Exrna Therapeutics: Advancing For Rare & Neurological Disorders

Satyam Tiwary, CEO

Satyam Tiwary

CEO

RNA’s role as a therapeutic tool is no longer theoretical, it’s reshaping the global biotech playbook. India, often viewed as the pharmaceutical hub of generics, is now becoming a frontrunner in RNAbased therapies, fueled by breakthroughs in mRNA vaccine development, CRISPR advancements, and surging interest in oligonucleotide research. Yet, the promise comes with hurdles. The molecular fragility of RNA, delivery challenges, immune system triggers, and the complexities of large-scale manufacturing remain persistent barriers. Harnessing the intricate functionalities of transfer RNA fragments, Exrna Therapeutics Ltd. is developing RNA-based therapies to address unmet needs in rare, neurological, and autoimmune diseases.

Exrna Therapeutics is a biotech firm specializing in antisense oligonucleotide therapies and RNA interference solutions. Originally established as Vastu Vihar Biotech, the company strategically narrowed its focus to RNAbased therapeutics in response to the evolving needs of the biotech sector. This deliberate transition resulted in the formation of Exrna Therapeutics, aligning the company with cutting-edge trends in genomics and cell and gene therapy. “Antisense therapy and RNA interference offer precision in gene regulation, making them ideal for treating genetic and autoimmune disorders”, says Satyam Tiwary, CEO, Exrna Therapeutics. “Our inspiration comes from studying extracellular RNA and tRNA-derived fragments, which we believe hold the key to both disease pathology and therapeutic intervention”, Satyam adds. Its product pipeline centers around ASO therapies targeting neuro disorders, cardiac complications, preeclampsia, infertility, and mitochondrial diseases.

Advanced Research Capabilities

Exrna Therapeutics leverages the regulatory potential of non-coding RNAs, particularly transfer RNA fragments (tRFs) and extracellular RNA, to modulate gene expression with remarkable precision. This approach allows the company to provide targeted solutions designed to recalibrate abnormal genetic activity associated with various medical conditions. It has established a comprehensive research infrastructure that spans molecular biology, bioinformatics, and preclinical studies. An integrated team of approximately 100 professionals, drawn from diverse disciplines such as neurology, cardiology, and immunology, drive the company’s development process.

Regular interdisciplinary consultations and AI-assisted data-sharing platforms ensure seamless coordination and foster a rigorous approach to research and innovation. The company’s research methodology is distinguished by its focus on novel secondary structure formation of ASOs and the use of lipid nanoparticle (LNP) formulations.

Exrna Therapeutics expects to deliver key advancements, particularly in ASO-based treatments for neurodisorders and mitoch

Satyam Tiwary, CEO

These methods are designed to enhance targeted delivery and optimize receptor-mediated uptake, ensuring that the therapies are not only effective but also safe for clinical applications.

On the manufacturing and quality front, Exrna Therapeutics' adherence to internationally recognized standards such as ISO 9001:2015 certification and DSIR recognition has enhanced its credibility among strategic partners and investors. The certifications validate its commitment to quality research and safety protocols and open avenues for government grants and biotech collaborations. Notably, the company operates under strict biosafety and bioethics protocols at its BSL-2 laboratory, supported by independent ethical review committees to ensure research transparency and integrity.

A Client-Centric Approach to RNA Therapeutics

Exrna Therapeutics’ research is not merely confined to the laboratory, it is carefully designed to translate into clinically viable therapies that address unmet medical needs. Its flagship ASO candidate, designated G4, is currently undergoing preclinical validation as it progresses toward Phase 1 clinical trials. G4 exemplifies the company’s methodical approach to drug development that emphasizes precision, scalability, and adherence to the highest regulatory standards.

Its focus on RNA interference and antisense technology translates into tangible benefits for patients. By targeting the underlying genetic mechanisms of disease, Exrna Therapeutics' therapeutic solutions aim to offer improved efficacy and reduced side effects compared to conventional treatments. Clients who partner with the company gain access to a platform that combines deep genomics research with state-of-the-art delivery mechanisms. The platform ensures that each therapeutic candidate is finely tuned to meet the specific needs of the target patient population.

Strategic Collaborations & Quality Assurance

The company strengthens its research and development efforts through strategic collaborations with esteemed institutions, including Banaras Hindu University, the University of Hyderabad, the Central University of South Bihar, and the Centre for Cellular and Molecular Platforms. These partnerships enhance its research capabilities and provide valuable insights that drive innovation in RNAbased therapeutics. Moreover, the company’s quality assurance framework is reinforced by internationally recognized certifications. Registration under the CPCSEA for its animal house facility, DSIR certification that validates in-house R&D capabilities, and ISO 9001:2015 certification for its research and theranostics laboratories collectively underscore Exrna’s commitment to excellence and operational integrity.

Exrna Therapeutics expects to deliver key advancements, particularly in ASO-based treatments for neurodisorders and mitochondrial-targeted therapies in the near future. In addition, it is actively working on nextgeneration RNA delivery mechanisms and expanding its research into clinical applications for fertility treatments, including sperm and oocyte enhancement. These efforts are complemented by an ongoing emphasis on strategic global partnerships, aimed at commercializing its therapies and broadening access to personalized medicine solutions. For healthcare organizations, biotech collaborators, and research institutions seeking to navigate the complexities of RNA-based therapeutics, Exrna Therapeutics aims to deliver precision-driven, clinically viable solutions grounded in science, compliance, and innovation.

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