Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa
BEDFORD, Texas, July 19, 2021 / Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that the first patient had been dosed in its Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with retinitis pigmentosa (RP).
“I want to congratulate the Nanoscope team, our clinical partners, and patients participating in the trial. The results from our Phase 2b trial may provide the basis for consideration by the FDA for accelerated approval of MCO-010 for the treatment of RP. We look forward to working with the FDA and other regulatory agencies so that we can bring MCO-010 to RP patients,” said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope.
Nanoscope’s optogenetics therapy uses an intravitreally delivered proprietary AAV2 vector to deliver Multi-Characteristic Opsin (MCO) genes into retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in a medical office setting without the need for goggles or other interventions. Based on preliminary evidence from the Company’s Phase 1/2a study, MCO-010 is potentially applicable not only for RP but other degenerative diseases of the eye irrespective of gene mutations. MCO-010 has received orphan drug designations for RP and Stargardt disease from the FDA.
The Phase 2b trial (NCT04945772) will include 27 participants in a randomized, double-blinded, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 or sham to confirm safety, tolerability, and efficacy in improving patients’ vision and visual function. The study is expected to complete enrollment by the end of 2021 with 12-month results available by the end of 2022.
“This is an important milestone for both Nanoscope and the field of ophthalmic research. We remain steadfast in our commitment to bringing a safe and differentiated optogenetic monotherapy to the ophthalmology community,” said Dr. Mohamed Genead, Chief Development and Chief Medical Officer of Nanoscope. “Accordingly, we are enthusiastic about our MCO-010 development program, which we believe holds significant potential for the treatment of a broad range of retinal diseases.”
A completed Phase 1/2a open-label study of MCO-010 in 11 patients with RP demonstrated that the ambient light activatable optogenetic monotherapy was well-tolerated with improved quality of life consistent with significant functional vision and visual function improvement in advanced RP patients.
“We are excited by the huge interest received from the RP patients, caregivers, and clinical community. Our team is working hard to further expand the MCO-platform for other retinal degeneration including Stargardt disease, dry age-related macular degeneration (dAMD), and others,” said Samarendra Mohanty, Ph.D., Nanoscope’s President and Chief Scientific Officer.
About RP
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina. When activated by light, these opsins trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetics-based retinal regeneration therapies for patients with RP, Stargardt disease, and dAMD.
Contact:
Ananta Ayyagari
pr@nanostherapeutics.com
817-857-1186
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Nanoscope Therapeutics Announces FDA Approval of IND for Phase 2b clinical trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa
PRESS RELEASE – BEDFORD, TX (June 23, 2021) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degenerative diseases, today announced that its Investigational New Drug application (IND) is now open for Phase 2b clinical trial of MCO-010, an ambient-light activatable optogenetic monotherapy to restore vision in patients with advanced retinitis pigmentosa (RP).
“Congratulations to the Nanoscope team and I am excited to be part of it. We hope our therapy will restore vision in ambient light without any external device, eliminating risk of photo-toxicity” said Sulagna Bhattacharya, CEO of Nanoscope.
The impressive clinical data presented at the American Academy of Ophthalmology 2020 annual meeting has persisted till the end of Phase1/2a study. “These are major milestones for Nanoscope with potential benefit for patients suffering from retinal degeneration,” said Board Chairman Al Guillem, PhD.
The Phase 2b trial which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients’ vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.
Nanoscope’s gene therapy uses an AAV2 vector to deliver the MCO genes into bipolar retinal cells, where they express polychromatic opsins enabling vision in different color environments. The therapy can be administered in an office setting. Additionally, the therapy is applicable for RP regardless of underlying gene mutations.
A Phase 1/2a open label study of MCO-010 demonstrated the optogenetic monotherapy was safe with improved quality of life consistent with functional vision and visual function in advanced RP patients.
“Our gene therapies reprogram retinal cells and make them photosensitive to restore vision. MCO delivery with proprietary viral vector has allowed MCO-transduction in patients’ retina, confirmed by sustained fluorescence reporter expression,” explained Samarendra Mohanty, PhD, Nanoscope’s President and Chief Scientific Officer.
“We are excited by the guidance received from FDA regarding improvising the characterization of our product, the primary endpoint and potency assays that will hopefully accelerate our clinical program to make the restorative drug available to RP patients,” Dr. Mohanty added.
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About RP
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells in the retina, and when activated by light they trigger the physiological process of vision. RP, linked to over 60 different gene mutations, encompasses a group of rare genetic disorders where photoreceptors degrade gradually, leading to impaired vision and eventual blindness.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company’s pipeline includes optogenetics based retinal regeneration therapy for patients with RP, Stargardt disease, and dry age-related macular degeneration.
