Deficiencies in the SLC6A1 gene, encoding the γ-aminobutyric acid transporter GAT-1, are associated with infantile encephalopathy with intellectual disability. Like other neurodevelopmental disorders, SLC6A1-related disorders lack effective treatments; therefore, gene therapy using viral vectors has been proposed as a potential therapeutic strategy.

2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.

Among the most profound results presented at the 2024 European Society for Medical Oncology (ESMO) Congress were the 10-year data from the Checkmate-067 and Keynote-006 trials of Opdivo and Keytruda as first-line agents in advanced or metastatic melanoma in which 10-year overall survival topped 40%. The success of checkpoint blockade, however, has not extended to all tumor types, but in 2024, molecular studies have led to advances in gene therapies and a multitude of approaches that have opened the door to hope.

AI pulled in major financings and approvals for Asia med-techs in 2024 as Asia Pacific countries played to individual strengths to maximize AI’s applications in the health care sector. While breakaway AI technologies like OpenAI’s ChatGPT reshaped and boosted many industries, AI also drove major financings for APAC med-techs weathering a wider macroeconomic downturn, with AI-based companies accounting for five out of 11 IPOs tracked on BioWorld’s med-tech IPOs list.

In the 1970s, scientists from several countries proposed to reconstruct, one by one, all the neurons in the brain as they appear under an electron microscope. They started with a small worm. Caenorhabditis elegans has only 302 neurons. It took 16 years. How much time would be required to repeat this arduous task for the 100 billion neurons in the human brain?

Gait instability and somnolence are the main hindbrain-related adverse effects associated with the inhibition of AMPA receptors (AMPARs). Transmembrane AMPAR regulatory proteins (TARPs) modulate AMPAR function, with the specific member TARPγ8 being highly expressed in brain regions associated with seizures and expressed at low or negligible levels in the hindbrain.

2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.

Sangamo Therapeutics Inc.’s second large, worldwide licensing deal for its capsid technology in the past five months is with Astellas Pharma Inc. The California-based company is getting $20 million up front and the chance to bring in up to $1.3 billion in fees and milestone payments in an agreement spanning five potential disease targets for gene therapies to treat neurological diseases.