Retina Foundation of the Southwest

Dallas, TX, United States

Retina Foundation of the Southwest

Dallas, TX, United States

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BALTIMORE, May 09, 2017 (GLOBE NEWSWIRE) -- Researchers at the Casey Eye Institute, the Retina Foundation of the Southwest, the Kellogg Eye Center and Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, reported results from a study of the natural history of X-linked retinoschisis (XLRS) and the impact of carbonic anhydrase inhibitors (CAIs) on disease progression. The data were presented this week at ARVO 2017, the Association for Research in Vision and Ophthalmology Annual Meeting, taking place in Baltimore from May 7-11. Maria A. Parker, M.D., Senior Project Manager at the Casey Eye Institute at the Oregon Health and Science University, presented the data in a poster titled “Natural History and Effect of Carbonic Anhydrase Inhibitor Use in X-Linked Retinoschisis” (Abstract #1490). XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual function in young boys, which can ultimately result in legal blindness in adult men. The study was designed to characterize the natural history of XLRS and to determine the effect of CAIs on retinal function and structure in XLRS patients. This observational study enrolled 56 patients six years of age and older (average 30.0 years) with a confirmed mutation in the RS1 gene. Of the 56 patients, 18 had no CAI use prior to or during the study (Group A), 18 had previously used CAIs and continued to do so during the study (Group B) and 20 had no history of CAI use but began these medications at the start of the study (Group C). All patients underwent functional [best corrected visual acuity (BCVA)] and structural [macular cystic cavity volume (CCV) calculated from spectral domain optical coherence tomography] evaluations at baseline, 6, 12 and 18 months, and Group C patients underwent additional exams at 1 and 3 months after starting CAI therapy. There were no significant differences in BCVA or CCV within each group at subsequent evaluations compared with baseline values. Comparison of Group C with Groups A and B at each follow up examination also showed no statistically significant differences in BCVA or CCV, although there was a suggestion of improved visual acuity in Group C earlier in the study. Researchers conclude that these results demonstrate that XLRS is stable over an 18-month time period and that topical CAI use was not associated with improvement in visual function or macular cyst volume at one year. However, they also noted that some individuals treated with CAIs demonstrated notable improvements at earlier time points, suggesting that these medications may have more nuanced effects. “There are limited data available on the natural course of XLRS, or the impact of using CAIs on disease progression, likely because XLRS is a rare condition,” said Sue Washer, President and CEO of AGTC. “This lack of information is one hurdle to overcome as we develop new treatment approaches. We believe these study results are an important advance in our understanding of the natural progression of XLRS, and will enhance our efforts to develop our AAV-based XLRS gene therapy candidate, which is currently being evaluated in a Phase 1/2 clinical trial.” AGTC is currently enrolling patients in a clinical trial for its XLRS product candidates, as part of the company's collaboration with Biogen. Patients and caregivers interested in participating in or learning more about this trial may learn more at www.agtc.com/patients-and-caregivers or by contacting advocacy@agtc.com. AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes ophthalmology programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration, and our optogenetics program with Bionic Sight. AGTC's non-ophthalmology programs include its adrenoleukodystrophy program and its otology program, which is in pre-clinical development, and the company expects to advance several otology product candidates into clinical development in the next few years. Each of AGTC's XLRS, XLRP and adrenoleukodystrophy programs is partnered with Biogen. AGTC employs a highly-targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need that it believes are clinically feasible and present commercial opportunities. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products. This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: no gene therapy products have been approved in the United States and only two such products have been approved in Europe; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; uncertainty inherent in the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2016, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.


