David Hutton

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Dr Mohamed Abou Shousha and Dr Richard Parrish II both of the University of Miami Health System Bascom Palmer Eye Institute, discuss their ARVO 2021 presentation regarding a study analyzing visual field measurements using the 

 Visual Field Multi-platform application downloaded on two different commercially available augmented reality devices.

Note: Dr Shousha is also CEO and founder of Hero

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Dr Mohamed Abou Shousha and Dr Richard Parrish II discuss their ARVO 2021 presentation regarding a study analyzing visual field measurements using the Heru Visual Field Multi-platform application downloaded on two different commercially available augmented reality devices.





Measuring visual fields with cloud-based AI platform

A patient performs a series of tests demonstrating how optogenetic methods can partially restore vision in a blind human patient. (Image courtesy of Institut de la vision)

Clinical trial results announced on Friday show for the first time that optogenetic methods can partially restore vision in a blind human patient. The achievement marks a milestone towards developing mutation-independent therapies for inherited photoreceptor diseases.

Breakthrough study restores partial vision in patient 

 by an international research team led by José-Alain Sahel, MD, and Botond Roska, MD, PhD, including members from the Paris Vision Institut (PVI), The Quinze-Vingts hospital (XV-XX), the University of Pittsburgh, the Institute of Molecular and Clinical Ophthalmology Basel (IOB), StreetLab, and GenSight Biologics.

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“Enabling a patient to regain partial vision by optogenetics could not have happened without the engagement of the patient, the efforts of our multidisciplinary team at the Institut de la Vision and GenSight, and the longstanding collaboration with Botond Roska, which was at the origin and core of this project,” said first and corresponding author Sahel, distinguished professor and chairman of Ophthalmology at the University of Pittsburgh, professor at Sorbonne University and the Quinze-Vingts hospital, and founding director of the Institut de la Vision .

Botond Roska, MD, PhD, founding director at IOB and professor at the University of Basel, noted that the findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible.

Optogenetic therapy stimulates photoreceptors

Optogenetics involves genetically altering cells so that they produce light-sensitive proteins called channelrhodopsins. The technique has a nearly 20-year history in neuroscience, but, so far, clinical benefit of optogenetics had not been demonstrated. A collaboration between the teams of Sahel and Roska culminated in the findings reported today that reflect 13 years of multidisciplinary effort.

The goal of the research is to treat inherited photoreceptor diseases, which are widespread causes of human blindness. Photoreceptors are light-sensing cells in the retina that use proteins called opsins to deliver visual information to the brain via the optic nerve. Photoreceptors progressively degenerate, and then blindness sets in. To restore light-sensing capability, the team uses gene therapy methods to deliver channelrhodopsins into the retina's ganglion cells.

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For the current study, the team delivered the gene coding for a channelrhodopsin called ChrimsonR. This particular protein senses amber light, which is safer for retinal cells than the blue light used for other types of optogenetic research. The team also developed specialized goggles outfitted with a camera that captures and projects visual images onto the retina at amber light wavelengths.

Training with the goggles began nearly five months after the injection, thus giving ChrimsonR expression time to stabilize in ganglion cells. Seven months later, the patient began reporting signs of visual improvement.

Test results showed he could locate, touch and count objects on a white table placed in front of him, but only with the aid of the goggles. Without the goggles, he was unable to perform these exercises. One test involved perceiving, locating, and then touching a large notebook or a smaller staple box. The patient touched the notebook during 36 of 39 separate evaluations (in other words, 92% of the time), but could pick out the smaller staple box only 36% of the time. In a second test, the subject correctly counted glass tumblers on the table 63% of the time.

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During a third test, the subject wore a skull-cap affixed with electrodes that took non-invasive electroencephalography (EEG) readings of his brain activity. A tumbler was alternately taken on or off the table, and the subject had to press a button indicating whether it was present or absent. Importantly, EEG readings showed that correlated changes in activity during this testing were concentrated in the visual cortex.

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The team also trained a software decoder to evaluate the EEG readings. By simply measuring neuronal activity, the decoder could tell with 78% accuracy if the tumbler was present or not in a given trial. This last evaluation, Roska said, helped to confirm that brain activity is indeed related to a visual object, and "therefore that the retina is no longer blind."

