Retinal degeneration (rhodopsin mutation)

New Preliminary Clinical Data on Potential of Opus AAV-based Gene Therapy for Rare Inherited Retinal Disease to be Presented at the American Academy of Ophthalmology Annual Conference 2023

Retrieved on: 
Friday, November 3, 2023
Opus, Retinal degeneration (rhodopsin mutation), Genetics, MD, Leber congenital amaurosis, Stem cell, LCA5, American Academy, Gene, Retina, American Academy of Ophthalmology, LCA, Pharmaceutical industry, Ophthalmology

RALEIGH, N.C., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6 in San Francisco.

Key Points: 
  • RALEIGH, N.C., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6 in San Francisco.
  • The data will be presented as part of a broader presentation by Tomas S. Aleman, MD, the principal investigator of the trial, focused on gene augmentation therapies for the treatment of inherited retinal degenerations.
  • Details of the presentation are as follows:
    Session: The Future of Retinal Disease Pharmacological, Stem Cell and Gene Therapy Treatments

Ray Therapeutics Closes Oversubscribed $100M Series A Financing to Advance Optogenetic Gene Therapies for Blinding Diseases

Retrieved on: 
Tuesday, May 16, 2023
Research, Genetics, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Optical, Science, Other Science, California Institute for Regenerative Medicine, Genotype, Therapy, Macular degeneration, Norwest Venture Partners, Eye, Retinitis pigmentosa, Quality of life, Hope, Brain, Cell, Regenerative medicine, Novo Holdings A/S, Retinal degeneration (rhodopsin mutation), Patient, Novo, Ophthalmology, CIRM, Growth, Merck, Merck & Co., Ray, Pharmaceutical industry, Medical device, Retina

The funding will advance multiple programs targeting blinding diseases of the eye through clinical development.

Key Points: 
  • The funding will advance multiple programs targeting blinding diseases of the eye through clinical development.
  • Visual optogenetics is a therapeutic modality that uses gene therapy to deliver a light sensitive protein to cells in the retina.
  • Light entering the eye can then stimulate these cells to produce a visual signal that is sent to the brain.
  • “This Series A financing marks a significant milestone in our mission to address the urgent unmet medical need of patients with blinding diseases,” said Paul Bresge, CEO & Co-Founder, Ray Therapeutics.

4BIO Capital Portfolio Company Ray Therapeutics Closes Oversubscribed $100M Series A Financing to Advance Optogenetic Gene Therapies for Blinding Diseases

Retrieved on: 
Tuesday, May 16, 2023
California Institute for Regenerative Medicine, Genotype, Therapy, Macular degeneration, Norwest Venture Partners, Investment, Eye, Retinitis pigmentosa, Hope, Brain, Jack London's San Francisco Stories, Merck & Co., Cell, Regenerative medicine, Novo Holdings A/S, Retinal degeneration (rhodopsin mutation), Patient, Ophthalmology, CIRM, Growth, Merck, Research, Ray, Pharmaceutical industry, Retina

London & San Francisco, CA, 16 May 2023 – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm unlocking the treatments of the future by investing in advanced therapies and other emerging technologies, today announces that its portfolio company, Ray Therapeutics , a leading optogenetics company, has raised an upsized and oversubscribed $100M Series A financing.

Key Points: 
  • London & San Francisco, CA, 16 May 2023 – 4BIO Capital (“4BIO” or “the Group”), an international venture capital firm unlocking the treatments of the future by investing in advanced therapies and other emerging technologies, today announces that its portfolio company, Ray Therapeutics , a leading optogenetics company, has raised an upsized and oversubscribed $100M Series A financing.
  • The funding will advance multiple programs targeting blinding diseases of the eye through clinical development.
  • Light entering the eye can then stimulate these cells to produce a visual signal that is sent to the brain.
  • Dr Dima Kuzmin will continue representing 4BIO Capital on the Board.

Verana Health to Present Findings of Studies Using American Academy of Ophthalmology IRIS® Registry Data to Advance Insights into Real World Outcomes in Treating AMD

Retrieved on: 
Wednesday, November 2, 2022
Millennium, American Academy, MD, NAMD, Blood, Retinal degeneration (rhodopsin mutation), Durability, Doctor of Philosophy, Brolucizumab, Geographic atrophy, Machine Intelligence Research Institute, HCPS, American Academy of Ophthalmology, Research, AMD, Patient, RWD, Comparison, VEGF, Department, Varnaq, Data management, American Academy of Neurology, Academy, Rush University Medical Center, CATT, HCP, Medicine, Novartis, FDA, American Urological Association, IgG4-related ophthalmic disease, Clinical trial, Poster, Severe cognitive impairment, SAN, Quality of life, Solution, Pharmaceutical industry, Medical imaging, Ophthalmology

SAN FRANCISCO, Nov. 2, 2022 /PRNewswire/ -- During The Retina Society's 55th Annual Scientific Meeting Nov. 2 to 5, Verana Health®—a digital health company elevating quality in real-world data—will present findings on two separate studies that utilize curated real-world data (RWD) from the American Academy of Ophthalmology (Academy) IRIS® Registry (Intelligent Research in Sight).

