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AI, the solution to accelerate drug discovery and the treatment of rare diseases

Ana-Maria Tapescu
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11 February 2026, 07:05
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Biotechnology now has advanced tools, but thousands of rare diseases remain without approved treatments. According to leaders at Insilico Medicine and GenEditBio, the problem is not the technology, but the lack of specialized researchers. AI emerges as a promising solution, having the potential to accelerate drug discovery.

Insilico has launched "MMAI Gym", an AI system that can analyze biological and chemical data, generating hypotheses and evaluating therapeutic options simultaneously, which reduces time and costs. GenEditBio, on the other hand, focuses on the precise delivery of CRISPR tools into organisms, having recently received FDA approval for CRISPR therapies.

However, AI faces challenges related to the quality of data needed to avoid bias. In the future, the development of digital twins for virtual clinical trials could revolutionize medical research, making personalized treatments more accessible.

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AI as a Force Multiplier in Rare Disease Treatment

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