New ‘zero-shot’ AI approach aims to jumpstart rare disease treatments
First Atom launches platform to create novel drugs for AADC, other conditions
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First Atom Therapeutics — newly rebranded from Asha Therapeutics — will use a drug design approach powered by artificial intelligence (AI) to develop entirely new therapeutic candidates for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency.
The platform will also be used to create novel drugs for treating neurodegenerative and neuro-oncology disorders, according to a company press release announcing the launch.
The new approach, branded as zero-shot, means the system is designed to propose promising molecules even when there’s little or no prior data regarding a specific target — a common challenge in rare disorders. It works by designing compounds from scratch rather than relying on the screening of preexisting chemical libraries, or being limited by known chemical templates.
By generating high-quality drug candidates faster, especially for difficult targets, the zero-shot approach could accelerate the path from biological insight to a therapy worth testing in clinical trials, according to the developer.
“The name First Atom incorporates our company’s approach to drug design from the outset — precise, foundational, and centered within the unlimited chemical space to create novel chemical entities with transformative potential,” said Rainer Metcalf, PhD, First Atom’s chief technology officer. “The origination of new drug concepts through rigorous design will define the next era of therapeutics.”
In rare disorders, therapeutic development can be slow because patient populations are small and traditional trial-and-error screening approaches may not efficiently find compounds that hit the right biological target.
First Atom’s approach aims to start with a specific disease target — for example, a protein involved in a rare neurologic condition — and computationally design brand-new chemical structures atom by atom to fit that target, rather than screening large premade libraries of compounds. The company says its platform combines AI with physics-based methods and can design compounds for targets with few or no existing drug-like examples.
Alongside the rebrand, the company said it has expanded its PRISM Platform as it builds a pipeline across “high unmet need” indications, including rare diseases. This platform is an AI-powered drug design system that builds new medicine compounds using physics-based models and helps identify targets, design molecules, predict how they’ll behave in the body, and assess safety, all before any actual synthesis in the lab.
“First Atom is assembling a unique combination of scientific expertise, proven drug design capability, and strategic execution skill,” said Wayne Guida, PhD, First Atom’s senior technology advisor. “I look forward to contributing to First Atom’s team and patient-centric culture as they continue to build out their drug design technology for the creation of critical new medicines.”