Key Points: 
  • treatment arms of the CATT trial using de-identified IRIS Registry data from a real-world population to compare treatment outcomes at one year to the monthly treatment arms of the CATT using patient-level clinical trial data.
  • Established in 2014, the IRIS Registry is one of the largest specialty society clinical data registries in all of medicine.
  • Using its clinician-informed and artificial intelligence-enhanced VeraQpopulation health data engine, Verana Health transforms structured and unstructured healthcare data into curated, disease-specific data modules, Qdata.
  • Verana Health's quality data and insights help drive progress in medicine to enhance the quality of care and quality of life for patients.

Frontera Therapeutics Receives Additional IND Clearance for its Lead Program FT-001

Retrieved on: 
Wednesday, September 21, 2022
Dai, Lists of diseases, Safety, Center for Drug Evaluation and Research, IRD, U.S. FDA, AAV, FDA, Food, Center, Visual impairment, Retinal degeneration (rhodopsin mutation), Ophthalmology, RPE65, RPE, Hematology, Occult macular dystrophy, Therapy, CDE, Patient, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Eye, CEO, Severe cognitive impairment, Retinal pigment epithelium, Neurology, CNS, Research, GLOBE, NMPA, Mutation, GMP, IRDS, Pharmaceutical industry, IND

Frontera previously announced that the U.S. Food and Drug Administration (FDA) also cleared an IND for FT-001 earlier this year in April.

Key Points: 
  • Frontera previously announced that the U.S. Food and Drug Administration (FDA) also cleared an IND for FT-001 earlier this year in April.
  • Inherited retinal degenerations or dystrophies are a heterogenous group of serious eye conditions that cause visual impairment that may lead to blindness.
  • This year, we have advanced our lead program, FT-001, and have received both U.S. FDA and China CDE IND clearances.
  • For additional information about Frontera Therapeutics, please visit the companys website at www.fronteratherapeutics.com .

Ray Therapeutics Appoints Dr. Peter Francis as Chief Scientific & Medical Officer and Jenny Holt as SVP, Program Portfolio Management

Retrieved on: 
Tuesday, September 6, 2022
Biotechnology, Health, Optical, Novartis, Physician, CEO, Immunotherapy, Cell, Ophthalmology, Retinal degeneration (rhodopsin mutation), Gene, Gladstone Institutes, Therapy, Retinitis pigmentosa, SVP, Retinal degeneration, Senior, Onyx, Program management, Allergan, AMD, Eye, Acquisition, Drug development, Mesa, Patient, Medical device, San Francisco Bay Area

Ray Therapeutics , a biotech company whose mission is to restore vision to patients with blinding eye diseases, today announced the appointment of Peter Francis, M.D., Ph.D., as Chief Scientific & Medical Officer, and Jenny Holt as Senior Vice President, Program Portfolio Management.

Key Points: 
  • Ray Therapeutics , a biotech company whose mission is to restore vision to patients with blinding eye diseases, today announced the appointment of Peter Francis, M.D., Ph.D., as Chief Scientific & Medical Officer, and Jenny Holt as Senior Vice President, Program Portfolio Management.
  • He was previously Chief Medical Officer of RetroSense Therapeutics, where he spearheaded the worlds first optogenetics program into clinical trials, positioning the company for acquisition by Allergan.
  • Jenny Holt joins Ray Therapeutics as SVP, Program Portfolio Management with more than 25 years of experience in gene therapy, working at Gladstone Institute, Onyx, Novartis, and was a Co-Founder of Ignite Immunotherapy.
  • Prior to joining Ray Therapeutics, Jenny was VP & Head of Program Management at 4D Molecular Therapeutics, where she drove gene therapy product development across multiple therapeutic areas, including ophthalmology.

