Antisense Therapeutics Completes Tens of Millions RMB Pre-A+ Financing and Deepens Collaboration with SCA Patient Organization Penguin Home

VCBeat learned第一时间 that recently, ASOcura (hereinafter referred to as "ASOcura" or "the company"), completed a multi-million yuan Pre-A+ round of financing. This round of financing was invested by Hery Ventures SCA3 Industrial Fund. At the same time, the company announced a comprehensive cooperation with Beijing Penguin House Cerebellar Atrophy Patient Care Center (hereinafter referred to as "Penguin House"), an organization for patients with Spinocerebellar Ataxia (SCA) in China, to accelerate the development of rare disease drugs and clinical research, benefiting as many rare disease patients in China as possible.

ASOcura, the first company in China to target RNA splicing and RNA editing for the development of antisense oligonucleotide (ASO) drugs, currently has multiple projects underway. Promising preclinical data has been achieved in drug development for diseases such as SCA3 (Spinocerebellar Ataxia Type 3) and DMD (Duchenne Muscular Dystrophy). The founder of the company, Dr. Yimin Hua, is a world-leading scientist in the ASO drug field and the principal inventor of Nusinersen (marketed as Spinraza), the best-selling small nucleic acid drug globally.

 Prof. Hua Yimin from ASOcura and the R&D team members

Professor Hua Yimin Introduces Product Features and Mechanism of Action at the Spinraza Training Workshop during the Biogen Academic Conference in Shanghai

In recent years, the development of orphan drugs has been encouraged and supported by global pharmaceutical regulatory agencies. However, the journey of developing an orphan drug is long and arduous, requiring joint efforts from all parties to bring a product from research and development to market and eventually benefit more patients. ASOcura has simultaneously announced a collaboration with "Penguin House" to establish shared resources in terms of patient needs, clinical research, and industry assets. This partnership truly integrates the pharmaceutical industry's R&D efforts with the social responsibility of addressing the unmet medical needs of rare disease patients. ASOcura is committed to becoming a leading enterprise driven by patient needs and original research in the future.

Beijing Penguin Home Spinocerebellar Ataxia Patient Care Center was established by patients and families affected by Spinocerebellar Ataxia (SCA). It is the only non-profit organization in China registered with the Ministry of Civil Affairs that focuses exclusively on the SCA field. Penguin Home is dedicated to caring for the physical and mental health of patients, promoting relevant medical knowledge and the latest research advancements, advocating for societal resources to pay attention to and assist patients, protecting the legal rights of patients, and improving their quality of life.

Since its establishment seven years ago, Penguin Home has closely collaborated with renowned expert teams in the SCA field in China. It has carried out numerous projects including doctor-patient exchange meetings, patient surveys, expert lectures, online disease education, and patient assistance, providing comprehensive care and support for Chinese SCA patients. With its professional expertise accumulated in the SCA field, Penguin Home will continue to take responsibility, enhance services, and fulfill its role by deepening communication and cooperation with government agencies, medical institutions, and pharmaceutical enterprises. By fully integrating resources and advantages from all parties, it aims to play an active role in patient support, disease management, drug research and development, clinical trials, and drug access, convey the genuine needs of patients, and maximize benefits for patients.

In the future, ASOcura will work with Penguin House to promote the progress of SCA drug research and development, jointly advance the development of China's rare disease initiatives, and contribute to the realization of the grand goal of Healthy China 2030.

 Penguin House holds the China Spinal Cerebellar Ataxia Doctor-Patient Exchange Meeting annually.

The collaboration between ASOcura and Penguin House was facilitated by Hery Ventures' SCA3 Industry Fund. This fund, initiated by SCA3 patients and Hery Ventures, is the first and currently the only private equity fund in China dedicated to investing in drugs and treatment methods for SCA3. Behind Hery Ventures is a group of seasoned professionals from the medical industry with extensive successful experience in product development, market sales, and healthcare investment. The vision of Hery SCA3 Industry Fund is to build an ecosystem for the development of SCA3 drug treatments, promote the development and iteration of SCA3 therapeutic drugs, and primarily invest in pharmaceutical R&D companies, introduce overseas products under research, seek team incubation, and invest in upstream and downstream industries related to SCA3.

When SCA3 Industry Fund of Healight Venture Partners was first established, it invested in ASOcura. With the better-than-expected progress in ASOcura’s R&D, Healight Venture Partners increased its investment in ASOcura and gradually expanded its portfolio to other related enterprises. Healight Venture Partners is firmly determined to tackle SCA3 and various other rare diseases that currently lack effective treatments. The firm is bringing together some of the best professionals in this field to work towards a common goal.

The organic combination of patients, capital, and industry forms an ecosystem in the field of rare disease development, jointly overcoming the challenges of orphan drug research and development to benefit more patients. ASOcura, Penguin House, and Huirui Venture Capital each have their own areas of expertise and have already carried out cooperation in multiple aspects. On one hand, they are closely collaborating with clinical doctors to explore faster ways for patients to access safe and effective products. On the other hand, they are fully communicating with relevant regulatory agencies, advocating for the acceleration of future product approval processes, and pushing for the inclusion of approved drugs into medical insurance at reasonable prices as soon as possible after market approval to enhance drug accessibility. In the future, the three parties will also explore several specific implementation plans in terms of project funding, patient recruitment for clinical trials, promotion of clinical drug use, and medical insurance payment access.

Most importantly, a large number of SCA patients have learned about ASOcura and Huirui Ventures through the Penguin House, and are able to access the most advanced treatments in the most direct way, truly gaining clinical benefits. They are also willing to contribute to drug development in various ways. Many hands make light work, and the urgent needs of patients are both the starting point and the end goal. Nusinersen, invented by Dr. Huo Yimin, the founder of ASOcura, has saved the lives of tens of thousands of children with spinal muscular atrophy worldwide. It is believed that in the future, the lives of millions more patients with rare diseases will be saved.