FDA Approves AI-Developed CRISPR RNA-Editing Therapy

HuidaGene Therapeutics, a global clinical-stage biotech company, has received the green light from the FDA to proceed with its investigational new drug application. The drug in question is HG202, a CRISPR/Cas13 RNA-editing therapy aimed at treating neovascular age-related macular degeneration (nAMD). This therapy has been developed with the assistance of machine learning and artificial intelligence.

The company utilized its HG-PRECISE platform, which stands for HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise, to create the therapy. This platform enables rapid discovery of Cas proteins through the use of AI and machine learning in the context of DNA sequencing. Additionally, HG-PRECISE provides assembly predictions from a metagenomic database, facilitating the development of CRISPR-based treatments.

HuidaGene is set to advance with a phase one study to evaluate the effectiveness of HG202 at various dosages in patients suffering from nAMD. The study, known as the BRIGHT trial, is expected to begin patient enrollment soon. According to Hui Yang, the co-founder and chief scientific advisor of HuidaGene, the AI/ML-driven HG-PRECISE platform was instrumental in the discovery of the Cas13X/Y system. On this foundation, Yang’s team managed to engineer high-fidelity Cas13Y, characterized by efficient editing capabilities and minimal off-target effects. This accomplishment lays the groundwork for future clinical applications of the technology.

In a broader context, CRISPR technology, which was developed over a decade ago, allows researchers to modify DNA sequences in living organisms. This technology has the potential to revolutionize the treatment of genetic disorders and the understanding of disease mechanisms. Additionally, gene editing technologies like SHERLOCK and DETECTR are transforming digital diagnostics, enabling rapid detection of infectious diseases such as COVID-19.

Despite these advancements, ethical, legal, and scientific concerns persist. Experts caution that gene editing can result in irreversible changes, with potential long-term effects that can impact multiple generations. The stability of these changes is also a concern, as any instabilities could have fatal consequences for individuals.

Profluent, another company in the field of CRISPR technology, has used AI to design an open-source gene editor named OpenCRISPR-1. This development demonstrates the capability of AI to create molecules capable of editing human DNA, further showcasing the potential of gene editing technologies.

The advances in gene editing and the application of AI in developing these technologies illustrate a promising future for medical treatments and diagnostics. However, it is crucial to proceed with caution, considering the profound and lasting impacts that these technologies can have on human health and genetics.