From the editors of Review of Ophthalmology and Retina Specialist
THE LATEST PUBLISHED RESEARCH
WELCOME to Review of Ophthalmology's Retina Online newsletter. Each month, Medical Editor Philip Rosenfeld, MD, PhD, and our editors provide you with this timely and easily accessible report to keep you up to date on important information affecting the care of patients with vitreoretinal disease.
Single-injection Response to Anti-VEGF Agents in Patients with Wet AMD
Researchers determined the incidence of complete resolution of choroidal neovascular membrane-associated exudation with a single anti-vascular endothelial growth factor injection in treatment-naïve wet age-related macular degeneration patients and its associated characteristics.
The retrospective, observational study included treatment-naïve wet AMD patients who received anti-VEGF therapy with ranibizumab/aflibercept and demonstrated complete resolution of retinal exudation with a single injection.
Complete resolution was defined as total disappearance of the intraretinal fluid, cysts and subretinal fluid, and a return of retinal thickness to <250 µm on spectral-domain optical coherence tomography. All relevant data was collected. Follow up was scheduled on days one, seven and 30 postoperatively, and then monthly, with at least nine mandatory visits per year if the macula remained fluid free. Appropriate statistical analyses were performed.
A total of 63 patients (29 males; mean age 67.25 ±4.40 years) were identified. Here were some of the findings:
- The mean baseline was 20/160 and final corrected distance visual acuity (CDVA) was 20/45.
- Patients completed a mean of 10.9 follow-up visits per year.
- Smaller CNVs (<200 µm), early presentation, better presenting CDVA, subfoveal CNVs, absence of blood/fibrosis and use of aflibercept (2 mg) favored resolution with one injection.
Researchers concluded that a subset (13.76 percent; 63/458, CI, 10.73 to 17.25) of patients with treatment-naïve wet AMD demonstrated resolution of CNV-associated exudation with a single anti-VEGF injection, sustained over two years or more. They noted that this strategy may reduce therapy costs, the number of treatments, the frequency of office visits and the potential risk of geographic atrophy.
SOURCE: Bilgic A, Kodjikian L, Mathis T, et al. Single injection response to anti-vascular endothelial growth factor agents in patients with wet age related macular degeneration: Incidence and characteristics. Retina 2021; Jan 4. [Epub ahead of print].
Extended Dosing Under Anti-VEGF Treatment of Exudative AMD
Researchers assessed disease stability (absence of intraretinal and/or subretinal fluid) and the percentage of eyes able to extend treatment intervals to ≥12 weeks in exudative age-related macular degeneration.
The authors performed a systematic literature search to identify clinical studies reporting treatment outcomes for ranibizumab, aflibercept and brolucizumab in exudative AMD under a treat-and-extend protocol with a follow-up of ≥12 months. Weighted mean differences and subgroup comparisons were used to integrate the different studies.
This meta-analysis of 29 published series included 27 independent samples and 5,629 patients. Here were some of the findings:
• In the pooled group, disease stability was reported in 62.9 percent and 56 percent, after 12 and 24 months of treatment, respectively.
• In the pooled group, treatment intervals were extended to ≥12 weeks in 37.7 percent and 42.6 percent, after 12 and 24 months of treatment, respectively.
• Ranibizumab, aflibercept and brolucizumab differed regarding their potential to achieve disease stability over 12 and 24 months, respectively:
o (after 12 months) 56.3 percent, 64.5 percent and 71.5 percent; and
o (after 24 months) 50 percent, 52.7 percent and 75.7 percent; (p=<0.001)
• Ranibizumab, aflibercept and brolucizumab differed regarding their potential to achieve disease stability after an interval extension to ≥12 weeks over 12 and 24 months, respectively:
o (after 12 months) 28.6 percent, 34.2 percent and 53.3 percent; and
o (after 24 months) 34.2 percent, 47.7 percent, and 41.7 percent; (p=< 0.001).
Researchers concluded that the percentage of eyes achieving disease stability regressed with two of three treatments in the second year, while the percentage of eyes receiving a ≥12-week interval increased disease stability in two of three treatments. They added that the discrepancy may reflect challenges in balancing treatment burden and undertreatment.
