BoGene Biotech Files IPO Prospectus: Pioneering Redosable Gene Therapy for Lifelong Treatment Solutions

After more than 30 years of development, gene therapy has gradually matured, becoming a new focus in the pharmaceutical industry following small molecules, recombinant proteins, and antibodies.

Adeno-associated virus (AAV) is a popular vector in the field of gene therapy, and gene therapies based on AAV-mediated delivery of target genes hold a significant position across the entire gene therapy landscape. With the successive approvals of three AAV-based drugs—Glybera, Luxturna, and Zolgensma—in Europe and the United States starting in 2012, industry confidence has been rekindled. Sixteen months after its launch, Zolgensma achieved sales of $1.027 billion, demonstrating that the success of gene therapy extends beyond clinical efficacy into commercial viability, thereby ushering in a wave of commercial enthusiasm.

AAV-based gene therapy is not flawless; as research has deepened, a series of issues have been identified, including toxicity associated with high-dose administration, the potential risk of abnormal clonal cell proliferation, and concerns regarding the permanence of mediated long-term expression.

As the founder of the startup Boyin Biotech, Dr. Pan Yukun possesses a profound understanding of this field. Dr. Pan earned his bachelor’s degree from the School of Life Sciences at Fudan University and began studying in the gene therapy research group at the same institution in 2005. He subsequently completed his training as a Medical Scientist Fellow at Yale University, obtained his Ph.D. from the Department of Genetics at Yale University, and conducted postdoctoral research at the Howard Hughes Medical Institute (HHMI) at Yale. With 15 years of experience in gene therapy and genetic engineering, Dr. Pan is committed to developing comprehensive gene therapy solutions spanning the entire lifecycle, leveraging his unique insights into medical molecular genetics.

Dr. Pan Yukun, Founder of Boyin Biology

On the eve of 2018, Pan Yukun led early-stage investments in cross-border AAV gene therapy intellectual property projects and license-in transactions in China. Now, standing at the new starting point of Boyin Biologics, Pan Yukun has decided to lead his team to directly address and overcome the critical challenges in the field of AAV gene therapy.

Adeno-associated virus (AAV) is a non-enveloped virus with a diameter of approximately 20 nm. Due to its advantages, including high-efficiency gene delivery, tissue tropism, and lack of significant pathogenicity, AAV has become one of the most widely used vectors in the field of gene therapy.

However, behind the seemingly perfect facade, AAV viral vectors also present an unavoidable technical challenge for gene therapy: the inability to administer repeat doses. Pan Yukun told VCBeat, “From any perspective, AAV is ultimately an exogenous pathogen. Its initial entry into the human body triggers the immune system to produce antibodies. Consequently, if a second infusion of the vector-based drug is required, or if gene therapy is administered to patients who already have pre-existing neutralizing antibodies, the vector will be directly cleared by the immune system, making therapeutic efficacy uncertain.”

“After AAV viral vectors deliver therapeutic genes into target cells, they exist in the nucleus of the target cells as extrachromosomal episomes and undergo independent expression. However, this expression is gradually diluted with individual growth and cell division, leading to a waning therapeutic effect.” Pan Yukun emphasized, “It is precisely due to these characteristics of AAV that we have observed age restrictions, such as requiring patients to be older than 18 years, in the inclusion criteria for some AAV gene therapy clinical trials. If patients fail to achieve the desired therapeutic effect after the initial dose, we cannot simply administer a second dose to cumulatively achieve the expected efficacy.”

In 2020, Dr. Pan Yukun co-founded Shanghai Boyin Biotechnology Co., Ltd. (hereinafter referred to as “Boyin Bio”) with two senior experts in the field, spearheading the Gene Therapy 2.0 revolution and focusing on addressing the critical challenge of non-readministrability within the sector.

BoYin Biotechnology has incorporated non-viral vector technologies into its technical strategy to enable repeat dosing of gene therapies. Dr. Pan Yukun stated that the company is actively advancing R&D and early preclinical pipeline development, with progress proceeding smoothly. BoYin Biotechnology expects to obtain large-animal in vivo study data by the end of 2021, and will submit regulatory applications for its candidate pipelines in 2022.

“We aim to develop a gene therapy drug that offers longer-lasting efficacy compared to traditional small-molecule and antibody drugs, while allowing for repeated administration from infancy through adolescence into adulthood, with cumulative therapeutic effects,” emphasized Dr. Pan Yukun. “We aspire to provide gene therapy solutions that cover the entire lifespan.”

Although China’s gene therapy sector currently lags behind its international counterparts, domestic gene therapy companies can pursue innovation and progress through two strategic approaches. Dr. Pan Yukun explained: “First, learn from Europe and the United States by pushing technological innovation and breakthroughs to their fullest potential, achieving global recognition. Second, reduce costs to develop ‘gene therapies that are affordable for the general public.’ Boyin Biologics has strategically positioned itself by taking both of these considerations into account.”

“All three of us founders come from the field of gene therapy. We have known each other for over ten years and share a common ideal, or perhaps an obsession: to develop drugs, specifically gene therapies,” said Dr. Pan Yukun with a smile. “We are currently working in an innovative technological sector characterized by the need to integrate multiple technologies into the final product, making it a highly interdisciplinary field.”

Boyin Biologics is building a young, dynamic team whose members come from diverse backgrounds but share a strong desire to step out of their comfort zones and integrate resources to strive toward a common goal. Furthermore, industry experience is highly valued. Recently, Boyin Biologics appointed Dr. Lu Yuan as Vice President of R&D. Dr. Lu is a talented professional with substantial industry experience and a willingness to tackle the challenges associated with developing repeatable injectable therapies. She earned her Ph.D. from the University of Florida, a renowned institution in gene therapy, and previously served as Assistant Director in the Oncology and Immunology Business Unit at WuXi AppTec, as well as Associate Director of R&D at a leading domestic gene therapy biotechnology company, where she reported directly to the Chief Scientific Officer (CSO). By joining Boyin Biologics as Vice President of R&D, Dr. Lu will significantly accelerate the company’s preclinical product pipeline and bring valuable industry expertise to the organization.

It is reported that Boyin Biology has secured tens of millions of RMB in angel-round funding from Fengrui Capital and Kaifeng Venture Capital, and its startup project has been selected for the Shanghai Pujiang Talent Program. The company also plans to initiate a new round of pre-A financing, with the proceeds primarily allocated to expanding the existing team and advancing its preclinical candidate pipeline.