BALTIMORE, May 09, 2017 (GLOBE NEWSWIRE) -- Researchers at the Casey Eye Institute, the Retina Foundation of the Southwest, the Kellogg Eye Center and Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, reported results from a study of the natural history of X-linked retinoschisis (XLRS) and the impact of carbonic anhydrase inhibitors (CAIs) on disease progression. The data were presented this week at ARVO 2017, the Association for Research in Vision and Ophthalmology Annual Meeting, taking place in Baltimore from May 7-11. Maria A. Parker, M.D., Senior Project Manager at the Casey Eye Institute at the Oregon Health and Science University, presented the data in a poster titled “Natural History and Effect of Carbonic Anhydrase Inhibitor Use in X-Linked Retinoschisis” (Abstract #1490). XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual function in young boys, which can ultimately result in legal blindness in adult men. The study was designed to characterize the natural history of XLRS and to determine the effect of CAIs on retinal function and structure in XLRS patients. This observational study enrolled 56 patients six years of age and older (average 30.0 years) with a confirmed mutation in the RS1 gene. Of the 56 patients, 18 had no CAI use prior to or during the study (Group A), 18 had previously used CAIs and continued to do so during the study (Group B) and 20 had no history of CAI use but began these medications at the start of the study (Group C). All patients underwent functional [best corrected visual acuity (BCVA)] and structural [macular cystic cavity volume (CCV) calculated from spectral domain optical coherence tomography] evaluations at baseline, 6, 12 and 18 months, and Group C patients underwent additional exams at 1 and 3 months after starting CAI therapy. There were no significant differences in BCVA or CCV within each group at subsequent evaluations compared with baseline values. Comparison of Group C with Groups A and B at each follow up examination also showed no statistically significant differences in BCVA or CCV, although there was a suggestion of improved visual acuity in Group C earlier in the study. Researchers conclude that these results demonstrate that XLRS is stable over an 18-month time period and that topical CAI use was not associated with improvement in visual function or macular cyst volume at one year. However, they also noted that some individuals treated with CAIs demonstrated notable improvements at earlier time points, suggesting that these medications may have more nuanced effects. “There are limited data available on the natural course of XLRS, or the impact of using CAIs on disease progression, likely because XLRS is a rare condition,” said Sue Washer, President and CEO of AGTC. “This lack of information is one hurdle to overcome as we develop new treatment approaches. We believe these study results are an important advance in our understanding of the natural progression of XLRS, and will enhance our efforts to develop our AAV-based XLRS gene therapy candidate, which is currently being evaluated in a Phase 1/2 clinical trial.” AGTC is currently enrolling patients in a clinical trial for its XLRS product candidates, as part of the company's collaboration with Biogen. Patients and caregivers interested in participating in or learning more about this trial may learn more at www.agtc.com/patients-and-caregivers or by contacting advocacy@agtc.com. AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes ophthalmology programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration, and our optogenetics program with Bionic Sight. AGTC's non-ophthalmology programs include its adrenoleukodystrophy program and its otology program, which is in pre-clinical development, and the company expects to advance several otology product candidates into clinical development in the next few years. Each of AGTC's XLRS, XLRP and adrenoleukodystrophy programs is partnered with Biogen. AGTC employs a highly-targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need that it believes are clinically feasible and present commercial opportunities. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products. This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: no gene therapy products have been approved in the United States and only two such products have been approved in Europe; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; uncertainty inherent in the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2016, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.


