QR-1123 / Ionis, ProQR - LARVOL DELTA

Genetic Therapies for Retinitis Pigmentosa: Current Breakthroughs and Future Directions. (PubMed, J Clin Med) - "Luxturna (voretigene neparvovec-rzyl), the first FDA-approved gene therapy targeting RPE65 mutations, represents a milestone in precision ophthalmology, while OCU400 is a gene-independent therapy that uses a modified NR2E3 construct to modulate retinal homeostasis across different RP genotypes...Moreover, QR-1123, a mutation-specific antisense oligonucleotide targeting the P23H variant in the RHO gene, is under clinical investigation for autosomal dominant RP and has shown encouraging preclinical results in reducing toxic protein accumulation and preserving photoreceptors. SPVN06, another promising candidate, is a mutation-agnostic gene therapy delivering RdCVF and RdCVFL via AAV to support cone viability and delay degeneration, currently being evaluated in a multicenter Phase I/II trial for patients with various rod-cone dystrophies. Collectively, these advances illustrate the transition from symptom management toward targeted, mutation-specific therapies, marking a..."

Journal • Review • Gene Therapies • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa • NR2E3 • PRPF31 • USH2A

RNA-based therapies in inherited retinal diseases. (PubMed, Ther Adv Ophthalmol) - "These RNA-based therapies bring several key advantages in the setting of IRDs, and the potential to bring meaningful vision benefit to individuals living with inherited blinding disorders. This review will examine the increasing breadth and relevance of RNA-based therapies in clinical medicine, explore the key features that make AONs suitable for treating genetic eye diseases, and provide an overview of the three-leading investigational AONs in clinical trials."

Journal • Review • Ophthalmology • Rare Diseases • Retinal Disorders

AURORA: A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene (clinicaltrials.gov) - P1/2 | N=11 | Active, not recruiting | Sponsor: ProQR Therapeutics | Recruiting ➔ Active, not recruiting | N=35 ➔ 11

Enrollment change • Enrollment closed • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

AURORA: A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene (clinicaltrials.gov) - P1/2 | N=35 | Recruiting | Sponsor: ProQR Therapeutics | Trial completion date: Oct 2021 ➔ Jul 2022 | Trial primary completion date: Oct 2021 ➔ Jul 2022

Trial completion date • Trial primary completion date • Genetic Disorders • Inherited Retinal Dystrophy • Ocular Inflammation • Ophthalmology • Retinal Disorders • Retinitis Pigmentosa

"https://t.co/UOpGKJGmbE from @lab_zhou lab? and QR-1123 https://t.co/EnKtaPIGQc??" (@Weekademia)

"$SYNACT: AP1189 Ph2 data in RA - Nov'21 $OBSN: Linzagolix Ph3 data in endometriosis - Q4'21 $PRQR: QR-1123 Ph1/2 in adRP - Q4'21 Conferences #ACAAI21 - 4-8 Nov CHMP Meeting - 8-12 Nov #CTAD21 - 9-12 Nov (3/4)" (@KempenLS)

Endometriosis • Gynecology

RNA therapeutics in ophthalmology - translation to clinical trials. (PubMed, Exp Eye Res) - "This review provides a detailed insight into the recent developments and clinical trials that have been conducted for several gene-silencing therapies, including ISTH0036, SYL040012, SYL1001, PF-04523655, Sirna-027, QR-110, QR-1123, QR-421a and IONIS-FB-L in glaucoma, dry eye disease, age-related macular degeneration, diabetic macular oedema and various inherited retinal diseases. Our aim is to explore the potential of these drugs whilst evaluating their associated advantages and disadvantages, and to discuss the future translation of RNA therapeutics in ophthalmology."

Clinical • Journal • Review • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Diabetic Macular Edema • Dry Age-related Macular Degeneration • Dry Eye Disease • Glaucoma • Macular Degeneration • Macular Edema • Ocular Infections • Ophthalmology • Retinal Disorders

"Dosing underway in study of ProQR's QR-1123 for #visionloss disorder $PRQR $ions #antisense #opthamology #clinicaltrials #genetherapy https://t.co/n36aqBq79h" (@IonisDisrupts)

Clinical

ProQR receives Orphan Drug Designation from FDA for QR-1123 for autosomal dominant retinitis pigmentosa (GlobeNewswire) - "ProQR Therapeutics...today announced that it received Orphan Drug designation (ODD) from the Food and Drug Administration (FDA) for QR-1123...The ODD program offers development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S. following market approval."

Orphan drug