Retinal pigment epithelium

PulseSight Therapeutics Launches to Advance Non-viral Gene Therapies with Disruptive Minimally-Invasive Delivery Technology for Severe Retinal Diseases

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Wednesday, February 28, 2024
Doctor of Philosophy, Decorin, Drug development, Sale, Iris (anatomy), Transferrin, Ciliary muscle, Geographic atrophy, Ranibizumab, Iron, Retinitis pigmentosa, Retina, CNV, Paratriathlon, Eye, Brolucizumab, VEGF, Therapy, AMD, Independent, DNA, Retinal pigment epithelium, KIP, Doctor of Pharmacy, Safety, Novartis, Oxidative stress, CSO, Conditional sentence, RPE, EMA, MD, FDA, SAS, Choroidal neovascularization, Cell, Human, Patient, Glaucoma, Gene expression

Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.

Key Points: 
  • Unique proprietary non-viral gene therapy platform with minimally invasive delivery technology providing long lasting gene expression and favorable distribution in the retina.
  • A substantially de-risked platform and two first-in-class gene therapies heading to the clinic, having the potential to become future blockbusters.
  • PARIS, Feb. 28, 2024 (GLOBE NEWSWIRE) -- PulseSight Therapeutics SAS, an ophthalmology biotech company developing disruptive non-viral gene therapies with minimally-invasive delivery technology, launches today with seed finance from Pureos Bioventures and ND Capital.
  • PulseSight is currently raising a Series A financing round to advance its programs into clinical proof-of-concept.

RxCell Announces $4,600,000 Investment from DMR Global Venture and Regenosis, Plans for Second Raise of $15,000,000 for IND Testing in Q4 2023

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Tuesday, December 5, 2023
Infectious Diseases, Finance, Clinical Trials, Professional Services, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, RPE, SERI, National University of Singapore, Notifiable disease, IND, Patient, USD, Investigational New Drug, Cell death, NUS, AMD, RPC, Retinal pigment epithelium, Cell, Name, National university, Pharmaceutical industry, Medical device

RxCell, a pioneering biotech company focusing on cellular therapeutics for age-related diseases, today proudly announces a $4,600,000 investment commitment from DMR Global Venture LLC and Regenosis.

Key Points: 
  • RxCell, a pioneering biotech company focusing on cellular therapeutics for age-related diseases, today proudly announces a $4,600,000 investment commitment from DMR Global Venture LLC and Regenosis.
  • Furthermore, RxCell is gearing up for a substantial second capital raise of $15,000,000 in Q4 2023.
  • The global Age-related Macular Degeneration (AMD) Market, an integral segment RxCell operates in, had a valuation of USD 7.47 billion in 2021.
  • Professor Xianmin Zeng, CEO of RxCell, remarked, "We're thrilled to align with DMR Global Venture and Regenosis.

Vesigen Highlights Non-Human Primate Biodistribution of Non-Viral Technology and Functional Delivery of Genome Editors In Vivo at the Cracking the Code: The Dawn of Nucleic Acid Medicines Meeting

Retrieved on: 
Thursday, October 19, 2023
Health, Genetics, Research, Pharmaceutical, Science, Biotechnology, Gene editing, Retinal pigment epithelium, Mouse, Dawn, RNA, ARMM, Histopathology, ARMM Regional Legislative Assembly, RPE, Disease, Cell death, Cytotoxicity, Therapy, Kupffer cell, Retina, Vaccine

Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex.

Key Points: 
  • Specifically, the Company presented new data demonstrating retinal biodistribution of engineered ARMMs across multiple species – including non-human primates – and data demonstrating successful in vivo functional delivery of a Cas9/guide RNA gene editing complex.
  • The data were presented at the Cracking the Code: The Dawn of Nucleic Acid Medicines meeting, taking place October 17-19, 2023, in Worcester, MA.
  • “We have shown that our ARMM technology can successfully deliver functionally active gene editing complexes to Kupffer cells and ameliorate disease in an acute liver injury model.
  • Additional data in mice, minipigs, and non-human primates demonstrated the efficient delivery of subretinally administered ARMMs to the RPE and photoreceptors, cell types associated with multiple retinal diseases.

