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A.I. Discovery Companies to Find Rare Disease Drugs

Synthetic biology

(Gerd Altmann, Pixabay)

8 Jan. 2020. A joint venture of two drug discovery and design companies aims to create new biotechnology businesses to find drugs for treating rare diseases. Financial and intellectual property details of the agreement between Cyclica Inc. in Toronto and Translational Medicine Accelerator LLC, in Boston were not disclosed.

Cyclica and Translational Medicine Accelerator say some 10,000 rare diseases affect one-tenth of the world’s population, with few drugs now approved to treat these conditions. Many rare diseases, say the companies, are chronic and progressive disorders, often misdiagnosed or not well-managed, leading to death without adequate treatment. Rare diseases are a particular burden in developing regions, where patients find few options for care and high costs.

To address these unmet needs, the two enterprises established the Rare Diseases Medicine Accelerator to combine Cyclica’s work in drug design and Translational Medicine Accelerator’s expertise in precision medicine, with both companies applying tools based on artificial intelligence. The Rare Diseases Medicine Accelerator plans to form new biotechnology businesses to discover treatments for rare diseases, initially focusing on the needs of patients in Latin America and sub-Saharan Africa.

Cyclica employs computational biology and biophysics for drug discovery to assess the total effects of proposed treatments on the body, not just the prime targets. The company’s Ligand Design service starts with what the company calls seed molecules, then designs more complex molecules with artificial intelligence to achieve specific chemical interactions in the body, as well as reflect desired ADMET — for absorption, distribution, metabolism, excretion, and toxicity — properties.

Cyclica’s Ligand Express is a complementary cloud-based service that screens small molecule compounds against a large simulated protein repository with artificial intelligence routines to visualize and predict multiple interactions in the body. The company takes part in other drug discovery joint ventures, as described by Science & Enterprise in November 2019, when Cyclica joined with ATAI Life Sciences to form a new company to find safer and more effective treatments for psychiatric disorders.

Translational Medicine Accelerator offers a subscription package of genomic, technical, and analytical services for health care providers to find personalized treatments for rare diseases, in what the company calls precision health as a service. The company’s technologies include artificial intelligence and other computational tools to identify, design, and develop personalized interventions for these difficult to treat cases. The personalized therapies can then be scaled-up and evaluated against larger groups of patients in similar conditions.

TMA’s scientific founder is Matthew Might, director of the precision medicine institute at University of Alabama in Birmingham, but also a professor computer science at the university. In 2012, Might’s son was diagnosed with an NGLY1 deficiency, a rare disorder affecting multiple systems in the body. Might’s efforts led to discovery of two therapies, as well as A.I.-enabled drug discovery software, and establishment of treatment and counseling services in Birmingham for rare disorders. He also served as an adviser to the White House during the Obama administration, developing a government-wide precision medicine initiative.

Naheed Kurji, president and CEO of Cyclica, says in a company statement that the Rare Diseases Medicine Accelerator, “will advance the discovery of medicines for those patients who are suffering from the most understudied and neglected diseases.” Joshua Resnikoff, CEO of TMA adds that the joint venture “can create an efficient and systematic approach for developing more effective medicines for individuals, furthering the deployment of precision health care at scale.”

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