Congratulations on FDA Approval for the GA001 Pipeline of Jianda Jiuzhou to Enter Phase II Clinical Trials

Recently, Jianda Jiuzhou (Beijing) Biotechnology Co., Ltd. (“Jianda Jiuzhou”) reached an important R&D milestone. Its independently developed gene therapy product GA001 Injection, being investigated for retinitis pigmentosa, has shown encouraging safety results in preclinical and early clinical studies. Following evaluation by the U.S. Food and Drug Administration (FDA), GA001 Injection has officially obtained authorization to proceed with clinical research, and is permitted to initiate Phase II studies to evaluate efficacy and safety.

As a strategic partner, Paiten Bio extends its sincere congratulations on this milestone achievement.

FDA IND approval for GA001 Injection marks a key step toward internationalization

FDA approval of the GA001 clinical trial application marks a significant step forward in Jianda Jiuzhou’s new drug development toward international standards. It not only further strengthens Jianda Jiuzhou’s leading position in ocular and major central nervous system (CNS) gene therapy fields, but also brings renewed hope for patients worldwide affected by retinitis pigmentosa.

Paiten Bio is honored to support Jianda Jiuzhou in advancing its gene therapy clinical program by providing high-quality CMC services. Going forward, Paiten Bio will continue to uphold its mission of “making gene therapy more affordable for the public,” further deepen R&D in vector manufacturing and technical development, and help advance more high-quality programs.

About GA001 Injection

GA001 Injection is a gene therapy new drug independently developed by Jianda Jiuzhou for patients with blindness. Its core technology is a high-sensitivity novel photosensitive protein discovered and developed by Jianda Jiuzhou’s research team.

The drug delivers the gene encoding the novel photosensitive protein to retinal ganglion cells via an adeno-associated virus (AAV) vector, with the aim of restoring their light-sensing function and ultimately helping patients regain vision.

Progress of the pre-investigator-initiated trial (IIT)

The investigator-initiated clinical study (IIT) for GA001 Injection conducted at Beijing Tiantan Hospital was completed for the first subject enrollment and administration in January 2024. To date, the study has produced interim results:

• All patientsachieved vision improvement to varying degrees. Some patients were able to recognize letters and patterns, leading to a significant enhancement in daily living routines and independent living abilities.
• No drug-related serious adverse eventshave been observed, preliminarily confirming that GA001 demonstrates good safety and promising therapeutic potential.

About Jianda Jiuzhou

Jianda Jiuzhou was founded by Prof. Rong Minmin, Director of the Beijing Institute for Brain Science and Brain-Inspired Intelligence. The company is committed to developing innovative gene therapy solutions targeting diseases of the central nervous system and the eye.

Its R&D pipeline covers multiple indications, including retinitis pigmentosa, epilepsy, intractable pain, depression, neurodegenerative diseases, and malignant brain tumors. The company aims to help patients restore durable neural function and provide effective treatment options for patients with refractory CNS and ocular diseases.

Jianda Jiuzhou will continue to pursue the philosophy of source innovation, continuously enhance its core competitiveness, and work toward becoming a global leader in brain science and ophthalmic gene therapy. By transforming innovative outcomes in the life sciences into benefits for human health, the company strives to create a better present and future for every life.