Choroid

Prevent Blindness Declares May as First-ever Inherited Retinal Disease (IRD) Genetic Testing Awareness Month

Retrieved on: 
Wednesday, April 24, 2024
Education, Paratriathlon, University, Blood, Birth defect, Retina, RP, Social media, Diagnosis, Stargardt disease, Stargardt, Marketing, Strabismus, Genetic, Johnson & Johnson, Gene, Spark Therapeutics, Choroid, Research, Visual impairment, Genetic testing, Pediatric ophthalmology, Disease, Visual Science, Patient, Eye, Cone dystrophy, MBA, Vision, Student, Therapy, Organization, IRD, Pharmaceutical industry

CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness, the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative. The group is providing a variety of tools to promote awareness and education for IRDs and the importance of genetic testing, including a free webinar, expert and patient videos, shareable social media graphics, and fact sheets in English and Spanish. IRD Genetic Testing Awareness Month is supported by funding from Johnson & Johnson, and Spark® Therapeutics.

Key Points: 
  • Prevent Blindness expands IRD and genetic testing awareness initiative to educate patients on various forms of IRDs, and the importance of genetic testing to confirm diagnosis and possible treatments.
  • CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness , the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative.
  • ET, the National Center for Children's Vision and Eye Health at Prevent Blindness will be hosting the free webinar, " Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases ."
  • To register for the "Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases" webinar, visit PreventBlindness.org/IRD-webinar-2024.

Inozyme Pharma Announces Positive Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency (PXE) and ENPP1 Deficiency

Retrieved on: 
Monday, April 8, 2024
Patient, Cerebrovascular disease, Stroke, Accelerated approval (FDA), European Medicines Agency, Doctor of Philosophy, Osteomalacia, PROMIS, Therapy, Conditional, Natural history, CTX, Pros, ADAS, Risk, DSM-IV codes, Cohort, Aes, Alkaline phosphatase, Rickets, EMA, PXE, Safety, Epilepsy, Clinical trial, Food, Hospital, Disability, BSAP, Cardiovascular disease, Disease, PD, Paresis, Consultant, Hypophosphatemia, Fatigue, Survivor, Sclera, FDA, Observational study, Pyrophosphate, Choroid, Incidence, Retina, Biomarker, Life, Event, Medical imaging

BOSTON, April 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive topline safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency (PXE, pseudoxanthoma elasticum) and ENPP1 Deficiency.

Key Points: 
  • “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.
  • The patients were assigned to three dose cohorts of INZ-701: 0.2 mg/kg (n=3), 0.6 mg/kg (n=3), and 1.8 mg/kg (n=4).
  • For trial design details, please see the section entitled “INZ-701 in ABCC6 Deficiency Phase 1/2 Clinical Trial Design” below.
  • For trial design details, please see the section entitled “INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design” below.

Everads Enters License Agreement with Kriya for Development of Ocular Gene Therapy Candidates Using Everads' Suprachoroidal Delivery Technology

Retrieved on: 
Wednesday, September 27, 2023
Sclera, Eye, TEL, Choroid, Translational Vision Science & Technology, Kriyā, Retina, Pharmaceutical industry, Medical device

The agreement follows a research collaboration between the parties whereby Everads' platform was evaluated for delivery of the company's gene therapy candidates in non-human primate models.

Key Points: 
  • The agreement follows a research collaboration between the parties whereby Everads' platform was evaluated for delivery of the company's gene therapy candidates in non-human primate models.
  • Under the terms of the agreement, Kriya was granted field-based exclusivity for use of Everads' platform using gene therapy against certain prespecified therapeutic targets including ophthalmic diseases that involve the complement cascade, while Everads retains the right to continue using and/or licensing its technology to other parties for the development of other therapeutic targets using gene therapy, as well as all non-gene therapy applications.
  • Everads received an undisclosed upfront payment and is entitled to receive development milestones and royalties on net sales of products using Everads' platform.
  • "We are extremely excited to enter into this license agreement after a thorough evaluation of our technology and its applicability for delivery of gene therapy.