Visit our website: nanostherapeutics.com
Contact:
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591
Nanoscope’s Optogenetic Gene Therapy Restores Clinically Meaningful Vision in 11 Patients Blinded by Retinitis Pigmentosa
Press Release – Bedford TX – June 3, 2021
- First reported clinically meaningful functional improvement by optogenetic therapy
- Significant dose-dependent improvement of visual acuity at 16 weeks, previously reported at the American Academy of Ophthalmology 2020 annual meeting, continues through 52 weeks in severe retinitis pigmentosa patients
- Vision restoration in the ambient light environment using optogenetic gene monotherapy without the need of stimulating retinal implants or explants (goggles)
- One therapy for many–The first proof of restoring vision in patients blinded by various single as well as multiple genetic defect(s) in a mutation-independent manner
- Significant improvement in multiple mobility tasks and quality of life measures
- First gene therapy demonstrating gene transduction in patients by fluorescence reporter
- The Company expects to advance the therapy by launching a late-stage Phase 2b trial this summer with gene therapy that delivers Multi-Characteristic Opsin (MCO) to retinal cells
Nanoscope Therapeutics Inc., a clinical-stage biotechnology company that is developing gene therapies for the treatment of retinal diseases, today announced that vision improvements for all evaluated advanced retinitis pigmentosa (RP) patients persisted through one year following a single intravitreal injection in a Phase 1/2a clinical study with MCO.
“We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the US this summer to further validate our gene therapy’s ability to improve clinically meaningful vision in RP patients. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide,” said Nanoscope CEO Sulagna Bhattacharya.
Three patients received low dose (1.75 × 1011 VG per eye) and eight received a high dose of 3.5 × 1011 VG per eye. Florescence imaging of retina revealed successful gene transduction. At completion, 6 out of 7 (86%) high dose MCO-therapy subjects gained >0.3 logMAR (15 letters). Due to COVID-19, one of the high dose subjects was not evaluated after 31 weeks of treatment.
“The safety and efficacy results from the clinical study have demonstrated that the benefit-risk balance is strongly in favor of MCO for the treatment of vision loss due to RP. The patients have improved irrespective of underlying gene mutation(s) that caused the disease,” said Nanoscope CMO and Ophthalmologist Sai Chavala, MD.
All subjects had objective and subjective improvement in functional vision. The shape discrimination accuracy improved to >90% in all subjects compared to baseline. Further, the performance in two different mobility tests improved by 50% reduction in time to touch lighted panel. These test outcomes were highly correlated with improved patient reported outcomes.
“After MCO-treatment, the patients reported long-lasting improvements in outdoor light sensitivity and daily activities. We were pleasantly surprised that after eight weeks of treatment, some subjects could attend their follow-up visits during the study without the assistance of a chaperone. Some of the patients even gained the ability to read letters on a wall or even the large text in a newspaper, use a cell phone, watch television, and could even thread a needle.” said the Principal Investigator Dr. Santosh Mahapatra, Ophthalmologist and Eye Surgeon.
Nanoscope’s RP gene therapy, which has received orphan drug designation from the US Food and Drug Administration, uses a proprietary AAV2 vector to deliver the MCO genes into the retina. This mutation-independent gene therapy involves a single injection through the eye administered in a doctor’s office.
Samarendra Mohanty, Ph.D., Nanoscope’s President, Chief Scientific Officer and inventor of the technology, said, “Optogenetics is a powerful research tool, but had limited scope of clinical benefit because the opsins had a narrow band of activation, unlike natural light environment. MCO is sensitive to broadband light and activatable by ambient light, thus eliminating the risk of photo-toxicity from long-term continuous use of external intense light stimulation devices.”
About RP
In normal eyes, opsins are expressed by photoreceptor cells and intrinsically photosensitive Retinal Ganglion Cells (ipRGCs) in the retina, and when activated by light they trigger the physiological process of vision. RP encompasses a group of rare genetic disorders in which the retina’s photoreceptor cells degrade over time, leading to impaired vision and eventual blindness. These disorders are believed to be linked to over 60 different gene mutations.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics of Bedford, TX, is developing optogenetic gene therapies using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. Nanoscope utilizes MCO molecules to re-sensitize the degenerated retina to restore vision in an ambient light environment. The company’s pipeline includes vision restoration in patients with retinitis pigmentosa, Stargardt disease, and age-related macular degeneration.
More information about Nanoscope Therapies
Contact:
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591
Nanoscope Therapeutics Announces New Clinical Advisory Board Appointments
BEDFORD, Texas, April 19, 2021 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of inherited retinal diseases, today announced an expansion of its Clinical Advisory Board with the appointment of four new members.
“We are honored to have such distinguished clinical experts in retinal disorders join us as advisers. Their guidance will be invaluable as we pioneer gene therapies to reprogram retinal cells and make them functionally active to regain vision,” said Nanoscope President and Co-Founder Samarendra Mohanty, Ph.D.
