ProQR to Start Clinical Trial of QR-110 in Leber’s Congenital Amaurosis Type 10 Patients

May 12, 2017: By Jon Swedien

ProQRThe US FDA has given clearance to ProQR Therapeutics’ investigational new drug application for QR-110, a proposed treatment for Leber’s congenital amaurosis Type 10 (LCA 10) patients, the Netherlands company announced April 27.

ProQR said it intends to move forward with a Phase I/II open-label trial to assess the safety, tolerability, pharmacokinetics, and efficacy of multiple administrations of QR-110 in one eye of each patient. ProQR said the trial will include six adults and six children with LCA 10.

Topline results are expected in 2018.

QR-110 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of LCA 10, a mutation in the CEP290 gene. It is intended to be administered through intravitreal injections in the eye. QR-110 has been granted orphan drug designation in the US and European Union, the company said.

LCA 10 leads to progressive loss of vision, causing most patients to lose their sight in the first few years of life. There are no approved treatments or products in clinical development that treat the underlying cause of the disease, ProQR said.

ProQR is focused on RNA medicines for the treatment of severe genetic rare diseases such as LCA 10, cystic fibrosis, and dystrophic epidermolysis bullosa.

Need Deeper Analysis? Sign up for Market Scope's monthly newsletter to get in-depth coverage of the ophthalmic market, including quarterly procedure volumes based on survey data and meeting coverage from around the world.

Recent Ophthalmic News

FDA Ophthalmic Devices Panel to hold Meeting Oct. 27

Novartis’ RTH258 Successful in Two Phase III Studies for nAMD

Ocugen Closes $7.5 Million Series B Round

Genentech Uses Vital Art and Science Vision-Testing App in Phase II Study of Sustained Delivery of Lucentis

US Supreme Court Ruling Shortens Time to Market for Biosimilars

Ellex Acquires Royalty Commitment for iTrack Device for $2.15 Million

Johnson & Johnson Vision Names Jonathan Talamo, MD, Chief Medical Officer

Teva Launches Generic Versions of Pataday in US

David Karcher Stepping Down as Executive Director, CEO of ASCRS

EyeGate Raises $10 Million in Public Offering

Aerie Raises $125 Million through Upsized Public Offering, ATM Program

pSivida’s Durasert for Posterior Segment Uveitis Successful in Second Phase III Study

Mynosys Zepto Receives US FDA 510(k) Clearance

Ozurdex, Humira Gain NICE Backing for Non-infectious Posterior Uveitis

Roger Steinert, Pioneer in Ophthalmology, Dies at 66

Carl Zeiss Meditec to Build New Innovation Center in Northern California

Gottlieb Says FDA to Publish List of Drugs Without Generic Competition

REGENXBIO Announces Phase I Clinical Trial for RGX-314 Gene Therapy for Wet AMD

Belkin Laser Closes Nearly $5 Million Financing Round

Valeant in Talks to Sell Surgical Business to Carl Zeiss Meditec

Coming soon

2017 Cataract Surgical Equipment: A Global Market Analysis for 2016 to 2022