SOURCE: Garweg JG, Gerhardt C. Disease stability and extended dosing under anti-VEGF treatment of exudative age-related macular degeneration (AMD) — a meta-analysis. Graefes Arch Clin Exp Ophthalmol 2021; Feb 2. [Epub ahead of print].
Anti-VEGF Dosing and Expected Acuity Outcome at One Year
Investigators determined dose-response characteristics of the anti-vascular endothelial growth factor agents ranibizumab and aflibercept in neovascular age-related macular degeneration using published randomized trials and observational series.
They conducted a literature review of published series from 2006 to 2018, as determined from electronic searches of PubMed and the Cochrane Library. Data extracted included treatment strategy, frequency and first-year visual acuity response. Monthly or bimonthly treatment schedules were classified as “fixed”; pro re nata studies were noted as PRN; treat-and-extend cases were classified as TE; and when no strategy was listed, the studies were noted as “variable.”
Of 2,062 citations, 96 eligible studies provided 120 data points of dose frequency vs. visual acuity change in year one of treatment. Here were some of the findings:
• The dose response curve was nonlinear, but a log transform of the number of injections per year yielded a linear relationship defined by the expression, letters of improvement= -6.66 + 15.7*log (Number of Injections Year 1).
• After accounting for the number of injections, neither the drug used (ranibizumab or aflibercept) nor the strategy employed (fixed, PRN, TE or variable) was a significant predictor of acuity change.
• Studies using the PRN approach had the lowest number of injections and the worst acuity improvements as a treatment strategy.
Investigators wrote that there appeared to be a predictable, mathematically defined relationship between dose frequency and visual acuity change at one year in nAMD. They added that the performance of current treatment efforts, as suggested by reported series and Medicare claims data, appeared to be substandard.
SOURCE: Spaide RF. Anti-vascular endothelial growth factor dosing and expected acuity outcome at 1 year. Retina 2021; Jan 11. [Epub ahead of print].
Predicting Incremental and Future Visual Change in nAMD Using Deep Learning
Investigators evaluated the predictive utility of quantitative imaging biomarkers, acquired automatically from optical coherence tomography scans of cross-sectional and future visual outcomes of patients with neovascular age-related macular degeneration starting anti-vascular endothelial growth factor therapy.
The retrospective cohort study included treatment-naïve, first-treated eyes of patients with neovascular AMD between 2007 and 2017 at Moorfields Eye Hospital undergoing anti-VEGF therapy.
Investigators used automatic segmentation and applied a deep learning segmentation algorithm to 137,379 OCT scans from 6,467 eyes of 3,261 patients with neovascular AMD. After applying selection criteria, 926 eyes of 926 patients were analyzed.
Main outcome measures included correlation coefficients (R2) and mean absolute error (MAE) between quantitative OCT (qOCT) parameters and cross-sectional visual-function. Investigators determined predictive value of these parameters for short-term visual change, i.e. incremental visual acuity resulting from an individual injection, as well as VA at distant timepoints (up to 12 months post-baseline).
Here were some of the findings:
- VA at distant timepoints could be predicted: R2 0.80 (MAE 5.0 ETDRS letters) and R2 0.7 (MAE 7.2) post-injection three and at 12 months post-baseline (both p<0.001), respectively.
- Best performing models included both baseline qOCT parameters and treatment-response.
- The incremental change in VA from an injection could be predicted: R2 0.14 (MAE 5.6) for injection two and R2 0.11 (MAE 5.0) for injection three (both p<0.001).
Investigators reported that automatic segmentation enabled rapid acquisition of quantitative and reproducible OCT biomarkers, with information to potentially inform treatment decisions in the care of neovascular AMD.
SOURCE: Fu DJ, Faes L, Wagner SK, et al. Predicting incremental and future visual change in neovascular age-related macular degeneration using deep learning. Ophthalmol Retina 2021; Jan 28. [Epub ahead of print].
