miRecule, EMS Unit Partner on RNA Delivery Technologies

miRecule has entered into a strategic research collaboration with Rio Biofarma Brasil Ltda. (RBBL), part of Brazilian pharmaceutical company EMS S.A., to develop peptide-mediated delivery technologies aimed at expanding the reach of RNA therapeutics beyond the liver and muscle.

The partnership will focus on developing new delivery approaches capable of transporting RNA medicines to extra-hepatic tissues, an area that remains one of the key challenges in the development of next-generation RNA therapeutics. While RNA-based medicines have advanced significantly in liver and muscle applications, delivering therapeutic RNA molecules to other tissues has proven more difficult.

Under the agreement, miRecule and RBBL will combine their respective technologies and expertise to develop novel peptide-RNA conjugates. The companies believe these delivery systems could enable RNA therapeutics to reach previously difficult-to-access tissue targets and potentially broaden the range of diseases that can be addressed with RNA medicines.

miRecule will contribute its proprietary NAVIgGator platform, which is designed to support the development of precision RNA medicines. The platform will be used to develop oligonucleotide payloads for up to three targets that can be paired with RBBL’s peptide delivery technology.

RBBL, meanwhile, will contribute expertise in peptide discovery, development and manufacturing. The company will manufacture the resulting products at its advanced manufacturing facility in Brazil, providing the partnership with a pathway from early technology development toward production.

The companies will co-own the technologies developed through the collaboration. They will also have opportunities to jointly develop products for global markets, potentially allowing successful delivery technologies to move beyond research and into therapeutic programs targeting broader patient populations.

The collaboration addresses a major limitation facing the RNA therapeutics industry. RNA medicines can be designed to influence biological processes at the genetic or molecular level, offering potential treatments for diseases that have historically been difficult to address with conventional drugs. However, getting RNA molecules safely and efficiently to the right cells and tissues remains a significant technical hurdle.

Many existing RNA delivery strategies rely on approaches that work particularly well in the liver. As researchers seek to develop RNA therapies for neuromuscular disorders, neurological diseases and other conditions involving tissues outside the liver, new delivery technologies are increasingly important.

miRecule is developing next-generation RNA therapeutics for rare genetic diseases, with a particular focus on neuromuscular conditions. The company expects the collaboration with RBBL to support its broader pipeline while providing access to peptide-based delivery capabilities that could potentially expand the range of tissues targeted by its RNA programs.

As part of the broader relationship, RBBL has also made an equity investment in miRecule. The investment will support development of the collaboration and help advance miRecule’s pipeline of neuromuscular assets. The companies did not disclose the financial terms of the research agreement or the size of the equity investment.

Anthony Saleh, CEO of miRecule, said the partnership with EMS-RBBL combines scientific expertise, manufacturing capabilities and global resources that could help move new RNA delivery technologies closer to patients. He said the collaboration could help address the challenge of delivering RNA therapies to difficult-to-reach extra-hepatic tissues.

Dan Salazar, CEO of Rio Biopharma, said the investment and partnership reflect the company’s confidence in miRecule’s scientific approach and the potential applications of the joint technology. He said the companies are looking to translate the delivery advances into future therapeutic opportunities.

For RBBL and EMS, the collaboration provides an opportunity to expand their involvement in RNA therapeutics while applying their peptide development and manufacturing expertise to an emerging drug modality. For miRecule, working with a major pharmaceutical organization in Brazil provides additional resources for technology development and manufacturing as the company advances its rare disease pipeline.

The collaboration could ultimately have implications across several therapeutic areas. Improved delivery to extra-hepatic tissues would potentially allow RNA medicines to address biological targets that are currently difficult to reach, including targets associated with neuromuscular and other rare genetic diseases.

The companies will now work on developing and evaluating peptide-RNA conjugates while advancing the underlying delivery platform. Success will depend on demonstrating that the technologies can efficiently transport RNA payloads to targeted tissues while maintaining appropriate safety and therapeutic activity.

The partnership highlights the growing importance of delivery technology in the RNA therapeutics field. As more companies move beyond liver-focused applications, innovations capable of directing RNA medicines to new tissues could become increasingly important in expanding the potential of the modality.