January 13, 2022, Wuhan Healthgen Biotechnology Corp. (hereinafter referred to as “Healthgen Biotech”) received the approval of the US Food and Drug Administration (For short "FDA") for the clinical trial of Alpha 1-antitrypsin (AAT) for injection (Project No. HY1003) independently developed by Healthgen Biotech. This is the world's first recombinant AAT and the second recombinant blood product of Healthgen Biotech approved by the FDA to enter clinical trials. The project was granted orphan drug designation by the U.S. FDA in 2020 (DRU-2019-7242), which can be used for the treatment of emphysema caused by AAT deletion or other causes. The FDA believes that the recombinant AAT produced by the rice expression platform can avoid contamination by infectious pathogens and has obvious advantages in safety compared with the same variety of drugs derived from human blood.
AAT is encoded by the SERPINA1 gene on human chromosome 14. Mutations in this gene cause low or denatured plasma levels of AAT, which damages the tiny air sacs (alveoli) in the lungs, causing Emphysema. When the alveoli are damaged, the lungs cannot expand and contract properly, resulting in reduced alveolar ventilation, and patients experience shortness of breath, coughing, or wheezing. As the lungs deteriorate, many patients develop pathological changes in lung tissue that cause asthma or chronic bronchitis, resulting in emphysema (AAT deficiency, AATD).
AATD is a higher incidence in Caucasians. The number of patients with emphysema caused by AATD is about 80,000 to 100,000 in the United States, and about 650,000 in the world. At present, clinical AAT drugs are completely extracted from human plasma. In the United States, only about 45% of patients can use this drug due to drug shortage, and the treatment cost is as high as 127,000 US dollars/person/year, and its market prospect is huge.
Healthgen Biotech is committed to the research and development of a series of new recombinant protein biological biopharmaceuticals using the self-developed rice endosperm cell-specific and high-efficiency expression platform OryzHiExp and purification technology platform OryzPur, and has established a world-leading plant biopharmaceutical platform and perfect industrialization. Healthgen Biotech is an internationally renowned biopharmaceutical enterprise focusing on biopharmaceuticals research and product development from plant platform. The clinical trial approval of AAT is licensed to follow Healthgen Biotech HY1001 (Recombinant Human Serum Albumin) and HY1002 (Recombinant Human Lactoferrin Lysozyme Oral Liquid), and AAT is the third original innovative biopharmaceutical that from plat platform, which demonstrating the great advantages and strong research and development capabilities of the Healthgen Biotech recombinant protein biopharmaceuticals technology platform.