ZM-02 / Augelux Therapeutics - LARVOL DELTA

Unexpected roles of aromatic ring-cleaving dioxygenases in oxygen-heterocycle degradation: Functional diversity, gene redundancy, and ecological implications. (PubMed, Water Res) - "By integrating affinity predictions with in vivo validation, we demonstrated that classical aromatic ring-cleaving dioxygenases, including the intradiol dioxygenases CatA1/CatA2 from Cupriavidus metallidurans ZM02 and the extradiol dioxygenase MhpB from Escherichia coli BL21, mediate the cellular cleavage of 2-hydroxytetrahydrofuran (2-OH THF), a key intermediate in THF metabolism, to yield 4-hydroxybutyric acid (GHB)...Furthermore, catA genes are frequently redundant in bacteria degrading both O-heterocycles and aromatics, and their active sites are highly conserved, suggesting an evolutionary conserved strategy for coping with mixed pollutants. These findings expand the substrate spectrum of well-known dioxygenases to include O-heterocycles, establish a novel catalytic route for cyclic ether degradation in aquatic systems, and offer new opportunities for bioremediation and enzyme engineering in water treatment."

Journal

Durable 52-Week Visual and Functional Gains Following First-in-human ZM-02 Optogenetic Therapy for Advanced Retinitis Pigmentosa (ASGCT 2026) - P1 | "MLSDT, Multi-luminance Shape Discrimination Test. MLTs, Multi-luminance Tests, including MLMT, MLCBT, and MLSDT."

First-in-human • Metastases • P1 data • Gene Therapies • Genetic Disorders • Inflammation • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • Uveitis

Next-Generation ON-Bipolar-Targeted Optogenetic Therapy Using Enhanced Photosensitive Protein and Promoter Achieves Durable Vision Restoration in Retinal Degeneration (ASGCT 2026) - "This study aimed to determine whether these innovations could provide long- term, safe, and meaningful visual restoration in a severe retinal degeneration model, building on the first-in-human experience of our previous product ZM-02, which has received IIT and IND clearance...(c) Photoresponses of retinal bipolar cells upon exposure to 470 nm, 530 nm, and 590 nm light at different light intensities, scale bar = 20 pA,1s. (d) Statistical analysis of peak photocuurrent in retinal bipolar cells at varied light intensities and wavelengths."

First-in-human • CNS Disorders • Gene Therapies • Infectious Disease • Ophthalmology • PRKCA

Durable 52-Week Visual and Functional Gains Following First-in-human ZM-02 Optogenetic Therapy for Advanced Retinitis Pigmentosa (ARVO 2026) - P1 | "Over 80% of treated subjects experienced meaningful improvements in vision, including better ability to recognize objects, move safely in low light, and even perceive colors. These lasting improvements suggest that ZM-02 may offer a new, long-term hope for people living with advanced RP."

First-in-human • Metastases • P1 data • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Next-Generation ON-Bipolar-Targeted Optogenetic Therapy Using Enhanced Photosensitive Protein and Promoter Achieves Durable Vision Restoration in Retinal Degeneration (ARVO 2026) - P1 | "This next-generation optogenetic therapy delivers durable, cortex-level vision restoration via a clinically practical intravitreal route. Supported by previous IIT and IND clearances for ZM-02, these data establish a powerful foundation for human translation and global clinical expansion of safe, effective gene therapies for end-stage retinal degeneration."

First-in-human • Ophthalmology • Retinal Disorders

PRISM: Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection) (clinicaltrials.gov) - P1/2 | N=21 | Not yet recruiting | Sponsor: Zhongmou Therapeutics | Initiation date: Feb 2026 ➔ Jun 2026

Trial initiation date • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

PRISM: Prospective, Randomized, Sham-controlled, Dose-finding I/II Trial of Safety and Efficacy of Modified Optogenetic Gene Therapy (ZM-02 Injection) (clinicaltrials.gov) - P1/2 | N=21 | Not yet recruiting | Sponsor: Zhongmou Therapeutics

New P1/2 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of ZM-02 Optogenetic Gene Therapy Clinical Study for Advanced Retinitis Pigmentosa (ASGCT 2025) - P1 | "This trial indicated that ZM-02 has a notable safety and tolerability profile with no drug-related AEs. Efficacy results showed significant BCVA improvement, supported by MLTs, with an unexpected bonus in color perception. These findings suggest that ZM-02 holds promise in advanced RP treatment."

Clinical • Gene therapy • Metastases • CNS Disorders • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy of ZM-02 Optogenetic Gene Therapy Clinical Study for Advanced Retinitis Pigmentosa (ARVO 2025) - P1 | "Surprisingly, many treated patients also reported improvements in color vision. These results suggest that ZM-02 could be a new way to help people with advanced RP regain some of their vision and improve their quality of life."

Clinical • Gene therapy • Metastases • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Zhongmou Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Zhongmou Therapeutics

Gene therapy • New P1 trial • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa