Time flies, and the seasons flow.
Suddenly we have passed 2022...
This year has been a year of hardship and testing,
but it has also been a year of struggle and harvest.

In 2022, as more and more rare disease gene therapy products move from scientific research to clinical practice, the treatment and rehabilitation of rare disease groups have gradually received more attention from all walks of life. Gene therapy drugs carry more hope for rare disease patients due to the long-term nature of their treatment, which also puts forward new requirements for GeneCradle's drug development and improvement. GeneCradle people have always adhered to the concept of "believing that gene therapy will solve more disease problems and benefit human health", and in 2022, the GeneCradle team has promoted multiple core products to the clinical trial stage and achieved phased milestones, and is committed to promoting Chinese gene drugs from basic to clinical and market to meet unmet clinical needs.

Looking back at 2022

Be down-to-earth and forge ahead

2022 is a challenging year for new drug research and development. In a rapidly changing environment, GeneCradle people have kept their mission in mind and worked closely together to persevere and strive for progress in the development of gene therapy drugs for rare diseases, achieving a series of milestones.

Core pipeline clinical licensing and clinical research

In 2022, the GeneCradle team completed preclinical studies of three gene therapy drugs, including GC101 adeno-associated virus injection, GC304 adeno-associated virus injection, and GC301 adeno-associated virus injection, and submitted clinical trial applications to the Drug Evaluation Center of the National Medical Products Administration. By the end of the year, four clinical trial implied licenses had been obtained:

GC101 adeno-associated virus injection for type 1 spinal muscular atrophy (CXSL2200324)

GC101 adeno-associated virus injection for type 2 spinal muscular atrophy (CXSL2200400)

GC304 adeno-associated virus injection for hypertriglyceridemia with recurrent acute pancreatitis (CXSL2200432)

GC301 adeno-associated virus injection for early-onset Pompe disease (CXSL2200480)

GeneCradle's clinical operations and medical teams will take over the baton of preclinical research for the subsequent development of the above projects, and will take the lead in launching systematic preparations for clinical trials, hoping to enter the clinical recruitment stage in the first half of 2023.

In addition to the above-mentioned formal clinical trials, GeneCradle conducted several investigator-initiated clinical studies (IITs) in 2022, covering patients with type 1, type 2 and type 3 spinal muscular atrophy and early-onset Pompe disease. Clinical observations showed that patients showed good drug safety and significant improvement in various indicators after treatment.

Qualification certification and industry recognition

In 2022, GeneCradle obtained the national high-tech enterprise qualification recognized by the Torch Center of the Ministry of Science and Technology, the qualification of a technology-based small and medium-sized enterprise recognized by the Ministry of Science and Technology, the qualification of a "specialized, refined and innovative" enterprise in Beijing, and the Beijing Intellectual Property Pilot Demonstration Unit certification issued by the Beijing Intellectual Property Office.

The qualification recognition of the national and Beijing science and technology departments is an encouragement to the GeneCradle team's independent research and development capabilities, scientific and technological achievements transformation and intellectual property system, and is also a driving force to continuously motivate the GeneCradle team to continue scientific research and innovation in subsequent work.

Quality Management Policy and System Certification

In 2022, GeneCradle formulated the quality management policy of "originating from demand, daring to innovate, focusing on design, strict in verification, sophisticated in manufacturing, continuous improvement, and benefiting patients", covering the process from R&D project establishment of gene therapy drugs to R&D technology innovation, and then to drug design, verification, manufacturing and listing to meet patient needs, and formulated the company's management and execution standards and requirements. On this basis, the company has successively passed the intellectual property management system audit and certification related to gene therapy R&D and ISO9001 quality management system certification, and obtained the "Intellectual Property Management System Certification Certificate" and "Quality Management System Certification Certificate" respectively, which indicates that the company attaches great importance to quality management and has initially established a standardized, standardized and scientific modern enterprise management system.

Improve the functions of production and clinical departments

In 2022, GeneCradle completed the functional improvement of the GMP production site, built upstream and downstream production, quality inspection and quality research departments around the production and preparation of AAV products, and successfully completed several batches of GMP-level AAV product production, providing product support for the project's clinical application and clinical trials; at the same time, the department functions of clinical operations and medical affairs were improved, and a clinical quality system and clinical project standard operating procedures were established to promote the clinical development of the project and enhance patient benefits from the institutional level. At the end of 2022, the company formed an organizational structure with the R&D center and the production center as the main business departments, and cooperated with several major support departments including the quality management department, project management department, strategic development department and comprehensive operation department. The company's organizational construction and personnel division of labor are becoming increasingly perfect and clear.

Practicing social responsibility

The treatment needs of patients have always been the driving force of drug development. "Derived from needs, beneficial to patients" is the original intention and goal of the GeneCradle team. We work hard to make drugs, pay more attention to clinical applications, pay attention to the real benefits of treatment to patients, and pay attention to cure and rehabilitation.

In 2022, the GeneCradle charity affairs team carried out patient-centered rare disease gene therapy collaboration and exchanges, and used science and technology charity to support rare disease research and publicity; assisted the Beijing Yicheng Cooperation and Development Foundation in holding the "3.18 Rare Disease Science and Technology Charity Annual Conference and Project Application Guidelines Online Release Conference", which received widespread attention from all walks of life; supported the establishment of the Ruixi Rare Disease Special Fund, and donated to fund rare disease scientific research projects, guiding social resources and scientific research forces to gather on rare disease-related research in the form of science and technology charity, and practicing the social responsibility of the GeneCradle team with actions.

Moving towards 2023

Working together to protect hope

Looking back at the path we came from, the vast expanse of greenery stretches across the horizon.

In the past year, GeneCradle people and the rare disease community have watched over each other together.

On the road to developing new drugs for rare diseases,

Always be grateful and enterprising,

With self-reliance and determination to move forward,

Respect the courage of every life to persevere.

Together with the rare disease community,

Welcome a tomorrow full of hope and warmth!