Neurophth is China’s first gene therapy company for ophthalmic diseases: with its mission to discover and develop gene therapies for patients suffering from genetic diseases. It is headquartered in Wuhan with subsidiaries in China (Shanghai and Suzhou) and US (San Diego, California).
The new 8,000 square-meter gene therapy-focused current good manufacturing practice (cGMP) production facility in Suzhou includes two drug substance production suites, two filling lines, a cell banking suite, a technology transfer laboratory and a quality-control laboratory.
The facility, located at the BioBay life science cluster of around 400 high-tech start-ups, went from design to completion in a year.
Neurophth's proprietary manufacturing processes will use single-use equipment for quick and effective product changeovers and equipment cleaning.
The in-house production capacity will eliminate the dependence on CDMOs, which the company says will allow it to speed up the process of bringing products from 10 programs from the research stage to the clinic and ultimately to patients.
"Neurophth's own cGMP production facility is designed to enable us to reach manufacturing scales suitable for future commercial production and the facility is also co-located with our development laboratories," said Bin Li, chairman and founder at Neurophth.
"This integrated approach allows for more efficient development, manufacturing and release of vectors for new indications and we will continue to adhere to the patient-centric innovation and contribute to the development of Suzhou BioBAY."
The Suzhou facility already employs over 100 people.
Neurophth’s AAV platform, which has been published in Nature - Scientific Reports, Ophthalmology, and EBioMedicine, has delivered proof-of-concept investigator-initiated trials data of 198 subjects with investigational gene therapies in the retina.
Its most advanced investigational candidate, NR082 (NFS-01 project, rAAV2-ND4), in development for the treatment of ND4-mediated Leber hereditary optic neuropathy (LHON), has been granted orphan drug designation (ODD) by the US FDA and its IND evaluating NR082 in a Phase 1/2/3 clinical trial has also been approved by the China NMPA in March 2021 with the first patient being dosed in June 2021.
The pipeline also includes ND1-mediated LHON, autosomal dominant optic atrophy, optic neuroprotection (eg. glaucoma), vascular retinopathy (eg. diabetic retinopathy), and five other preclinical candidates.