AskBio shares technology

Opens nonprofit focused on orphan drug development

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CHAPEL HILL, N.C.—Responding to repeated requests by the scientific community for access to its proprietary technologies, Asklepios BioPharmaceutical (AskBio) formed the Chapel Hill Project, a nonprofit organization dedicated to the development of cell- and gene-based therapies for orphan diseases (see Orphan success). The company hopes the project will serve as the basis for wider collaboration within the research community and will be of particular interest to groups that can't afford the up-front fees typically associated with technology-access agreements.

"Without the exposure and potential capital associated with pursuing traditionally sized pharmaceutical markets, resources can be rate limiting to translational research, which has a direct impact on the timing of getting therapies to patients," says Jade Samulski, AskBio director of program management, explaining the key challenge of tackling orphan diseases.

To facilitate this effort, AskBio has provided the Project access to its Biological Nano Particles (BNP) and self-complementary vector technologies, both based on recombinant adeno-associated virus (rAAV) gene delivery vehicles.

"AskBio has developed a library of chimeric BNPs based on integrating capsid amino acids from AAV and other viruses," Samulski explains. "The BNPs are designed to be tissue-selective, immunologically distinct, and engineered specifically to support scale-up manufacturing and purification platforms.

The self-complimentary or "dimeric" vector, meanwhile, is designed to overcome a limitation of traditional AAV technology, he continues.

Traditional AAV vectors generate their DNA cargo in a single-stranded form that has to be converted to double-stranded DNA, slowing down the process. AskBio's dimeric technology, however, produces fully double-stranded AAV or BNP vectors. Samulski says the dimeric vector-BNP vector combination offers significant advantages in vector delivery efficiency, drug kinetics, and protein expression levels.

Based on its earlier experiences with academic and nonprofit collaborations, AskBio expects researchers to submit abstracts of their research and development proposals to the Project for evaluation by a scientific peer-review panel comprised of key industry clinicians and researchers, who will be looking at questions of scientific merit and translational logistical planning.

"The Project intends to be able to assist research at several different levels," Samulski says. "First is getting our most advanced technology to the researchers in an expedited fashion. Next, we intend to allow AskBio's regulatory files to be cross-referenced, which includes safety data on the BNPs, and where appropriate, we will assist with program planning and logistics to ensure the researchers have the highest probability of success."

As he explains, removing IP barriers to development will hopefully increase the ability of researchers to get the financial backing required to support their efforts.

"The primary focus of the Project is to provide a space in which researchers pursuing rare and neglected diseases are empowered to relieve the suffering of under-served patients of these devastating diseases," he says. "Insofar that the Project's interactions grow organically into collaborations and increase use of AskBio's technology, we believe that will only strengthen the field of gene delivery and provide an effective path to securing our success."

Initially, the company expects most of the interest to come from within the United States, but according to Samulski, AskBio is very conscientious of the fact that genetic diseases have no borders, and that the Project's IP may support global development of treatments for neglected diseases.

"As the Project's vision gains momentum, we are anticipating significant interest from developing nation companies wanting to contribute to the effort, and look forward to empowering researchers in the developed and developing world to bridge some of the gaps in public health that exist."

If the company can get other pharmaceutical and biotechnology firms to join the Project—or its efforts induce other companies to start their own projects—so much the better; for everyone.

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