Healx and the Foundation for Angelman Syndrome Therapeutics (FAST) are partnering to identify new therapies for Angelman syndrome from among already-approved and available medications, according to a company press release.
Finding new uses for old medications — a process called drug repositioning or repurposing — provides a faster, cheaper, and safer alternative to the traditional drug discovery process.
Several experimental therapies are under development to treat Angelman syndrome, but none have been approved yet. Those living with Angelman manage symptoms through physical, speech, and occupational therapy, as well as with anti-epileptic medications.
Healx plans to combine its expertise in artificial intelligence with insights from patient groups and research to predict and validate suitable therapies. By including patient insights in its therapeutic development strategy from the start, Healx hopes to build a more collaborative drug discovery model.
“Patients and their caregivers are the real experts in rare diseases,” said Bruce Bloom, MD, chief collaboration officer at Healx. “They are the ones driving forward research efforts to find new treatments, and have a wealth of experience and insight about what it’s actually like to live with these conditions. By integrating their expertise early on, and connecting to their well developed scientific and clinical network, we have a better chance of finding critical new therapies.”
The collaboration comes from Healx’s Rare Treatment Accelerator (RTA), a partnership program that provides up to $1 million per project to bring therapies to clinical trials within a roughly two-year timeframe.
This is the third RTA-supported project and the first to focus on Angelman syndrome.
Other projects focus on genetic disorders involving nerves and muscles. A collaboration with Muscular Dystrophy UK focuses on facioscapulohumeral muscular dystrophy, which affects muscles of the face, shoulder blades, and upper arms. Another partnership with the Children’s Tumor Foundation seeks treatments for a rare genetic disorder called neurofibromatosis.
“FAST is very excited to partner with Healx and we welcome them to the Angelman syndrome community,” said Allyson Berent, DVM, chief scientific officer at FAST.
“Healx’s use of cutting edge artificial intelligence, combined with our scientific team’s expertise in cell lines and animal models, makes this innovative program incredibly promising,” she said. “This program creates a unique opportunity to quickly bring therapies from bench to clinic, which could potentially have a huge impact on all individuals living with Angelman syndrome.”