RuiFeng Bio Advances AI-Driven Gene Editing Therapy with Successful Cure of Adult β-Thalassemia and Files IPO Prospectus

Since the completion of the Human Genome Project, we have been experiencing a rapid wave of industrial development across three tiers: reading genes, editing genes, and, in recent years, designing genomes de novo. In reality, the development of gene-editing therapeutics remains in its early stages, with indications limited to genetic disorders and clinical approvals being exceedingly rare; in China, there is currently only one product with an Investigational New Drug (IND) application. In recent years, as global regulatory agencies have continued to refine regulations and guidelines in a targeted manner, gene-editing therapy has become a sector in which major international pharmaceutical companies are deeply involved and place high expectations. Furthermore, this therapeutic approach is gradually expanding into areas of high-prevalence complex diseases and oncology.

In May 2022, VCBeat New Medicine interviewedDr. Liang Junbin, Chairman and CEO of RayWind Biologics, discussing the current status, pain points, and breakthroughs in China’s gene editing therapy sector. Ruifeng Biotechnology is a Chinese gene-editing therapeutics company and one of the few domestic firms possessing proprietary foundational patents and development capabilities for CRISPR/Cas gene-editing technology. Its team is also among the earliest globally to engage in the clinical translation of gene editing. Dr. Liang Junbin previously worked at the Guangdong Academy of Sciences and BGI Shenzhen. As a serial entrepreneur in the field of genetic diagnosis and therapy, he has accumulated over 10 years of innovation and business management experience in molecular biology and genomics.

The following is a transcript of the interview between Dr. Liang Junbin and VCBeat New Medicine. For ease of reading, VCBeat New Medicine has made editorial adjustments to the text without altering its original meaning:

VCBeat: Could you please introduce your previous research and work experience? What technological and resource foundations led you to establish Ruifeng Biologics?

Dr. Liang Junbin:My academic, research, and entrepreneurial journey has been firmly rooted in the field of genetics, allowing me to develop a profound technical and industrial understanding of human genetics and genomics. This expertise serves as a solid foundation and starting point for the development of gene-editing therapeutics, which is why I chose to embark on an entrepreneurial path.

Looking back over the past few years, gene editing technology has advanced at an extraordinary pace. It has not only gained prominence as a core research and development tool but has also given rise to a new class of therapeutics. The advent of CRISPR gene-editing technology first opened the door to hope for the treatment of genetic diseases,Addressing a common question among clinicians: “We’ve identified genetic abnormalities, but how do we address them?”

Our co-founder, Professor Huang Junjiu, was among the earliest scholars to explore this path, applying gene-editing technology to the treatment of thalassemia. In recognition of this work, Professor Huang was named one of Nature’s “Ten People Who Mattered” in 2015. When the founders of RayWind Bio established the company in 2019, they were unequivocal in their commitment to advancing gene-editing therapeutics, with their primary goal being the cure of thalassemia.

VCBeat New Medicine: Why did Ruifeng Bio choose gene-editing therapies among the many niche segments of the pharmaceutical industry? What pain points and prospects did it identify?

Dr. Junbin Liang:As mentioned above, this is a natural choice following our understanding of genes. There are still numerous diseases that remain incurable. I firmly believe that gene editing will propel drug development to unprecedented heights, bringing significant benefits to patients—a value that cannot be replaced by any other technology.As an emerging field in its early stages of development, gene editing therapies have most products still in the preclinical stage. In addition to some common issues in drug development, more specific problems need to be addressed. For example, various aspects such as editing strategies and efficiency improvement, delivery specificity, control of off-target effects from cutting or base editing point mutations, on-target/off-target analysis, and quality evaluation.

But in reality,It is precisely because gene-editing therapies are still in their early stages globally that they represent one of the pharmaceutical sectors where Chinese companies can compete on equal footing with their European and American counterparts.From an industrial application perspective, China has witnessed rapid development, with indications gradually expanding from rare diseases to more prevalent conditions, including chronic degenerative diseases and malignant tumors.

