miRecule and Rio Biofarma Brasil Partner to Advance Next-Generation RNA Delivery Technologies
miRecule, Inc., a biotechnology company focused on developing next-generation RNA therapeutics for rare genetic diseases, has entered into a strategic research collaboration with Rio Biofarma Brasil Ltda. (RBBL), part of EMS S.A., Brazil’s largest pharmaceutical company. The partnership is designed to accelerate the development of innovative peptide-mediated delivery technologies that can improve the targeted delivery of RNA therapeutics to tissues beyond the liver, an area that remains one of the most significant scientific challenges in the field of RNA medicine.
The collaboration combines the complementary strengths of both organizations. miRecule brings its proprietary NAVIgGator™ RNA therapeutic discovery platform and expertise in precision RNA medicine, while RBBL contributes extensive capabilities in peptide discovery, process development, large-scale manufacturing, and pharmaceutical commercialization. Together, the companies aim to create advanced peptide-RNA conjugates capable of delivering RNA-based medicines to previously difficult-to-target tissues, potentially opening new therapeutic opportunities across a broad range of diseases.
Addressing a Critical Challenge in RNA Therapeutics
RNA therapeutics have rapidly emerged as one of the most promising areas in modern biotechnology. These therapies work by targeting genetic material directly, allowing scientists to regulate disease-causing genes, replace missing proteins, or silence harmful genetic mutations. Their ability to precisely address the underlying causes of disease has generated significant interest for treating rare genetic disorders, metabolic diseases, neurological conditions, and cancer.
Despite remarkable advances in RNA medicine over the past decade, one major obstacle has limited the full potential of these therapies: effective delivery to tissues outside the liver.
Many currently approved RNA medicines naturally accumulate in liver tissue because of the body’s biological processes and existing delivery technologies. While this has enabled significant progress in treating liver-related disorders, researchers have struggled to safely and efficiently transport RNA molecules to other organs such as the brain, heart, lungs, kidneys, peripheral nerves, and various connective tissues.
Expanding delivery capabilities beyond hepatic tissues is considered one of the next major frontiers in RNA therapeutics. Successfully overcoming this challenge could dramatically broaden the number of diseases that can be treated using RNA-based medicines.
The new partnership between miRecule and RBBL is specifically focused on solving this problem.
Combining Complementary Scientific Expertise
Under the collaboration, miRecule will leverage its proprietary NAVIgGator™ platform, which is designed to accelerate the identification and optimization of precision RNA therapeutics. The platform supports the development of customized oligonucleotide-based medicines that can selectively target disease-causing genetic pathways.
RBBL will contribute its extensive expertise in peptide engineering and manufacturing. Peptides have emerged as attractive delivery vehicles because they can be engineered to recognize specific cell types and transport therapeutic molecules directly into target tissues.
By combining these capabilities, the companies plan to develop novel peptide-RNA conjugates capable of improving tissue-specific delivery while maintaining the safety and efficacy of RNA therapies.
The collaboration aims to create delivery technologies that can potentially reach organs and tissues that have historically been inaccessible using conventional RNA delivery systems.
Development Program Includes Multiple Therapeutic Targets
The agreement outlines a collaborative development strategy involving multiple therapeutic programs.
Under the terms of the partnership, miRecule will develop RNA oligonucleotide payloads directed against up to three therapeutic targets using the jointly developed peptide delivery platform.
Meanwhile, RBBL will be responsible for manufacturing the resulting therapeutic candidates at its advanced pharmaceutical manufacturing facilities in Brazil. The company’s infrastructure is expected to support both clinical and potential future commercial production.
The companies also intend to explore opportunities for jointly developing and commercializing resulting therapies in international markets, extending the impact of the collaboration beyond Brazil.
Shared Ownership Encourages Long-Term Innovation
Unlike traditional licensing agreements, the collaboration includes co-ownership of the technologies developed during the partnership.
This shared intellectual property framework allows both companies to participate in the future development and commercialization of the platform while encouraging continued scientific collaboration.
Such arrangements are becoming increasingly common in biotechnology partnerships where both organizations contribute substantial technological expertise and expect to jointly benefit from future innovations.
Strategic Investment Strengthens the Partnership
As part of the broader collaboration, Rio Biofarma Brasil has also made an equity investment in miRecule.
The investment is intended to support not only the peptide delivery research collaboration but also the continued advancement of miRecule’s internal pipeline of RNA therapeutics, particularly its programs targeting neuromuscular diseases.
Although financial terms of both the research agreement and the investment were not disclosed, the equity participation demonstrates RBBL’s long-term confidence in miRecule’s technology platform and scientific strategy.
