Short CV

Annalisa received her PhD in Neuroscience from the University of Genova in collaboration with the Italian Institute of Technology (IIT) in 2019. Afterward, she worked as a postdoctoral researcher at Telethon Foundation and IIT, where she continued the PhD work on the discovery and development of a novel drug candidate for neurodevelopmental disorders (NDDs). Since 2022, she has been working as Head of Preclinical Development at IAMA Therapeutics, the biotech company advancing the drug candidate she contributed to developing during her PhD and postdoctoral work. Her research focuses on developing therapeutic strategies to modulate GABAergic transmission in neurodevelopmental disorders, with a particular interest in small‑molecule modulating NKCC1 transporter to improve brain-related core symptoms in Down syndrome, autism spectrum disorders, and other NDDs. With the drug candidate now progressing into clinical development, her work has evolved into a translational research role, bridging preclinical discovery with early‑phase clinical studies.

Title of the talk

The Translational Journey of a Small‑Molecule: from in vitro models to patients

Abstract

In this talk, Annalisa will present the translational development of IAMA‑6, a first‑in‑class small‑molecule NKCC1 inhibitor designed to modulate GABAergic transmission in neurodevelopmental disorders. The presentation will introduce the scientific and therapeutic rationale behind targeting NKCC1 and GABAergic transmission, outlining the unmet clinical needs in conditions such as Down syndrome, autism spectrum disorders, and other NDDs. It will then describe the preclinical discovery and characterization of IAMA‑6, including in vitro assays, pharmacological profiling, and efficacy studies in rodent models demonstrating improvements in core behavioral and cognitive symptoms. Finally, the talk will highlight the successful transition of IAMA‑6 from preclinical research to early‑phase clinical development, discussing the implications for translational neuroscience and the potential of NKCC1 modulation as a therapeutic strategy for neurodevelopmental disorders.