The four new members include:
- Michael Singer, M.D., Clinical Professor of Ophthalmology at the University of Texas Health Science Center in San Antonio, Texas. He is also the Director of Clinical Research at Medical Center Ophthalmology Associates in San Antonio.
- Stephen H. Tsang, M.D, Ph.D., the Laszlo T. Bito Professor of Ophthalmology, and Pathology and Cell Biology at Columbia University Irving Medical Center.
- Paul Yang, M.D., Ph.D., Assistant Professor in Ophthalmic Genetics and Immunology at the Oregon Health & Science University Casey Eye Institute.
- SriniVas R. Sadda, M.D., President and Chief Scientific Officer of the Doheny Eye Institute, the Stephen J. Ryan – Arnold and Mabel Beckman Endowed Chair, and Professor of Ophthalmology at the University of California – Los Angeles David Geffen School of Medicine.
The new members have joined current advisory board members: Samuel Barone,M.D.; Vittorio Porciatti, DSc; Weldon Wright, M.D.; and Thomas Yorio, Ph.D.
Nanoscope’s lead product is an optogenetic gene therapy, vMCO-010, that delivers light-sensitive Multi-Characteristics Opsin (MCO) into retinal cells to restore vision in patients with retinitis pigmentosa (RP) and Stargardt disease. Both applications have received orphan drug designation from the FDA. If successful, the optogenetic therapy would be the first treatment aimed at correcting these conditions.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics, of Bedford, TX, is advancing gene therapy using light-sensitive molecules for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. Nanoscope utilizes an ambient light-sensitive MCO molecule to re-sensitize the retina toward ambient light level. The company’s pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with RP, Stargardt disease, and dry/end-stage wet age-related macular degeneration (AMD). For more information visit: https://nanostherapeutics.com/
Contact:
Dan Eramian
Opus Biotech Communications
danieleramian@comcast.net
425-306-8716
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591
Nanoscope® team conferred Phase II Retinal Organoid Challenge (ROC) award by National Institute of Health
BEDFORD, Texas, Feb. 2, 2021 /PRNewswire/ — To meet the advance challenge for generating a physiologically relevant three-dimensional (3D) retinal organoid, Nanoscope researchers® in collaboration with University of Colorado, and Miami University were recently awarded the top prize from the 3D-ROC challenge by the National Institute of Health (NIH). https://www.nei.nih.gov/about/goals-and-accomplishments/nei-research-initiatives/3-d-retina-organoid-challenge-3-d-roc/2017-ideation-challenge/2017-ideation-winner-and-honorable-mention
The organoids provide a unique opportunity for evaluating novel gene therapies including Multi-Characteristic Opsin (MCO) gene therapy for vision restoration. This is an active therapeutic development area for Nanoscope Therapeutics to evaluate effectiveness of MCO optogenetic therapy, which has shown promise in restoring sight in blind subjects. The opsin-based (and other gene therapy) treatment requires evaluation of potency in-vitro (for release characterization of drug) and objective clinical measurements. While we are currently using fluorescence imaging for objective measurement of potency of MCO-drug, there is a need for label-free evaluation of light activated activities in opsin-sensitized neurons. This is especially important because fluorescence methods inherently perturb the light-sensitive opsin-expressing neurons.
“Nanoscope continue to develop breakthrough technologies to assess neural activities without use of excitation light (as used in fluorescence) and is currently working on implementing this approach for objective label-free detection of activities of natural photoreceptors of eye as well as opsin-sensitized cellular activities in response to light stimulation in-vivo,” said Samarendra Mohanty, PhD, President and Chief Scientific Officer.
For the 3D ROC challenge, Nanoscope used near-infrared light-based Optical Coherence Tomography (OCT) integrated with electro and opto-physiology device to measure both structure and function of layer-specific cells in the targeted regions of retina.
https://www.youtube.com/watch?v=Gh0IqFZV_I0&list=LL&index=3&t=19s
This label-free activity detection technology has already attracted several Biopharmaceutical companies to evaluate their therapeutic approaches.
“We are also advancing OCT guided targeted laser microirradiation technology for creating models of degenerative eye diseases such as Retinitis Pigmentosa (RP) and dry age-related macular degeneration (dry-AMD) as well as for targeted delivery of therapeutic genes,” said Sulagna Bhattacharya, CEO of Nanoscope.
About NANOSCOPE THERAPEUTICS Inc.
Nanoscope Therapeutics is advancing gene therapy using light-sensitive MCO-molecules for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. Our pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with RP, Stargardt disease, and dry-AMD.
Contact:
Sulagna Bhattacharya
Investor Relations and Corporate Communications
817-719-2692
info@nanostherapeutics.com
