Archive for the Nanoscope Press Release Category

Nanoscope Awarded $1.5M Phase 2B SBIR Grant to Advance Ambient Light Activatable Opsin Gene Therapy to Restore Vision for AMD Patients

Nanoscope Awarded $1.5M Phase 2B SBIR Grant to Advance Ambient Light Activatable Opsin Gene Therapy to Restore Vision for AMD Patients

Nanoscope Awarded $1.5M Phase2B SBIR Grant to Advance Ambient Light Activatable Opsin Gene Therapy to Restore Vision for AMD Patients

BEDFORD, TX (September 14, 2021)—Nanoscope researchers were awarded a $1.5 million Phase2b Small Business Research Innovation (SBIR) grant by the National Eye Institute (NEI)of the National Institutes of Health (NIH) to advance the company’s ambient light activatable optogenetic gene therapy for age-related macular degeneration(AMD).

“Winning the NIH grant via a highly competitive review process is a testament to the innovativeness and clinical significance of our first-in-class optogenetic therapy based on our Multi-Characteristic Opsin (MCO),which is a patent-protected ambient light activatable protein for restoration of vision in people with AMD,”said Sulagna Bhattacharya, CEO of Nanoscope.“Our goal is to apply our therapy to relieve the suffering of millions of people worldwide with AMD.”’

Nanoscope’s lead MCO gene therapy, MCO-010, is in a late-stage Phase 2b trial for retinitis pigmentosa (RP) in the US. MCO-010 has orphan drug designations for RP and Stargardt disease from the US Food and Drug Administration. A Phase 1/2a trial of MCO-010 showed patients blinded by RP experienced clinically meaningful vision restoration.

Nanoscope’s optogenetic gene therapy uses a proprietary AAV2 vector to deliver MCO genes into bipolar retinal cells where they express MCOs engineered to be fast and polychromatic, enabling vision in different color environments. The MCOs reprogram bipolar cells to act like photoreceptor cells damaged not only by AMD, but also the full range of inherited retinal disorders (IRDs) regardless of underlying disease-causing gene mutations. The therapy involves a single intravitreal injection and can be administered in a medical office setting.

“Our preliminary data show MCO therapy can be applied in a mutation-independent manner for specific IRDs and can serve as a retinal disease-agnostic platform therapy,” said Samarendra Mohanty, PhD, Principal Investigator of the recently awarded NIHgrant and Nanoscope’s President and Chief Scientific Officer.“ With this grant we can accelerate advancement of our MCO therapy for geographic atrophies of the macula for juveniles as well as adults.”

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 other IRDs as well as age-related macular degeneration.

Contact:
Charles Craig
Opus Biotech Communications
pr@nanostherapeutics.com
404-245-0591

Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference

Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy  for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference

Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference

Company’s lead product, an optogenetic gene therapy to restore vision for patients blinded by retinitis pigmentosa, is currently in late-stage Phase 2b trials in US

BEDFORD, TX (September 8, 2021) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degeneration diseases, today announced Dr. Santosh Mahapatra, principal investigator of the company’s clinical study on optogenetic gene therapy for vision restoration in people blinded by different gene mutations including ABCA4, will present findings from the study at the EURETINA 2021 Virtual Conference September 9 – 12. The conference is conducted by the European Society of Retina Specialists.

Dr. Mahapatra’s presentation, “52 Weeks Safety and Efficacy of Multi-Characteristic Opsin Enabled Vision Restoration in Patients with ABCA4 Mutation, is Sunday, September 12, in Prize Papers 13 session on New Drug Treatments & Technologies.

ABCA4 mutations are associated with retinal degeneration diseases, including retinitis pigmentosa and Stargardt disease. Patients in the study received a single intravitreal injection of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy and were followed for 52 weeks. All patients experienced improvement in visual acuity and function with no serious adverse events.

Nanoscope’s optogenetics therapy delivers MCO genes in proprietary AAV2 vectors into retinal cells leading to expression of ambient light-sensitive polychromatic opsins for vision restoration in different color environments. The therapy, which can be administered in a medical office, focuses on disease phenotype, enabling treatment of retinal diseases regardless of genetic mutations.

Nanoscope researchers also developed a Low-Vision Multi-Parameter Test (LVMPT) for assessing different attributes of functional vision. Michael Carlson’s presentation of the “Low-Vision Multi-Parameter Test for Monitoring Visual Function of Patients with Advanced Retinal Diseases” is Thursday, September 9, in the New Drug Treatment and Technology session.

