Genecradle Therapeutics Receives Multiple Annual Industry Attention Drug Awards, Ascends the Innovation Influence List

2024-12-19

Recently, the "6th China Biomedical Industry Innovation and Transformation Forum and the 1st Guangdong-Hong Kong-Macao Greater Bay Area Biomedical Industry Innovation and Transformation Forum" was grandly held in Guangzhou. The event gathered top experts and industry elites from the domestic and international biomedical fields to discuss the innovation and development of the biomedical industry. At this forum, the "2024 China Biomedical Industry Innovation Influence List" was officially released, and Beijing Genecradle Therapeutics Technology Co., Ltd. (hereinafter referred to as "Genecradle Therapeutics") won multiple awards for its outstanding R&D achievements and innovative capabilities.

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Genecradle Therapeutics' two independently developed adeno-associated virus (AAV) injections, GC301 and GC101, were awarded the "Annual Industry Attention Rare Disease Drug" and "Annual Industry Attention Gene Drug" awards, respectively, fully showcasing Genecradle Therapeutics' innovative strength and industry influence in the field of gene therapy.

Introduction to Genecradle Therapeutics' Award-Winning Drugs

GC301 adeno-associated virus injection is an AAV gene therapy drug for Pompe disease, with indications including infantile-onset Pompe disease (IOPD) and late-onset Pompe disease (LOPD). The drug achieves widespread compensation for GAA enzyme gene defects throughout the body through a one-time intravenous injection, covering key tissues such as the liver, heart, skeletal muscles, and central nervous system. GC301 is not only the world's first drug using AAV delivery technology to treat infantile Pompe disease but also successfully completed the first subject dosing in a registered clinical trial for late-onset Pompe disease AAV gene therapy in China. In clinical trials conducted, GC301 has shown significant safety and efficacy.

GC101 adeno-associated virus injection is the first domestically developed AAV gene therapy drug for spinal muscular atrophy (SMA) administered intrathecally, with indications including Type 1 SMA, Type 2 SMA, and Type 3 SMA. GC101 injection delivers the therapeutic gene SMN1 precisely to the central nervous system and stably expresses it in motor neuron cells through a one-time intrathecal injection, thereby improving the function of affected cells such as motor neurons and enhancing the respiratory and motor abilities of SMA patients. With good safety and efficacy demonstrated in clinical trials, GC101 has been included in the Breakthrough Therapy list by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.

While focusing on the research and clinical application of gene drugs, Genecradle Therapeutics also places great emphasis on talent team building and training. In this industry influence list selection, Ma Wenhao from Genecradle Therapeutics was awarded the "2024 China Biomedical Industry Innovation Influence List · U45 Influential Young Talent Award." This is not only a recognition of individual ability but also an affirmation of the company's talent training system.

Genecradle Therapeutics will continue to adhere to the development concept driven by innovation, stick to the original intention of "originating from needs, benefiting patients," continuously explore new frontiers in gene therapy, and promote the continuous progress of medical science and technology. In the future, the company will further deepen cooperation between industry, academia, research, and application, commit to providing more and better gene therapy solutions for patients worldwide, and work with industry peers to create a better future for human health.

Genecradle Therapeutics Honored as TOP2 in the Biomedical Field at the 8th International Frontier Technology Competition

Genecradle Therapeutics' GC101 Injection Recognized as a Breakthrough Therapy

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