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Advancements in Retina and Vitreous Treatments Highlighted in Recent Studies and Collaborations

Recent developments in the treatment of retinal diseases include strategic collaborations, clinical trials, and studies highlighting advancements in personalized treatment and the increasing prevalence of retinopathy of prematurity.

Strategic Collaboration for Geographic Atrophy Treatment City Therapeutics and Bausch + Lomb have announced a strategic collaboration aimed at developing an RNA interference-based treatment for geographic atrophy. This partnership underscores the ongoing efforts to address complex retinal diseases through innovative therapeutic approaches.
Home OCT's Role in Personalized Retinal Disease Treatment Dr. Majda Hadziahmetovic from Duke University discussed the growing importance of Home OCT in providing precise personalization in the treatment of retinal diseases. This technology represents a significant step forward in tailoring treatments to individual patient needs.
Clinical Trials for X-linked Retinoschisis Atsena Therapeutics has initiated part B of the phase 1/2 LIGHTHOUSE study, focusing on the treatment of X-linked retinoschisis with ATSN-201. This study is part of the broader effort to find effective treatments for rare retinal conditions.
Increase in Retinopathy of Prematurity Cases A 2023 study analyzing over 23 million births revealed that the proportion of premature infants diagnosed with retinopathy of prematurity nearly doubled in the U.S. from 2003 to 2019. This alarming trend highlights the need for continued research and intervention strategies.
Rare Pediatric Disease Designation for Retinitis Pigmentosa Treatment The FDA has granted rare pediatric disease designation to VG901 for the treatment of retinitis pigmentosa caused by mutations in the CNGA1 gene. This designation is a critical step in the development of treatments for rare genetic conditions affecting the retina.
Guidelines for Proliferative Diabetic Retinopathy Treatment Dr. Carl D. Regillo from Wills Eye Hospital provided guidelines for the treatment of proliferative diabetic retinopathy, emphasizing the importance of early detection and management to prevent vision loss.
These developments reflect the dynamic nature of retina and vitreous research, with ongoing efforts to improve treatment outcomes for patients with a wide range of retinal diseases.
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Ocugen's OCU400 Shows Promise in Retinitis Pigmentosa Treatment with Positive Clinical Data and Regulatory Milestones

Ocugen's OCU400 gene therapy demonstrated a statistically significant improvement in low-luminance visual acuity in patients with retinitis pigmentosa (RP).
The European Medicines Agency (EMA) has granted OCU400 an Advanced Therapy Medicinal Product (ATMP) classification, expediting its regulatory review process.

Children's Hospital Los Angeles Celebrates Milestone with 100th Retinal Gene Therapy Procedure

Children's Hospital Los Angeles (CHLA) celebrates administering its 100th retinal gene therapy, Luxturna, since 2018 for Leber congenital amaurosis (LCA).
The gene therapy, Luxturna, targets mutations in the _RPE65_ gene, with CHLA utilizing in-house genomic support for precise patient screening.

Mayo Clinic Offers First FDA-Approved Retinal Gene Therapy for Inherited Retinal Disease

Mayo Clinic now provides voretigene neparvovec-rzyl, the first FDA-approved gene therapy for inherited retinal diseases caused by biallelic _RPE65_ mutations.
This one-time treatment has demonstrated the potential to restore the visual cycle and improve functional vision in eligible patients.

Emerging Therapies Offer Hope for Geographic Atrophy Treatment

Several therapies are under evaluation in clinical trials for geographic atrophy (GA) secondary to age-related macular degeneration (AMD), addressing the unmet need for effective treatments.
Complement pathway inhibitors like ANX007, danicopan, and AVD-104 are being investigated to modulate the inflammatory response and cell lysis associated with GA progression.

CRISPR-Cas13bt3 Effectively Silences VEGFA in Retinal Cells, Showing Promise for Gene Therapy

Researchers demonstrated that CRISPR-Cas13bt3, a compact and specific variant, effectively silences VEGFA in retinal cells.
The study highlights the potential of Cas13bt3 for RNA-targeted gene therapy due to its AAV-compatible size.

Ocugen to Highlight Retinal Gene Therapy Progress at Clinical Showcase

Ocugen will present updates from the Phase 3 liMeliGhT trial for retinitis pigmentosa and new data from Phase 1/2 trials on November 12, 2024.
Preliminary safety and efficacy data from the Phase 1/2 ArMaDa clinical trial for geographic atrophy will be shared at the event.

LENZ Therapeutics' LNZ100 NDA Accepted by FDA for Presbyopia Treatment

The FDA has accepted the New Drug Application (NDA) for LENZ Therapeutics' LNZ100, a potential treatment for presbyopia.
The NDA is supported by Phase 3 CLARITY study data, which demonstrated significant near vision improvement in participants.

CRISPR-Cas9 Reduces VEGF Levels in Monkeys, Shows Retinal Toxicity at High Doses

CRISPR-Cas9 targeting VEGFA delivered via AAV vectors reduces VEGF levels in monkeys, potentially treating choroidal neovascularisation (CNV).
Low AAV doses (6 x 1011 vg/eye) led to a 33% reduction in VEGF protein concentrations and decreased CNV severity.

Advancements in Ocular Therapies: Updates on Dry Eye, AMD, and Myopia Management

Aldeyra Therapeutics resubmitted its NDA for reproxalap, targeting dry eye disease symptoms with both acute and chronic activity demonstrated in trials.
Aviceda Therapeutics completed enrollment for its Phase 2b SIGLEC study, evaluating AVD-104 for geographic atrophy secondary to age-related macular degeneration.

FDA Grants Fast Track Status to Treg Therapy for Multiple Sclerosis and Orphan Drug Designation to Gene Therapy for Retinitis Pigmentosa

Abata Therapeutics' ABA-101, an autologous Treg cell therapy, received FDA Fast Track designation for progressive multiple sclerosis, aiming to reduce inflammation in the central nervous system.
Orgenesis Inc. reported encouraging data from a real-world study of ORG-101, a CD19 CAR-T therapy, showing high complete response rates in both adult and pediatric patients with B-cell ALL.

Reference News

[1]
Retina & Vitreous News
healio.com · Jan 6, 2025

City Therapeutics and Bausch + Lomb collaborate on RNA interference-based treatment for geographic atrophy. Atsena Thera...

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