BALTIMORE, May 09, 2017 (GLOBE NEWSWIRE) -- Researchers at the Casey Eye Institute, the Retina Foundation of the Southwest, the Kellogg Eye Center and Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, reported results from a study of the natural history of X-linked retinoschisis (XLRS) and the impact of carbonic anhydrase inhibitors (CAIs) on disease progression. The data were presented this week at ARVO 2017, the Association for Research in Vision and Ophthalmology Annual Meeting, taking place in Baltimore from May 7-11. Maria A. Parker, M.D., Senior Project Manager at the Casey Eye Institute at the Oregon Health and Science University, presented the data in a poster titled “Natural History and Effect of Carbonic Anhydrase Inhibitor Use in X-Linked Retinoschisis” (Abstract #1490). XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual function in young boys, which can ultimately result in legal blindness in adult men. The study was designed to characterize the natural history of XLRS and to determine the effect of CAIs on retinal function and structure in XLRS patients. This observational study enrolled 56 patients six years of age and older (average 30.0 years) with a confirmed mutation in the RS1 gene. Of the 56 patients, 18 had no CAI use prior to or during the study (Group A), 18 had previously used CAIs and continued to do so during the study (Group B) and 20 had no history of CAI use but began these medications at the start of the study (Group C). All patients underwent functional [best corrected visual acuity (BCVA)] and structural [macular cystic cavity volume (CCV) calculated from spectral domain optical coherence tomography] evaluations at baseline, 6, 12 and 18 months, and Group C patients underwent additional exams at 1 and 3 months after starting CAI therapy. There were no significant differences in BCVA or CCV within each group at subsequent evaluations compared with baseline values. Comparison of Group C with Groups A and B at each follow up examination also showed no statistically significant differences in BCVA or CCV, although there was a suggestion of improved visual acuity in Group C earlier in the study. Researchers conclude that these results demonstrate that XLRS is stable over an 18-month time period and that topical CAI use was not associated with improvement in visual function or macular cyst volume at one year. However, they also noted that some individuals treated with CAIs demonstrated notable improvements at earlier time points, suggesting that these medications may have more nuanced effects. “There are limited data available on the natural course of XLRS, or the impact of using CAIs on disease progression, likely because XLRS is a rare condition,” said Sue Washer, President and CEO of AGTC. “This lack of information is one hurdle to overcome as we develop new treatment approaches. We believe these study results are an important advance in our understanding of the natural progression of XLRS, and will enhance our efforts to develop our AAV-based XLRS gene therapy candidate, which is currently being evaluated in a Phase 1/2 clinical trial.” AGTC is currently enrolling patients in a clinical trial for its XLRS product candidates, as part of the company's collaboration with Biogen. Patients and caregivers interested in participating in or learning more about this trial may learn more at www.agtc.com/patients-and-caregivers or by contacting advocacy@agtc.com. AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes ophthalmology programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration, and our optogenetics program with Bionic Sight. AGTC's non-ophthalmology programs include its adrenoleukodystrophy program and its otology program, which is in pre-clinical development, and the company expects to advance several otology product candidates into clinical development in the next few years. Each of AGTC's XLRS, XLRP and adrenoleukodystrophy programs is partnered with Biogen. AGTC employs a highly-targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need that it believes are clinically feasible and present commercial opportunities. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products. This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: no gene therapy products have been approved in the United States and only two such products have been approved in Europe; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; uncertainty inherent in the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2016, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.