Comparing the Effects of Anti-VEGF Treatments on PED in AMD
The aim of this study was to compare structural and visual outcomes of treatment-naive neovascular age-related macular degeneration patients with significant pigment epithelial detachment, treated with ranibizumab and aflibercept.
This retrospective case series included 33 treatment-naive patients treated with ranibizumab and 25 treated with aflibercept. The patients were followed with pro re nata after first three intravitreal injections. LogMAR visual acuity, PED height and radius on spectral-domain optical coherence tomography findings were compared.
Here were some of the findings:
• For ranibizumab and aflibercept groups, respectively:
o baseline mean PED height was 270.39 ±114.14 µm and 315.24 ±115.8 µm (p=0.14).
o baseline mean PED radius was 2,063.64 ±942.75 µm and 1,958.88 ±452.22 µm (p=0.61); and
o baseline BCVA was 1.16 ±0.73 and 1.09 ±0.69 (p=0.73).
• In the aflibercept group, a statistically significant decrease was found in PED height at the first, third and 12th months.
• The decrease in PED radius was greater in the aflibercept group, though not statistically significantly.
• Visual acuity was better in the aflibercept group at all three months, though not statistically significantly.
Researchers found the maximum improvement was seen at the third month, and final visual acuity and parameters of PED were better in the aflibercept group. They added that both drugs had efficacy for choroidal neovascularization, but suggested that, in cases with significant PED, aflibercept could be considered first-line treatment.
SOURCE: Ulusoy MO, Kal A, Yilmaz G. Comparison of the effects of anti-vascular endothelial growth factor treatments on pigment epithelial detachment in age-related macular degeneration. Int Ophthalmol 2021; Jan. 22. [Epub ahead of print].
nAMD Treatment Burden Among Different Types of Neovascularization
Researchers evaluated differences in the treatment burden among different types of neovascular age-related macular degeneration.
This retrospective, observational study included 431 patients diagnosed with neovascular AMD. Patients were divided into three groups: type 1 or 2 neovascularization group (n=167); type 3 neovascularization group (n=50); and polypoidal choroidal vasculopathy (PCV) group (n=214). The number of hospital visits per year and the number of anti-vascular endothelial growth factor injections per year were compared among the groups. Furthermore, the incidence of bilateral involvement during follow-up was compared among the groups.
The mean follow-up period was 50.6 ±11.3 months. Here were some of the findings:
• The number of hospital visits per year was significantly higher in the type 1 or 2 neovascularization group (mean: 6.1 ±1.5) and type 3 neovascularization (6.6 ±1.6) than in the PCV group (6 ±1.5) (p<0.001).
• The number of anti-VEGF injections per year was significantly higher in type 3 neovascularization group (3.1 ± 1.7) than in the type 1 or 2 neovascularization group (2.3 ± 1.5) or the PCV group (2.3 ±1.2)(p=0.042).
• A significant difference was found in the incidence of bilateral involvement among patients in type 1 or 2 neovascularization group (20.4 percent); type 3 neovascularization group (46 percent); and the PCV group (15.4 percent) (p<0.001).
Researchers found that the high frequency of hospital visits and high number of anti-VEGF injections in patients with type 3 neovascularization suggested a high treatment burden in these patients. They added that the high incidence of bilateral involvement could be a primary reason for the greater treatment burden in patients with type 3 neovascularization.
SOURCE: Lee JH, Kim JH, Kim JW, et al. Difference in treatment burden of neovascular age-related macular degeneration among different types of neovascularization. Graefes Arch Clin Exp Ophthalmol 2020; Jan 6 [Epub ahead of print].
Ranibizumab for nAMD With or Without PCV: DRAGON Study
Investigators evaluated the efficacy and safety of monthly and pro re nata (guided by visual acuity stabilization and disease activity criteria) ranibizumab regimens in Chinese patients with neovascular age-related macular degeneration and polypoidal choroidal vasculopathy.
This double-masked study randomized nAMD patients (1:1) to ranibizumab monthly from baseline to month (M) 11 to a PRN regimen from M12 to M23 (monthly group, n=167) vs. ranibizumab, three monthly doses followed by a PRN regimen up to M23 (PRN group, n=166). Subgroups were assessed based on the presence/absence of PCV (indicated by indocyanine green angiography).