Kriya Announces Exclusive License and Collaboration Agreement with Everads to Advance Gene Therapies for Prevalent Diseases in Ophthalmology Including Geographic Atrophy

Retrieved on: 
Wednesday, September 27, 2023
Drug delivery, Retina, Injection, CR2, Pediatrics, IgG4-related ophthalmic disease, Sclera, Eye, Geographic atrophy, Quality of life, Intravitreal injection, Inflammation, AAV, Complement, RESEARCH, Administration, C3, Choroid, Stanford University School of Medicine, Patient, CR1, PARK, Entrepreneurship, FDA, Therapy, Emotional dysregulation, Disease, Pharmaceutical industry, Medical device, Vaccine, Ophthalmology, Kriyā, C5

PALO ALTO, Calif. and RESEARCH TRIANGLE PARK, N.C., Sept. 27, 2023 (GLOBE NEWSWIRE) -- Kriya Therapeutics, Inc. ("Kriya"), a biopharmaceutical company developing gene therapies for conditions affecting millions of people around the world, has entered into an exclusive license, collaboration and supply agreement with Everads Therapy, Ltd (“Everads”) to advance Kriya’s portfolio of gene therapies for retinal diseases using Everads' suprachoroidal delivery device. Everads is a private biotech company that has developed a novel technology enabling targeted delivery of therapies to the retina via the suprachoroidal space, an anatomical compartment located between the sclera and choroid that traverses the circumference of the posterior segment of the eye. Suprachoroidal delivery is an emerging, non-surgical route of administration in ophthalmology. Specifically in delivery of gene therapy, this route of administration offers the potential to enhance the efficiency of gene therapy delivery to the retina while minimizing intraocular inflammation.

Key Points: 
  • Specifically in delivery of gene therapy, this route of administration offers the potential to enhance the efficiency of gene therapy delivery to the retina while minimizing intraocular inflammation.
  • The transaction enables Kriya’s access to Everads’ suprachoroidal delivery technology to deliver multiple prespecified gene therapy product candidates for several ophthalmic diseases, including those involving the complement cascade.
  • “I am quite optimistic and convinced that innovative therapies will continue to improve the lives of people with geographic atrophy and other serious ophthalmic diseases.
  • A gene therapy targeting the C3 and C5 pathways delivered by a suprachoroidal injection may be a significant improvement in the treatment of geographic atrophy.”

Adverum Biotechnologies Announces Positive Aflibercept Protein Level Data from the LUNA Phase 2 Trial

Retrieved on: 
Tuesday, September 26, 2023
Retina, Injection, Maintenance, Adverum Biotechnologies, Anti-VEGF, AAO, Life, Eye, ELISA, IVT, Choroid, Patient, Humour, M.A, Visual acuity, Safety, Pharmaceutical industry, Vaccine, Medical device, Dietary supplement, Medical imaging, OPTIC

REDWOOD CITY, Calif., Sept. 26, 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 announced initial aflibercept protein expression data from its ongoing Phase 2 LUNA trial evaluating ixoberogene soroparvovec (Ixo-vec) for the treatment of wet age-related macular degeneration (wet AMD). New data announced today include aflibercept protein levels for both the 2E11 and 6E10 vg/eye doses, suggesting that both doses are within the therapeutically active range. Notably, these protein expression levels are consistent with levels observed in the OPTIC trial, in which aflibercept levels and corresponding clinical activity have been sustained in patients through multiple years of follow-up after a single Ixo-vec injection.

Key Points: 
  • New data announced today include aflibercept protein levels for both the 2E11 and 6E10 vg/eye doses, suggesting that both doses are within the therapeutically active range.
  • “This first data cut from the LUNA trial is highly encouraging, showing similar aflibercept expression levels at the 2E11 and 6E10 doses,” stated Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies.
  • Aqueous Aflibercept Protein Expression Levels:
    Aflibercept protein data suggest Ixo-vec at the 2E11 and 6E10 doses deliver similar levels of aflibercept that are both within the therapeutically active range based on OPTIC and non-human primate data.
  • Presentation of OPTIC four-year Ixo-vec aflibercept protein expression data and three-year extension efficacy and safety data at the American Academy Ophthalmology (AAO) Meeting in November 2023.

