Glioblastoma (GBM) is an aggressive cancer from the CNS usually characterized by a very bad prognosis. It is known that around 30%-35% of patients with GBM develop epilepsy as a comorbidity of the disease.

Being able to detect and monitor the aggregation of α-synuclein in situ could lead to more objective, earlier diagnosis of Parkinson’s disease as well as allow real-time monitoring of whether patients are responding to treatment.

Huidagene Therapeutics Co. Ltd. has presented data for HG-303, a new CRISPR-hfCas12Max-based therapeutic approach that knocks down ATXN2 expression for the treatment of amyotrophic lateral sclerosis (ALS).

Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.

The credibility gap in psychedelic drug development continues to narrow with positive top-line data from a Beckley Psytech Ltd. phase IIa study in depression. The results keep the treatment into a tight race with GH Research plc, which also has an inhalable drug in development.

The merger of Protagenic Therapeutics Inc. and Phytanix Bio Inc. combines two different approaches to treating obesity and metabolic issues. The all-stock deal will merge Protagenic’s peptide candidate in IND-enabling development for treating depression, anxiety, posttraumatic stress disorder and additional indications, along with Phytanix’s cannabinoid and cannabinoid-like molecules for bladder pain syndrome and treatment-resistant focal seizures.

Mirroring results reported in a phase IIb study, Apnimed Inc.’s first of two pivotal trials testing AD-109 as an oral therapy in obstructive sleep apnea hit primary and secondary endpoints. Should similar findings emerge from a second phase III trial, expected to read out in a couple of months, the privately held firm anticipates a U.S. NDA filing in early 2026.