NGGT Bio: Innovating with Differentiation to Deliver Clinically Needed Gene Therapies

Since 2017, the Cell and Gene Therapy (hereinafter referred to as CGT) industry has continued to achieve breakthrough progress. Milestone CGT products such as Kymriah, Luxturna, and Zolgensma have successively received FDA approval for market launch, delivering impressive commercial performance, while investment and financing in the related fields have remained robust. Currently, the CGT industry has become one of the most promising global frontiers in pharmaceuticals.

China has demonstrated remarkable agility in keeping pace with developments in this cutting-edge field. Since 2015, the number of cell and gene therapy (CGT)-related clinical trials in China has grown rapidly—according to data from a Frost & Sullivan report,From 2015 to 2020, China conducted approximately 250 CGT clinical trials, becoming the region with the second-highest number of clinical trials after the United States. With a compound annual growth rate (CAGR) exceeding 60%, it ranked first globally.

Behind the booming development of the CGT market is a large number of overseas talents returning to China to start businesses, bringing with them cutting-edge technologies and extensive industry experience. Suzhou NoJieBei Biotechnology Co., Ltd. (NGGT, hereinafter referred to as “NoJieBei”) is one of the most representative companies among them—established byDr. Jiang Lixin and Dr. Qu Guang, senior pharmaceutical scientists with successful end-to-end experience in the gene therapy sectorCo-founded in March 2020, it is committed to becoming a globally leading gene therapy company with international influence.

VCBeat learned that the two founders of NoJieBeiWhether in the early-stage development of gene therapy projects, the large-scale manufacturing processes and quality standard establishment for viral vectors, or the conduct of international multi-center clinical trials and marketing authorization applications for gene therapy products,All possess successful experience across the entire industry chain. This lays a solid foundation for NoJieBei Biotechnology to independently develop more safe, effective, and accessible gene therapy drugs.

The extensive experience of the company’s core team in the large-scale manufacturing of gene therapy products gives NoJieBei greater technical advantages and accumulated expertise in future gene therapy drug development compared to typical startups.

Recognizing the strong scientific and industrial background of NoJieBei’s core team, and expressing confidence in the prospects of the gene therapy industry, multiple renowned investment institutions—including Aurora Venture Capital, Yipu Capital, Yuansheng Venture Capital, and Weidu Capital—participated in NoJieBei’s Series A financing round, which raised $30 million (approximately RMB 200 million). The funds from this round will primarily be used to prepare clinical products for NoJieBei’s multiple gene therapy pipelines and to construct cGMP-compliant production facilities.

To gain a deeper understanding of NoJieBei’s advantages in its technology platform and its product pipeline layout, VCBeat interviewedDr. Qu Guang, Co-founder of NoJieBeiConducted an exclusive interview.

Dr. Qu Guang, Co-founder of NoJieBei

Dr. Qu Guang, Co-founder of NoJieBei, earned his Ph.D. from The Ohio State University in the United States and conducted postdoctoral research in the laboratory of Professor James Wilson, a renowned expert in gene therapy. With over 30 years of experience in the development of gene therapy drugs, Dr. Qu previously held positions at Avigen, one of the earliest gene therapy companies in the U.S., and the Viral Vector Core at Children’s Hospital of Philadelphia (CHOP). In these roles, he was responsible for process and new technology development for large-scale viral vector manufacturing, with a particular focus on adeno-associated virus (AAV) vector process development.

In 2013, Spark Therapeutics was founded, with Dr. Guang Qu serving as the Head of Viral Vector Manufacturing Process and New Technology Development. In this role, he oversaw process development for all of the company’s products and led the end-to-end development and establishment of manufacturing processes for Luxturna, the world’s first ophthalmic gene therapy product (approved by the FDA in 2018), from laboratory scale to commercial launch. Additionally, he produced the lentiviral vectors used in the pioneering CAR-T therapy led by Dr. Carl June at CHOP, which was among the earliest such trials conducted globally. To date, Dr. Qu has led process development for one approved gene therapy product, two products currently in Phase III clinical trials, and three products in Phase I clinical trials.

