ExoRNA Bio's First-in-Class siRNA Therapy ER2001 Receives FDA IND Approval for Huntington’s Disease

Dance always evokes associations of grace and elegance.The physique. But there is a disease named after dance——Huntington's Disease (Huntington's Disease (HD) causes patients to lose control of their movements.Trapped in the "dance." It is estimated that this irreversible rare disease affects approximately30,000 people, the social reality issues triggered by Huntington's disease are increasingly receiving attention.

Recently, according to the U.S. Food and Drug Administration (FDA) Email confirmation, ExoRNA Bio (Nanjing) Co., Ltd. (hereinafter referred to as ExoRNA Bio) nucleic acid drug "ER2001 Injection" clinical trial application (IND)It has been approved. According to reports, the drug is the first candidate product developed on ExoRNA Bio's third-generation in vivo self-assembling siRNA nucleic acid delivery technology platform and is being developed for the treatment of Huntington's disease, which currently has no cure. Previously, theThe drug also receivedFDA Orphan Drug Designation.

"Based on the preclinical experimental results, with the support of the company's unique third-generation in vivo self-assembling siRNA delivery technology platform, ExoRNA Bio is expected to overcome the challenges in treating central nervous system diseases such as Huntington's disease.""Dr. Xia Meng, CEO and co-founder of ExoRNA, said."

Innovative Nucleic Acid Drugs May Crack the "Dance of Death"

As a rare autosomal dominant genetic disorder, patients with Huntington's disease often exhibitNeurological dysfunction, motor, cognitive, and psychiatric abnormalities. Current clinical information shows that the peak onset age for Huntington's disease patients is between 30 to 50 years old, with an average life expectancy of approximately 15 to 20 years post-onset. The disease has extremely high rates of disability and mortality, with patients gradually losing the ability to care for themselves amidst uncontrollable "dancing" movements. Existing medications and surgical treatments can only alleviate some symptoms and cannot address or cure the root cause of the disease.

The research results indicate that Huntington's disease is caused byMutant Huntingtin Protein (mHTT) Abnormally aggregates into large molecular clusters within cells, leading to neuronal cell death. Currently, ReduceThe mHTT protein is considered the fundamental and optimal strategy for treating this condition. ExoRNA Bio's self-developed ER2001It is precisely along this therapeutic approach that the First-In-Class Class I innovative drug was formed.

As the first product globally to adopt the third-generation in vivo self-assembling siRNA delivery technology platform, preclinical experimental results have confirmed that after intravenous administration of ER2001 injection, it can cross the blood-brain barrier to reach the affected area, exerting a therapeutic effect. Safety evaluation studies show that the intended human dose is far below the maximum feasible dose in animals. Additionally, ExoRNA Bio has previously completed extensive analyses, including off-target risk assessments and more.Evaluation, ExclusionPotential Safety Risks of ER2001 After Intravenous Administration, successfully achieving IND tacit approval.

ExoRNA Bio's Innovative Nucleic Acid Drug Delivery Platform Draws Capital Attention

The key to ER2001 achieving its expected efficacy and safety lies in ExoRNA Bio's independently developed third-generation nucleic acid delivery technology platform. According to reports, this is also the world’s first self-assembling nucleic acid drug delivery platform capable of targeting the central nervous system via intravenous administration.

Based on this platform, ExoRNA Bio has applied the concept of synthetic biology. By modifying different components such as targeting markers and active siRNA sequences, specific targeted endogenous exosomes are produced in cells for the assembly of therapeutic small nucleic acids. Upon secretion, various nucleic acid drugs can be delivered to different organs, tissues, and cells., achieving a breakthrough in the "effective delivery system of nucleic acid drugs outside the liver." The highly promising technology platform and rapidly advancing innovative pipeline have also garnered attention from investors for ExoRNA Bio.2023In the year, ExoRNA Bio completed a multi-million-dollar angel+ round of financing led by the well-known fund Yuexiu Industrial Fund, despite the adverse market conditions. The earlier angel round of financing was also led by the renowned institution CDH Investments. It is reported that ExoRNA Bio's new round of financing is currently underway.

“ER2001"The approval of the FDA's IND for the injectable solution also excites us, as it represents a significant milestone in the company’s development. This clinical approval also signifies comprehensive and high recognition from international drug regulatory authorities for our original, differentiated third-generation small nucleic acid delivery system, highlighting the company's innovation capabilities and international competitiveness in the field of nucleic acid therapeutics, especially in treating central nervous system diseases."Dr. Mengxia stated.

According to Dr. Meng Xia, the safety and efficacy of ER2001 injection have also been preliminarily validated in clinical IIT studies. The company is actively promoting the IND registration application for this product in China, with the hope of officially entering the registration clinical study within the year.In the future, ExoRNA Bio will continue to focus on unmet clinical needs in the field of central nervous system diseases and other areas by utilizing its unique third-generation in vivo self-assembling siRNA delivery system, aiming to build a world-leading platform-based RNA innovative drug company.。