BALTIMORE, May 09, 2017 (GLOBE NEWSWIRE) -- Researchers at the Casey Eye Institute, the Retina Foundation of the Southwest, the Kellogg Eye Center and Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, reported results from a study of the natural history of X-linked retinoschisis (XLRS) and the impact of carbonic anhydrase inhibitors (CAIs) on disease progression. The data were presented this week at ARVO 2017, the Association for Research in Vision and Ophthalmology Annual Meeting, taking place in Baltimore from May 7-11. Maria A. Parker, M.D., Senior Project Manager at the Casey Eye Institute at the Oregon Health and Science University, presented the data in a poster titled “Natural History and Effect of Carbonic Anhydrase Inhibitor Use in X-Linked Retinoschisis” (Abstract #1490). XLRS is characterized by abnormal splitting of the layers of the retina, resulting in poor visual function in young boys, which can ultimately result in legal blindness in adult men. The study was designed to characterize the natural history of XLRS and to determine the effect of CAIs on retinal function and structure in XLRS patients. This observational study enrolled 56 patients six years of age and older (average 30.0 years) with a confirmed mutation in the RS1 gene. Of the 56 patients, 18 had no CAI use prior to or during the study (Group A), 18 had previously used CAIs and continued to do so during the study (Group B) and 20 had no history of CAI use but began these medications at the start of the study (Group C). All patients underwent functional [best corrected visual acuity (BCVA)] and structural [macular cystic cavity volume (CCV) calculated from spectral domain optical coherence tomography] evaluations at baseline, 6, 12 and 18 months, and Group C patients underwent additional exams at 1 and 3 months after starting CAI therapy. There were no significant differences in BCVA or CCV within each group at subsequent evaluations compared with baseline values. Comparison of Group C with Groups A and B at each follow up examination also showed no statistically significant differences in BCVA or CCV, although there was a suggestion of improved visual acuity in Group C earlier in the study. Researchers conclude that these results demonstrate that XLRS is stable over an 18-month time period and that topical CAI use was not associated with improvement in visual function or macular cyst volume at one year. However, they also noted that some individuals treated with CAIs demonstrated notable improvements at earlier time points, suggesting that these medications may have more nuanced effects. “There are limited data available on the natural course of XLRS, or the impact of using CAIs on disease progression, likely because XLRS is a rare condition,” said Sue Washer, President and CEO of AGTC. “This lack of information is one hurdle to overcome as we develop new treatment approaches. We believe these study results are an important advance in our understanding of the natural progression of XLRS, and will enhance our efforts to develop our AAV-based XLRS gene therapy candidate, which is currently being evaluated in a Phase 1/2 clinical trial.” AGTC is currently enrolling patients in a clinical trial for its XLRS product candidates, as part of the company's collaboration with Biogen. Patients and caregivers interested in participating in or learning more about this trial may learn more at www.agtc.com/patients-and-caregivers or by contacting advocacy@agtc.com. AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC's product pipeline includes ophthalmology programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration, and our optogenetics program with Bionic Sight. AGTC's non-ophthalmology programs include its adrenoleukodystrophy program and its otology program, which is in pre-clinical development, and the company expects to advance several otology product candidates into clinical development in the next few years. Each of AGTC's XLRS, XLRP and adrenoleukodystrophy programs is partnered with Biogen. AGTC employs a highly-targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need that it believes are clinically feasible and present commercial opportunities. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products. This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. Forward-looking statements include information concerning possible or assumed future results of operations, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities and the effects of competition. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: no gene therapy products have been approved in the United States and only two such products have been approved in Europe; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; uncertainty inherent in the regulatory review process; risks and uncertainties associated with drug development and commercialization; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended June 30, 2016, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.


Birch D.G.,Retina Foundation of the Southwest | Weleber R.G.,Oregon Health And Science University | Duncan J.L.,University of California at San Francisco | Jaffe G.J.,Duke University | Tao W.,Neurotech
American Journal of Ophthalmology | Year: 2013

Purpose: To evaluate the safety and effect on visual function of ciliary neurotrophic factor delivered via an intraocular encapsulated cell implant for the treatment of retinitis pigmentosa (RP). Design: Ciliary neurotrophic factor for late-stage retinitis pigmentosa study 3 (CNTF3; n = 65) and ciliary neurotrophic factor for early-stage retinitis pigmentosa study 4 (CNTF4; n = 68) were multicenter, sham-controlled dose-ranging studies. Methods: Patients were randomly assigned to receive a high- or low-dose implant in 1 eye and sham surgery in the fellow eye. The primary endpoints were change in best-corrected visual acuity (BCVA) at 12 months for CNTF3 and change in visual field sensitivity at 12 months for CNTF4. Patients had the choice of retaining or removing the implant at 12 months for CNTF3 and 24 months for CNTF4. Results: There were no serious adverse events related to either the encapsulated cell implant or the surgical procedure. In CNTF3, there was no change in acuity in either ciliary neurotrophic factor- or sham-treated eyes at 1 year. In CNTF4, eyes treated with the high-dose implant showed a significant decrease in sensitivity while no change was seen in sham- and low dose-treated eyes at 12 months. The decrease in sensitivity was reversible upon implant removal. In both studies, ciliary neurotrophic factor treatment resulted in a dose-dependent increase in retinal thickness. Conclusions: Long-term intraocular delivery of ciliary neurotrophic factor is achieved by the encapsulated cell implant. Neither study showed therapeutic benefit in the primary outcome variable. © 2013 Elsevier Inc. All rights reserved.


