POSTERS & PUBLICATIONS

An image of a male scientist sitting at a bench in a lab. He is wearing a white lab coat, clear goggles and blue gloves. There are a series of red test tubes set up in front of him. He is holding a spare red test tube in his left hand and is squeezing fluid from a scientific device into the remaining test tubes with his right hand.

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

TITLE
DOWNLOAD
AUGUST 2026 | A Phase 1 Study of PYC-003, a Peptide-Oligonucleotide Conjugate Designed to Increase PC1 Expression in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
MARCH 2026 | PINACLE-1: A Phase 1 Clinical Trial of PYC-003 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
SEPTEMBER 2025 | A Phase 1a Clinical Trial of PYC-003 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Phelan-McDermid Syndrome (PMS)

TITLE
DOWNLOAD
JULY 2026 | 2026 PMSF Family Conference - PYC-002, a disease modifying RNA therapeutic approach to treat Phelan-McDermid syndrome
OCTOBER 2025 | PYC-002: Targeting SHANK3 with an RNA therapeutic approach for the treatment of Phelan-McDermid syndrome
JUNE 2025 | PMS Global Conference – PYC-002: Targeting SHANK3 with an RNA Therapeutics Approach for Phelan-McDermid Syndrome

Autosomal Dominant Optic Atrophy (ADOA)

TITLE
DOWNLOAD
MAY 2026 | SUNDEW: A Phase 1A Single Ascending Dose Study of PYC-001; a peptide conjugated oligonucleotide designed to treat OPA1 mutation-associated Autosomal Dominant Optic Atrophy
MARCH 2026 | A Phase 1A Single Ascending Dose Study To Evaluate Safety Of PYC-001; A Peptide-Conjugated Oligonucleotide Designed To Treat OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy
OCTOBER 2023 | PYC-001, a peptide-conjugated phosophorodiamidate morpholino oligomer for the treatment of autosomal dominant optic atrophy

Retinitis Pigmentosa type 11 (RP11)

TITLE
DOWNLOAD
MAY 2026 | A Phase 1B Multiple Ascending Dose Study of VP-001; a peptide conjugate of oligonucleotide designed to treat PRPF31-related Retinitis Pigmentosa
MAY 2026 | Adaptive optics imaging, microperimetry and optoretinograhy following intravitreal injection of VP-001 in patients with PRPF31-associated retinitis pigmentosa
MAY 2025 | Foundation Fighting Blindness 2025 Presentation: Advancing PRPF31 Related Retinitis Pigmentosa Treatment: Key Insights from the VP-001 Phase 1B Study
NOVEMBER 2024 | RANZCO 2024 presentation: a Phase 1 first-in-human study of VP-001 designed to treat retinitis pigmentosa type 11
MAY 2024 | ARVO 2024 presentation: a peptide conjugated oligonucleotide for the treatment of retinitis pigmentosa type 11
MAY 2023 | VP-001 as an interventional therapy for patients with PRPF31 mutation-associated retinal dystrophy
JUNE 2021 | Modulation of CNOT3 expression using antisense oligomers to treat retinitis pigmentosa type 11
SEPTEMBER 2020 | Exploring microperimetry and autofluorescence endpoints for monitoring disease progression in PRPF31-associated retinopathy

Platform

TITLE
DOWNLOAD
OCTOBER 2022 | Enhancement of antisense oligomer cell penetration in retinal layers using a modular cell penetrating peptide platform
JUNE 2021 | RNA therapeutics in the treatment of retinal disease: delivering the potential
DECEMBER 2015 | GFP-complementation assay to detect functional CPP and protein delivery into living cells