Home - RogCon

Pioneering Breakthroughs for SCN2A Mutations

RogCon — The SCN2A Company — is leading the discovery and development of novel treatments for SCN2A mutations that cause epilepsy, autism and other neurological disorders.

Join the International SCN2A Natural History Study

The SCN2A Natural History Study is a central element to facilitating new drug development for SCN2A patients. An NHS such as this one – being conducted by the top epileptologists and neurologists around the world – is a critical part of designing and implementing clinical trials and provides important regulatory information to help increase the FDA’s overall understanding of the condition and the impact of the novel treatments.

Join the Natural History Study

Learn More About Clinical Trials

RogCon is advancing several investigational new drugs for those affected by SCN2A mutations. We will call RogCon a success only when every child with an SCN2A mutation has access to a disease-modifying treatment. Clinical trials for our lead program are projected to begin shortly. Learn more about our clinical trials and how to participate.

More about Clinical Trials

RogCon was founded by two parents of children affected by SCN2A mutations, Alex Nemiroff and Kelley Dalby, and Dr. Steven Petrou, a renowned neuroscientist specializing in genetic epilepsy. Our founders came together to address the severe, unmet medical need of children and their families who are suffering from mutations in SCN2A, where no solutions currently exist. At RogCon, we are walking a path of relentless pursuit, taking deliberate action on a daily basis to bring novel, disease-modifying treatments to all patients.

The Latest on What’s Happening at RogCon

RogCon and Prometheus Research Enter into Partnership to Support International Natural History Study on SCN2A Mutations

NEW HAVEN, Conn., Jan. 14, 2019 /PRNewswire/ — RogCon Biosciences, Inc., (RogCon), a biotechnology company leading the discovery and development of novel treatments for patients suffering from SCN2A (Nav1.2) mutations, has partnered with Prometheus Research to support the creation of a scalable clinical data registry (CDR) that will assist a planned international natural history study of patients with […]

Antisense Oligonucleotide Therapy For SCN2A Gain-of-Function Epilepsies: AES 2018 Poster

Click here to view poster RATIONALE: The clinical spectrum associated with SCN2A de novo mutations includes early and late seizure onset developmental and epileptic encephalopathy (DEE) as well as autism. Patients with early seizure-onset DEE are most frequently associated with SCN2A variants that show gain of function biophysical changes that would lead to enhanced neuronal […]

Partners on our Path to Discovery

Our path is enhanced through strong strategic collaborations and partnerships. Due to our expertise with SCN2A and our commitment to discover new therapies for SCN2A disease, RogCon has forged partnerships with Ionis Pharmaceuticals and The Florey Institute of Neuroscience, and has formed research collaborations with the foremost academic experts in genetics and epilepsy from around the world to support a number of ongoing programs.

In 2016, Ionis Pharmaceuticals (“Ionis”) [NASDAQ: IONS] and RogCon began a collaboration with the goal of developing new disease-modifying antisense oligonucleotide drugs to treat SCN2A disorders.

Ionis is the leading company in RNA-targeted drug discovery and development with a pipeline of first-in-class or best-in-class medicines with the potential to provide high value for patients with significant unmet medical needs. Over the last 30 years, Ionis has created an efficient and broadly applicable drug discovery platform using its proprietary antisense technology.

Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over forty drugs in development. Including SPINRAZA® (nusinersen), an approved treatment for patients with spinal muscular atrophy, or SMA, a severe motor-neuron disease that is the leading genetic cause of infant mortality. Ionis is well-suited as a partner for RogCon given their leading position in antisense technology and proven success in rare, genetic disorders and neurological disease.

Our co-founder, Dr. Steven Petrou, leads The Florey Institute of Neuroscience and Mental Health, one of the largest and most highly respected brain research centers in the world.

The research collaboration between The Florey and RogCon is critical to ensure RogCon is at the forefront of scientific and medical knowledge on SCN2A mutations. A large group of scientists at the Ion Channels and Human Diseases Laboratory at the Florey Institute have played a pivotal role in RogCon’s work to research and understand SCN2A mutations through a multidisciplinary approach including ion channel biophysics, mouse transgenesis, genetic analysis, computational modeling and in vitro and in vivo electrophysiology.

RogCon U.R.

RogCon U.R., a sister company of RogCon Biosciences, is focused on the research and development of novel, disease-modifying compounds that up-regulate SCN2A for the treatment of loss-of-function mutations in SCN2A causing autism and epilepsy. RogCon U.R. is working in partnership with the Florey Institute, among others, on this platform.

Learn More