Miriam Corredor, CEO of noctuRNA: "Thanks to the support of CDTI Innovation we are promoting a new generation of therapies based on circRNA"

In recent years, RNA has established itself as one of the most promising platforms of modern biomedicine. After the turning point that RNA vaccines represented, the field has entered a new stage: that of the rational design of molecules capable of intervening directly in the molecular mechanisms that cause complex diseases. In this context, noctuRNA was born, a spin-off from the Universitat Pompeu Fabra that develops innovative therapies based on circular synthetic RNA.

Miriam Runner, CEO of the company with more than a decade of experience in the biotechnological entrepreneurial ecosystem, explains: "noctuRNA is the materialization of years of academic research in a therapeutic proposal with a clear clinical and social vocation", and adds: "Our goal is to address diseases where today there are no real solutions, acting directly on their molecular root."

With the support of CDTI Innovation, the company is advancing a strategic project focused on neuromuscular and neurodegenerative diseases such as myotonic dystrophy type 1 (DM1) and amyotrophic lateral sclerosis (ALS), with a technology that aims to open a new generation of therapies based on circRNA.

Part of the team of noctuRNA Therapeutics, a biotechnology company that develops innovative therapies based on circular synthetic RNA

From the laboratory to the company: transforming knowledge into clinical development

The origin of noctuRNA is found in more than seven years of academic research in RNA structure and biology. Turning that knowledge into a biotechnology company was a profound change.

"The transformation was not easy; it involved a very significant cultural and operational change," Corridor said. "We move from an environment focused on understanding the 'why' of biological mechanisms to one geared towards generating measurable milestones, robust data and results aligned with regulatory and investment expectations."

The previous experience of the founding team in the creation and management of biotechnology companies facilitated this transit. In addition, as a university spin-off, it was necessary to articulate a solid strategic relationship with the institution of origin. “It was essential to professionalize the technology and structure it as a business project with international projection,” says the CEO.

Today, the company combines scientific excellence, female leadership and a clear orientation to social impact, with a multidisciplinary team that integrates experts in molecular biology, computational design and business development.
 

A new therapeutic strategy based on circRNA

The project supported by CDTI Innovation on new therapeutic strategies based on synthetic circular RNAs for the treatment of neurodegenerative diseases, constitutes the central axis of current development. The proposal is based on designing circRNAs capable of specifically binding to pathological secondary structures present in certain cellular RNAs and destabilizing them.

As Sillo well explains: "Our circRNAs are designed to act on secondary RNA structures that play a key role in the development of these diseases." In addition, the CEO points out that "currently there are no therapies that specifically act on the causal mechanism in pathologies such as DM1 or ALS; treatments are mainly limited to symptomatic management."

Corridor also stresses that the scientific moment is especially propitious: "In recent years, the molecular mechanisms of these pathologies have been characterized with greater precision, and the field of RNA has reached a technological maturity that facilitates its clinical translation."

Added to this is the availability of computational design tools and synthetic biology that allow the development of highly specific molecules.

Research in the facilities of noctuRNA Therapeutics, where the scientific team works on the development of new therapeutic strategies based on circRNA

Experimental validation: from proof of concept to translation

The validation of the action mechanism has been one of the pillars of the project. Initially, the platform was tested on viral infectious diseases. "We demonstrated in vitro that our circRNAs could bind to viral RNA structures and inhibit their replication in models such as SARS-CoV-2, dengue or West Nile virus," Corridor says. “We even validated in vivo efficacy with our best candidate against SARS-CoV-2.”

Based on this conceptual validation, the company extended the application to genetic and neurodegenerative diseases. "In type 1 myotonic dystrophy we have demonstrated the reduction of nuclear aggregates and the correction of splicing defects associated with aberrant RNA structures," he explains. Similar results have been observed in cellular models of ALS and Huntington.

