STK-002 / Stoke Therap - LARVOL DELTA

Antisense Oligonucleotide STK-002 Increases OPA1 in Retina and Improves Mitochondrial Function in Autosomal Dominant Optic Atrophy Cells. (PubMed, Nucleic Acid Ther) - "STK-002 and enhanced OPA1 immunofluorescence were visualized in retinal ganglion cells of cynomolgus monkeys treated with the ASO. Cumulatively, these data support the progression of STK-002 toward the clinic as the first potential disease-modifying treatment for ADOA."

Journal • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

FALCON: A Prospective Natural History Study of Patients with Autosomal Dominant Optic Atrophy (ADOA) (ARVO 2024) - "FALCON will provide data to inform us about the natural history of ADOA and to support the development of the antisense oligonucleotide (STK-002) as a potential disease-modifying treatment for patients with ADOA. Stoke plans to initiate a Phase 1 study (OSPREY) of its investigational ASO (STK-002)."

Clinical • Ocular Inflammation • Ophthalmology

OSPREY: An Open-label Study to Investigate Safety, Tolerability, and Exposure of the Antisense Oligonucleotide (ASO) STK-002 in Patients with Autosomal Dominant Optic Atrophy (ADOA) (ARVO 2024) - "STK-002 has the potential to be the first disease-modifying therapy for patients with ADOA, addressing the genetic cause of the disease by restoring OPA1 levels. OSPREY data will support the continued STK-002 development informing us of appropriate dosing as well as efficacy measures."

Clinical • Ocular Inflammation • Ophthalmology

OSPREY: An Open-Label Study Investigating the Safety, Tolerability, and Exposure to the Antisense Oligonucleotide (ASO) STK-002 in Patients with ADOA (AAPOS 2024) - No abstract available

Clinical • Ophthalmology

Mitochondrial dysfunction in Autosomal Dominant Optic Atrophy (ADOA) assessed in FALCON, a non-interventional, natural history study (SOE 2023) - "The natural history of visual neurodegeneration in ADOA is understudied.FALCON, STK-002-OA-901, is a multicenter, prospective natural history study of patients with ADOA who are ≥8 to ≤60 years old and have confirmed heterozygousOPA1 gene variant...FPF may add to current diagnostic tools for earlier detection of mitochondrial dysfunction and may help to inform future clinical studies of ADOA. _x000D_ _x000D_ _x000D_"

Ocular Inflammation • Ophthalmology