Artery New Medicine: Reflecting on the industry's development in recent years, how has the perception of domestic venture capital firms regarding CRISPR gene-editing technology evolved? What is your own perspective on the medical value of this technology?

Dr. Liang Junbin:This is a rather intriguing question. I believe that gene sequencing, gene editing, and synthetic biology constitute the “three major pillars” of the entire life sciences field, with profound implications for medicine. Among these, gene editing technology holds disruptive, curative value, akin to performing surgery on the human “book of life.” At this stage, early-stage companies like ours play the role of pioneers: progressing from addressing monogenic diseases with clear causal genetic links to tackling complex diseases—such as chronic conditions, hematologic malignancies, and solid tumors—where genetic mutations or dysregulation act as contributing factors.

As to how far we can go, we cannot say for sure. But one thing is certain: this boundary will continue to be expanded. At present, the industrialization of gene editing is still in a relatively early stage.The overall understanding among stakeholders depends on their proficiency with gene-editing tools and the depth of their knowledge about diseases.However, the fact that it is in a relatively early stage of development does not mean its future applications will be limited. Gene editing is not a niche technological field; it has profound implications for all aspects of life sciences and will give rise to a substantial industrial landscape.

In 2018, when I first began engaging with investment institutions, most adopted a wait-and-see approach, as they did not regard this technology as particularly distinctive. However, two significant events shifted their stance: one was the clinical success of gene-editing therapy for thalassemia, and the other was the joint awarding of the 2020 Nobel Prize in Chemistry to two pioneers of CRISPR gene-editing technology. These developments bolstered confidence, spurred growth in the related industry and investment landscape, heightened public interest, and enhanced recognition. Consequently, several leading investment firms explicitly designated the gene-editing sector as a key focus for their strategic investments.

Currently, Ruifeng Bio’s thalassemia product has demonstrated curative efficacy in certain cases, while early-stage R&D is concurrently underway in several chronic disease areas within ophthalmology and the nervous system.On the other hand, we are also witnessing a trend in immune cell therapy shifting from personalized to universal approaches, with gene editing technology playing a pivotal role. Notably, a diagnostic industry based on CRISPR/Cas technology is emerging, with foreign companies Mammoth Biosciences and Sherlock Biosciences currently establishing their presence in this field. Undoubtedly, gene editing, as a foundational technology in life sciences, will bring revolutionary changes to human health and medical care.

VCBeat New Medicine: CRISPR Gene-Editing Drugs Boast Highly Promising Prospects—Will This Industry Rapidly Shift from a Blue Ocean to a Red Ocean?

Dr. Liang Junbin:Valuable industry directions will inevitably attract more entrants, but I believe this field will not quickly become a red ocean.First, gene editing is a technology that is easy to learn but difficult to master, making it unlikely to lend itself to quick-fix entrepreneurial ventures. Second, drug development involves long cycles and substantial capital intensity; few companies possess the patience or capability to commit to such demanding endeavors.Making friends with time is a challenge that stumps many people.On the other hand, gene editing is a foundational technology with broad applications in the field of disease treatment.There are approximately 8,000 rare diseases explicitly defined by humans, along with many non-genetic disorders. With numerous potential indications for development, companies can pursue their own unique technological pathways, thereby avoiding intense homogenous competition.

Arterial Network New Medicine: Discussing the Clinical Research Progress of Ruifeng Biotechnology's Gene-Editing Drug for β-Thalassemia.

Dr. Liang Junbin:This is Ruifeng Bio’s first R&D product. Thalassemia is the monogenic inherited disease with the widest global prevalence. Gene-editing therapies are currently regarded internationally as the optimal option for a definitive cure of thalassemia. CTX001, a therapy developed by CRISPR Therapeutics in the United States for transfusion-dependent β-thalassemia, has achieved highly promising clinical trial results and is widely considered a blockbuster drug poised for market launch.