The MCO-010 gene therapy and LVMPT device are being evaluated in a Phase 2b trial (NCT04945772) in the US. MCO-010 has received orphan drug designations from the US Food and Drug Administration for RP and Stargardt disease. 

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 ocular gene therapies for patients with RP, Stargardt disease, and geographic atrophies.

 

Contact:

Dan Eramian
Opus Biotech Communications
pr@nanostherapeutics.com

425-306-8716

Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

Nanoscope Therapeutics Announces First Patient Dosed in Phase 2b Clinical Trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

BEDFORD, TexasJuly 19, 2021Nanoscope 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

Related Links

http://www.nanostherapeutics.com

Nanoscope Therapeutics Announces FDA Approval of IND for Phase 2b clinical trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

Nanoscope Therapeutics Announces FDA Approval of IND for Phase 2b clinical trial of Optogenetic Gene Monotherapy to Restore Vision in Patients with Retinitis Pigmentosa

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.

####

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

Nanoscope’s Optogenetic Gene Therapy Restores Clinically Meaningful Vision in 11 Patients Blinded by Retinitis Pigmentosa

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

Nanoscope Therapeutics Announces New Clinical Advisory Board Appointments

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’s Optical Gene Therapies to be Featured at Optogenetics and Optical Manipulation 2021 Conference

Nanoscope’s Optical Gene Therapies to be Featured at Optogenetics and Optical Manipulation 2021 Conference

Nanoscope’s Optical Gene Therapies to be Featured at Optogenetics and Optical Manipulation 2021 Conference

Company’s President Samarendra Mohanty will co-chair the conference, which is part of the SPIE Photonics West Digital Forum

BEDFORD, Texas, March 5, 2021 /PRNewswire/ — Researchers of Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal diseases, will make multiple scientific presentations featuring its groundbreaking research on optical gene delivery and opsin based neuromodulation therapy at the Optogenetics and Optical Manipulation 2021 virtual conference, March 6 – 11, 20201.

Nanoscope President Samarendra Mohanty, Ph.D., is serving as a co-chair of the event, which is part of SPIE Photonics West’s week long series of online conferences and industry programs. SPIE is an international society for optics and photonics dedicated to advancing light-based technologies.

“Nanoscope is on the verge of significant breakthroughs in optical gene therapies for improving quality of life in people with retinal and neurological diseases,” said Dr. Mohanty. “We are developing both viral vector and non-viral light delivery of genes to reprogram neurons and make them light-activatable, allowing therapeutic modulation.”

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 retinal degeneration. If successful, the optogenetic therapy would be the first treatment aimed at correcting these retinal diseases.

Nanoscope also is developing an alternative, non-viral light-based gene delivery method that is designed to be more targeted with less immunogenicity.

Below are the sessions highlighting Nanoscope’s optical gene therapy research. All three sessions will be available on-demand starting March 6.

Neuroprotection by optical delivery of therapeutic gene into retina
Author(s): Subrata Batabyal, Sanghoon Kim, Michael Carlson, Houssam Al-Saad, Jazlyn Gallego, Adnan Dibas, Samarendra Mohanty

Development and functional characterization of retinal dystrophy model using targeted layer-specific laser microirradiation
Author(s): Micahel Carlson, Sanghoon Kim, Subrata Batabyal, Samarendra Mohanty

Pain modulation by multi-characteristic opsin sensitization of inhibitory network of brain
Author(s): Darryl Narcisse, Robert Benkowski, Arman Fijany, Samarendra Mohanty

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is advancing gene therapy 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 an ambient light-sensitive MCO molecule to re-photosensitize the retina. The company’s pipeline includes optogenetics based retinal regeneration therapy for vision restoration in patients with retinitis pigmentosa and dry age-related macular degeneration.

Contact:
Charles Craig
Opus Biotech Communications
charles.s.craig@gmail.com
404-245-0591

Nanoscope President to Deliver Talk on Gene Therapy for Retinal Diseases at Association for Ocular Pharmacology and Therapeutics Conference

Nanoscope President to Deliver Talk on Gene Therapy for Retinal Diseases at Association for Ocular Pharmacology and Therapeutics Conference

Nanoscope President to Deliver Talk on Gene Therapy for Retinal Diseases at Association for Ocular Pharmacology and Therapeutics Conference

Company’s lead product, an optogenetic gene therapy to restore vision for patients with retinitis pigmentosa, expected to enter seminal clinical trials in 2021

BEDFORD, Texas, March 3, 2021 /PRNewswire/ — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of inherited retinal diseases, today announced that its President, Samarendra Mohanty, Ph.D., will participate in the Association for Ocular Pharmacology and Therapeutics (AOPT) 15th Biennial (Virtual) Meeting, March 4-7, 2021.