News Article | February 15, 2017
Site: www.technologyreview.com

The companies, GenSight Biologics of Paris and Bionic Sight, a startup out of Weill Cornell Medical College in New York, both say a combination of wearable electronics and gene therapy has a chance to restore vision by re-creating the retina’s ability to sense light. Both companies are aiming to help patients with a degenerative eye disease called retinitis pigmentosa, which destroys light-sensing cells in the retina. If the approach works, it could in theory be used to treat any type of retinal disease that involves the loss of these cells, called photoreceptors. Optogenetics, a form of gene therapy, offers an unconventional but potentially powerful way to bypass damaged photoreceptors. Using the technique, scientists add genetic instructions to a different type of retinal cells, ganglions, so that they become light-sensitive instead. Working with the Institut de la Vision in Paris, GenSight has developed a pair of goggles containing a camera, a mircroprocessor, and a digital micromirror that will convert images the camera captures into bright pulses of red light in order to stimulate the modified cells. When tested in blind monkeys and rats, the technology appeared to restore their ability to see, says GenSight CEO Bernard Gilly, but only a test in human volunteers who are able to describe what they perceive after being treated will be definitive. He expects a human study to start this year. The companies are also closely tracking results from an initial human test of optogenetics carried out last March in Texas. In a trial being led by RetroSense Therapeutics, recently acquired by Allergan, a blind woman became first person to receive an optogenetic treatment to help restore her vision. That study has so far enrolled four patients, according to David Birch of the Retina Foundation of the Southwest, where the trial is taking place. Each patient gets three injections into the eye of an engineered virus carrying a gene from algae, which instructs cells to make the light-sensitive protein. The team hasn’t yet reported its results, so it’s unknown whether the subjects have gotten any of their vision back. The RetroSense study relies on natural light to activate the cells. That could limit the treatment’s effectiveness, because the light-sensing proteins only respond to specific wavelengths of light, and low levels of ambient or natural light may not be bright enough to trigger them. Richard Masland, an ophthalmology professor at Harvard Medical School and a scientific advisor for RetroSense, says that is why companies are looking into goggles or other “light adaptation machinery” as way to beam light of the right wavelengths and intensity into the eye. Also pursuing a combination of goggles and optogenetics is Bionic Sight, a startup founded by Sheila Nirenberg, a neuroscientist at Weill Cornell Medical College. The company said in January that it would partner with the gene-therapy company Applied Genetic Technologies to begin clinical trials by 2018. It’s still unclear what sort of vision will result from stimulating the ganglion cells, as these cells normally act to relay nerve impulses and don’t receive light directly. Nirenberg says her goggles will convert light into a “neural code,” or a pattern of pre-processed pulses, which will look to the ganglion cells as if they are coming from other cells in the retina. Daniel Palanker, an ophthalmology professor and director of the Hansen Experimental Physics Laboratory at Stanford University, is skeptical that Nirenberg’s neural code will help. That’s because there are around 30 types of retinal ganglion cells, some of which respond to light while some respond to motion and some to differences in contrast. No one set of light patterns would be able to communicate with all of them, he says.


