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Rett Syndrome Research Trust

Melior Enters Drug Discovery Collaboration with Rett Syndrome Research

Melior Discovery, Inc. announced today that it has entered into collaboration with the Rett Syndrome Research Trust to screen drug-candidates in an in vivo model of Rett Syndrome.

Melior Discovery has developed world-class expertise in screening compounds for potential therapeutic activity in animal models. Melior will apply this expertise to the evaluation of large numbers of drug-candidates in a specialized model of Rett Syndrome. “This partnership is further illustration of Melior’s truly unique capabilities and competence in high throughput in vivo pharmacology,” said Andrew Reaume PhD, CEO, Melior Discovery.

Rett Syndrome, the most physically disabling of the autism spectrum disorders, strikes young girls almost exclusively, with first symptoms usually appearing before the age of 18 months. These children lose speech, motor control and functional hand use, and many suffer from seizures, orthopedic and severe digestive problems, breathing and other autonomic impairments. Most live into adulthood, and require total, around-the-clock care.

The disorder is caused by alterations of an X-linked gene, MECP2. The animal models that have been developed mimic the disorder well and provide an excellent system to test potential drug candidates.

“We are pleased to be working with Melior and have been impressed with their ability to rapidly and effectively evaluate compounds in animal models. We look forward to our collaboration as we strive to identify compounds to ameliorate the devastating symptoms of Rett,” added Monica Coenraads, Executive Director of the Rett Syndrome Research Trust.

Our Melior Discovery, Inc.

Melior Discovery is leading the transformation of pharmaceutical drug repositioning with its unique theraTRACE® platform of multiplexed in vivo models. Melior is using this capability to build an internal pipeline of development candidates. Melior Discovery also partners with pharmaceutical and biopharmaceutical companies to expand their drug development pipelines by using the theraTRACE® platform, and individual animal models, in conjunction with the Company’s in-depth in vivo pharmacology expertise.

Melior Discovery is located in Exton, PA. The Company is privately held with investors that include Cammeby’s Capital Group, VenturEast, Osage Ventures, Mid-Atlantic Angel Group, and BioAdvance.

About Rett Syndrome

Rett Syndrome is a genetic neurological disorder that almost exclusively affects girls. It strikes randomly, typically at the age of 12 to 18 months, and is caused by random mutations of the MECP2 gene on the X chromosome. Rett Syndrome is devastating as it deprives young girls of speech, hand use, normal movement often including the ability to walk. As the girls enter childhood the disorder brings anxiety, seizures, tremors, breathing difficulties, severe GI issues. While their bodies suffer, it is believed that their cognitive abilities remain largely intact. Although most children survive to adulthood, they require total round-the-clock care.

About the Rett Syndrome Research Trust

RSRT is a non-profit organization with a highly focused and urgent mission: eradicate Rett Syndrome and related MECP2 disorders. In search of a cure and effective treatment options, RSRT operates at the center of global scientific activity, funding bold projects that are unlikely to be supported by the NIH or other more traditional funding agencies. RSRT refutes the conventional practice of labs working in isolation, instead seeking out, promoting and funding collaborations and consortia in which scientists work across multiple disciplines. These relationships enable the development and execution of a research agenda that neither academia nor industry could achieve alone. Since 2008, RSRT has provided $25 million of financial support to: 4 clinical trials testing 3 compounds, 33 scientists in 27 academic institutions and 3 biotech firms. To learn more about the Trust, please visit www.ReverseRett.org.