Technology | BNP Platform
DNA, which is the blueprint for the human body is organized into segments called genes. Each individual gene provides specific instructions to the body's cells on how to manufacture proteins which are critical to maintaining normal body functions. Occasionally, the DNA's genes can be corrupt causing aberrant protein expression in the cells. Many of the disease states in humans today are caused by improper production or the absence of these proteins. Isolating and then transporting a corrected gene safely into the cells can increase or decrease protein production, as well as introduce a replacement gene which can express a missing therapeutic protein.
Historically there have been varying delivery methods and vehicles used in the attempt to safely deliver therapeutic genes. Use of viruses, called viral vectors, takes advantage of a viruses' natural ability to introduce DNA to cells. Viral vectors have been the more recent delivery vehicle employed to safely and efficiently deliver a therapeutic genetic payload. Several different recombinant viral vectors have been harnessed for the delivery of genes, including adenovirus, herpes simplex virus, lentivirus, and adeno-associated virus. However due to its non-pathogenic properties and absent immune response, ability to maintain efficient and long-term expression, and ease of genetic manipulation, recombinant adeno-associated virus (rAAV) has quickly become the gene-delivery vector of choice for a multitude of targets.
Asklepios BioPharmaceutical, Inc., has now developed a library of rAAV Biological Nano Particles (BNPTM), the next generation of gene delivery platform technology. These synthetically designed particles provide unsurpassed safety, transduction efficiency, "ramp-up" expression capabilities, with multiple tissue targeting transcapsidation. The proprietary rAAV BNPTM technology combined with the company's complimentary enabling technologies, (which includes proprietary gene, clinically proven production protocols, regulation system, scaleable production, proprietary purification system, and bio-stint technology), poises Asklepios as the company to bridge the current technical gap of reliable, safe, and efficient genetic cargo delivery (e.g. genes, RNAi, transcriptional targeting promoters, etc.).
AskBio is addressing Duchenne's muscular dystrophy as well as other diseases with a proprietary break through technology, rAAV Biological Nano ParticlesTM ("BNP"). BNPsTM are synthetically designed from components of different associated adeno viruses as well as other parvoviruses. These bio-particles have been selected and engineered specifically for their biological propensities for targeting and transducing muscle tissue, as well as being immune evasive and pathogenically safe. Once administered to the patient, the BNPsTM transport the gene, RNAi, small molecules or other genetic cargo of choice which have been coded for a desired therapeutic result.
The BNPTM technology can be directly introduced to the patient or combined with the proprietary Matrix Systems, which allows AskBio to apply the bio-particles to stints, sutures, and other medical devices. These devices are implanted into the patient, which slowly elute the therapeutic particles directly into the diseased tissue. Upon particle delivery the muscle tissue acts as its own bioreactor and begins producing a sustained dosage of therapeutic protein for years. Having been studied extensively in animal models for over two decades, the biology of BNPsTM is well known. With an expected successful implementation of a Phase-I safety muscular dystrophy clinical trial in 2005, AskBio's BNPTM technology is fast becoming a proven reality, setting a new standard for safe and efficient bio-particle therapeutics.