Ocumension Therapeutics Announces 2023 Interim Results

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Thursday, August 24, 2023
Retina, Tissue, Glaucoma, Chorioretinitis, Betaxolol, Marketing, Culture, Uveitis, COVID-19, NDA, Visual impairment, Quality of life, Vitreous body, Research, Paratriathlon, Inflammation, Philosophy, Hospital, Latanoprost/timolol, Patient, Optic nerve, CDE, Growth, Near-sightedness, Emedastine, Therapy, Safety, III, NMPA, Cryopyrin-associated periodic syndrome, Retinal pigment epithelium, Disease, Pharmaceutical industry, Fine chemical, Period, PRC

HONG KONG, Aug 24, 2023 - (ACN Newswire) - Ocumension Therapeutics ("Ocumension" or the "Company", together with its subsidiaries, collectively the "Group", stock code: 1477), a leading China ophthalmic pharmaceutical platform company, announced its interim results for the six months ended 30 June 2023 ("the Period") today.

Key Points: 
  • HONG KONG, Aug 24, 2023 - (ACN Newswire) - Ocumension Therapeutics ("Ocumension" or the "Company", together with its subsidiaries, collectively the "Group", stock code: 1477), a leading China ophthalmic pharmaceutical platform company, announced its interim results for the six months ended 30 June 2023 ("the Period") today.
  • During the Period, OcuMension maintained its focus on the pilot scale production and validation batch production for its products, such as Emadine, at its Suzhou manufacture site.
  • The Company expects to complete the enrollment of subjects for phase II clinical trials in the second half of 2023.
  • Mr. Ye LIU, CEO and Executive Director of Ocumension, said: "Over the past three years, we have made many remarkable achievements.

Clearside Biomedical Announces Second Quarter 2023 Financial Results and Provides Corporate Update

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Monday, August 14, 2023
Pharming, Safety, New Drug Application, General partnership, TKI, Experiment, Patient, Research and development, R, Axitinib, SCS, SAB, ARVO, Retinal pigment epithelium, Therapy, ISO, Experimental & Molecular Medicine, Research, Genome, Choi, International Organization for Standardization, Conference call, Partner, The Association, Conference, ASRS, Retina, Eye, Administration, Chairperson, Certification, Webcast, International Organization (journal), OASIS, Association for Research in Vision and Ophthalmology, AMD, NDA, Needle, ET, Physician, Annual general meeting, Visual acuity, Society, Pharmaceutical industry, Medical device, Video game, Clearside, Ophthalmology, MD

ALPHARETTA, Ga., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.

Key Points: 
  • ET -
    ALPHARETTA, Ga., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), today reported financial results for the second quarter ended June 30, 2023 and provided a corporate update.
  • License Revenue: License and other revenue for the second quarter of 2023 was $1.0 million, compared to $0.4 million for the second quarter of 2022.
  • Other Income: Other income for the second quarter of 2023 was $0.5 million, compared to $24,000 for the second quarter of 2022.
  • Other Expense: Non-cash interest expense for the second quarter of 2023 was $2.3 million, compared to $0 in the second quarter of 2022.

Apellis Announces Seven Abstracts Accepted for Oral Presentation at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting

Retrieved on: 
Monday, July 10, 2023
Abstract, Caregiver, APLS, AMD, GALE, Geographic atrophy, GA, Angiography, OAKS, Retinal pigment epithelium, MD, Digital, FDA, Society, MOSAIC, Clinical, ASRS, Pharmaceutical industry, Communications satellite, Patient

WALTHAM, Mass., July 10, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that seven abstracts will be presented as oral presentations at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 28 to August 1 in Seattle.