Of 334 randomized patients, 41.7 percent had PCV at baseline. Here were some of the findings:
• Mean average best-corrected visual acuity change from M3 to M4 through M12 was 3.3 letters with monthly, and 1.7 letters with PRN (mean difference: 1.6; CI: -2.95, -0.20, primary endpoint).
• Mean change in BCVA from baseline (monthly/PRN, 53.8/53.7) to M12 and M24 was 12.3 and 11.3 letters, respectively, in the monthly group; and 9.6 and 9.3 letters, respectively, in the PRN group.
• Corresponding values for patients with PCV/without PCV were 12.7/12.1 letters (M12) and 12.3/10.6 letters (M24) in the monthly group; and 9.4/9.4 letters (M12) and 9.7/8.7 letters (M24) in the PRN group.
• The mean number of injections was 11.4 (monthly) and 8.2 (PRN) from day 1 to M11 and 4.8 (monthly), and 5 (PRN) from M12 to M23.
• No new safety findings were reported.
Investigators wrote that the study results supported the use of ranibizumab monthly or PRN in Chinese patients with nAMD, regardless of presence of PCV.
SOURCE: Li X, Zhu Q, Egger A, et al. Two different treatment regimens of ranibizumab 0.5 mg for neovascular age-related macular degeneration with or without polypoidal choroidal vasculopathy in Chinese patients: Results from the Phase IV, randomized, DRAGON study. Acta Ophthalmol 2020; Dec 30. [Epub ahead of print].
Fundus Autofluorescence in nAMD & Clinicopathologic Correlation to MA
Investigators wrote that macular atrophy of retinal pigment epithelium and photoreceptors leads to vision loss in neovascular age-related macular degeneration despite successful treatment with anti-angiogenic agents. To better understand macular atrophy, confirm the cellular basis of fundus autofluorescence (FAF) imaging and inform management of nAMD, they performed a histological analysis of an eye with multimodal clinical imaging and apparent prior exudation due to nAMD.
The case study and clinicopathologic correlation involved a White woman in whom AMD findings of inactive subretinal fibrosis (right eye) were followed for nine years, using FAF and optical coherence tomography. The woman had no detectable subretinal fluid or other recurrent exudation, and no intravitreal injections before her death at age 90 years. The right eye was preserved 6.25 hours after death, post-fixed in osmium tannic acid paraphenylenediamine and prepared for submicrometer epoxy resin sections (n=115), with 19 matched to clinical OCT B-scans.
Main outcome measures included light microscopic morphology of a hyperautofluorescent (hyperFAF) area attributed to prior exudation (“floodplain” hyperFAF), hypoautofluorescent (hypoFAF) spots of MA and areas of unremarkable FAF.
Here were some of the findings:
• Floodplain hyperFAF was visible throughout the nine-year follow-up, with several hypoFAF atrophic spots expanding within it over time.
• The hyperFAF pattern corresponded to outer retinal atrophy (ORA) on OCT and photoreceptor loss over dysmorphic, yet continuous, RPE in histology. The hypoFAF spots inside the floodplain corresponded with complete retinal pigment epithelium and outer retinal atrophy (cRORA) on OCT, and loss of both photoreceptors and RPE in histology.
• In contrast, areas of unremarkable FAF showed continuous RPE accompanied by full-length photoreceptors and a thick outer nuclear layer.
Investigators wrote that FAF imaging involved optical signal modulation by photoreceptors as well as emission signal sources in RPE. They found that HyperFAF attributed to an exudative floodplain signaled loss of photoreceptors over continuous RPE, and in MA signaled loss of both cell layers. Investigators suggested that FAF imaging should be interpreted with the multilayer perspective provided by OCT for maximal value. Finally, they added, prevention of exudation in nAMD may preserve photoreceptors.
SOURCE: Chen L, Messinger JD, Ferrara D, et al. Fundus autofluorescence in neovascular age-related macular degeneration, a clinicopathologic correlation relevant to macular atrophy. Ophthalmol Retina 2021; Feb 1. [Epub ahead of print].
