Global clinical-stage biotech company HuidaGene Therapeutics has received FDA clearance to move forward with its application for its investigational drug, a CRISPR/Cas13 RNA-editing therapy to treat neovascular age-related macular degeneration (nAMD), dubbed HG202, developed using machine learning and AI.

The company used its HG-PRECISE platform (HuidaGene – Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise) to devise the therapy.

HG-PRECISE allows for the quick discovery of Cas proteins using AI and machine learning in DNA sequencing. It also provides assembly predictions from a metagenomic database. 

The company will move forward with a phase one study to evaluate HG202's effectiveness at different doses in patients with nAMD. The company says the BRIGHT trial will begin enrolling patients soon. 

"Our AI/ML-driven HG-PRECISE platform led us to discover the Cas13X/Y system," Hui Yang, cofounder and chief scientific advisor of HuidaGene, said in a statement. 

"Building on this basis, my team engineered high-fidelity Cas13Y with efficient editing and low off-target effects, laying out the technical foundation for future clinical applications."

THE LARGER TREND

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, developed more than a decade ago, allows scientists to modify DNA sequences of living organisms.

Potential applications range from treatments for genetic disorders to researching disease mechanisms.

Gene editing technologies, such as SHERLOCK and DETECTR, are transforming digital diagnostics, enabling rapid detection of infectious diseases like COVID-19.

Still, ethical, legal and scientific factors warrant consideration, and instabilities can happen that can be fatal for someone, according to experts. Gene editing can cause irreversible changes, and changes to a DNA sequence can last for generations. 

Another company working with CRISPR technology is AI-enabled protein design company Profluent, which has leveraged AI to design an open-source gene editor called OpenCRISPR-1, demonstrating the technology can be used to create molecules with the power to edit human DNA.