ProQR Announces Top-Line Results from Phase 2/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10

Retrieved on: 
Friday, February 11, 2022
Foundation, Prevalence, Annual report, RNA, U.S. Securities and Exchange Commission, Union, Western world, ANCOVA, CEP290, Therapy, Retinal degeneration (rhodopsin mutation), COVID-19, Retina, Usher syndrome, CME, Retinitis pigmentosa, Philadelphia, RNA transfection, Research, Perfect digit-to-digit invariant, Eye, Life, Children's Hospital of Philadelphia, EMA, Risk, Cataract, LCA, Nasdaq, Lists of diseases, Safety, Intravitreal injection, ProQR, FST, Mutation, Disease, Ghent University, Visual impairment, Leber congenital amaurosis, GLOBE, Month, Intellectual property, Hospital, Patient, CEO, Forward-looking statement, Security (finance), Event, Illuminate, FDA, Severe cognitive impairment, Ageing, PRIME, Children, Pharmaceutical industry, Vaccine, Ophthalmology

Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10, said Daniel A. de Boer, Founder and CEO of ProQR Therapeutics.

Key Points: 
  • Given the results observed in earlier studies of sepofarsen, the Illuminate trial results are unexpected and disappointing, especially for people living with LCA10, said Daniel A. de Boer, Founder and CEO of ProQR Therapeutics.
  • We are deeply grateful to all of the participants, their supporters, and investigators who participated in the Illuminate study.
  • We will continue to work with ProQR to understand the data as they work to advance therapies for inherited retinal diseases.
  • Consistent with the findings observed in the Phase 1/2 trial, cataracts, CME, and retinal thinning were observed.

Vedere Bio II Appoints Martina Schinke, Ph.D., as Vice President, Program Leader

Retrieved on: 
Tuesday, February 8, 2022
People, Beth Israel Deaconess Medical Center, Multimedia, File, Severe cognitive impairment, Time, Harvard Medical School, Research, Gastrointestinal tract, Biomedical Research, Twitter, Bluebird, IND, Leadership, Quality of life, Program, Patient, Bluebird bio, Atlas, Novartis, Heidelberg University, Novartis Institute for Tropical Diseases, Biomedicine, AAV, Retinal degeneration (rhodopsin mutation), Geographic atrophy, CAR, Harvard University, LinkedIn, Autism spectrum, Doctor of Philosophy, AMD, Atlas Venture, Genomics, Management, Pharmaceutical industry, Els Veder-Smit, Medicine

CAMBRIDGE, Mass., Feb. 8, 2022 /PRNewswire/ -- Vedere Bio II, Inc., a company developing next-generation therapies for vision restoration and preservation in patients with vision loss due to photoreceptor death, today announced the appointment of Martina Schinke, Ph.D., as Vice President, Program Leader.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 8, 2022 /PRNewswire/ -- Vedere Bio II, Inc., a company developing next-generation therapies for vision restoration and preservation in patients with vision loss due to photoreceptor death, today announced the appointment of Martina Schinke, Ph.D., as Vice President, Program Leader.
  • "With nearly 20 years of experience in program leadership, strategic alliances, and program management, we are thrilled to welcome Martina to Vedere," said Cyrus Mozayeni, M.D., Chief Executive Officer & President, Vedere Bio II and Atlas Venture Entrepreneur in Residence.
  • Dr. Schinke joins Vedere Bio II from her role as Vice President, Program Management & Leadership at Finch Therapeutics, where she provided program leadership for the development of a novel class of biological drugs aiming to restore microbiome functionality in Autism Spectrum Disorder and disorders of the gastrointestinal tract.
  • For more information, please visit www.vederebio.com or follow Vedere Bio II on Twitter and LinkedIn .

Aequus Strengthens Clinical Experience in Stargardt Disease with Pediatric Ophthalmology Expertise

Retrieved on: 
Wednesday, August 25, 2021
Cell death, Revision, Adult, FRCS, Pediatric surgery, Master of Science, Retina, Research, Safety, Risk, Pediatrics, AMD, OTCQB, University, Lists of diseases, Ophthalmology, Doctor of Philosophy, Humanitarian use licenses, Therapy, Forward-looking statement, SEDAR, Technology, Degenerative disease, MD, Legislation, Disease, AQS, Retinal degeneration (rhodopsin mutation), Company, Retinal degeneration, Acquisition, GLOBE, Patient, MCH, Lipofuscin, Pharmaceutical industry

REV-0100 is a potential therapy for patients with Stargardt disease that is designed to bind and clear a toxiclipidcalled lipofuscin.

Key Points: 
  • REV-0100 is a potential therapy for patients with Stargardt disease that is designed to bind and clear a toxiclipidcalled lipofuscin.
  • Aequus Pharmaceuticalsand Dr.Koenekooplook forward to working closelywithreVision Therapeutics to support the development of REV-0100for Stargardt disease.
  • Stargardt disease is also called Stargardt macular dystrophy, juvenile macular degeneration, or fundusflavimaculatus.
  • Stargardt disease is one of several but the most common cause of genetic disorders that cause juvenile macular degeneration.