Recurrence of ME in BRVO Following Discontinuation of Anti-VEGF
Researchers identified factors predicting the recurrence of macular edema following the discontinuation of intravitreal anti-vascular endothelial growth factor injection in patients with branch retinal vein occlusion.
This retrospective study included subjects who had discontinued injections at three months after the final bevacizumab injection due to fully resolved macular edema. Fifty-two eyes meeting the criteria were included and divided into two groups (“Recurrence” and “No Recurrence”). Researchers analyzed clinical features and measurements of retinal thickness at the time of the diagnosis and when the decision to stop injections was made (stopping point).
Here were some of the findings:
• At the stopping point, the No Recurrence group showed a thinner parafoveal inner retina, better best-corrected visual acuity and lower incidence of ellipsoid zone disruption in multivariate logistic regression analysis (all p<0.05).
• Parafoveal inner retinal thinning of more than 30 μm, when compared with the corresponding region of the fellow eye or the unaffected region of the affected eye, was significantly related to less recurrence of macular edema.
Researchers determined that thinning of the parafoveal inner retina, as well as better vision and intact outer retinal layers were associated with a lack of recurrence of macular edema. They added that the findings suggested that inner retinal atrophy after BRVO may result in a reduction in oxygen demand in the affected retinal tissue and less production of VEGF.
SOURCE: Lee GW, Kang SW, Kang MC, et al. Associations with recurrence of macular edema in branch retinal vein occlusion following the discontinuation of anti-vascular endothelial growth factor. Retina 2021; Jan 13 [Epub ahead of print].
Retinal Venous Pressure After Anti-VEGF Therapy in RVO-Related Macular Edema
Investigators wrote that, while the pathogenesis leading to retinal vein occlusion is unclear, mechanical compression, thrombosis and functional contractions of veins are discussed as reasons for the increased resistance of venous outflow. Investigators evaluated changes in retinal venous pressure following intravitreal injection of an anti-vascular endothelial growth factor agent to determine the effect on RVO-related macular edema.
Twenty-six patients with RVO-related macular edema (16 branch RVOs and 10 central RVOs, ages 72.5 ±8.8 years) who visited the investigators’ hospital were included in the prospective study. Visual acuity, intraocular pressure, central retinal thickness determined by macular optical coherence tomography, and RVP measured using an ophthalmodynamometer were obtained before intravitreal injection of ranibizumab and one month later.
Comparison of BRVOs and CRVOs showed that acuity was significantly improved by a single injection in BRVOs (p<0.0001; p=0.1087 for CRVOs), but CRT and RVP were significantly decreased without significant difference in IOP after treatment in both groups (p<0.0001).
Investigators wrote that anti-VEGF treatment resulted in a significant decrease in RVP, although RVP remained significantly higher than IOP. They added that an increased RVP plays a role in the formation of macula edema, so reducing it is desirable.
SOURCE: Kida T, Flammer J, Konieczka K, et al. Retinal venous pressure is decreased after anti-VEGF therapy in patients with retinal vein occlusion-related macular edema. Graefes Arch Clin Exp Ophthalmol 2021; Jan 15. [Epub ahead of print].
Detection of DR from UWF-SLO Images
Scientists aimed to develop a deep-learning system that can detect referable and vision-threatening diabetic retinopathy (RDR and VTDR) from images obtained on ultra-wide field scanning laser ophthalmoscope (UWF-SLO), as part of an observational, cross-sectional study.
A total of 9,392 UWF-SLO images of 1,903 eyes from 1,022 subjects with diabetes from Hong Kong, the U.K., India and Argentina were included.
All images were labeled according to the presence or absence of RDR and the presence or absence of VTDR. Labeling was performed by retina specialists from fundus examination according to the International Clinical Diabetic Retinopathy Disease Severity Scale. Three convolutional neural networks (ResNet50) were trained with transfer-learning procedures for assessing gradeability, and identifying VTDR and RDR. External validation was performed on four datasets spanning different geographical regions.