News Article | November 11, 2016
Site: news.yahoo.com

Kids with lazy eye — or amblyopia, the medical term for the condition — may improve their vision by playing a specially designed iPad game, a new study finds. What's more, kids in the study who played the game experienced greater improvements in their vision after just two weeks, compared with those who received the standard treatment for lazy eye, in which the child wears a patch over his or her "good" eye. The findings suggest that treatments for lazy eye that require children to use both eyes, as the iPad game does, "may yield faster gains than patching," the researchers, from a nonprofit eye research institute called the Retina Foundation of the Southwest, in Dallas, wrote in the Nov. 10 issue of the journal JAMA Ophthalmology. Children with lazy eye don't see as well with one of their eyes as they do with the other, and this weaker eye may wander from side to side, according to the Mayo Clinic. About 3 percent of U.S. children have a lazy eye. The standard eye-patch treatment helps to stimulate the weaker eye in kids with lazy eye. Although most children have improved vision after this treatment, it doesn't always work, and some kids experience a return of their lazy eye after the treatment, the researchers said. In addition, children with lazy eye may have trouble using both of their eyes together, so treatments that allow them to use both eyes may work better than those that allow them to use only one of their eyes (as is the case with eye patching), the researchers said. In the new study, the researchers randomly assigned 28 children with lazy eye to either wear an eye patch or undergo treatment with the iPad game. In the game, called Dig Rush, children control characters who are mining for gold, and the goal is to return pieces of gold to a cart as fast as possible while avoiding obstacles, such as monsters. [7 Ways to Short-Circuit Kids' Mobile Addiction] To play the game, the children wear special glasses that affect how the game looks to each eye. The stronger eye sees some elements of the game (such as the gold cart) in reduced contrast, while the weaker eye sees other elements (such as the miner characters) in high contrast.. Both eyes see elements of the background (such as rocks) in high contrast. The children in the iPad group played the game for 1 hour a day, five days a week, for two weeks. Those in the eye-patch treatment group wore an eye patch for 2 hours a day, seven days a week, for two weeks. After two weeks, the children in the iPad group were able to read an average of 1.5 more lines on a letter chart than they could before the treatment, while those in the eye-patch group could read 0.7 more lines on the letter chart, on average, than they could before the treatment. About 40 percent of the children in the iPad group reached 20/32 vision or better (which is near-normal vision), compared with just 7 percent in the eye-patch group. After two weeks, the children in the eye-patch group switched over to the iPad group, and all of the children played the game for another two weeks. After four weeks, there was no difference in vision between the two groups, the researchers found. However, it's still not clear whether the vision improvements in the children after they played the game will last over the long term, the researchers said, so future research is needed to look at that question.


News Article | November 10, 2016
Site: www.sciencedaily.com

A special type of iPad game was effective in treating children with amblyopia (lazy eye) and was more effective than the standard treatment of patching, according to a study published online by JAMA Ophthalmology. Amblyopia is the leading cause of monocular visual impairment in children, affecting 3 percent in the United States. Amblyopia has traditionally been viewed as a monocular disorder that can be treated by patching the fellow (opposite) eye to force use of the amblyopic eye, but it does not always restore 20/20 vision or teach the eyes to work together. Because amblyopia arises from binocular discordance, binocular treatments are likely to yield better vision outcomes. However, it is unclear whether binocular treatment is comparable to patching in treating amblyopia. Krista R. Kelly, Ph.D., of the Retina Foundation of the Southwest, Dallas, and colleagues randomly assigned 28 children (average age, 7 years) with amblyopia to binocular game treatment (n = 14) and to patching treatment (n = 14). The action-oriented adventure iPad game required children to wear special glasses that separate game elements seen by each eye so that reduced-contrast elements are seen by the fellow eye, high-contrast elements are seen by the amblyopic eye, and high-contrast background elements are seen by both eyes. For successful game play, both eyes must see their respective game components. Children were asked to play the game at home for 1 hour a day, 5 days a week for 2 weeks (10 hours total). The primary outcome was change in amblyopic eye best-corrected visual acuity (BCVA) at the 2-week visit. The researchers found that at the 2-week visit, improvement in amblyopic eye BCVA was greater with the binocular game compared with patching, with the average visual acuity improvement after binocular treatment being more than double the improvement found with patching, and this was achieved with less than 50 percent treatment time required for patching (10 vs 28 hours assigned treatment). Five of 13 children (39 percent) with binocular treatment reached 20/32 or better visual acuity compared with 1 of 14 children (7 percent) with patching. At 2 weeks, patching children crossed over to binocular game treatment, and all 28 children played the game for another 2 weeks. At the 4-week visit, no group difference was found in BCVA change, with children who crossed over to the binocular games catching up with children treated with binocular games. "We show that in just 2 weeks, visual acuity gain with binocular treatment was half that found with 6 months of patching, suggesting that binocular treatment may yield faster gains than patching. Whether long-term binocular treatment is as effective in remediating amblyopia as patching remains to be investigated," the authors write.