Key Points: 
  • WALTHAM, Mass., July 10, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) announced that seven abstracts will be presented as oral presentations at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting to be held July 28 to August 1 in Seattle.
  • The upcoming data further reinforces the efficacy and safety profile of SYFOVRE® (pegcetacoplan injection), the first and only treatment for geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
  • “The breadth of SYFOVRE data at ASRS is a testament to the excitement surrounding the first and only FDA approved treatment for GA, which provides flexible dosing and slows GA lesion growth with increasing treatment effects over time,” said Caroline Baumal, MD, chief medical officer, Apellis.
  • “Among our seven oral presentations this year, we are thrilled to present data for the first time from the GALE long-term extension study of SYFOVRE.”
    Long-Term Efficacy of Pegcetacoplan in Patients with Geographic Atrophy – Nathan Steinle, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:06-9:12 a.m. PT
    Effect of Pegcetacoplan on Photoreceptor and Retinal Pigment Epithelium Integrity in Geographic Atrophy in the Phase 3 Trials and GALE Extension Study – Ursula Schmidt-Erfurth, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:12-9:18 a.m. PT
    Retinal Tissue Preservation with Intravitreal Pegcetacoplan in Patients with Geographic Atrophy – Sunir Garg, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:18-9:22 a.m. PT
    Pegcetacoplan vs Avacincaptad Pegol in Patients with Geographic Atrophy: An Anchored Matching-Adjusted Indirect Comparison of the Phase 3 Trials – Paul Hahn, MD – Dry AMD Symposium 2 – Sunday, July 30 – 9:42-9:46 a.m. PT
    The MOSAIC Study: A Clinical and Humanistic Burden of Illness Study of Patients with Geographic Atrophy and their Caregivers in the United States and Canada – Sophie J. Bakri, MD – Dry AMD Symposium 1 – Saturday, July 29 – 9:15-9:19 a.m. PT
    Utility of Outer Retinal Tubulation in Predicting Lesion Growth in Both Subfoveal and Nonsubfoveal Geographic Atrophy – Srinivas Kondapalli, MD – Imaging, Digital, Angiography Symposium – Saturday, July 29 – 2:57-3:01 p.m. PT
    Exudative Age-Related Macular Degeneration Events from the OAKS and DERBY Clinical Trials of Pegcetacoplan in Geographic Atrophy – Roger Goldberg, MD – Wet AMD Symposium 3 – Monday, July 31 – 1:05-1:09 p.m. PT

Adverum Biotechnologies Introduces an Intravitreal Gene Therapy Program for Geographic Atrophy and Presents Data on Its Ocular Gene Therapy Platform

Retrieved on: 
Thursday, May 18, 2023
Injection, CFI, Vance DeGeneres, Gene, MOI, AMD, CMC, Optogenetics, Research, GA, Publication, AAV, Geographic atrophy, IVT, Protein, Patient, Complement factor I, Kaay Raav Tumhi, Annual general meeting, Cell, Retinal pigment epithelium, Sf9 (cells), Poster, RPE, Kinetics, Society, Disease, Vaccine, Chemistry, Adverum Biotechnologies

REDWOOD CITY, Calif., May 18, 2023 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today presented new nonclinical data on an IVT gene therapy for the treatment of GA secondary to dry age-related macular degeneration (dry AMD) via expression of CFI at the American Society of Gene & Cell Therapy (ASGCT) 2023 Annual Meeting, in Los Angeles, California. Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.

Key Points: 
  • Adverum also presented new research pipeline data supporting the utility of its proprietary adeno-associated virus (AAV) vector platform in ocular gene therapy with presentations on optogenetics and on Chemistry, Manufacturing and Controls (CMC) strategies for AAV-mediated gene therapies in highly prevalent diseases.
  • “The ability to deliver both CFI and an engineered melanopsin leveraging our proprietary capsids via IVT administration demonstrates the strength of our platform at Adverum,” commented Brigit Riley, Ph.D., chief scientific officer of Adverum Biotechnologies.
  • “Similar to wet AMD, patients with GA are forced either to receive frequent, often monthly, injections or to experience faster lesion growth and vision deterioration.
  • The ASGCT poster and oral presentations will be made available on the Publications page of the Adverum website.

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

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Tuesday, May 16, 2023
Inhibitor, Neutralizing antibody, Hemolysis, Gene, NOD, MLD, PAH, Serum, SCID, Phenylketonuria, Trabecular meshwork, Gene editing, Gene expression, FIX, Publication, Metachromatic leukodystrophy, Messenger, PKU, Future, Administration, Immunosuppression, Genome, Intravitreal administration, Homologous recombination, Treatment, Vivisection, Immune system, PNH, Cell, Retinal pigment epithelium, NHP, Poster, RPE, Detection, Identification, Virus, ARSA, Society, Vaccine, Pharmaceutical industry, Truffle, Mouse, IND, C5

BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

Doheny Eye Institute Announces Upcoming Presentations at ARVO 2023 Annual Meeting

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Friday, April 21, 2023
Breakfast, ARVO, The Historic New Orleans Collection, Association for Research in Vision and Ophthalmology, AMD, Disease, Research, Ernest, RSVP, Doctor of Philosophy, Retinal pigment epithelium, Cell death, Annual general meeting, New Orleans Marriott, Diabetic retinopathy, CDT, Doheny Eye Institute, Ophthalmology, Symposia

PASADENA, Calif., April 21, 2023 (GLOBE NEWSWIRE) -- Doheny Eye Institute , one of the nation’s leading vision research institutions, announced that more than 30 of its scientists and clinician-scientists will present their latest research during the upcoming Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.

