New Treatment to Autism Spectrum Disorder

Amal Haitham, HUJI, School of Medicine - IMRIC, School of Pharmacy- Institute for Drug Research
Domb Abraham, HUJI, School of Medicine - IMRIC, School of Pharmacy- Institute for Drug Research



Life Sciences and Biotechnology   


autism, treatment

Current development stage

For Pharmaceutical development: TRL4 - PoC&Safety of candidate device or system is demonstrated in a defined lab or animal model



Autism spectrum disorder (ASD) is a complex developmental disability, typically appearing during childhood and affecting a person's ability to communicate and interact with others.

In 2020, the Centers for Disease Control and Prevention (CDC) reported that approximately 1 in 54 children in the U.S. is diagnosed with ASD. According to 2016 data: 1 in 34 boys and 1 in 144 girls were identified with autism.

Currently, there are no medications that can cure ASD or treat the core symptoms. The existing medications and methods target ASD symptoms, helping to manage high energy levels, inability to focus, anxiety, depression, and behavioral reactivity.

In 2019 nearly a fifth of children with autism were reported to have unmet healthcare needs.

Our innovation

We have found that inhibiting the formation of a CNS-specific multifunctional signaling molecule, by inhibiting its synthesis in a brain-specific manner, improved the autistic behavior of ASD model mice. We are currently developing chemical derivatives of the inhibitor with better pharmacokinetic properties as well as novel sustained release formulation of the inhibitors, which may improve patient compliance.

Moreover, administration of this signaling molecule to healthy wild type mice induced autistic like behavior, thus validating the role of this molecule in ASD induction.


The technology offers a novel treatment to ASD. Unlike the existing ASD therapy today which aims mainly to reduce symptoms, the suggested therapy directly targets the mechanism responsible for ASD, and therefore may substantially improve the quality of life of the fast growing number of people, suffering from ASD.



Contact for more information:

Mel Larrosa
VP Business Development Healthcare
Contact ME: