Autifony Therapeutics Archives • Fragile X Research

Neurodevelopmental Drug Development Summit Presentation

FRAXA Research Foundation Research Updates January 7, 2022Autifony Therapeutics, Prader-Willi

FRAXA president and co-founder, Katie Clapp was one of three patient advocacy leaders invited to kick off the Neurodevelopmental Drug Development Summit with a presentation on Fragile X, and FRAXA Scientific Advisor, Dr. Elizabeth Berry-Kravis also presented lessons learned from clinical trials in Fragile X Syndrome.

Fragile X Syndrome: In Pursuit of a Cure Webinar

FRAXA Research Foundation Research Updates • Seminar Series July 29, 2021November 22, 2021Autifony Therapeutics, autism, cancer, Fragile X Research Foundation of Canada, Healx, Tetra Therapeutics

A global webinar titled “Fragile X Syndrome: In Pursuit of a Cure,” took place on July 22, 2021 to commemorate World Fragile X Day. This complimentary event is co-organized with WuXi AppTec. We are delighted that more than 5,000 registered from more than 50 countries worldwide, coming together to raise awareness of Fragile X, and to foster collaborations towards effective treatments and ultimately a cure.

Correcting Sensory Processing in Fragile X Mice by Modulating Kv3.1

FRAXA Research Foundation 2019 Grants • Current Research Grants • Disease Mechanisms • Kourdougli Nazim • Portera-Cailliau, Carlos • Treatment Targets • University of California at Los Angeles June 19, 2019June 19, 2019Autifony Therapeutics, hypersensitivity, neural network

FRAXA has awarded a $90,000 grant to Carlos Portera-Cailliau, PhD and Nazim Kourdougli, PhD at UCLA to investigate whether a novel drug can rescue sensory processing deficits in Fragile X mice. People with Fragile X have similar problems in sensory processing. This new drug acts on Kv3.1, a promising Fragile X treatment target also being pursued by UK-based Autifony Therapeutics based on FRAXA-funded research done at Yale.

Novel Modulators of Potassium Channels to Treat Fragile X

FRAXA Research Foundation 2015 Grants • 2016 Grants • 2017 Grants • Disease Mechanisms • Kaczmarek, Leonard • Research Grant Results • Treatment Targets • Yale University January 2, 2019October 10, 2019Autifony Therapeutics, BK channel, hypersensitivity, ion channel, potassium channels, sound

With funding from FRAXA over 2015-2017, the Yale University team of Leonard Kaczmarek, PhD showed that the firing patterns of auditory neurons in response to repeated stimulation is severely abnormal in Fragile X mice. Based on these results, they are collaborating with the UK-based company Autifony to develop advanced compounds which may reverse these deficits.

FRAXA Research Grants Drive Big Investments in Fragile X

Dave Bjork Bhattacharyya, Anita • Canal, Clinton E. • Kaczmarek, Leonard • Research Updates • Zhao, Xinyu March 28, 2018November 22, 2021Autifony Therapeutics, FRAXA-DVI, Fulcrum Therapeutics, seizure, serotonin, sound

Most people know that FRAXA supports academic research at many institutions such as Harvard University, University of Pennsylvania, Massachusetts Institute of Technology, and Yale University. However, FRAXA is also working with more than 30 pharmaceutical companies around the world. Mike spends a lot of his time advising and collaborating with industry partners.

Newly Discovered Regulatory Pathways in Fragile X

J. Dora Levin Kaczmarek, Leonard • Research Updates October 31, 2017July 30, 2018Autifony Therapeutics, BK channel, FDA, glutamate, hyperexcitability, hypersensitivity, ion channel, mGluR5, potassium channels, Roche, seizure, sound

Studies at Yale University and elsewhere are showing that FMRP plays a significant role in the regulation of potassium channels. Looking forward, potassium channel opener drugs could rescue some symptoms of Fragile X in humans.

Neural Markers of Fragile X: A Powerful New Tool for Clinical Trials

J. Dora Levin Biomarkers • Boston Children's Hospital • Clinical • Harvard University • Nelson, Charles A. • Research Updates • Wilkinson, Carol August 17, 2017June 20, 2019Autifony Therapeutics, autism, EEG, hypersensitivity, ion channel, Pierce Family Fragile X Foundation, seizure, sleep, sound

Once the neural marker is identified for a particular challenge, such as kids with poor language versus good language, neural markers can be measured during drug and behavioral therapy trials to see if a child is improving based on objective biological measures.