HuidaGene Therapeutics to Attend BIO-Europe Fall 2024 to Expand Global Collaborations in Genome Medicines

SHANGHAI and MIDDLETOWN (DE), October 28, 2024 –HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company pioneering CRISPR-based programmable genome medicines, is excited to announce its participation at BIO-Europe^® 2024 Fall Conference and One-on-One PartneringONE^®. The event will take place from November 4-6, 2024, in Stockholm, Sweden, with digital partnering sessions scheduled from November 12-13.

BIO-Europe^®, a leading life sciences conference, gathers over 4,000 life science professionals from more than 2,200 companies across 60+ countries, aiming to drive biotech innovation and global industry growth. Dr. Alvin Luk, Co-founder and CEO of HuidaGene, along with Ms. Xixi Zhu, Senior Director of International Business Development, will present the company’s cutting-edge gene-editing technology and its innovative gene therapy product pipeline, designed to address unmet medical needs across ophthalmology, neurology, and other therapeutic areas. They will also host one-on-one meetings during the three-day event to explore global collaboration and expand HuidaGene’s international presence.

In 2024, HuidaGene continued its trajectory of innovation and excellence in the field of genome medicines. Key highlights from the year include:

1. Advancement of Retinal Diseases:

• HG004 first-in-human “LIGHT” trial (NCT06088992) demonstrates that a better viral vector of RPE tropism, reduced in the total vector dose and injection volume, can avoid progressive chorioretinal atrophy, as seen in real-world studies of Luxturna.

• Preliminary data from the world’s first RNA-targeting therapy (HG202) in the ‘SIGHT” trial (NCT06031727) shows visual improvement in AMD patients who developed treatment-resistant to anti-VEGF therapies.

2. Orphan Drug and Rare Pediatric Disease Designations Highlight the Potential of the Company’s Innovative Therapies:

• HG302 hfCas12Max/DNA-editing for Duchenne muscular dystrophy (DMD) received orphan drug designation (ODD) from the U.S. FDA.

• HG205 base-editing for OTOF-related hearing loss received ODD and rare pediatric disease designation (RPDD) from the U.S. FDA.

• HG204 Cas13/RNA-editing for MECP2 duplication syndrome received ODD from EMA.

3. Initiation of First-in-Human Trial of HG204 and HG302:

• In August 2024, HuidaGene completed the site initiation visit for Cas12/DNA-editing therapy for DMD (HG302). The “MUSCLE” trial (NCT06594094) of Cas12/DNA-editing is the world’s first clinical trial beyond Cas9.

• In September 2024, HuidaGene completed the site initiation visit for Cas13/RNA-editing therapy for MECP2 duplication syndrome (HG204). The “HERO” trial (NCT06615206) of HG204, which has received ODD from EMA after receiving ODD and RPDD from the FDA in 2023, is the world’s first Cas13 RNA-editing clinical trial for neurodevelopment disease.

• The initiation of these two first-in-human clinical trials marked a groundbreaking advancement in CRISPR-based in-vivo genome medicines beyond Cas9.

4. Preclinical Pipeline in CNS:

• CNS pipeline covering rare (ALS, Huntington, and Angelman syndrome) and common (Alzheimer’s and Parkinson’s) diseases.
  

5. Global Scientific Impact in Innovations and CRISPR-based AI-driven HG-PRECISE^® Platform:

• HuidaGene’s research was showcased through more than 30 oral/poster presentations at prestigious international conferences, including the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, ARVO, ASGCT, ESGCT, and late-breaking abstracts at the EURETINA and World Muscle Society (WMS) Annual Conference.
  

2024 marked another year of breakthroughs for HuidaGene, reinforcing its leadership in gene-editing technology and its commitment to delivering life-changing treatments. We look forward to discussing the Company’s innovation at BIO-Europe^®.

About HuidaGene

HuidaGene utilizes its proprietary CRISPR-based HG-PRECISE^® platform to develop potentially curative genome medicines. The Company is advancing clinical programs, including trials of HG004 (granted ODD & RPDD by FDA) ‘LIGHT’ trial (NCT06088992) and Phase 1/2 international, master-protocol ‘STAR’ clinical trial (NCT05906953) in RPE65-associated retinal disease, HG202 RNA-editing therapy ‘SIGHT-I’ first-in-human trial (NCT06031727) and Phase 1 ‘BRIGHT’ clinical trial (NCT06623279) for nAMD, HG204 RNA-editing therapy (granted ODD & RPDD by FDA and ODD by EMA) ‘HERO’ trial (NCT06615206) for MECP2 duplication syndrome, and HG302 DNA-editing therapy (granted ODD & RPDD by FDA) first-in-human ‘MUSCLE’ trial (NCT06594094) for DMD. The preclinical programs include HG303 DNA-editing for ALS and CRISPR RNA-editing therapy for Alzheimer’s. With an extensive intellectual property portfolio, HuidaGene is a leader in genome medicines for neurology and ophthalmology. Learn more at huidagene.com or on LinkedIn.