The next step is to move towards more complex preclinical studies. "In order to make the leap to the clinic, it is essential to consolidate this evidence into more advanced in vivo models," says the CEO.

first-in-class molecules with differential advantages

The circRNAs developed by noctuRNA are considered first-in-class because of their innovative mechanism and structural properties.

"As they are circular RNAs — molecules that already exist naturally in cells — they have a very robust safety profile," says Sillo. “In our in vivo studies we have not observed immune activation,” he adds.

The absence of free ends gives these molecules a high resistance to degradation, which translates into prolonged stability. "We have demonstrated stability of at least one month in vivo, without the need for frequent chemical modifications in other RNA formats," explains the CEO.

From the pharmaceutical point of view, they also offer relevant logistical advantages. "Its stability in aqueous solution facilitates storage and distribution, which is key when we think of industrial scalability."

Detail of the work in the noctuRNA laboratory, where synthetic circular RNA molecules with therapeutic potential are designed and validated

Artificial intelligence to accelerate development

A differential element of the platform is the integration of artificial intelligence and computational design. noctuRNA has its own software capable of identifying relevant secondary structures and designing optimized circRNAs. "Our algorithms allow us to significantly reduce experimental trial error and increase the likelihood of success," Corridor explains. "Approximately 70% of circRNAs designed using our systems show therapeutic activity in in vitro assays."

The integration between in silico design and experimental validation generates a cycle of continuous improvement. "Each iteration feeds the system and improves the predictive capability of the platform," he adds.
 

Impact on patients and families

Beyond technological advancement, the ultimate goal is to improve the lives of patients. Diseases such as DM1, ALS or Huntington’s are progressive, incapacitating and generate a high emotional and assistance burden.

"They are serious, multisystemic pathologies and, today, without therapies that significantly modify their evolution," says Sillo. "Our approach seeks to act on the underlying molecular origin with the aim of modifying that progression."

The stability of circRNAs could involve fewer therapeutic administrations. "If we achieved that effect, the benefit would not only be clinical, but also social and economic, reducing the burden on patients, families and healthcare systems."
 

The role of the CDTI Innovation

The CDTI Innovation has supported this project through Neotec, a line of subsidies aimed at financing the start-up of small technology-based companies. In this regard, Sillo assures that "the collaboration with the CDTI has been a decisive impulse for noctuRNA", and adds: "It has allowed us to address key preclinical studies that represent a turning point in the development of any advanced therapy."

These studies in murine models represent a high cost and a fundamental stage to attract private investment. “Neotec financing allows us to significantly reduce technological risk and position the company in a much stronger position for future rounds of financing and strategic alliances.”
 

Looking to the future: consolidating the leadership in circRNA

Five years later, the company aspires to have several programs in clinical development. "Our goal is to have graduated the candidate for myotonic dystrophy during Phase I/IIa and complete that same clinical phase with the candidate in ALS," explains Runner. They also plan to start trials in the field of viral diseases and advance programs for Huntington or other genetic pathologies.

Ten years on, the ambition is clear: "We aspire to position ourselves as an international reference in therapies based on circRNAs and, above all, to have a real impact on patients who today do not have therapeutic alternatives."

With frontier science, artificial intelligence and the impetus of the CDTI Innovation, noctuRNA is at the forefront of a new generation of therapies that seek not only to treat symptoms, but also to intervene in the molecular origin of diseases hitherto intractable and thus improve the health of society.
 

Facilities of noctuRNA Therapeutics, spin-off of the Universitat Pompeu Fabra

CDTI Innovation

The Center for Technological Development and Innovation, CDTI E.P.E. It is the innovation agency of the Ministry of Science, Innovation and Universities, whose objective is the promotion of technological innovation in the business environment. The mission of the CDTI is to ensure that the Spanish business fabric generates and transforms scientific and technical knowledge into globally competitive, sustainable and inclusive growth. In 2025, within the framework of the Strategic Plan 2024-2027, the CDTI provided more than 2 billion euros of support to Spanish companies and startups.

 
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