The following is an excerpt from the interview:

VCBeat: Could you please provide a brief overview of NoJieBei’s current key pipeline portfolio and strategic approach?

Dr. Qu Guang:NoJieBei's current pipeline layout strategy is“Progressive advancement,” “integration of short- and long-term strategies,” and “differentiated pipeline layout based on innovative technologies.”Initially, we will focus on developing products targeting monogenic diseases, while also engaging in international cooperation through “license-in” arrangements to introduce additional products and further enrich our portfolio. As our product pipeline advances and achieves milestone results, Suzhou NoJieBei Biotechnology Co., Ltd. will begin to lay out products for polygenic diseases, which involve more complex pathogenic mechanisms and longer R&D cycles. The company’s development pace will remain aligned with that of the scientific community, gradually completingFrom Monogenic Diseases to Polygenic Diseases, from Rare Diseases to Common Diseasesbreakthrough.

In addition, NoJieBei’s unique"One Target, Two Drugs"patented technology, which can provide effective therapeutic options for various genetic disorders that currently lack treatment methods worldwide. Currently, we primarily focus onOphthalmology (crystalline retinitis pigmentosa), metabolic diseases (phenylketonuria), and neurological disordersDevelop relevant gene therapy drugs.

Crystalline retinitis pigmentosa is a monogenic hereditary disease that causes vision loss and blindness due to lipid metabolism disorders in retinal pigment epithelial cells. It is more prevalent in Asian populations, while related research in Europe is relatively limited, providing an excellent opportunity for Suzhou NoJieBei Biotechnology Co., Ltd. to develop therapies for this condition. As a domestic enterprise, we aim to prioritize addressing clinical needs in China and the Asia-Pacific region, while also striving for breakthroughs in innovative drug development. Phenylketonuria (PKU) is an amino acid metabolism disorder and the most common type within this category. Although classified as a rare disease, the global number of patients is not insignificant, with an estimated worldwide incidence of approximately 1 in 15,000.

VCBeat: Given the extensive experience you and other core team members have in large-scale manufacturing processes and quality standard development for viral vectors, will NoJieBei consider strategically prioritizing the development of its CDMO business alongside its gene therapy drug R&D efforts?

Dr. Qu Guang:Yes, in fact, we are currently establishing an 8,500-square-meter production facility in Suzhou. Construction is scheduled to commence this June, and we anticipate moving into the facility around November of this year. Our decision to build this production plant is driven by two key considerations: first, to meet NoJieBei’s own manufacturing needs for gene therapy-related products; and second, to leverage our team’s expertise in gene therapy product manufacturing processes and quality standards research, thereby providing professional CDMO outsourcing services to more domestic and international CGT companies.

Our goal remains to position ourselves internationally. There is a saying in the United States that goes,“Your process is your product”, meaning that a product's manufacturing process directly impacts its quality. Dr. Jiang and I bring extensive personal experience in the gene therapy field, as well as proven success in developing gene therapy products across the entire value chain. This will give NoJieBei certain technical advantages and accumulated expertise over typical startups as it establishes its CGT CDMO business.

VCBeat: Over the past few decades, the cell and gene therapy (CGT) industry has experienced significant ups and downs. As a scientist who has witnessed both the “troughs” and “peaks” of the CGT industry’s development, how do you view its current resurgence? What are your thoughts on the current state and future trajectory of this field?

Dr. Qu Guang:Objectively speaking, as an emerging technology in the biopharmaceutical sector, gene therapy remains at a relatively early stage of development from an industrialization perspective. This field is fraught with uncertainties, necessitating deeper understanding and exploration of disease pathogenesis and the in vivo behavior of viral vectors, while also addressing various potential challenges.