Main outcome measures included:
- area under the receiver operating characteristic curve (AUROC);
- area under the precision-recall curves (AUPRC);
- specificity; and
- accuracy of the DL system in gradeability assessment, and detection of RDR and VTDR.
Here were some of the findings:
- For gradeability assessment, the system achieved, for the primary validation dataset:
- an AUROC of 0.923 (CI, 0.892 to 0.947);
- sensitivity of 86.5 percent (CI, 77.6 to 92.8); and
- specificity of 82.1 percent (CI, 77.3 to 86.2)
- For gradeability assessment, the system achieved, for the geographical external validation datasets:
- >0.82 AUROCs:
- >79.6-percent sensitivity; and
- >70.4-percent specificity.
- For detecting RDR and VTDR for the primary validation dataset:
- the AUROCs were: 0.981 (CI, 0.977 to 0.984) and 0.966 (CI, 0.961 to 0.971), with:
- sensitivities of 94.9 percent (CI, 92.3 to 97.9) and 87.2 percent (CI, 81.5 to 91.6);
- specificities of 95.1 percent (CI, 90.6 to 97.9) and 95.8 percent (CI, 93.3 to 97.6); and
- positive predictive values (PPVs) of 98 percent (CI, 96.1 to 99.0) and 91.1 percent (CI, 86.3 to 94.3).
- The AUROCs and accuracies for detecting RDR and VTDR were >0.9 and >80 percent for the geographical external validation datasets.
- The AUPRCs were >0.9, and sensitivities, specificities and PPVs were >80 percent for the geographical external validation datasets for RDR and VTDR detection.
Scientists wrote that the “excellent performance” achieved with the DL system for automated image quality assessment and detection of RDR and VTDR in UWF-SLO images highlighted its potential as an efficient and effective diabetic retinopathy screening tool.
SOURCE: Tang F, Luenam P, Ran Ran A, et al. Detection of diabetic retinopathy from ultra-wide field scanning laser ophthalmoscope images: A multi-center deep-learning analysis. Ophthalmol Retina 2021; Jan 31. [Epub ahead of print].
Genentech’s Faricimab Meets Endpoints in Multiple Trials
Genentech announced topline results from two identically designed global Phase III studies, TENAYA and LUCERNE, evaluating its investigational bispecific antibody, faricimab, in people with neovascular age-related macular degeneration. Both studies met their primary endpoint and showed that people receiving faricimab injections at fixed intervals of up to every 16 weeks achieved visual acuity outcomes that were non-inferior to those receiving aflibercept injections every eight weeks, the company says. Nearly half (45 percent) of people in both studies were treated with faricimab every 16 weeks during the first year. Read more.
SOURCE: Genentech, January 2021
Adverum Announces Publication of Preclinical Safety Data on ADVM-022
Adverum Biotechnologies announced the publication of preclinical data on ADVM-022 intravitreal gene therapy in Translational Vision Science & Technology. One finding included that a single IVT injection of ADVM-022 (2 x 1012 vg/eye in this study) appeared to be safe and well-tolerated, and resulted in sustained expression of aflibercept with no detectable adverse effects on normal retinal structure or function measured out to 30 months. ADVM-022 is in clinical trials for wet age-related macular degeneration and diabetic macular edema. Read more.
SOURCE: Adverum Biotechnologies, February 2021
Gemini Initiates GEM103 Phase IIA Study
Gemini Therapeutics announced the start of a Phase IIa trial advancing GEM103 as a potential add-on therapy for patients with wet age-related macular degeneration who have, or may be at risk for, macular atrophy but require ongoing anti-vascular endothelial growth factor treatment. Topline data related to safety, tolerability, effect on intraocular CFH levels and biomarkers is expected in the second half of 2021. Read more.
SOURCE: Gemini Therapeutics, February 2021
Inflammasome Publishes Dry AMD Data
Inflammasome Therapeutics says that data published in Proceedings of the National Academy of Sciences confirms that the company’s proprietary compounds “hold promise for preventing and treating age-related macular degeneration.” The paper reveals that patients taking certain nucleoside reverse transcriptase inhibitors (NRTIs) to prevent HIV infection have a 40 percent lower risk of developing dry AMD compared to matched controls. Researchers at Inflammasome Therapeutics are developing these drugs as treatments for dry AMD. Read more.
