Healx collaborates on Angelman syndrome therapies

By Jenni Spinner contact

- Last updated on GMT

(Nobi_Prizue/iStock via Getty Images Plus)
(Nobi_Prizue/iStock via Getty Images Plus)

Related tags: Artificial intelligence, Rare disease, Precision medicine, Drug discovery, Neurology

The AI-centric drug discovery technology firm is working with a foundation to discover potential treatments for the rare neurogenetic disorder.

UK-based Healx will collaborate with the Foundation for Angelman Syndrome Therapeutics (FAST) to discover and develop treatments for the disorder, which affects approximately one in 15,000 people worldwide. The partnership will use the company’s AI-powered technology to explore potential treatments for Angelman syndrome, which attacks the nervous system.

The partnership is the latest collaboration originating from Healx’s Rare Treatment Accelerator (RTA) program. Launched in October 2019, the initiative is designed to bring together the company’s artificial intelligence (AI) based drug discovery technology with patient insights to predict and validate new uses from already approved drugs, a process reportedly proving quicker, cheaper and safer than traditional discovery methods.

Bruce Bloom, chief collaboration officer with Healx, told Outsourcing-Pharma that incorporating insights from patients is important in the pursuit of treatments for rare conditions.

Patients and their families are at the very heart of everything we do, and we believe that by involving them from the outset, we can close the discovery research loop earlier, meaning we can find treatments faster​,” Bloom said. "The Accelerator gives patient groups the opportunity to collaborate with us to potentially find life-changing treatments for their rare disease - in a matter of years, as opposed to decades​.”

Working together, we combine our AI technology, data, disease and drug discovery expertise to understand the disease, develop novel therapies and take them towards clinical trials in a typical timeframe of 24 months​,” Bloom added.

Angelman syndrome is caused by multiple genetic mutations, including the loss of function in protein-making gene UBE3A; this leads to issues with the nervous system, such as severe problems with movement, balance and speech. Currently there are no approved treatments for the disorder.

Allyson Berent, FAST’s chief scientific officer, said the collaboration is hopeful news for this particular rare-disease population.

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​,” Berent 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​.”

Previous RTA projects include a collaboration with Muscular Dystrophy UK; started in June 2020, the project is focusing on a condition called facioscapulohumeral muscular dystrophy, which causes muscle-weakening in the face, shoulder blades and upper arms. Additionally, Healx announced a partnership with the Children’s Tumor Foundation to develop new therapies for neurofibromatosis, a rare genetic disorder that affects 1 in 3,000 people worldwide.

Outside of the RTA umbrella, Bloom said, Healx is working with FRAXA Research Foundation to develop treatments for Fragile X, believed to be the leading genetic cause of autism, which affects around 1 in 6000 people. To date, the collaboration has identified eight drugs and 17 combination therapies, progressing the most promising candidates through pre-clinical trials towards Phase 2a clinical trials later this year. 

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