Advancing RNA Therapeutics with miRecule

Tachyon Ventures is thrilled to announce its first investment in miRecule, a biotech company dedicated to developing RNA therapeutics aimed at treating and potentially curing a range of challenging diseases. 

Understanding RNA Therapies

RNA therapies represent a cutting-edge approach to treating the root cause of genetic diseases by targeting disease-mediating RNA molecules. In many genetic diseases, mutations in the DNA lead to the production of abnormal RNA. This mutated RNA is then translated into abnormal proteins that lead to disease symptoms. Traditional therapies often target the diseased proteins themselves, or alleviate symptoms without addressing the genomic cause.

RNA therapies work by intercepting and blocking these disease-causing messenger RNA (mRNA) molecules, preventing them from being translated into harmful proteins. By preventing the production of dysfunctional proteins, RNA therapies can effectively repress the disease at its root, offering the potential for more durable and profound treatment outcomes. This can be achieved through various methods, including: 

• Antisense Oligonucleotides (ASOs): ASOs are short, single-stranded pieces of DNA or RNA that bind to the target mRNA, leading to its degradation or blocking its translation into protein. They can be designed to correct splicing defects or reduce the expression of harmful proteins. ASOs are highly sequence specific and can be tailored to target virtually any gene.
• Small Interfering RNA (siRNA): siRNAs are double-stranded RNA molecules that bind to mRNA and incorporate into the RNA-induced silencing complex (RISC). This complex then degrades the target mRNA, preventing protein production. siRNAs are used to silence specific genes associated with disease, and can achieve potent gene silencing with relatively low doses.
• MicroRNA (miRNA) Therapies: miRNAs are small, naturally occurring RNA molecules that regulate gene expression by binding to complementary sequences on target mRNAs, resulting in translational repression or mRNA degradation. Functional studies have confirmed that dysregulation of miRNA is causal in many cases of cancer, with miRNAs acting as tumor suppressors and oncogenes. miRNA therapies can modulate the expression of multiple genes simultaneously, making them a suitable target for complex diseases. Developing therapeutics that either replace miRNA using miRNA mimics, or inhibit miRNA function, is an attractive avenue for novel therapeutic approaches. 
• Antibody-RNA Conjugate (ARC) Therapies: These therapies use antibodies to deliver RNA molecules directly to specific tissues or cells. The antibodies are designed to bind to unique markers or antigens present on the surface of target cells, thereby delivering the therapeutic RNA (such as siRNA or miRNA) specifically to the diseased cells. This approach is particularly valuable for targeting cancer cells or other cells where expression of specific surface markers is known. It ensures that the RNA therapy affects only the target cells, minimizing side effects on healthy cells.

A New Era in RNA Therapeutics

miRecule's mission is to create RNA therapies that not only treat symptoms but also target the underlying genomic causes of disease, offering the potential for long-term remission. First, the company's flagship DREAmiR™ platform leverages genomic data to identify novel RNA therapeutic targets and select patient groups most likely to respond effectively. Then, Mirecule uses its NAVIgGator technology to develop antibody-RNA conjugate (ARC) therapies – enabling precision targeting and ensuring that treatments are delivered specifically to diseased tissues. miRecule’s ARC technology combines highly potent RNA therapeutics that are biologically enhanced to improve safety, potency, and stability alongside a conjugated antibody that is directed to a tissue-specific target. miRecule’s pipeline is primarily focused on neuromuscular diseases and oncology. The company has discovered a novel, proprietary antibody receptor with high specificity for ARC delivery to human muscles – the target tissue in neuromuscular diseases.

Transformative Programs

MC-30 for Head and Neck Cancer

Affecting over 100,000 patients annually in the United States, head and neck cancer encompasses a variety of malignancies that arise in the head and neck region, including the oral cavity, throat, and larynx. Current treatment options for head and neck cancer include surgery, radiation therapy, and chemotherapy. While these treatments can be effective, they are often associated with severe side effects and a significant risk of recurrence. Head and neck cancer patients often experience recurrence of their cancer within 18 months of treatment – as such,  the prognosis for patients with advanced head and neck cancer remains poor, with many experiencing a limited progression-free survival period.

Tumor heterogeneity—the presence of diverse cell populations within a tumor—is a significant driver of relapse and remission in cancer patients, as it can lead to drug resistance and treatment failure. miRecule aims to break this cycle with its innovative approach to RNA therapeutics. 

Targeted microRNA (miRNA) therapy offers a promising solution to overcoming drug resistance by simultaneously targeting multiple genes involved in tumor growth and survival. This multi-targeted approach can effectively manage diseases with high tumor heterogeneity, ensuring that diverse cancer cell populations are addressed, thereby reducing the likelihood of drug resistance and improving treatment outcomes.

miRecule's MC-30 is an miRNA mimic that replaces the potent tumor suppressor activity of microRNA-30 which is lost in half of head and neck cancer patients. Set to begin Phase 1 clinical trials soon, MC-30 represents a promising new miRNA approach to treating head & neck cancer. Preclinical studies have shown that MC-30 has strong efficacy and a low toxicity profile, potentially doubling progression-free survival for patients. This innovative RNA therapeutic could offer a much-needed alternative for head and neck cancer patients, providing more effective and less harmful treatment options.

MC-DX4 for Facioscapulohumeral Muscular Dystrophy (FSHD)

Facioscapulohumeral muscular dystrophy (FSHD) is a severe, progressive disease affecting over 500,000 patients worldwide. FSHD leads to muscle weakness and atrophy, severely impacting the quality of life and mobility of those affected. Currently, there are no approved treatments for FSHD, leaving a significant unmet need in the medical community.

MC-DX4 is designed to address this critical gap. MC-DX4 is an antibody-RNA conjugate (ARC) designed to be a disease-modifying treatment for the underserved FSHD patient population. By targeting the underlying genetic causes of FSHD, miRecule aims to provide a therapeutic option that can halt or even reverse disease progression, offering hope to patients who currently have limited options. This ambitious programme is bolstered by miRecule's strategic partnership with Sanofi. This collaboration will combine Sanofi's proprietary muscle-targeted NANOBODY technology and miRecule’s MC-DX4 to join the two molecules into an antibody-siRNA conjugates (ARC) utilizing miRecule’s NAVIgGatorTM conjugation and formulation chemistry. 

Supporting miRecule's differentiated platform

miRecule is advancing the landscape of RNA therapeutics by addressing the genetic root causes of diseases with precision and innovation. Its cutting-edge technologies for targeted RNA Antibody Conjugates (ARCs) have been validated through partnerships with top tier pharma companies and promising programs in oncology and neuromuscular disease. Tachyon Ventures is proud to support miRecule as it advances these transformative therapies and moves closer to providing life-changing treatments for patients in need.