Dr. Kaustabh Ghosh Recognized With Prestigious Catalyst Award for Innovative Age-Related Macular Degeneration Research

Retrieved on: 
Thursday, June 1, 2023
Cerebral edema, Visual impairment, AMD, Eye, Research to Prevent Blindness, Cancer, Research, Paratriathlon, Doctor of Philosophy, Atherosclerosis, Associate, Blood, Choroid, Doheny Eye Institute, RPB, Green, RPD, Principal, Pharmaceutical industry, Ophthalmology

PASADENA, Calif., June 01, 2023 (GLOBE NEWSWIRE) -- Doheny Eye Institute , one of the nation’s leading vision research institutions, announced that Kaustabh Ghosh, PhD , was recently recognized by Research to Prevent Blindness (RPD) and the International Retinal Research Foundation (IRRF) with the Catalyst Award for Innovative Approaches for Age-Related Macular Degeneration (AMD).

Key Points: 
  • PASADENA, Calif., June 01, 2023 (GLOBE NEWSWIRE) -- Doheny Eye Institute , one of the nation’s leading vision research institutions, announced that Kaustabh Ghosh, PhD , was recently recognized by Research to Prevent Blindness (RPD) and the International Retinal Research Foundation (IRRF) with the Catalyst Award for Innovative Approaches for Age-Related Macular Degeneration (AMD).
  • RPB and IRRF are two of the preeminent non-profits supporting eye research.
  • The Catalyst Award brings a grant of $300,000 for Dr. Ghosh to continue his important research.
  • For more information about the groundbreaking ocular research that takes place every day at Doheny Eye Institute, visit www.doheny.org .

Curative Biotechnology, Inc. Files Patent on Ophthalmic Formulation

Retrieved on: 
Wednesday, May 24, 2023
Retina, Patent Cooperation Treaty, PCT, Application, OTC, Metformin, RPE, Rare, Choroid

Boca Raton, FL, May 24, 2023 (GLOBE NEWSWIRE) -- Curative Biotechnology Inc. (OTC: CUBT) (“Curative Biotech” or the “Company”), a development-stage biomedical company focused on novel therapies for rare diseases and conditions, announced today that a Patent Cooperation Treaty (PCT) application was duly filed in the U.S. Receiving Office on May 22, 2023, claiming priority to U.S. Provisional Application No. 63/345,759, filed May 25, 2022. 

Key Points: 
  • Topical Administration Capable of Delivering Metformin to Choroid, RPE, and Retina
    Boca Raton, FL, May 24, 2023 (GLOBE NEWSWIRE) -- Curative Biotechnology Inc. (OTC: CUBT) (“Curative Biotech” or the “Company”), a development-stage biomedical company focused on novel therapies for rare diseases and conditions, announced today that a Patent Cooperation Treaty (PCT) application was duly filed in the U.S.
  • Receiving Office on May 22, 2023, claiming priority to U.S.
  • 63/345,759, filed May 25, 2022.
  • The Application has been accorded International Application No.

Ashvattha Therapeutics Presents Preclinical Data on anti-VEGF Nanomedicine D-4517.2 at the 2023 ARVO Annual Meeting

Retrieved on: 
Wednesday, April 26, 2023
Retina, Injection, Drug, ARVO, Therapy, Macrophage, DME, CNV, AMD, Eye, National Center for Advancing Translational Sciences, Nanomedicine, Diabetic retinopathy, Inflammation, Cell, Choroid, Johns Hopkins University, Patient, VEGFR, Microglia, Association for Research in Vision and Ophthalmology, Poster, RPE, Research, III, Ashvattha, Pharmaceutical industry, Vaccine, Ophthalmology

REDWOOD CITY, Calif., April 26, 2023 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company advancing a new class of nanomedicine therapeutics that transverse tissue barriers to selectively target activated cells in regions of inflammation, today announced two preclinical data sets demonstrating the efficacy of its anti-angiogenic precision nanomedicine, D-4517.2.

