See official press release issued by 4MTx here, which is replicated below.
Skillman, NJ – Nov. 12, 2024 – 4M Therapeutics Inc. (4MTx), an early-stage biotechnology company developing treatments for neuropsychiatric and neurodegenerative diseases, today announced that the Company has been awarded a $120,000 grant from SynGAP Research Fund dba Cure SYNGAP1 (SRF) to evaluate central nervous system effects of novel glycogen synthase kinase 3 beta (GSK3β) inhibitors 4MT2001 and 4MT1060. SynGAP is a protein that plays an important role in neuroplasticity and cognition. SYNGAP1 loss of function variants are associated with intellectual disability, autism spectrum disorder, and seizures, among other symptoms.
The project, entitled, “Dosing and Administration of GSK3β inhibitors in Mice to Support SynGAP1 Research” intends to expose mice to different dose levels of 4MT2001 and 4MT1060 and examine central nervous system target engagement of both single and repeat (7-day) dosing. The project will examine both blood and brain levels of 4MT2001 and 4MT1060, obtain brain homogenates, and evaluate the expression of pCRMP2 and PKC substrates.
“We are pleased that the scientific review by SRF recognized the potential of GSK3β inhibition, and in particular our lead asset, 4MT2001, as well as our 4MT1060 compound, to address a key underlying mechanism behind SynGAP1 disorder,” said Dr. Pablo Lapuerta, Chief Executive Officer and Co-Founder of 4M Therapeutics. “It has been well characterized that GSK3β inhibition through lithium enhances synaptic plasticity and neurogenesis, and our novel GSK3β inhibitors could provide a significant therapeutic advancement in this space, with higher potency and selectivity combined with improved safety.”
“As a parent of a child with SYNGAP1-Related Disorders (SRD), I am acutely aware of the need to find a treatment for the underlying condition that causes SRD that is not only effective but safe,” said J. Michael Graglia, Co-Founder and Managing Director of SRF. “We look forward to supporting 4MTx in this project and the key insights it will provide towards our mission of finding better therapeutic options for people living with SYNGAP1-Related Disorders.”
About 4M Therapeutics Inc.
4M Therapeutics Inc. (4MTx) is advancing treatments for neuropsychiatric and neurodegenerative conditions. The Company focuses on targets for a wide array of disorders and indications. 4MTx applies unique insights from its living human brain cell platform, which was developed through a collaboration between Harvard, MIT, and the University of Washington to identify and design more effective and safer therapeutics. The Company’s pipeline includes potential breakthrough treatments for bipolar mania, agitation in Alzheimer’s disease, neurodegeneration, and other CNS disorders. For more information, visit www.4mtx.net.
For investor and media inquiries, please contact:
Kimberly Lee, DO
Chief Business Officer
ir@4mtx.net
About SYNGAP1-Related Disorders (SRD)
SYNGAP1-Related Disorders (ICD-10 F78.A1) is a rare genetic disorder caused by variants on the SYNGAP1 gene that reduce SynGAP protein levels. SRF has identified over 1,497 patients to date, and the number grows weekly. This protein acts as a regulator in the synapses (where neurons communicate with each other). When SynGAP protein levels are too low, we see an increase in excitability in the synapses making it difficult for neurons to communicate effectively. This leads to many neurological issues seen in SynGAP patients.
Symptoms of SRD include primarily neurological issues including autism spectrum disorder (ASD), intellectual disability, epilepsy, hypotonia (low muscle tone), gross and fine motor delays, global developmental delay, and visual abnormalities such as strabismus (crossed eyes) as well as gastrointestinal challenges and disordered sleep.
About SRF’s Seven Scientific Programs
SynGAP Research Fund has seven scientific programs. These programs reflect SRF’s urgency to develop disease modifying treatments. These include the following:
- BTS – Basic and Translational Science
- Purpose – Drug Repurposing
- SMART – SYNGAP1 Missense Analysis, Research & Therapeutics
- SBOM – SYNGAP1 Biomarkers & Endpoints
- Facilitate – Develop and Share Research Tools and Reagents
- SRDC – SYNGAP1– Related Disorders Characterization
- ProMMiS: Prospective Multidisciplinary, Multisite Study for Clinical Excellence – Natural History Study at Multidisciplinary Clinics
This grant falls into the Purpose program. Drug repurposing looks to reduce suffering in patients on a short time scale. SRF’s process of Find, Assess, Survey, and Trial existing medicines for use in SRD may improve care and reduce medication burden. SRF has funded over $1.65M in Purpose grants.
About the SynGAP Research Fund
The mission of the SynGAP Research Fund (SRF) dba Cure SYNGAP1 is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies, and support systems.
SRF was founded in the US in 2018 as a 501(c)(3) US public charity. There are sister organizations founded by local families in the UK in 2020, Europe (Netherlands) in 2022, as well as both Australia & Latin America (Colombia) in 2023.
Completely family-led, SRF is a leading funder of SynGAP research having committed over $6.2 million in grants to date. The founders cover operational costs, ensuring that donations fund science & patient-related programs. SRF’s grant program awards one or two-year grants to investigators, physician residents, and clinicians interested in studying SYNGAP1. SRF grants are intended to help researchers explore novel ideas and answer open questions related to the clinical aspects of and therapies for SRD.
For more on SRF, visit cureSYNGAP1.org or follow @cureSYNGAP1 on LinkedIn, YouTube, Instagram, Facebook, TikTok, or X.SRF is a member of FasterCures, COMBINEDBrain, Global Genes Foundation Alliance, Everylife Foundation Community Congress, Epilepsies Action Network, Personalized Medicine Coalition, Rare Epilepsy Network, Epilepsy Leadership Council, Alliance for Genetic Etiologies in Neurodevelopmental Disorders and Autism (AGENDA), California Action Link for Rare Diseases, American Brain Coalition, Genetic Alliance UK, Rare Disease UK, Syndromes Without a Name (SWAN UK), Jumpstart Program, Patient Worthy, Autism Brain Net, Innovation and Value Initiative, Rare Disease Diversity Coalition, Cambridge Rare Disease Network, Breaking Down Barriers, Rare-X, Mencap, IndoUSRare, and The World Orphan Drug Congress.