Congratulations to GenAns Biotech on FDA Clearance to Advance Its GA001 Pipeline into Phase II Clinical Trial

Recently, GenAns Biotech Co., Ltd. (“GenAns Biotech”) achieved a major R&D milestone: its independently developed gene therapy drug, GA001 Injection, for the treatment of retinitis pigmentosa, has demonstrated favorable safety in previous clinical studies. Following review by the U.S. Food and Drug Administration (FDA), GA001 has officially received clinical trial clearance and has been approved to proceed into a Phase II clinical study to evaluate its efficacy and safety. As a strategic partner, PackGene Biotech extends its sincere congratulations on this milestone achievement.

FDA clearance of the clinical trial application for GA001 marks an important step in GenAns Biotech’s international drug development journey. It not only further consolidates GenAns Biotech’s leading position in gene therapy for ophthalmic and major brain disorders, but also brings new therapeutic hope to patients worldwide who are blinded by retinitis pigmentosa. PackGene Biotech is honored to have supported the clinical development of GenAns Biotech’s gene therapy program by providing high-quality CMC services. Looking ahead, PackGene Biotech will continue to uphold its mission of “making gene therapy affordable and accessible to the public,” further deepen its expertise in vector process and technology development, and help advance more high-quality programs.

About GA001 Injection

GA001 Injection is an innovative gene therapy drug independently developed by GenAns Biotech for patients with blindness. Its core technology is a highly sensitive novel photosensitive protein discovered by the GenAns Biotech team. The drug uses an adeno-associated virus vector to deliver the gene encoding this novel photosensitive protein to retinal ganglion cells, thereby restoring their photosensitivity and ultimately helping patients regain vision.

Progress of Previous IIT Study

An investigator-initiated clinical study (IIT) of GA001 Injection conducted at Beijing Tiantan Hospital completed enrollment and dosing of its first subject in January 2024. To date, the study has achieved interim results:

All patients experienced varying degrees of visual improvement, with some patients able to recognize letters and patterns, resulting in significant improvements in daily living and independent living abilities.

No drug-related serious adverse events were observed, preliminarily confirming GA001’s favorable safety profile and significant therapeutic potential.

About GenAns Biotech

GenAns Biotech was founded by Professor Luo Minmin, Director of the Chinese Institute for Brain Research, Beijing. The company is committed to developing innovative gene therapy solutions for central nervous system and ophthalmic diseases. Its R&D pipeline covers multiple diseases, including retinitis pigmentosa, epilepsy, refractory pain, depression, neurodegenerative diseases, and malignant brain tumors, with the aim of helping patients restore durable neurological function and providing effective therapeutic options for patients with refractory central nervous system and ophthalmic diseases.

GenAns Biotech will continue to uphold the philosophy of original innovation, continuously enhance its core competitiveness, and strive to become a global leader in brain science and ophthalmic gene therapy. The company is dedicated to translating innovations in life sciences into meaningful improvements in human health, creating a better present and future for every life.