SOURCE: Inflammasome Therapeutics, February 2021
MeiraGTx’s AAV-CNGA3 Granted FDA Fast Track Designation for Achromatopsia Treatment
MeiraGTx announced the FDA granted Fast Track designation to its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM), caused by mutations in the CNGA3 gene. AAV-CNGA3 is an investigational gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection. Read more.
SOURCE: MeiraGTx, January 2021
Rezolute Initiates Phase I Study of RZ402 for DME Treatment
Rezolute announced the initiation of dosing in a Phase I first-in-human clinical study of RZ402, an investigational oral plasma kallikrein inhibitor, for the treatment of diabetic macular edema. The single-center, randomized, double-blind, placebo-controlled, single ascending dose study will evaluate the safety, tolerability and pharmacokinetics of RZ402 in healthy adult volunteers. Read more.
SOURCE: Rezolute, January 2021
Nanoscope Receives Orphan Drug Designation for Gene Therapy, Wins Retinal Organoid Challenge From NIH
Nanoscope Therapeutics received FDA Orphan Drug Designation for its gene therapy-based treatment of Stargardt’s disease. The therapy uses light-sensitive molecules to re-sensitize the retina toward ambient light levels. Nanoscope’s pipeline includes optogenetics-based retinal regeneration therapy for vision restoration in patients with retinitis pigmentosa, Stargardt’s disease and dry age-related macular degeneration. Read more.
In addition, company researchers, in collaboration with the University of Colorado and Miami University (Ohio), were awarded top prize from the 3D-ROC challenge from the National Institute of Health, for generating a physiologically relevant three-dimensional retinal organoid. The organoids provide a unique opportunity for evaluating novel gene therapies, including multi-characteristic opsin (MCO) gene therapy for vision restoration. Nanoscope is evaluating the effectiveness of MCO optogenetic therapy, which the company says has shown promise in restoring sight in blind subjects. Read more.
SOURCE: Nanoscope Therapeutics, January, February 2021
Asymmetric Divisions are Crucial to Form a Functional Retina
Researchers at the Instituto Gulbenkian de Ciência and the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, Germany, have discovered that, in the developing retina, the divisions leading to the first differentiating neurons are asymmetric and that this asymmetry is necessary to generate the correct types of neurons in the right numbers and proportions. The study, published in eLife, is the first to report this asymmetry and the molecular processes underlying it, increasing understanding on how the brain develops its architecture and function. Read more.
SOURCE: Instituto Gulbenkian de Ciencia, December 2020
Scientists Take Step Toward Using Retinal Cell Transplants to Treat Blindness
In what’s been touted as an “important advance in the development of cell therapy to treat blindness,” retinal cells derived from a cadaver human eye survived when transplanted into the eyes of primate models, according to a study published on January 14 in Stem Cell Reports
. To restore retinal pigment epithelial cells, the researchers extracted retinal stem cells from donated cadaver adult eyes, which can enable donor compatibility matching and serve as a recurring source of human RPE. The team then assessed the safety and feasibility of implanting adult retinal stem cells into non-human primates, and found that RPE patches transplanted under the macula remained stable and integrated in vivo
for at least three months without serious side effects. The researchers also found that the stem cell-derived RPE at least partially took over the function of the original RPE and was able to support the endogenous photoreceptor. Read more
Source: Mount Sinai, January 2021
US Ophthalmic Introduces Eyer Portable Retinal Camera
US Ophthalmic introduced the Eyer Portable Retinal Camera, which connects to a smartphone for images of the retina. The company says the device can “detect fundus disease at a lower cost than conventional methods.” The device, which can be used for telemedicine exams, illuminates and images the retina. It connects to a smartphone's camera, and an app sends the images over the internet to Eyer Cloud. The device further offers panoramic images of more 110 degrees, iCloud connectivity, telemedicine capability, anterior and posterior segment imaging, and 12 megapixel images, according to the company. Read more.
SOURCE: US Ophthalmic, January 2021
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