▎Edited by the WuXi AppTec content teamCentral Nervous System(CNS)With a broad disease impact and significant unmet treatment needs, the development of innovative drugs in this field holds substantial value. In the third quarter of 2024, at least 12 Chinese CNS innovative drug companies completed new rounds of financing, with a total amount exceeding 700 million yuan. The following highlights are worth noting:Cell and Gene Therapy (CGT) Rising StarEmerging:Through fundamental repair and regeneration, CGT therapies are expected to provide breakthrough treatments for CNS diseases. At least five funded companies have ventured into the research and development of these innovative therapies, including SINEUGENE, Leapcell Bio, Renyuan Bio, Xueji Bio, and Xiatong Bio, with stem cell therapy being the most popular. Apart from one round of unknown financing, all other companies have received early-stage funding prior to Series B.
Nucleic Acid Drugs Gain Attention:Nucleic acid drugs provide an opportunity for precise regulation of gene expression in CNS diseases, enabling targeted intervention in pathogenic mechanisms and offering more effective and personalized treatment options. At least five financed companies are advancing the development of nucleic acid therapies, including Rona Therapeutics and Hegia Biotech, which focus on siRNA, and Antishengshi, which is dedicated to antisense oligonucleotides.

Rona Therapeutics Completes $35 Million Series A+ Financing

On July 19, Rona Therapeutics, a clinical-stage company focusing on nucleic acid drug development, announced the completion of a $35 million Series A+ financing round. This round was led by LongRiver Jiangyuan Investment, with participating investors including Zhaode Investment, Boyuan Capital, Zhongqi Capital, and Lilly Asia Ventures. According to the press release, Rona Therapeutics will use the proceeds to...Advance the innovative siRNA metabolism pipeline into global development and expand the solidification of extrahepatic delivery platforms in the central nervous system and other fields.。Rona Therapeutics is a nucleic acid innovative drug platform company,Dedicated to the research and development of potential "best-in-class" siRNA drugs,Focused on the treatment of metabolic diseases and central nervous system degenerative diseases. In the field of central nervous system degenerative diseases, the company has deeply explored the potential of extrahepatic nucleic acid delivery, successfully establishing a proprietary extrahepatic delivery platform aimed at treating a series of complex diseases, such as amyotrophic lateral sclerosis, Alzheimer's disease, and other conditions where traditional drug therapies have proven ineffective.SINEUGENE Completes Nearly 100 Million Yuan Pre-A Financing Round

On August 1, SINEUGENE announced the completion of a nearly 100-million-yuan Pre-A round of financing. This round was led by Zhongguancun Capital, with participation from ZB Power, Chuanghua Capital, and Sanmei Investment. The funds raised will primarily be used for...For the IND application of SINEUGENE's first pipeline SNUG01 and the advancement of other pipeline productsetc.
SINEUGENE, established in 2021, is a biopharmaceutical company focused on gene therapy for neurological diseases. Its core pipeline originates from over a decade of fundamental research achievements in the field of neuroscience by Professor Yi-Chang Jia's laboratory at the School of Medicine, Tsinghua University. By integrating technical approaches such as AAV-mediated gene expression and editing, as well as small nucleic acid-mediated gene expression regulation, the company has developed multiple product pipelines aimed at addressing neurological disorders such as Amyotrophic Lateral Sclerosis (ALS), stroke, and Parkinson’s disease. SNUG01 is SINEUGENE's first gene therapy drug to enter clinical trials, with ALS as its indication. Using AAV as a vector and targeting a potential “first-in-class” therapeutic target, it is currently undergoing investigator-initiated clinical research (IIT).
Antacura Completes Nearly 100 Million Yuan in Series A Financing

On July 1, Antishengshi announced the completion of a nearly 100-million-yuan A round of financing, led by SDIC Venture Capital, with follow-up investments from existing shareholders and Ruihe Venture Capital's SCA3 Industry Fund. The funds raised in this round will be used forAccelerate the advancement of product development and application for clinical trials targeting indications such as Duchenne Muscular Dystrophy, Spinocerebellar Ataxia, and Huntington's Disease., and promote the product development of multiple subsequent innovative pipelines.Antishengshi was founded in 2017, dedicated to the development of antisense oligonucleotide (ASO) drugs targeting RNA processing. The company's founder, Dr. Yimin Hua, is a scientist in the field of ASO drugs. In recent years, Antishengshi has made breakthrough progress in areas such as RNA splicing regulation and novel RNA processing, and has established more than ten R&D pipelines, with some products already showing promising preclinical data.Hedgehog Bio Completes RMB Tens of Millions in A2 Round Financing