Ruifeng Bio has completed an investigator-initiated clinical study on thalassemia. The data demonstrate significant therapeutic efficacy of the product, with participants cured and discharged to enter the follow-up phase. Key clinical indicators, such as hemoglobin levels and transfusion independence duration, are at the industry-leading level.This is also the first clinical study globally of a gene-editing therapy for thalassemia targeting a novel therapeutic target, and marks the first successful treatment of adult thalassemia patients in China. Our data will be presented at the upcoming 2022 European Hematology Association (EHA) Congress, representing the first clinical report on Chinese gene therapies for thalassemia at this conference.

VBInsight: What are Ruifeng Bio’s leading advantages and achievements in gene-editing therapeutics?

Dr. Liang Junbin:Ruifeng Bio has adopted innovative DNA and RNA editing as its therapeutic development strategy, expanding its pipeline beyond hematopoietic stem cells. This includes ophthalmic product development targeting retinitis pigmentosa and two other blinding diseases, with some data reaching internationally advanced levels. In the field of Parkinson’s disease, Ruifeng Bio has successfully achieved neuronal regeneration through RNA editing, and positive preliminary efficacy results have been observed in non-human primate models. Additionally, we have other promising early-stage data that will be disclosed gradually in the future.

VCBeat New Medicine: How is Ruifeng Bio leveraging AI to accelerate the development of gene-editing drugs? Why pursue this line of work?

Dr. Liang Junbin:We are committed to pursuing genuine innovation by taking the difficult yet correct path. On one hand, this is driven by the inherent characteristics of gene editing itself: it is a highly programmable drug technology that can be understood as a digital form of therapeutics. On the other hand, gene editing involves altering the nucleic acid sequences of genetic material. To excel in this field, mastery and application of genomics are indispensable, and genomics is, at its core, a data science.Ruifeng Bio possesses a proprietaryTianbing^TM（TIPBIG^TM, Therapeutic Innovation Platform Based on Informatics and Genomics) technical innovation platform, aiming toIntegrating Bio and ITcapabilities to accelerate drug R&D efficiency and foundational innovation. We have built a dedicated AWS cloud platform that integrates technologies such as machine learning, data mining, and protein structure prediction.

Technological innovation is no easy feat; it demands a mindset of “building strong fortifications and fighting methodical battles.” Leveraging their unique interdisciplinary backgrounds in genomics, molecular and cellular biology, and computer science, the founding team of RayWind Biosciences has already achieved several innovative breakthroughs in CRISPR editing tools through a combination of independent R&D and strategic collaborations.It is worth noting that RayWind Bio is one of the few companies in China possessing foundational CRISPR patent technologies. Notably, our proprietary CRISPR/Cas13m system features high cleavage efficiency, low off-target effects, and a compact size that facilitates easy delivery. The CRISPR/Cas13m technology can be broadly applied to RNA-level knockdown, modification, and base editing. Compared with RNA interference (RNAi) technology, it offers higher target specificity and presents unique therapeutic advantages for disease treatment.

VBInsight New Medicine: Leveraging the aforementioned advantages and achievements, RayPharm Biotech completed three rounds of financing within just over two years. Last year, it also gained recognition from multiple institutions, including Yansheng Ventures, Boyuan Capital, Hillhouse Investment, and Sinovation Ventures. With dual support from capital and technology, what are RayPharm Biotech’s short-term goals and long-term plans for the future?

Dr. Liang Junbin：Ruifeng Biologics is dedicated to developing curative therapies for serious diseases. We have recently achieved milestone progress in our hematopoietic system and ophthalmology product portfolios. Centered on gene editing, we are strategically building our pipeline in the fields of genetic disorders and major diseases, including products targeting liver, neurological, and oncological indications. These areas represent our long-term strategic focus. Meanwhile, we will adhere to a dual-engine strategy of parallel advancement in both products and technologies, akin to the twin engines of an aircraft, serving as the driving force for the company’s growth. Ruifeng Biologics aims to become an innovative pharmaceutical company with proprietary technologies, global competitiveness, and international influence.