News Article | November 10, 2016
Site: www.eurekalert.org

A special type of iPad game was effective in treating children with amblyopia (lazy eye) and was more effective than the standard treatment of patching, according to a study published online by JAMA Ophthalmology. Amblyopia is the leading cause of monocular visual impairment in children, affecting 3 percent in the United States. Amblyopia has traditionally been viewed as a monocular disorder that can be treated by patching the fellow (opposite) eye to force use of the amblyopic eye, but it does not always restore 20/20 vision or teach the eyes to work together. Because amblyopia arises from binocular discordance, binocular treatments are likely to yield better vision outcomes. However, it is unclear whether binocular treatment is comparable to patching in treating amblyopia. Krista R. Kelly, Ph.D., of the Retina Foundation of the Southwest, Dallas, and colleagues randomly assigned 28 children (average age, 7 years) with amblyopia to binocular game treatment (n = 14) and to patching treatment (n = 14). The action-oriented adventure iPad game required children to wear special glasses that separate game elements seen by each eye so that reduced-contrast elements are seen by the fellow eye, high-contrast elements are seen by the amblyopic eye, and high-contrast background elements are seen by both eyes. For successful game play, both eyes must see their respective game components. Children were asked to play the game at home for 1 hour a day, 5 days a week for 2 weeks (10 hours total). The primary outcome was change in amblyopic eye best-corrected visual acuity (BCVA) at the 2-week visit. The researchers found that at the 2-week visit, improvement in amblyopic eye BCVA was greater with the binocular game compared with patching, with the average visual acuity improvement after binocular treatment being more than double the improvement found with patching, and this was achieved with less than 50 percent treatment time required for patching (10 vs 28 hours assigned treatment). Five of 13 children (39 percent) with binocular treatment reached 20/32 or better visual acuity compared with 1 of 14 children (7 percent) with patching. At 2 weeks, patching children crossed over to binocular game treatment, and all 28 children played the game for another 2 weeks. At the 4-week visit, no group difference was found in BCVA change, with children who crossed over to the binocular games catching up with children treated with binocular games. "We show that in just 2 weeks, visual acuity gain with binocular treatment was half that found with 6 months of patching, suggesting that binocular treatment may yield faster gains than patching. Whether long-term binocular treatment is as effective in remediating amblyopia as patching remains to be investigated," the authors write. Editor's Note: This research is supported by a grant from the Thrasher Research Fund and from the National Eye Institute. This study was conducted at the Retina Foundation of the Southwest. All authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and none were reported.


Birch E.E.,Retina Foundation of the Southwest | Birch E.E.,Southwestern Medical Center
Progress in Retinal and Eye Research | Year: 2013

Amblyopia is the most common cause of monocular visual loss in children, affecting 1.3%-3.6% of children. Current treatments are effective in reducing the visual acuity deficit but many amblyopic individuals are left with residual visual acuity deficits, ocular motor abnormalities, deficient fine motor skills, and risk for recurrent amblyopia. Using a combination of psychophysical, electrophysiological, imaging, risk factor analysis, and fine motor skill assessment, the primary role of binocular dysfunction in the genesis of amblyopia and the constellation of visual and motor deficits that accompany the visual acuity deficit has been identified. These findings motivated us to evaluate a new, binocular approach to amblyopia treatment with the goals of reducing or eliminating residual and recurrent amblyopia and of improving the deficient ocular motor function and fine motor skills that accompany amblyopia. © 2012 Elsevier Ltd.

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