Neurodevelopmental disorders represent significant areas of unmet need in healthcare. People affected by these disorders, predominantly children and young adults, can face a range of severe and devastating symptoms. In many cases, there are only limited or no treatments available. GRIN Therapeutics is dedicated to the research and development of precision therapeutics for pediatric neurodevelopmental disorders with the goal of bringing hope to patients and caregivers.
GRIN Therapeutics is advancing promising research and development to assess the safety and efficacy of the investigational therapy radiprodil for the treatment of serious neurodevelopmental disorders including GRIN-related disorders, tuberous sclerosis complex (TSC), and focal cortical dysplasia (FCD).
In many cases, pediatric neurodevelopmental disorders are rare diseases that are not widely known or understood. In our effort to potentially bring new options to people living with pediatric neurodevelopmental disorders, GRIN Therapeutics is privileged to work with many of the global leaders in research, patient care, and advocacy to help build broader awareness of the impact of these disorders among patients, caregivers and communities.
About GRIN-related Disorders
GRIN-related disorders were first classified in 2010 and are a family of rare, genetically defined pediatric neurodevelopmental disorders caused by mutations in a group of genes known as “GRIN” genes that includes GRIN1, GRIN2A, GRIN2B, and GRIN2D. While symptoms of GRIN-related disorders can present as early as infancy, a diagnosis is often not confirmed until age two or later when a child fails to reach developmental milestones. A diagnosis can be established through genetic testing. Most people living with GRIN-related disorders develop disabilities that require continuous supervision. Currently, there are no FDA approved therapies for GRIN-related disorders.
There are several known classes of GRIN-related disorders, including:
GRIN1-related Disorders
Individuals affected by GRIN1-related disorders experience mild-to-severe developmental delays and intellectual disabilities. Other common manifestations include epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties, and behavioral problems.
GRIN2A-related Disorders
Individuals affected by GRIN2A-related disorders may experience developmental delays or intellectual disabilities. Many also develop speech disorders, epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties, and behavioral problems.
GRIN2B-related Disorders
Individuals affected by GRIN2B-related disorders experience developmental delays and intellectual disabilities. Other common manifestations include epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties, and behavioral problems.
GRIN2D-related Disorders
Individuals affected by GRIN2D-related disorders experience moderate-to-severe developmental delays and intellectual disabilities. Other common manifestations include epilepsy, muscular hypotonia, movement disorders, spasticity, feeding difficulties, and behavioral problems.
Additional information about GRIN-related disorders is available from international organizations including:
Progress in Research
GRIN Therapeutics is advancing the development of radiprodil, an investigational drug that has been shown in preclinical studies to target a receptor on the NMDA subunit called “NR2B.” Research has shown that abnormal activity of the NMDA receptor leads to the clinical manifestations of GRIN-related disorders and other neurological conditions including tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II.
About TSC
TSC is a multi-system genetic disorder caused by a mutation in the TSC1 or TSC2 genes that causes non-cancerous tumors to form in many different organs, including the brain, eyes, heart, kidneys, skin and lungs. Tumors impacting the central nervous system can cause cognitive impairment, developmental delays, seizures, behavioral problems and autism spectrum disorder. TSC patients can experience a range of symptoms and they often vary in level of severity from person to person. An estimated one million people worldwide are living with TSC, with approximately 50,000 in the United States.
TSC is the leading genetic cause of epilepsy and autism. While some patients are treated with antiseizure drugs, only about one-third of treated patients become seizure-free[1]. Currently, there are no approved therapies that address the root cause of TSC.
[1] Salussolia CL et al. Annu Rev Genomics Hum Genet 2019;20:217-40.
More information about TSC is available from international organizations including:
About the Astroscape Study
GRIN Therapeutics is planning to launch the Astroscape study, an open-label proof of concept study to assess the safety and tolerability of different doses of radiprodil in people living with TSC or FCD Type II. The study will evaluate the effect of radiprodil on the frequency and severity of epileptic seizures and on other symptoms related to behavior, motor skills, sleep and quality of life. Approximately 30 patients will be treated with radiprodil. Participants may have the option to participate in a long-term extension phase of the study.
For additional information about the Astroscape Study, visit astroscapestudy.com or clinicaltrials.gov.
Our Commitment to Patients
and Families
At GRIN Therapeutics, we are privileged to work closely with leaders from different advocacy and treatment communities to continually ensure that our efforts reflect the unique needs of the individuals and families we serve.
To contact GRIN Therapeutics patient advocacy, click here.
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GRIN Therapeutics is a subsidiary of Neurvati Neurosciences, a division of Blackstone Life Sciences.