Gene therapies are employed to treat or prevent diseases caused by single gene mutation or expression failure. They offer an unprecedented opportunity, even a cure, for patients with diseases that existing interventions are inadequate. A viral vector is commonly used to deliver a normal copy of the gene to the cells. As a result, the transgene expresses normal proteins in vivo, which will rescue or enhance function and provide clinical benefits to patients.
The recombinant adeno-associated virus (AAV) vector used in gene therapy drugs developed by Belief BioMed is the industry's leading platform for in vivo gene delivery. Compared with other delivery vectors, AAV brings significant benefits due to its non-pathogenicity, low immune response, high transduction efficiency, improved tissue/cell specificity and long-term transgene expression activity. In addition, promoter, as well as other components, is designed and incorporated into cassette to drive different levels of gene expression or to limit it to specific cell types.
Belief BioMed holds comprehensive patent portfolio covering a variety of gene therapy targets, capsids & expression cassettes. Leveraging internal AAV capsid development platform, we have developed a number of innovative capsids with high tissue specificity and transduction efficiency，and continued evolving them to design a therapy candidate specific to the disease being targeted. We believe the internal customization capability enables us to develop the best-in-class gene therapies in the industry.
With years of accumulation of scientific and engineering expertise in Adeno-associated Virus (AAV), Belief BioMed has established a process that includes the world's leading serum-free culture process for HEK293 cell suspension and a full-scale chromatographic purification process. The Minhang R&D and manufacturing center has successfully passed the validation test and is capable of handling standardized mass production of AAV from 200L to 500L. In addition, with a production capacity of up to 2000L for a single batch, our manufacturing site located in Shanghai Lingang Special Area is GMP compliant, and can meet the needs for commercial batch production. As of today, it has successfully produced the Investigational Medicinal Products (IMPs) for our first Phase III clinical trial.
Minhang R&D and manufacturing center is designed and operated in compliance with GMP-like standards. The 5,000 square meter facilities include plasmid production and purification, cell expansion, virus packaging, virus purification, finished product filling, and quality control to meet the needs of innovative gene therapies from preclinical stage to clinical development.
A large GMP production base in the Lingang Special Area of Shanghai, with a plant area of 15,000 square meters, including three 200L plasmid production lines; three rAAV production lines with flexible scale switching between 200L, 500L and 2000L; fully automated sterile filling system; Clean In Place (CIP) / Sanitize in Place (SIP) dispensing center; high standard quality control laboratory; The Lingang production base has a pilot production line, including a 50L plasmid fermentation and purification line, a 200L virus packaging and purification line, and a GMP-certified sterile isolator-based filling line, that can meet the production needs from pre-clinical to clinical development stages. The plant equipment and facilities have obtained a variety of validations and are fully compliant with GMP standards, to meet the requirements of phase III clinical study and commercialization of innovative gene therapies.
The management and science team at Belief BioMed is consisted of hundreds of experienced members with expertise in process development, analytical method development, manufacturing, quality control, regulatory policy, and project management for gene therapy. Our team continues to optimize and upgrade the production process and analytical methods of rAAV vectors, and has completed product development, pilot production and IND filing for several gene therapy projects, some of which have entered the clinical trial stage. The in-house development process and people development programs are designed in a way to ensure that the know-how acquired during the development process remains within the company, which is also one of the core competitiveness and technical barriers we strive to build.
Parkinson's disease is a progressive disorder of the nervous system. The disorder affects several regions of the brain, especially an area called the substantia nigra that controls balance and movement.
Generally, Parkinson disease that begins after age 50 is called late-onset disease. The condition is described as early-onset disease if signs and symptoms begin before age 50. Early-onset cases that begin before age 20 are sometimes referred to as juvenile-onset Parkinson disease.
Parkinson's disease affects more than 7 million people worldwide. In China, the prevalence of Parkinson's disease in people over the age of 65 is about 1.7 percent. In the United States, Parkinson's disease occurs in approximately 13 per 100,000 people, and about 60,000 new cases are identified each year.
Hemophilia is a bleeding disorder that slows the blood clotting process. People with this condition experience prolonged bleeding or oozing following an injury, surgery, or having a tooth pulled. In severe cases of hemophilia, continuous bleeding occurs after minor trauma or even in the absence of injury (spontaneous bleeding). Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms of hemophilia do not necessarily involve spontaneous bleeding, and the condition may not become apparent until abnormal bleeding occurs following surgery or a serious injury.
The major types of this condition are hemophilia A (also known as classic hemophilia or factor VIII deficiency) and hemophilia B (also known as Christmas disease or factor IX deficiency). Although the two types have very similar signs and symptoms, they are caused by mutations in different genes. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood but have few bleeding problems after puberty.
The two major forms of hemophilia occur much more commonly in males than in females. Hemophilia A is the most common type of the condition; 1 in 4,000 to 1 in 5,000 males worldwide are born with this disorder. Hemophilia B occurs in approximately 1 in 20,000 newborn males worldwide.