Sailxen Bio Receives FDA Orphan Drug Designation for Autologous Polyclonal Regulatory T Cell Therapy in ALS
Novabio Therapeutics
Developer of Multi-Modal Treg Cell Therapy
On August 22, 2024, Shanghai Saierxin Biomedical Technology Co., Ltd. (referred to as "NOVABIOTX") announced that the company's self-developed autologous human polyclonal regulatory T cells (Treg) for the treatment of amyotrophic lateral sclerosis (ALS) has been granted Orphan-drug Designation by the U.S. Food and Drug Administration (FDA). This is the first Orphan-drug Designation received since the company’s establishment, and another significant milestone following the ethical review approval of the NP001 project by the First Affiliated Hospital of Zhengzhou University.
Drug-related Situations
Orphan drugs, also known as rare disease drugs, are drugs or biologics recognized by the U.S. FDA for the prevention, diagnosis, or treatment of rare diseases or conditions. The recent granting of orphan drug designation by the FDA will help Treg cell therapy drugs enjoy certain policy support in the subsequent research and development, registration, and industrialization in the United States, including but not limited to tax credits for clinical trial costs, exemption from new drug application fees, and a seven-year market exclusivity period after marketing. This designation will, to a certain extent, expedite the progress of clinical trials and marketing registration.
Many autoimmune diseases and neurodegenerative diseases currently lack effective and ideal therapeutic drugs. Amyotrophic lateral sclerosis, abbreviated as ALS and commonly known as "Lou Gehrig's disease," is a progressive and fatal motor neuron degenerative disease. ALS is caused by the degeneration of motor neurons in the central nervous system that control skeletal muscles. Due to the degeneration and death of upper and lower motor neurons, muscles gradually weaken and atrophy. Eventually, the brain completely loses the ability to control voluntary movements, leading to speech, swallowing, and respiratory difficulties. Currently, there are no clinically significant effective treatments for ALS, and the average life expectancy for patients from onset to death is 3 to 4 years.
Research over the past 50 years has proven that natural Treg cells, which specifically express FOXP3 in the nucleus and CD25 on the cell surface, exist in normal individuals and actively participate in regulating immune responses to self, microbial, and environmental antigens that are either free or excessive. Their dysfunction can indeed lead to various immune-related diseases, such as autoimmune disorders and allergies. The NP001 project is a therapy based on autologous Treg cell infusion. Although the exact pathogenesis of ALS remains unclear, with only an estimated 5%-10% linked to genetic mutations, at different stages of disease progression, there is evidence of immune cell infiltration into the central nervous system, attacking myelin and causing neuronal damage. The NP001 product aims to utilize intrathecal injection of Tregs to more directly suppress immune reactions at the site of pathology and recruit oligodendrocytes for neural repair, thereby playing a positive role in ALS treatment.
NOVABIOTX was founded in 2022, focusing on the development of multi-modal Treg cell therapies for treating neurodegenerative and autoimmune diseases. It is the only biopharmaceutical innovation company in China dedicated to the development of Treg cell therapeutic drugs and has made the fastest progress. Treg cells can effectively suppress the proliferation and function of pro-inflammatory cells through multiple mechanisms. After years of research, it has been discovered that Treg cells also possess tissue repair capabilities. Based on the pathogenic mechanisms of different indications, NOVABIOTX has designed a fully independent and innovative R&D pipeline, progressively achieving disease remission through immune suppression, tissue repair, and immune tolerance. Adhering to the core mission of "addressing unmet clinical needs," and upholding the core values of "patient-centricity, integrity, pragmatism, and perseverance," NOVABIOTX is leading the industrial development of Treg therapies.
E.N.D
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