Fragile X research news

Seaside Therapeutics Awards $4.5 Million Collaborative Research Contract to Vanderbilt University Medical Center

Funding supports development of novel therapeutics for fragile X syndrome

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Seaside Therapeutics announced today the award of a $4.5 million collaborative research contract to Vanderbilt University Medical Center to discover novel compounds to potentially suppress the manifestations of fragile X syndrome. Fragile X syndrome is the most common inherited disorder of brain development and the most common known genetic cause of autism. Individuals with fragile X can suffer from impaired cognitive function, developmental delay, attention deficit and hyperactivity, anxiety, obsessive-compulsive and autistic behaviors.

Research conducted by Seaside founders and others in the field has indicated that excessive signaling through metabotropic glutamate receptor subtype 5 (mGluR5) may be responsible for the neurological and psychiatric consequences of fragile X syndrome. Seaside believes that selective inhibition of this receptor could potentially reduce or eliminate the devastating effects of fragile X syndrome.

Scientists at Vanderbilt, led by Dr. Jeffrey Conn, Director of the Vanderbilt Program in Drug Discovery, principal investigator of the fragile X project and a member of Seaside’s Scientific Advisory Board, have identified more than 400 novel compounds belonging to multiple chemical classes that inhibit mGluR5. With the support of the Seaside Therapeutics’ funding, Vanderbilt researchers will use medicinal chemistry, molecular biology, pharmacology, and efficacy studies to develop compounds that have the properties required for drugs to be used for further study in fragile X. Seaside Therapeutics will collaborate with Vanderbilt on this project by contributing scientific and drug development expertise, particularly as related to fragile X syndrome, autism and other disorders of brain development. Seaside will also select compounds from the collaboration to carry forward into clinical development.

“There are currently no effective treatments for fragile X syndrome,” said Dr. Randall Carpenter, Co-Founder, President and CEO of Seaside Therapeutics. “Seaside believes the best approach to identifying new treatments is to use our own research to discover and validate specific biological sites that play a role in fragile X, and then, either internally or in collaboration with others, develop therapeutics that modulate these biologic targets. We’re excited to work with the team at Vanderbilt given their expertise in drug discovery and, most importantly, because they share Seaside’s passion for helping children with fragile X—creating a strong partnership focused on rapidly translating new discoveries in neurobiology into desperately needed novel treatments.”

“Selectively inhibiting mGluR5 to treat fragile X is an innovative idea and, with continued success, has the potential to change the way people think about developmental disorders,” said Dr. Jeffrey Conn. “While we are at the very earliest stages in the drug discovery process, my team members and I are hopeful we can help advance research efforts in fragile X.”

About Fragile X

Fragile X syndrome is relatively rare, affecting approximately 90,000 people in the United States. It is caused by a mutation in the FMR1 gene on the X chromosome that prevents expression of a single protein, the fragile X mental retardation protein (FMRP). The absence of FMRP gives rise to the major symptoms of fragile X syndrome in humans—impaired cognitive function, developmental delay, attention deficit and hyperactivity, anxiety, obsessive-compulsive and autistic behaviors. A key advance for understanding fragile X was identification of the FMR1 gene and subsequent generation of the Fmr1 knockout mouse—an animal model that lacks FMRP and mimics the human condition. By studying the brain of these mice, Seaside scientific founder Mark Bear, Ph.D., the Picower Professor of Neuroscience at the Massachusetts Institute of Technology’s Picower Center for Learning and Memory, discovered a connection between metabotropic glutamate receptor subtype 5 (mGluR5) signaling and fragile X syndrome. Metabotropic glutamate receptors are activated by the neurotransmitter glutamate. Studies by Bear and others indicate that excessive signaling through mGluR5 may be responsible for the neurologic and psychiatric consequences of fragile X syndrome, and suggest that selective mGluR5 inhibitors will provide therapeutic benefit to this population.

About Seaside Therapeutics

Seaside Therapeutics is creating new drug treatments to correct or improve the course of fragile X syndrome, autism and other disorders of brain development. We are dedicated to translating breakthrough discoveries in genetics and neurobiology into therapeutics that improve the lives of patients and their families.

About Vanderbilt University Medical Center and the Vanderbilt Program in Drug Discovery

Vanderbilt University Medical Center is a major referral center for the Southeast and nation. It is made up of Vanderbilt University Hospital, The Vanderbilt Clinic, The Monroe Carell Jr. Children’s Hospital at Vanderbilt, Vanderbilt School of Medicine and Vanderbilt School of Nursing. VUMC is the largest private employer in the region, employing more than 10,000 employees and generating an annual regional economic impact of over $1 billion. The primary mission of the Vanderbilt Program in Drug Discovery is to facilitate the application of chemical and other technologies to answer fundamental questions in the biological sciences that may ultimately lead to the development of novel therapeutic strategies. Vanderbilt scientists led by Dr. Jeffrey Conn, Director of the Vanderbilt Program in Drug Discovery, have pioneered the discovery of “allosteric” compounds that modulate (“turn up” or “turn down”) the activation of certain receptors, called metabotropic glutamate receptors, when the neurotransmitter glutamate binds to them. Using Vanderbilt’s high-throughput screening facility, which is capable of testing tens of thousands of small molecules for drug-like activity in a single day, Dr. Conn and his colleagues have identified more than 400 compounds with mGluR5 inhibitory effects.Source: BusinessWire.com

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