RDH12

Opus Genetics Sponsors Uni-Rare Natural History Study for People with Inherited Retinal Diseases

Retrieved on: 
Monday, November 14, 2022
AAV, Retinal degeneration, PARK, Severe cognitive impairment, RESEARCH, Ophthalmology, RDH12, Retina, Science, Triangle Park (Quezon City), Research, IRDS, Retinal dehydrogenase, IRD, Visual impairment, Natural history, Organization, Genetics, Gene, Patient, Pharmaceutical industry, Opus

RESEARCH TRIANGLE PARK, N.C., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the company will co-sponsor the Uni-Rare Study, a new natural history study for approximately 1,500 people with mutations in one of more than 300 rare genes associated with inherited retinal diseases (IRDs).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced the company will co-sponsor the Uni-Rare Study, a new natural history study for approximately 1,500 people with mutations in one of more than 300 rare genes associated with inherited retinal diseases (IRDs).
  • Opus is building a strong portfolio of programs focused on treating genetic mutations that cause inherited retinal diseases.
  • The natural history data collected as part of the Foundations Uni-Rare Study, a first of its kind in retinal diseases, will be critical to the future research and development of new therapies for inherited retinal diseases.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

Foundation Launching its Largest Natural History Study to Date for 1,500 People with Inherited Retinal Diseases Caused by Rare Mutated Genes

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Thursday, November 10, 2022
IRDS, RPE65, Retina, Macular degeneration, Research, Gene, Population, Ophthalmology, Therapy, Nanoparticle, USH2A, Severe cognitive impairment, Company, CEP290, Genetics, University of Pittsburgh School of Medicine, Foundation Fighting Blindness, Wilfrid Sellars, USH1B, Environment, MYO7A, Doctor of Pharmacy, Triangle Park (Quezon City), CNGA3, AAV, Science, Patient, CEO, RHO, CNGB3, MD, RPGR, Doctor of Philosophy, Research Triangle, Foundation, HIV disease progression rates, Retinitis pigmentosa, CHM, Biotechnology, Retinal degeneration, Leber congenital amaurosis, PROM1, IRD, Technology, RDH12, Usher syndrome, PCDH15, University, Visual impairment, Atsena Otie Key, MBA, LCA, Natural history, Pharmaceutical industry, Medical device, Vaccine, Antibiotics, Opus, GUCY2D

COLUMBIA, Md., Nov. 10, 2022 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, is committing at least $8.6 million for its Uni-Rare Study, a new natural history study for approximately 1,500 people with one of more than 300 rare genes associated with inherited retinal diseases (IRDs) including: retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions.

Key Points: 
  • "A majority of the 300 genes linked to inherited retinal diseases have not been well characterized in the clinic.
  • "We are excited to launch a highly inclusive study to benefit a large segment of the population affected by IRDs, which are so genetically diverse."
  • Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

Opus Genetics Appoints Jennifer Hunt Chief Development Officer

Retrieved on: 
Wednesday, May 11, 2022
Hunting, Drug development, LCA5, Foundation Fighting Blindness, Jennifer Hunt, State university system, GLOBE, Triangle Park (Quezon City), RDH12, AAV, Food, Hemoglobinopathy, CDO, Patient, Editas Medicine, Syracuse, Foundation, Genzyme, Program, Gene editing, Retina, RESEARCH, Lesley University, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Severe cognitive impairment, Ophthalmology, Forestry, NMNAT1, PARK, Visual impairment, Science, LCA, CRISPR, Genetics, State University of New York College of Environmental Science and Forestry, CEO, Environmental science, Leber congenital amaurosis, Therapy, Pharmaceutical industry, Opus, Biology

RESEARCH TRIANGLE PARK, N.C., May 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Jennifer Hunt as Chief Development Officer (CDO).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., May 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Jennifer Hunt as Chief Development Officer (CDO).
  • Hunt brings over 25 years of drug development experience to Opus, with specific expertise in global clinical operations, product development and program management in biologics, small molecules, gene editing and gene therapy.
  • Prior to joining Opus, she held key clinical and regulatory positions at several biopharmaceutical companies, including Genzyme, Voyager Therapeutics and Editas Medicine.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Treatment of Rare Inherited Retinal Diseases

Retrieved on: 
Wednesday, May 4, 2022
LCA5, Mutation, University, IND, NHP, GLOBE, Triangle Park (Quezon City), RDH12, AAV, Safety, ARVO, Retinal degeneration, Patient, Foundation, Association, Retina, RESEARCH, Association for Research in Vision and Ophthalmology, Perelman School of Medicine at the University of Pennsylvania, Severe cognitive impairment, Leadership, PARK, Science, LCA, Genetics, Leber congenital amaurosis, Pharmaceutical industry, Medical device, Opus, Ophthalmology, Research

RESEARCH TRIANGLE PARK, N.C., May 04, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced promising new preclinical data from studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA), a group of rare inherited retinal diseases characterized by photoreceptor degeneration, due to mutations of LCA5 or RDH12 genes, respectively.