Dr. Mohanty will deliver a presentation titled, “Ready for the Future: Refunctionalizing, Reprogramming, Revitalizing and Regenerating Retina for Restoring Vision,” during an AOPT Special Session, Saturday, March 6, at 12 pm EST.

“Hundreds of millions individuals are suffering from vision loss due to damage to the retina and optic nerve,” said Dr. Mohanty, who also serves as Nanoscope’s Chief Scientific Officer. “We are developing gene therapies to reprogram retinal cells and make them functionally active to regain vision.”

In addition, Nanoscope will participate in a session Friday, March 5, at 1:30 pm EST, and will make a presentation titled: “Low-Vision Multi-Parameter Test (LVMPT) to Quantitatively Measure Visual Level of Low-Vision Patients.”

“The LVMPT measures functional vision which can serve as a novel key endpoint for our clinical trial on Low-Vision Patients,” said Sai Chavala, M.D., Chief Medical Officer of Nanoscope.

AOPT is a global, not-for-profit organization for scientists and individuals from all disciplines related to ocular pharmacology and its therapeutic applications. Members are from academic institutions, pharma and biotech industries, device companies, and clinics.

Nanoscope’s lead product is an optogenetic gene therapy, vMCO-010, that delivers light-sensitive Multi-Characteristic Opsin (MCO) into retinal cells to restore vision in patients with retinitis pigmentosa and Stargardt disease. Both applications have received orphan drug designation from the US-FDA. If successful, the optogenetic therapy would be the first treatment aimed at correcting these conditions.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics, 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 retinitis pigmentosa, Stargardt disease, and dry age-related macular degeneration (AMD).

Contact:
Dan Eramian
Opus Biotech Communications
danieleramian@comcast.net
425-306-8716

Nanoscope® team conferred Phase II Retinal Organoid Challenge (ROC) award by National Institute of Health

Nanoscope® team conferred Phase II Retinal Organoid Challenge (ROC) award by National Institute of Health

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

Nanoscope Therapeutics Receives Orphan Drug Designation for Gene Therapy of Blindness

Nanoscope President to Deliver Talk on Gene Therapy for Retinal Diseases at Association for Ocular Pharmacology and Therapeutics Conference

This is the second program based on Nanoscope’s Multi-Characteristic Opsin Platform to receive orphan designation. This designation highlights the unmet need for therapy of patients with rare form of inherited macular degeneration.

BEDFORD, Texas, Jan. 25, 2021 /PRNewswire/ — NANOSCOPE THERAPEUTICS Inc., a clinical-stage biotechnology company determined to change lives through the ocular gene therapy, today announced that it has received an Orphan drug designation from FDA for gene therapy-based treatment of Stargardt disease, a form of inherited retinal degenerative disease caused by gene mutation and passed on to children.

Central vision loss due to poor functioning RPE cells and loss of photoreceptors in the macula is the hallmark of Stargardt disease. According to Federation for Fighting Blindness, Stargardt disease is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S. The loss of vision is devastating to both children and adults, and significantly impacts their quality of life.  There is an immense need to restore vision in these patients.

“Currently, there are no approved therapies for Stargardt Disease” said Sulagna Bhattacharya, Chief Executive Officer of Nanoscope. “We are excited by the potential of ambient light activatable Multi-Characteristic Opsin (MCO) based photosensitization of retinal neurons for treating Stargardt disease in a gene agnostic manner.”

“vMCO-010 expands the portfolio of orphan drug designations obtained in multiple pipeline programs, expanding its therapeutic potential from Retinitis Pigmentosa (RP) with peripheral retinal degeneration to macular degeneration as in Stargardt disease”, said Al Guillem, Chairman of Nanoscope Therapeutics Board.

Sai Chavala, Chief Medical Officer of Nanoscope, commented “We look forward to developing our innovative optogenetic platform technologies for visually-challenged patients through continued interaction with the FDA.  The orphan designation will aid in accelerating our clinical development program.”

The Orphan Drug Act encourage the development of medicines for rare diseases, and benefits include tax credits and application fee waivers designed to offset clinical development costs, as well as eligibility for seven years of post-approval market exclusivity.

