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.
Cutting-edge bioprocessing is critical to guarantee yield and clinical performance of AAV gene therapies. With multiple-year expertise for AAV sciences and engineering, BBM has established suspension system and high-yield cell line for AAV production. We have validated and standardized manufacturing for a spectrum of batch sizes from 200L to 500L in Minhang development & manufacturing center. Large-scale cGMP manufacturing facility in Lingang Special Area of Shanghai Pilot Free Trade Zone is under construction and will launch commercial lot in 2022.
Design and operation of Minhang development & manufacturing center is compliant with GMP standards. The 5000 m2 facility comprises of functions of plasmid production & purification, cell expansion, scalable production & purification of virus & therapies, fill & finish, and quality control, all of which are designed in a manner of fully integrated production. The facility was designed to accommodate needs of innovative gene therapy candidates from pre-clinical stages to clinical development and commercialization.
Meanwhile, the company has a bioprocessing development team and an analytical/quality team under the same roof. This enables us to develop state-of-the-art processes and analytical methodologies in house, which can be transferred to (c)GMP manufacturing by following internal project procedure. The mechanism for BBM to develop its own processes and talents is aimed to ensure that know-how gained during process development is retained within the company, foundation of our long-term vision.
Belief BioMed manufacturing team consists of hundreds of well-trained multidisciplinary staffs with expertise in process development, manufacturing, quality control and analytics, regulatory policy and project management of gene therapies.
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.