Key Points: 
  • REDWOOD CITY, Calif., April 26, 2023 (GLOBE NEWSWIRE) -- Ashvattha Therapeutics (“Ashvattha”), a clinical-stage company advancing a new class of nanomedicine therapeutics that transverse tissue barriers to selectively target activated cells in regions of inflammation, today announced two preclinical data sets demonstrating the efficacy of its anti-angiogenic precision nanomedicine, D-4517.2.
  • The data were shared in an oral presentation and poster at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting held on April 23-27, 2023 in New Orleans, LA.
  • This data supports the development of an oral formulation of D-4517.2 as an alternative to injections while greatly reducing the treatment burden on patients,” said Jeff Cleland, CEO, co-founder, and chairman of Ashvattha Therapeutics.
  • The data further support that systemic administration of D-4517.2 is effective in reducing neovascular lesions associated with AMD and selectively targets activated microglia and macrophages.

Wet Age-Related Macular Degeneration - Pipeline Insight, 2023: Comprehensive Analysis on 80+ Upcoming Drugs - ResearchAndMarkets.com

Retrieved on: 
Wednesday, March 22, 2023
Health, Pharmaceutical, SEQUOIA, Retina, Biologics license application, Injection, Ranibizumab, Therapy, Laser, DME, RVO, Symantec Endpoint Protection, AMD, Eye, Fluorescein angiography, Clinical trial, NAMD, NDA, Route of administration, Geographic atrophy, Visual impairment, DR, Diabetic retinopathy, Inflammation, AAV, Novartis, IVT, Blood, Route, Administration, Choroid, Discovery, Acquisition, Peripheral vision, Patient, Molecular Partners, Risk, VEGFR, AbbVie, Macular edema, Diagnosis, Paratriathlon, Growth, Cytokine, Visual acuity, News, Macular degeneration, Medical imaging, Pharmaceutical industry, Publishing, Vaccine, Allergan, Wet, Phase

This report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Wet Age-related Macular Degeneration pipeline landscape.

Key Points: 
  • This report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Wet Age-related Macular Degeneration pipeline landscape.
  • A detailed picture of the Wet Age-related Macular Degeneration pipeline landscape is provided which includes the disease overview and Wet Age-related Macular Degeneration treatment guidelines.
  • The companies which have their Wet Age-related Macular Degeneration drug candidates in the most advanced stage, i.e.
  • Wet Age-related Macular Degeneration pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.

Akari Therapeutics Announces Pre-Clinical Development Progress Toward Potential IND on Long-Acting PAS-Nomacopan for Geographic Atrophy (GA)

Retrieved on: 
Wednesday, February 15, 2023
Choroid, CNV, LTB4, Vance DeGeneres, Eye, Engineering, Drug discovery, Professor, ARVO, Intravitreal injection, Anxiety, VEGF, VEGF-A, C5, E pluribus unum, Tufts University School of Medicine, Leukotriene B4, Depression, Food, Risk, Choroidal neovascularization, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Endothelium, GMP, Leukotriene, Hot game, Geographic atrophy, American Society for Clinical Investigation, Blood, Therapy, Andrew Conway Ivy, Patient, Incidence, Retina, IVT, Visual system, AAO, Intellectual property, Kinetics, AMD, Inflammation, Medscape, PAS, PEGylation, Injection, McClard, Infection, IND, Pharmaceutical industry, Vaccine, Ophthalmology, Medicare, Akari

PASylation (PAS) technology is a biological alternative to the chemical conjugate polyethylene glycol (PEGylation) in the development of extended half-life recombinant protein therapeutics.

Key Points: 
  • PASylation (PAS) technology is a biological alternative to the chemical conjugate polyethylene glycol (PEGylation) in the development of extended half-life recombinant protein therapeutics.
  • Complement overactivation is a validated target and an important aspect in the pathophysiology of GA, while LTB4 may contribute to overexpression of VEGF-A, a key driver of CNV.
  • “Patients who have geographic atrophy also are at risk of developing an additional sight-threatening condition known as choroidal neovascularization or CNV, which presents more hurdles in the fight to preserve sight.
  • Medscape article on 24-month data presentation at AAO 2022 With Approval Pending, Pegcetacoplan Shows Mixed Results for Treating Geographic Atrophy https://www.medscape.com/viewarticle/981813#vp_2
    McClard CK, et al.