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Home » Keywords » retinitis pigmentosa

Items Tagged with 'retinitis pigmentosa'

ARTICLES

Eye and DNA
Ocular

Variant advances ocular gene therapy VAR-002 based on feedback from EMA

April 4, 2025
Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.
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Ocular

Gene editing in iPSCs corrects inherited retinal dystrophies

Feb. 25, 2025
Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a recent publication, researchers from the Institute of Ocular Microsurgery applied clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and transcription activator-like effector nucleases (TALEN) gene-editing tools to precisely correct induced pluripotent stem cell (hiPSC) lines derived from IRD patients.
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PYC advances PYC-003 to clinic for polycystic kidney disease

Dec. 3, 2024
By Tamra Sami
After raising AU$40 million (US$25.9 million) earlier this year to advance lead candidate VP-001 for treating retinitis pigmentosa type 11, PYC Therapeutics is now advancing PYC-003 to the clinic for polycystic kidney disease.
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Ocular

RNA-editing strategy to treat RHO-related retinitis pigmentosa

Nov. 7, 2024
Retinitis pigmentosa (RP) is the most common hereditary degenerative eye disease that leads to progressive vision loss, primarily caused by retinal degeneration.
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Illustration of human eye
Ocular

Skyline Therapeutics’ gene therapy awarded US orphan drug designation for retinitis pigmentosa

Sep. 4, 2024
Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.
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Eye and DNA illustration
Ocular

Rznomics gains Australian clearance to advance RZ-004 into clinic for retinitis pigmentosa

July 15, 2024
Rznomics Inc. has received clinical trial notification (CTN) from Australia’s Therapeutic Goods Administration (TGA) for the initiation of a phase I/IIa trial evaluating RZ-004, a gene therapeutic candidate for autosomal dominant retinitis pigmentosa with rhodopsin mutation.
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Optogenetics illustration
Ocular

Opus Genetics awarded funding to advance preclinical programs for inherited retinal diseases

June 14, 2024
Opus Genetics Inc. has received $1.7 million in project-based funding from the Foundation Fighting Blindness to help advance two preclinical candidate programs for inherited retinal diseases.
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Illustration demonstrating structure of the human eye and organization of retinal cells
Biomarkers

COQ8B variants identified as cause of nonsyndromic retinitis pigmentosa

June 6, 2024
University of Basel investigators have reported an association between mutations in the COQ8B gene and nonsyndromic retinitis pigmentosa (RP). RP is a genetically heterogeneous retinal degeneration disorder, which has been found to be associated with mutations in more than 70 genes. However, there are still cases for which no genetic cause has been found.
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Vial and syringe with DNA
Ocular

ASGCT: ‘From darkness to light’ in ocular gene therapy

May 14, 2024
By Mar de Miguel
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
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Vial and syringe with DNA
Ocular

ASGCT: ‘From darkness to light’ in ocular gene therapy

May 10, 2024
By Mar de Miguel
From glaucoma to Stargardt disease, age-related macular degeneration (AMD) to retinitis pigmentosa, or a corneal transplant to Bietti’s crystalline dystrophy, the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) is working to bring some light to patients with age and congenital diseases that affect vision. From May 7-11, 2024, thousands of scientists are gathering in Baltimore to show their advances against the challenges of delivering genes and cells to the correct place, avoiding immunogenicity and improving diseases.
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More Articles Tagged with 'retinitis pigmentosa'

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