Global biotech innovator HuidaGene Therapeutics has received FDA approval to advance with its investigational drug application for HG202, a CRISPR/Cas13 RNA-editing therapy designed to treat neovascular age-related macular degeneration (nAMD). HG202 leverages AI and machine learning to optimize its development process.
Developed on HuidaGene’s proprietary HG-PRECISE platform (Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise), HG202 benefits from advanced computational tools. This platform enables rapid discovery of Cas proteins using AI and machine learning integrated with DNA sequencing data, allowing the company to predict Cas protein assemblies from a metagenomic database swiftly.
The upcoming phase one trial, named BRIGHT, aims to evaluate HG202’s safety and efficacy across varying dosages in nAMD patients, with enrollment expected to commence shortly. Hui Yang, HuidaGene’s co-founder and chief scientific advisor, emphasized, “Our AI/ML-driven HG-PRECISE platform enabled us to identify the Cas13X/Y system. With this foundation, our team engineered Cas13Y for high-precision editing with minimal off-target effects, creating a solid foundation for clinical applications.”
Industry Context
CRISPR technology, first established over a decade ago, has revolutionized genetic research, allowing scientists to precisely modify DNA sequences in living organisms. Applications now extend from genetic disorder treatments to innovations in disease research. The technology has sparked the creation of rapid diagnostics tools, such as SHERLOCK and DETECTR, which have been crucial in identifying infectious diseases like COVID-19 swiftly.
Despite its transformative potential, CRISPR raises ethical and technical concerns. Editing DNA can result in irreversible and multi-generational effects, and some applications may pose unforeseen risks. As gene editing becomes more widespread, balancing innovation with ethical safeguards remains paramount.
HuidaGene joins other leaders in CRISPR advancements, such as Profluent, a company using AI to develop OpenCRISPR-1, an open-source gene editor.
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