process with several parties involved” led to the deal centered on the firm’s Huntington’s disease program with Novartis AG, an arrangement that brings $1 billion up front along with as much as $1.9 billion in development, regulatory, and sales milestone rewards.

Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.

The collaboration aims to identify a small molecule that targets mutant androgen receptor (AR) mRNA splicing and causes selective destruction of the disease-causing mRNA.

Our immune cells are not just “defenders” against deadly viruses and pathogens but also a great balancer for tissue homeostasis. For neurological disorders, understanding the neuro-immune axis could be key to treating previously untreatable conditions such as autism spectrum disorder, according to Jun R. Huh, professor of immunology at Harvard Medical School.

Sarepta Therapeutics Inc. has signed a licensing and collaboration agreement with Arrowhead Pharmaceuticals Inc. to obtain exclusive global rights to multiple clinical, preclinical and discovery-stage programs for rare genetic diseases of the muscle, central nervous system (CNS) and the lungs. The deal includes ARO-DUX4, ARO-DM1, ARO-MMP7 and ARO-ATXN2.

Enveric Biosciences Inc. has completed preclinical pharmacokinetic (PK) studies of EB-003 in rats and dogs, further supporting EB-003’s oral bioavailability and targeted nonhallucinogenic profile. EB-003 is a neuroplastogen designed to promote neuroplasticity without inducing hallucinations in patients with difficult-to-address mental health disorders.

Oxford Nanopore Technologies Ltd. has established a new collaboration with UK Biobank to create the world’s first comprehensive, large-scale epigenetic dataset. The project will utilize Oxford Nanopore’s DNA/RNA sequencing technology to map the epigenome of 50,000 blood samples from UK Biobank to unlock insights into disease mechanisms, with the aim of improving patient outcomes.

Diagnosing depression and other mental illnesses is largely subjective, but that could soon change as Sydney-based Trivarx Ltd. developed an AI-based algorithm that uses heart rate and heart rate variability coupled with sleep disturbance to diagnose major depressive episodes.