Key Points: 
  • In preparation for IND-enabling trials of OPGx-001 and OPGx-002, studies were conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases.
  • In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies.

New Data on Potential of Opus AAV-based Gene Therapies for Rare Inherited Retinal Diseases to be Presented at Association for Research in Vision and Ophthalmology Annual Meeting 2022

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Thursday, April 28, 2022
Medicine, RDH12, Foundation, Penn Presbyterian Medical Center, Retina, Association, Department, Patient, AAV, GLOBE, Research, IRD, Leber congenital amaurosis, Genetics, MDT, Center, Perelman School of Medicine at the University of Pennsylvania, Science, Eye, Severe cognitive impairment, Association for Research in Vision and Ophthalmology, Gene, University, LCA5, LCA, ARVO, Pharmaceutical industry, Ophthalmology, Opus

The annual summit, held prior to the ARVO Annual Meeting, features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients.

Key Points: 
  • The annual summit, held prior to the ARVO Annual Meeting, features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Backed by Foundation Fighting Blindnesss venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases.
  • Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies.

Opus Enters Strategic Collaboration with Resilience for AAV-based Gene Therapy Development and Manufacturing for Inherited Retinal Diseases

Retrieved on: 
Monday, April 11, 2022
Doctor of Science, Retina, IRD, AAV, IND, Toxicology, Visual impairment, CEO, Research Triangle Park, NMNAT1, Retinal dehydrogenase, Severe cognitive impairment, LCA5, GLOBE, RDH12, Science, Foundation, Foundation Fighting Blindness, Genetics, GMP, Patient, Pharmaceutical industry, Vaccine, Resilience, Opus

RALEIGH, N.C., April 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced a strategic manufacturing services agreement with National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, to support the development and manufacturing of Opus pipeline.

Key Points: 
  • RALEIGH, N.C., April 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced a strategic manufacturing services agreement with National Resilience, Inc. (Resilience), a technology-focused biomanufacturing company dedicated to broadening access to complex medicines, to support the development and manufacturing of Opus pipeline.
  • Helping Opus advance their gene therapies for inherited retinal diseases to the clinic will hopefully lead to new treatments for underserved patients, said Rahul Singhvi, ScD, Chief Executive Officer of Resilience.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies.

Opus Genetics Announces Agreement with Massachusetts Eye and Ear and Harvard Medical School to License Third Program for Inherited Retinal Disease

Retrieved on: 
Thursday, November 11, 2021
Leber congenital amaurosis, Science, LCA5, Retinal dehydrogenase, LCA, Massachusetts Eye and Ear, Harvard Medical School, Research, Program, NMNAT1, Severe cognitive impairment, Foundation, Retina, GLOBE, Ear, RDH12, Patient, AAV, IND, Genetics, Pharmaceutical industry

The new program, OPGx-003, is based on the work of Eric Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and scientific co-founder of Opus.

Key Points: 
  • The new program, OPGx-003, is based on the work of Eric Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and scientific co-founder of Opus.
  • OPGx-003 is a gene augmentation therapy designed to halt functional deterioration in pediatric patients with retinal degenerative disease caused by mutations in the nicotinamide mononucleotide adenylyltransferase 1 (NMNAT1) gene.
  • Recent preclinical data have demonstrated the potential for this gene augmentation approach to achieve stable rescue of retinal structure and function.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

RD Fund Launches Opus Genetics with $19M Seed Funding to Advance Gene Therapy Treatments for Blinding Conditions

Retrieved on: 
Wednesday, September 22, 2021
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RALEIGH, N.C., Sept. 22, 2021 (GLOBE NEWSWIRE) -- The Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness aimed at rapidly driving research toward preventions, treatments and cures for the entire spectrum of retinal degenerative diseases, today announced the launch of Opus Genetics, a patient-focused gene therapy company efficiently developing therapies for orphan inherited retinal diseases. The $19 million in seed financing was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners.

Key Points: 
  • The $19 million in seed financing was led by the RD Fund with participation from the Manning Family Foundation and Bios Partners.
  • The initial seed funding will allow Opus to advance the preclinical research of its scientific founders, Jean Bennett, M.D., Ph.D.,the F.M.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Dr. Bennetts laboratory at Penn receives sponsored research funding from Opus Genetics.