“We are extremely excited and pleased to announce a second orphan drug designation for MCO based treatment for retinal diseases. Since the pathology of degenerated macula in Stargardt disease is similar to that of dry-AMD, we are very excited about the opportunity to learn from our Stargardt program and advance MCO-based dry-AMD program” said Samarendra Mohanty, President and Chief Scientific officer of Nanoscope that received an orphan designation in 2017 for MCO for treatment of RP.

About NANOSCOPE THERAPEUTICS Inc.

Nanoscope Therapeutics 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. We utilize an ambient light-sensitive MCO molecule to re-sensitize the retina toward ambient light level. 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

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Nanoscope Announces Presentation at the 2020 Annual Meeting of American Academy of Ophthalmology

Nanoscope Therapeutics Announces Presentation at the 2020 Annual Meeting of American Academy of Ophthalmology

BEDFORD, Texas, Nov. 13, 2020 /PRNewswire/ — NANOSCOPE THERAPEUTICS Inc., a clinical- stage biotechnology company determined to change lives through the ocular gene therapy based on its proprietary ambient light activatable MCO Optogenetic Technology Platform, today announced that an oral presentation at the Late Breaking Developments session of Retina Subspecialty Day will be made during the American Academy Ophthalmology (AAO) 2020 Annual Meeting, taking place November 13-15, 2020.

The presentation will feature data from the vMCO-010 Phase 1/2a intravitreal trial for the treatment of Retinitis Pigmentosa (RP). Details are as follows:

Title: Phase 1/2a Study of Intravitreal Optogenetics Gene Therapy for Vision Restoration in Advanced Retinitis Pigmentosa
Presenter: Dr. Santosh Mahapatra, Chief Medical Ofcer, JPM Rotary Eye Hospital and Research Center
Date/time: 13th November 2020, 3.00 PM PST; also available on-demand Session title: Late Breaking Developments, Part IThe Nanoscope co-authors of the presentation are Sai Chavala (Chief Medical Ofcer), Samuel Barone (Advisor), Samarendra Mohanty (President and Chief Technology & Innovation Ofcer), Subrata Batabyal, Michael Carlson, Ananta Ayyagari, and Kissaou Tchedre.
About NANOSCOPE THERAPEUTICS Inc.
Nanoscope Therapeutics is advancing gene therapy using light-sensitive molecules and light- assisted gene delivery for giving sight to the millions of blind individuals suffering from retinal degenerative disease, for which no cure exists. We utilize an ambient light-sensitive Multi- Characteristic Opsin (MCO) molecule to re-sensitize the retina toward low light level. Our light- assisted gene therapy is highly targeted to geographic atrophies without perturbing the intact retina circuitry. Our team is highly experienced in Ophthalmology product development and clinical translation of innovative research. Our pipeline includes light based retinal regeneration therapy and light-based cortical prosthetics for vision restoration.
Contact:
Sulagna Bhattacharya
Investor Relations and Corporate Communications
Nanoscope Therapeutics, Inc.
1312 Brown Trail, Suite A
Bedford, TX 76022
(817) 857-1186
  • Dr. Samarendra Mohanty, PhD |  Co-Founder & President

    Samarendra Mohanty (Co-Founder/President) is an inventor & serial entrepreneur with 20+ years experience in Biomedical Sciences. He is Co-Founder of several Biotechs and Biomedical device /diagnostic companies (developed & commercialized $100K+ biomedical instruments.)

    Samar obtained M.Tech in Applied Optics from the Indian Institute of Technology-Delhi and a PhD in (Bio)Physics from the Indian Institute of Sciences-Bangalore.

    His extensive Biomedical Technologies experience includes serving as Professor/Senior Scientist at the University of Texas;  University of California, Irvine; Center for Adv. Tech (India); Int. Mol. Biotech (Germany); Univ. Pavia (Italy); NUS (Singapore); and University of St. Andrews (UK). He has authored over 200 international patents and publications in leading journals including Nature and Nature Photonics.

    He is the Principal Investigator for major grants from National Eye Institute including Audacious Goal Initiative and Bioengineering Research grants. He serves on the editorial board of journals and chairs international conference on optogenetics. He is winner of 2019 Healthcare Heroes award (Fort Worth Business Press), Retinal Organoid Challenge Award, Audacious Goal Initiative Award (NIH), Finalist of Tech Titan, and NIH-Director’s Innovator Award.