Healx Archives

Fragile X Syndrome: In Pursuit of a Cure Webinar

FRAXA Research Foundation FRAXA Updates • Seminar Series July 29, 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.

2021 FRAXA Awards – Recognizing Perseverance and Dedication

Eric Welin FRAXA Updates July 22, 2021July 22, 2021Healx

In conjunction with World Fragile X Day 2021, FRAXA Research Foundation is proud to recognize its annual award recipients. This year’s recipients exemplify the perseverance and dedication that has made FRAXA a global leader in Fragile X research for nearly 30 years. We are fortunate to partner with these individuals on research, community support and awareness efforts.

FRAXA Drug Validation Initiative (FRAXA-DVI)

FRAXA Research Foundation 2011 Grants • 2012 Grants • 2013 Grants • 2014 Grants • 2015 Grants • 2016 Grants • 2017 Grants • 2018 Grants • 2019 Grants • 2020 Grants • 2021 Grants • Biomarkers • Carullo, Paulina • Cogram, Patricia • Current Research Grants • Deacon, Robert • Drug Repurposing • FLENI Foundation • Research Resources • University of Chile • University of Oxford May 6, 2021May 6, 2021BPN14770, drug repurposing, EEG, FRAXA-DVI, Healx, Neuren Pharmaceuticals, NIH, Ovid Therapeutics, PDE4, Tetra Therapeutics, trofinetide

The FRAXA Drug Validation Initiative (FRAXA-DVI) provides speedy, cost-effective, objective preclinical testing of potential Fragile X treatments. FRAXA-DVI uses in-vitro systems, behavior batteries, and gene expression and peripheral biomarker platforms to validate investigational new drugs and repurposed available compounds in Fragile X syndrome (FXS).

Healx Drug Repurposing Programme for Fragile X Syndrome

FRAXA Research Foundation Research Updates • Seminar Series October 1, 2020October 1, 2020autism, drug repurposing, EEG, Healx, PAK (p21-activated kinase), Roche

David Brown, MD, PhD, Ivan Angulo-Herrera, PhD and Anthony Hall of Healx present about the Drug Repurposing Programme for Fragile X syndrome.

Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse

FRAXA Research Foundation Binder, Devin • Razak, Khaleel • Research Updates • Seminar Series August 27, 2020August 27, 2020astrocytes, DoD, drug repurposing, EEG, glutamate, Healx, hypersensitivity, matrix metalloproteinase, NIH, seizure, sound

In this Fragile X research webinar we hear from Devin K. Binder, MD, PhD, Professor, University of California at Riverside Medical School and Khaleel Razak, PhD, Professor, University of California at Riverside as they present about Mechanisms and Biomarkers of Sensory Hypersensitivity in the fmr1 Knockout Mouse.

Drug Repurposing for Rare Disease and the Future of Health – The Genetics Podcast

FRAXA Research Foundation FRAXA Updates • Healx • Research Updates February 20, 2020February 20, 2020autism, drug repurposing, Healx, metformin, minocycline

In this double-bill episode of The Genetics Podcast, Dr. Patrick Short talks to two key rare disease researchers in the field: Dr. Bruce Bloom, CCO of Healx, and Dr. Mike Tranfaglia, CSO of FRAXA. Both draw on their wide-ranging personal and professional experiences to discuss the successes and opportunities of drug repurposing, the power of using machine learning, and the work they’ve been doing to aid in finding effective treatments for Fragile X.

New Partners Aim to Accelerate the Discovery and Repurposing of Medicines for Rare Neurological Diseases

Fragile X News Today FRAXA Updates December 13, 2019drug repurposing, Healx

First Healx secured $56M in new financing to launch a global Rare Treatment Accelerator program to tackle Fragile X syndrome and 39 other rare diseases. Now they have built a partnership with Boehringer Ingelheim worth millions. It all started with a small FRAXA grant to Healx to repurpose available drugs for Fragile X.

Healx Raises $56M to use AI to Find Treatments for Fragile X & Other Rare Diseases

FRAXA Research Foundation Drug Repurposing • FRAXA Updates • Healx October 16, 2019October 16, 2019baclofen, drug repurposing, FRAXA-DVI, Healx, lithium, lovastatin, metformin, minocycline

Healx has secured $56M in new financing to build a clinical-stage portfolio for rare diseases, including treatments for Fragile X syndrome, and to launch a global Rare Treatment Accelerator program. Where the traditional drug discovery model takes more than a decade and can run into the billions of dollars, Healx’s AI-driven approach makes the process faster, more efficient and cost-effective.

Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X

FRAXA Research Foundation 2016 Grants • 2017 Grants • 2018 Grants • Current Research Grants • Drug Repurposing • Healx February 8, 2018July 26, 2018drug combinations, drug repurposing, FDA, FRAXA-DVI, Healx

FRAXA Research Foundation initially partnered with Healx in 2016 to identify existing drugs with potential to treat Fragile X syndrome, using machine learning algorithms and computational biology. The study produced results, and now FRAXA and Healx have launched a new round of studies to evaluate combinations of compounds, including both drugs and natural products.

Drug Repurposing Study Results Accelerate Progress Towards Fragile X Treatments

J. Dora Levin Drug Repurposing • Healx • Research Updates October 29, 2017July 11, 2018autism, drug repurposing, FRAXA-DVI, Healx, seizure, sleep

While there are over 8,000 rare diseases affecting an estimated 350 million people worldwide, only around 200 of these conditions have effective treatments. Due to the high cost of developing new drugs, rare diseases have historically been less attractive to pharmaceutical companies. Drug repurposing systematically leverages the detailed information available on approved drugs and reduces the time and money needed to deliver safe “new” treatments, but with greater success rates and a potentially more immediate impact on health care.

Healx team David, Dan, Narissa - FRAXA (1)

Repurposing Available Drugs to Treat Fragile X Syndrome – FRAXA Initiatives

Michael Tranfaglia, MD Drug Repurposing • Research Updates February 10, 2017December 26, 2019arbaclofen, autism, baclofen, drug repurposing, FRAXA-DVI, glutamate, GSK3, Healx, hypersensitivity, lithium, lovastatin, MAP1b, matrix metalloproteinase, metformin, mGluR, minocycline, NIH

FRAXA Research Foundation was founded in 1994 to fund biomedical research aimed at finding a cure for Fragile X syndrome and, ultimately, autism. We prioritize translational research with the potential to lead to improved treatments for Fragile X in the near term. Our early efforts involved supporting a great deal of basic neuroscience to understand the cause of Fragile X. By 1996, these efforts had already begun to yield results useful for drug repurposing. To date, FRAXA has funded well over $25 million in research, with over $3 million of that for repurposing existing drugs for Fragile X.

University of Cambridge Startup Healx is Rapidly Identifying Existing Drugs to Help Fragile X Patients

Theodore Coutilish Drug Repurposing • Healx • Research Updates January 9, 2017July 2, 2018drug repurposing, Healx

FRAXA awarded $44,000 to Healx in 2017 for drug repurposing to find new treatments for Fragile X syndrome. The results of this study include eight top “hits” which show promise for Fragile X. FRAXA is further investigating these hits.