Core Technology Platforms

The foundational core of Huida Gene's business model is the discovery of novel, ultra-compact CRISPR systems that solve the delivery limitations of traditional gene editing.

1. HG-PRECISE® Discovery Engine

Standing for "Platform for Rational Engineering of CRISPR-Cas Identification by Synergic Expertise," this system utilizes artificial intelligence and deep machine learning to mine metagenomic databases. It automates DNA sequencing and assembly prediction to identify uncharacterized Cas proteins. The platform rationalizes these enzymes to ensure high on-target precision, a compact molecular footprint, and drastically reduced off-target activity in human cells.

2. CRISPR-Cas12 Systems (DNA Editing)

Huida Gene discovered and engineered the hfCas12Max nuclease system. Traditional Cas9 and Cas12 enzymes are often too large to pack alongside their guide RNAs (gRNAs) into a single delivery vehicle. The hfCas12Max is structurally compact, meaning the entire gene-editing apparatus can be packaged safely into a single adeno-associated virus (AAV) vector, facilitating highly efficient mammalian cell delivery.

3. CRISPR-Cas13 Systems (RNA Editing & Base Editing)

To circumvent the risks of permanent genomic double-strand breaks—such as unintended translocations—Huida Gene pioneered the development of the hfCas13X and hfCas13Y RNA-editing systems. These tools alter the transient RNA message rather than rewriting the permanent DNA sequence. Building on top of this, the company engineered mini-dCas13X-mediated RNA base-editing solutions (such as glycosylase-based guanine base editors) capable of correcting point mutations directly at the transcript level.

4. HG-AAV Vector Engineering Platform

Developing an editing enzyme is ineffective without a targeted method to deliver it to the correct tissue. Huida Gene actively develops novel recombinant Adeno-Associated Virus (AAV) capsids. The company screens and isolates customized capsids engineered with high tissue-tropism, allowing them to selectively cross the blood-brain barrier or infect specific retinal cell layers at a much lower dose, mitigating systemic toxicity.

Therapeutic Pipeline & Products

Huida Gene translates its software and screening platforms into an active drug pipeline geared toward "one-and-done" curative therapeutics.

Ophthalmology

• HG004: A gene-replacement therapy targeting RPE65 mutation-associated inherited retinal dystrophies (blindness). It is designed to deliver a functional wild-type RPE65 gene using a novel AAV vector. Preclinical data showed significant functional recovery at a 10-fold lower vector dose compared to existing treatments. It has achieved multi-national Phase 1/2 clinical milestone dosing and has been granted Orphan Drug (ODD) and Rare Pediatric Disease Designations (RPDD) by the U.S. FDA.
• HG202: A first-in-class CRISPR/Cas13 RNA-editing therapy engineered to treat neovascular age-related macular degeneration (nAMD). It functions by knocking down the vascular endothelial growth factor (VEGF) transcripts to block abnormal blood vessel growth in the eye without altering host DNA permanently.
• HG203 / DNA Editing for adRP: Preclinical optimization programs targeting Rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP) via targeted DNA disruption.

Neurology & Otology

• HG204: An RNA-editing therapeutic leveraging the Cas13Y tool to treat MECP2 Duplication Syndrome (MDS), a severe neurodevelopmental disorder. It has received ODD and RPDD clearances from the U.S. FDA and European Medicines Agency (EMA).
• Congenital Hearing Loss Program: Utilizing the mini-dCas13X RNA base-editing platform to correct OTOF (Otoferlin) point mutations, aiming to restore auditory function in children born with congenital deafness.
• Neurodegenerative Research: Early-stage pipeline solutions utilizing DNA-editing configurations to treat Huntington's Disease (HD) and Amyotrophic Lateral Sclerosis (ALS).

Neuromuscular

• HG302: A DNA-editing therapy centered on the hfCas12Max nuclease system tailored for Duchenne Muscular Dystrophy (DMD). Delivered via a single AAV vector, it targets specific deletions to restore the reading frame of the dystrophin gene. It holds both FDA ODD and RPDD status.

B2B Licensing & Commercial Services

Beyond internal drug development, Huida Gene monetizes its intellectual property platform through corporate out-licensing agreements, granting commercial access to its compact nucleases for research and therapeutic development:

• Synthego Partnership: Huida Gene entered a global licensing agreement with Synthego Corporation, granting them the manufacturing and commercial rights to distribute the hfCas12Max nuclease and optimized gRNA for research use, alongside sublicensing mechanisms for therapeutic developers.
• Kactus Partnership: A strategic licensing partnership with Kactus to scale the production and availability of high-fidelity hfCas12Max reagents.
• NovoCrops Alliance: Extending its CRISPR IP outside of human health, Huida Gene licensed its hfCas12Max system to NovoCrops for use in gene-edited crop development and agricultural engineering.

Infrastructure & Manufacturing

To maintain absolute control over product quality and speed to market, Huida Gene has established dedicated internal technical facilities: * CMC Laboratory Infrastructure: Located in the Shanghai Waigaoqiao Free Trade Zone, this chemistry, manufacturing, and controls (CMC) center manages pilot-scale production of high-titer, clinical-grade AAV vectors. * In-House Animal Facility: A specialized animal testing facility engineered to support non-human primate (NHP) and humanized mouse model studies, accelerating IND-enabling safety, toxicology, and tissue tropism evaluation.