Andlit Therapeutics is a unique biotech company, which rose from compassion and patient oriented values of Our Medison Ventures and is dedicated to developing new and innovative therapeutic agents in collaboration with partners from the industry and academia for children with HNRNPH2
Co-founder and President
Executive Chairman and Co-Founder
CEO and co-founder
Prof. Rotem Karni
Our Scientific Advisors
Dr. Silvi Noiman
Prof. Roger Kornberg
Prof J. Paul Taylor
|PRODUCT||DISCOVERY||PHASE 1||PHASE 2||PHASE 3|
|SMALL MOLECULE FOR|
About HNRNPH2 Mutations
HNRNP family protein can bind to heterogeneous nuclear RNAs and control the splicing fate of those RNA targets. This splicing regulation has an important role in directing cell differentiation and cell function. The splicing function on differential mRNA are controlled by shuttling complexes that bind to the HNRNP NLS domain and direct HNRNPs in and out of the nucleus. Recently, using whole exome sequencing revealed a set of de novo missense mutations that leads to a variant in HNRNPH2 protein that is associated with neurodevelopmental disorder. The HNRNPH2 gene is located on the X chromosome and most of the mutations are restricted to the NLS domain of the HNRNPH2
HNRNPH2-related disorders have recently been described in association with tone abnormalities, developmental delay, intellectual disability, autism, and seizures with a range of severity. These phenotypic observations, mainly in females, resemble many X linked mental retardation, and may have a common pathological pathway.In a mutual effort with the YBRP, we have located ~80 patients, all with de-novo mutations. Now that we have identified the causal gene responsible for the neurobehavioral challenges in these children, we have an opportunity to potentially use this gene as a target for future therapies and/or an opportunity to correct the gene through gene editing.
We are recruiting a project alliance manager, working full time to continue searching for new technologies for the
correction of HNRNPH2 mutations
- Position: Project manager
- Commitment: Full time
- Andlit Therapeutics inc.
10 Hashiloach St,.
We work closely collaboration with the Yellow Brick Road Project yellowbrickroadproject
We recommend patients and families work closely together.
Global natural history study
A global natural history study for the patients is conducted by Dr. Jennifer Bain columbianeurology at Columbia university
please see clinicaltrials