Leber Congenital Amaurosis-2 Gene Therapy Trial Doses First Patient


The company expects to provide initial data from the trial in the second half of this year.

The first patient has been dosed in a phase 1 clinical trial for Frontera Therapeutics’ FT-001, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Leber Congenital Amaurosis-2 (LCA-2).1,2

FT-001 is administered as a single injection to the subretinal space of the eye and is intended to provide a functional copy of RPE65, the disease-targeted gene, to retinal cell nuclei. The investigational new drug (IND) application for the trial was originally cleared by China’s Center for Drug Evaluation (CDE) in September of last year. The study, which is being carried out in China, is assessing safety, tolerability, pharmacokinetics, and therapeutic effects for patients with LCA-2. The company expects to provide initial data from the trial in the second half of this year. In addition to the IND cleared by the CDE, an IND for FT-001 was also cleared by the FDA in April of last year.

“This is an exciting achievement for Frontera Therapeutics as we begin our first clinical trial and take a significant step forward as a company,” Yong Dai, PhD, chief executive officer and founder, Frontera Therapeutics, said in a statement regarding the dosing of the first patient.1

Another ophthalmologic therapy in Frontera’s pipeline is FT-003, an investigational gene therapy intended to treat neovascular age-related macular degeneration (nAMD), for which a phase 1 clinical trial is planned (NCT05611424). The open-label, single-center, dose-escalation trial will seek to recruit approximately 18 patients aged 45 years or older who have been diagnosed with nAMD. Participants are required to have active choroidal neovascularization and a best corrected visual acuity (BCVA) in the study eye of 53 letters or fewer. Patients with other intraocular disease in the study eye that could interfere with improvement of visual acuity will be excluded from participation. Participants in the study will be assigned to 1 of 3 dose levels arms. The study’s primary end point is the incidence and severity of adverse events. The secondary end point is the change in BCVA from baseline in the study eye.

“Since Frontera’s founding, we have rapidly progressed a significant number of programs into IND enabling studies,” Dai said in a September 2022 statement.2 “This year, we have advanced our lead program, FT-001, and have received both US FDA and China CDE IND clearances. Building on our product development momentum, we intend to advance a number of programs into clinical studies in the next 6 to 9 months. We will also leverage our APEX Technology & Manufacturing platform to create new innovative programs targeting large global markets such as cardiovascular, metabolic, and central nervous system diseases.”

Frontera Therapeutics announced the completion of a $160 million round of Series B financing in July 2022.3 Among the investors were Boyu Capital, Sequoia China, OrbiMed, and Creacion Ventures. A previous series A round raised $35 million, with OrbiMed and Creacion Ventures listed as contributors. The company noted that it has raised $195 million in total since its founding in 2019.

1. Frontera Therapeutics doses first patient in phase 1 clinical trial for gene therapy FT-001 for the treatment of leber congenital amaurosis-2. News release. Frontera Therapeutics. January 6, 2023. https://www.fronteratherapeutics.com/frontera-therapeutics-doses-first-patient-in-phase-1-clinical-trial-for-gene-therapy-ft-001-for-the-treatment-of-leber-congenital-amaurosis-2/ 
2. Frontera Therapeutics receives additional IND clearance for its lead program FT-001. News release. Frontera Therapeutics. September 21, 2022. https://www.fronteratherapeutics.com/frontera-therapeutics-receives-additional-ind-clearance-for-its-lead-program-ft-001/ 
3. Frontera Therapeutics completes $160 million series B financing to fund clinical development and manufacturing capabilities. Frontera Therapeutics. July 19, 2022. https://www.fronteratherapeutics.com/frontera-therapeutics-completes-160-million-series-b-financing-to-fund-clinical-development-and-manufacturing-capabilities/
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