Healx Archives • Fragile X Research - FRAXA Research Foundation

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, 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.

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, 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, 2018FDA, 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, FRAXA-DVI, Healx, 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)

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, 2018Healx

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.