“Importantly, blind patients with different kinds of neurodegenerative photoreceptor disease and a functional optic nerve will potentially be eligible for the treatment. However, it will take time until this therapy can be offered to patients,” Sahel said.

Articles

An international research team has shown that optogenetic therapy has helped to partially regain visual function in a patient with retinitis pigmentosa. This is a milestone towards a gene therapy that could restore vision.

Gene therapy helps patient with retinitis pigmentosa achieve partial vision

 announced on Tuesday it has formed Vyluma Inc., which will focus on the development and commercialization of therapies to treat ophthalmic diseases.

Vyluma will operate as a wholly owned subsidiary of Nevakar, a privately held biopharmaceutical company based in Bridgewater, NJ, with a portfolio of products in the ophthalmic and injectable areas.

According to the company, Vyluma will focus on ophthalmic pharmaceutical therapies, with an emphasis on 

. The company noted that there currently are no pharmaceutical treatment options approved for refractive errors such as childhood myopia, presbyopia, night vision disturbance, and hyperopia.

It takes a village to beat visual system diseases in children

Vyluma’s lead product candidate is NVK-002, which is in late Phase III clinical development with readouts anticipated during the fourth quarter of 2022. If approved, it would be the first pharmaceutical therapy to slow the progression of myopia in children. Vyluma's pipeline also includes programs for treatment of 

 myopia, presbyopia, night vision disturbance, acute ocular pain, and hyperopia.

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Navneet Puri, PhD, founder, chairman and CEO of Vyluma, pointed out that Nevakar has gained traction in advancing its product pipeline which includes hospital injectable and ophthalmic products.

“After validation of our ophthalmology franchise by establishing key partnerships with Zhaoke in China and Théa in Europe for NVK002, and early strong clinical data on other ophthalmic programs, it was clear we needed to separate the ophthalmic portfolio from our injectable business to bring increased focus to this area of fundamental human need,” Puri said in a statement.

Exploring possibilities of myopia prevention and control in the US

“With the establishment of Vyluma, our ophthalmic portfolio will have a dedicated team of professionals focused on assuring Vyluma meets its commitment to patients, prescribers, shareholders and other key constituents,” Puri added in the statement.

In addition to Puri, Vyluma's leadership team will include Marshall Woodworth, chief financial officer; Raul A. Trillo, Jr., MD, chief commercial officer; and Houman Hemmati MD, PhD, chief medical officer.

Increased focus on ophthalmic development

Hemmati noted in a statement that the new company has an “opportunity to improve patient care and quality of life developing a pipeline of products focused on the biggest unmet need, myopia, as our leading pipeline candidate, followed by additional indications outside of refractive errors in the cornea and anterior segments.”

The company also noted in the statement that board of directors with a focus on ophthalmology will be appointed. One board member already announced is Tuyen Ong, MD, CEO of Ring Therapeutics and former senior vice president at Biogen, with more than 20 years of clinical and drug development experience in ophthalmology.

Use of virtual reality training in amblyopia

Zhaoke Ophthalmology, based in China, and Laboratories Théa, headquartered in Europe, are key commercial partners for Vyluma. Earlier this year, Nevkar entered into a licensing agreement with Théa for the commercialization of NVK-002.

Nevkar also announced it has formed Nevakar Injectables Inc., also a wholly owned subsidiary of the parent company. The newly formed company has a portfolio of products focused on critical patient care, acute pain management, long acting injectables and hospital infections.

Vyluma Inc. will focus on the development and commercialization of therapies to treat ophthalmic diseases, including paediatric myopia.

Nevakar unveils ophthalmic subsidiary, Vyluma

Dr Michael A. Singer of the Medical Center Ophthalmology Associates in San Antonio, Texas, speaks on the highlights of his presentation focusing on intraocular pressure (IOP) in the PALADIN 24-month data intravitreal steroid (ILUVIEN), presented during ARVO 2021. 

Dr Michael A. Singer speaks on the highlights of his presentation focusing on IOP in the PALADIN 24-month data intravitreal steroid (ILUVIEN).

Author | David Hutton

Articles

Gene therapy helps patient with retinitis pigmentosa achieve partial vision

Nevakar unveils ophthalmic subsidiary, Vyluma

Focusing on 24-month data of the PALADIN study

Measuring visual fields with cloud-based AI platform