On September 3, Hygieia announced that it had recently completed a multi-million RMB A2 round of financing, led by Huangpu Biomedical Industry Fund and participated by Nanwan Biofund. Meanwhile, Hygieia's A+ round of financing is underway, with plans...To raise additional funds for multiple pipelines in Phase 1 clinical trials, provide financial support for the development of more subsequent IND-enabling stage projects, and further the early-stage research of extrahepatic delivery platform technology.。Hedya Bio is dedicated to the construction of a delivery technology platform in the siRNA field and drug development. The company has established an end-to-end siRNA drug development platform from target discovery to clinical proof-of-concept, focusing on disease areas with urgent clinical needs or no available treatments. NSDP is the company's siRNA delivery technology platform developed for neurological disorders, which can target not only the central nervous system but also the peripheral nervous system.In the company's pipeline,NSDP-02Intended for the treatment ofAlzheimer's disease, is currentlyLinBefore the BedPhase.YueSai Bio Completes Multi-Million A+ Round Financing

On July 11, Leap Biotech announced that it had received another multimillion-dollar A+ round of financing after securing over 100 million yuan in its Series A round in April this year.This round of financing was participated by the well-known investment institution Guoke Innovation Investment, and the funds will be mainly used forAdvance the company's innovative pipeline in the field of stem cell therapy and accelerate clinical translation.。Since its establishment, LeapCell BioTech has been committed to developing a new generation of cell therapy drugs based on human pluripotent stem cell technology, with research pipelines covering Parkinson's disease and other neurodegenerative diseases. The company possesses an independently developed innovative technology platform for human pluripotent stem cells, including neural differentiation technology, SISBAR lineage tracing technology, stem cell gene editing technology, and GMP-grade hiPSC construction. Several of the company’s research achievements have been published.Nature Methods、Cell Stem Celland other well-known academic journals.Ren Yuan Bio Completes Millions of Yuan in Seed Funding

On August 7, Renyuan Bio, which focuses on vascular repair stem cell therapy, completed a seed round of financing worth several million yuan, with Puhuo Capital as the investor. The funds will be used to advance the R&D process of its products, build a broad system of technical R&D and application cooperation resources, and prepare for clinical trials and various pre-market activities.RenYuan BioFocused on Developing Cardiovascular and Cerebrovascular Disease Treatment Products through Vascular Repair Stem Cell TechnologyThe company utilizes vascular repair stem cell technology, which is expected to open new avenues for the treatment of cardiovascular and cerebrovascular diseases by participating in the regulation of angiogenesis, repairing damaged blood vessels, and unblocking vessels. Rona Therapeutics currently has four pipeline products under development, including three investigational drugs—VCP01, VCP02, and VCP03—and the MV3D-01 3D vascular chip product under research. Among them, VCP03 is intended for the treatment of stroke and is currently in the preclinical animal experimental stage.Readers, please star⭐Chuangjianhui to receive push notifications in the first place.
Disclaimer: The content team of WuXi AppTec focuses on introducing the research progress in global biopharmaceuticals and health. This article is for information exchange purposes only, and the views expressed in the article do not represent the position of WuXi AppTec, nor does it indicate that WuXi AppTec supports or opposes the views mentioned. This article is not a recommendation for treatment plans. For guidance on treatment options, please visit a正规 hospital.
Copyright Statement: This article was compiled and edited by the content team of WuXi AppTec based on publicly available information. Individuals are welcome to share it on their social media platforms, but unauthorized forwarding or reproduction by media or institutions in any form to other platforms is strictly prohibited. For authorization to forward, please contact"Chuangjian Hui"Contact us via the WeChat Official Account message.
More Data Content RecommendationsClick "InSee", Sharing New Health Trends from Sineugene"