Strategic investments of this type often strengthen collaboration by aligning the long-term interests of both organizations while providing additional resources for research and development.
Expanding Opportunities for RNA Medicines
The partnership reflects growing industry interest in solving one of the biggest remaining barriers in RNA drug development.
Recent years have seen tremendous success for RNA therapeutics in several therapeutic areas, particularly following advances in messenger RNA vaccines and antisense oligonucleotide therapies. However, researchers widely recognize that improved delivery systems will determine how broadly these technologies can be applied in the future.
Peptide-based delivery systems represent one promising approach because they can potentially offer:
- Improved tissue specificity
- Enhanced cellular uptake
- Reduced off-target exposure
- Better therapeutic efficiency
- Expanded treatment possibilities for rare and complex diseases
If successful, the technologies emerging from this collaboration could enable RNA therapeutics to reach a much wider range of organs than current delivery methods allow.
Leadership Highlights the Scientific Potential
Dr. Anthony Saleh, Chief Executive Officer of miRecule, emphasized that partnering with an established pharmaceutical organization provides significant advantages for advancing innovative delivery technologies.
He noted that EMS-RBBL brings substantial scientific expertise, manufacturing capabilities, and global experience that complement miRecule’s strengths in RNA medicine. According to Dr. Saleh, the collaboration represents an important opportunity to accelerate the development of transformative RNA delivery solutions capable of reaching challenging extra-hepatic tissues and ultimately expanding treatment options for patients with serious diseases.
Dan Salazar, Chief Executive Officer of Rio Biopharma, Inc., also highlighted the strategic importance of the collaboration.
He stated that the company’s investment reflects confidence in miRecule’s scientific vision and in the long-term potential of combining the organizations’ complementary technologies. Salazar added that RBBL looks forward to working closely with miRecule to translate these research innovations into future therapeutic opportunities capable of addressing significant unmet medical needs.
Strengthening the Future of Precision Medicine
The collaboration also reflects broader trends within the biotechnology industry, where partnerships increasingly focus not only on developing individual therapies but also on enabling platform technologies that can support multiple future products.
Delivery technology is widely viewed as a foundational component of next-generation precision medicine. Improvements in targeted delivery have the potential to benefit numerous therapeutic modalities beyond RNA medicines, including gene editing, gene silencing, and other nucleic acid-based approaches.
For miRecule, the partnership provides additional resources and manufacturing expertise to support the continued growth of its RNA therapeutic pipeline. For RBBL, it offers an opportunity to participate in the development of cutting-edge RNA delivery technologies while expanding its presence in advanced biologics and precision medicine.
As research progresses, the collaboration aims to generate innovative peptide-mediated delivery systems capable of broadening the therapeutic reach of RNA medicines, ultimately supporting the development of more effective treatments for rare genetic disorders and other diseases with significant unmet medical needs. Together, miRecule and Rio Biofarma Brasil hope to accelerate the next generation of RNA therapeutics by overcoming one of the field’s most important scientific challenges: delivering precision medicines safely and effectively to tissues throughout the human body.
About miRecule:
miRecule, Inc. is a biotechnology company focused on advancing next-generation RNA therapeutics for treating rare neuromuscular diseases. The company’s proprietary and validated Antibody RNA Conjugate (ARC) platform delivers RNA therapies that target and knock down disease-causing genes in muscle, addressing the underlying genetic causes of these disorders. miRecule’s most advanced candidate, MC-DX4, has been in-licensed by Sanofi and is designed for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD), the third most common form of muscular dystrophy.
miRecule’s second program, MC-DNM2, targets Centronuclear Myopathy (CNM), a rare and often lethal pediatric disease with no approved therapies. CNM often presents at birth, with a mortality rate of approximately 50% by age two. By targeting the underlying genetic drivers, miRecule aims to deliver transformative therapies for patients suffering from these debilitating conditions, for which there are currently no treatments. For more information visit: www.mirecule.com.
About Rio Biopharmaceuticals and EMS
Rio Biofarma Brasil Ltda. (RBBL) is an EMS Group company focused on the development and manufacturing of complex sterile injectable and peptide-based therapeutics. As part of EMS’s global innovation and manufacturing strategy, RBBL supports the advancement and large-scale production of next-generation medicines for both Brazilian and international markets.
EMS is the largest pharmaceutical company in Brazil, holding this market leadership position for more than 20 years. EMS has invested more than R$1.2 billion in advanced technology platforms, including a dedicated peptide manufacturing facility in São Paulo with the capacity to support large-scale production of complex peptide-based therapeutics. With operations in more than 60 countries and a robust pipeline of innovation and patents, EMS is committed to advancing in-house technology development and delivering highly complex therapies to patients worldwide.