Key Points: 
  • PASADENA, Calif., April 21, 2023 (GLOBE NEWSWIRE) -- Doheny Eye Institute , one of the nation’s leading vision research institutions, announced that more than 30 of its scientists and clinician-scientists will present their latest research during the upcoming Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
  • The meeting will take place April 23-27, 2023 in New Orleans at the Ernest N. Morial Convention Center.
  • Doheny Eye Institute and UCLA Stein Eye Institute will also be exhibiting onsite in a shared booth space, #737.
  • “Doheny Eye Institute remains at the forefront of groundbreaking research to conserve, preserve and restore eyesight in people, and we are eager to share our latest findings and insights at ARVO 2023,” said Deborah A. Ferrington, PhD, Chief Scientific Officer at Doheny Eye Institute.

Turn around, bright eyes. Here's how to see the eclipse and protect your vision

Retrieved on: 
Monday, April 17, 2023
Newton, People, Eclipse, Retina, Radical, Radiation, Human, Headache, Diabetic retinopathy, ISO, Oxygen, Eye, Sun Blindness Music, GP, Astronomical Society of Australia, X-ray detector, Moon, Polaroid, Blood, Pain, AWST, Australian Chamber of Commerce and Industry, Patient, Time, Map, Hallucination, Retinopathy, Welding, Retinal pigment epithelium, Commerce, Communications satellite, Cosmetics, Telescope, Copper, Telescopic sight, Ancient Greece, Industry

Thousands of people will visit the small coastal town of Exmouth to witness this spectacular event, while Australians elsewhere will experience a partial eclipse.

Key Points: 
  • Thousands of people will visit the small coastal town of Exmouth to witness this spectacular event, while Australians elsewhere will experience a partial eclipse.
  • It is imperative to take steps to protect your eyes from solar retinopathy – permanent eye damage caused by looking at directly at the sun.
  • Expert guidelines advise people to never look at the sun or an eclipse with the naked eye.

Looking too closely

    • While some experienced initial improvements, the damage in others was unchanged 15 years later.
    • In 1999, 45 people presented to the Eye Casualty of Leicester Royal Infirmary after an eclipse seen there.
    • And after the solar eclipse of August 2017, 27 patients in the American state of Utah presented with concerns about vision.

What happens to your eye when you stare at the sun?

    • Solar retinopathy is damage to the back of the eye (the fovea centralis in the retina) from exposure to intense light.
    • A process called “phototoxicity” happens when the energy in the light forms damaging free radicals and reacts with oxygen within the retina.
    • This disrupts the retinal pigment epithelium (a layer of supportive cells beneath the retina) as well as the choriocapillaris (blood vessels) beneath.

There is no effective treatment

    • There is no proven treatment for solar retinopathy.
    • Antioxidant medications are used in some eye diseases, but there are no studies showing a benefit in solar retinopathy.

How to spot proper eclipse specs. 5 tips:


    Only approved glasses will absorb the appropriate wavelengths of visible, ultraviolet and infrared light. They must:
    In Exmouth in WA, the Chamber of Commerce and Industry has purchased some 20,000 eclipse viewing glasses, which meet the international ISO safety standard. Regular sunglasses, polaroid filters, welding shields, X-ray film, neutral density filters, red glass filters, mobile phones and homemade sun filters are not safe for viewing the sun or an eclipse.

What if you think the damage is done?

    • Symptoms to watch out for include blurred vision in one or both eyes within one to two days of exposure.
    • People may also experience blind spots, altered colour vision, visual distortion (straight lines appearing kinked or wavy), micropsia (objects appearing smaller than normal), light sensitivity and headache.
    • There may be no symptoms at all in the first few hours to a day.

What about totality though?

    • The Astronomical Society of Australia has an interactive map which eclipse viewers should visit, to determine the precise timing of totality in their location.
    • Eclipse glasses should only be removed after totality has commenced, when the moon has completely covered the sun and it suddenly becomes dark.
    • Remember, totality will only occur over the Ningaloo region, so it not safe to view the eclipse without protection anywhere else in Australia.