But at the same time, I still believe that the field of gene therapy is a“Thriving Now” and “Promising Future”Gene therapy, an emerging field of development, offers novel therapeutic concepts and modalities for rare diseases, chronic conditions, cancers, and other refractory disorders. It provides the potential for single-dose administration with long-term benefits—advantages often unattainable by conventional drugs—and holds promise for achieving curative outcomes in certain diseases. Compared with traditional treatment approaches, gene therapy represents a significant leap and breakthrough.

VCBeat: How do you view the current development constraints and challenges facing the gene therapy industry? What are the core barriers or difficulties in this sector? How does NoJieBei plan to leverage its team’s strengths to provide solutions for these pain points in the future?

Dr. Qu Guang:In the current stage of gene therapy, there are certainly many areas that require further learning and breakthroughs. However, like any other field of drug development, the progress of gene therapy is inevitably driven by scientific breakthroughs made in response to problems identified during its development. Moreover, with deepening scientific research and substantial involvement of industrial capital and resources, many new challenges emerging in the gene therapy sector can be anticipated. As scientists continue to overcome one challenge after another, this field will steadily mature. This is a normal course of development.

As for specific difficulties and challenges, I believe that gene therapy products developed for different disease areas face distinct challenges. Taking the well-known production capacity constraints associated with viral vectors in gene therapy as an example, the limitations imposed on ophthalmic therapies differ from those on hepatic therapies, as these two types of treatments have different requirements for viral load. Therefore, the production capacity constraints of viral vectors pose a lesser limitation for some product pipelines, while posing a greater limitation for others. In my view, future solutions to such issues will necessarily involve the development of more advanced and mature technologies, such asDevelopment of New Serotypes, Exploration of Novel Routes of Administration and New Formulation TypesWait, there should be some breakthroughs in this area.

VCBeat: How do you view the current development status of the CGT market both domestically and internationally? How are Chinese companies currently performing in CGT drug R&D? As high-caliber scientists with extensive industry experience, like yourself, continue to return to China, bringing back the most advanced theoretical knowledge, technologies, and product manufacturing processes from the global industrial and academic sectors, how do you assess the potential for domestic companies to catch up in these areas in the future?

Dr. Qu Guang:Europe and the United States were the pioneers in gene therapy research, boasting extensive and mature reserves of knowledge and technology related to cell and gene therapy (CGT), as well as substantial experience in regulatory oversight and the formulation of relevant regulatory policies by government authorities. However, when tracing the development history of China’s gene therapy industry, our country’s entry into this field was not significantly delayed.

Everyone says“Favorable timing, favorable location, and harmonious human relations”From the perspective of national policy, after more than a decade of encouragement and standardized policy guidance, China’s gene therapy sector has entered a stage of standardized development. In terms of talent and technology, a large number of outstanding overseas professionals have returned to China, and corporate technological capabilities have gradually accumulated to a significant level. From the standpoint of the capital market, China’s domestic capital market is becoming increasingly mature and is showing an increasingly open and welcoming attitude toward the development of emerging technologies. All these factors have laid the foundation for the rapid development of gene therapy drugs in China.

Moreover,As a country with a large population, China has a significant number of patients suffering from severe conditions such as rare diseases, chronic diseases, and cancer, resulting in substantial unmet medical needs. These patients require truly effective therapeutic agents, particularly high-quality drugs in the field of gene therapy.Broad market demand is firmly rooted here, and given time, China’s gene therapy industry will undoubtedly achieve substantial progress. Meanwhile, we must solidly advance research efforts across all fronts to ensure the healthy and orderly development of this field.

Some gene therapy companies in China currently,From the perspective of pipeline layout, there is a high degree of redundancy and significant homogenization, which results in substantial waste of R&D resources. This may lead to a narrowing of competitive tracks in the future and is detrimental to the long-term development of China’s gene therapy industry.While the risks associated with new drug development are indeed high, there must be individuals willing to undertake the research and development of innovative therapies. Such courage is essential to driving the gene therapy field further and fostering its healthier growth. Suzhou NoJieBei Biotechnology Co., Ltd. hopes to collaborate with industry peers to jointly promote the maturation of China’s gene therapy sector and contribute its share to this endeavor.