Cell therapy weekly: European facility to accelerate advanced therapies
This week: DNA Nanobots (OH, USA) and the University of California Berkeley (CA, USA) signed an exclusive commercial licensing agreement for non-viral gene delivery technology, Thermo Fisher Scientific (MA, USA) unveiled a new ultra-cold facility to support cell and gene therapy clinical trials in Europe and Be Biopharma (MA, USA) received Orphan Drug Designation from the US Food and Drug Administration (FDA: MD, USA) for its engineered B-cell therapy for hemophilia B.
The news highlights:
- Exclusive commercial licensing agreement for non-viral gene delivery technology
- European facility to accelerate advanced therapies
- Engineered B-cell medicine receives Orphan Drug Designation
Exclusive commercial licensing agreement for non-viral gene delivery technology
DNA Nanobots, a biotechnology company developing customizable DNA nanoparticles, has entered an exclusive commercial licensing agreement with UC Berkeley. This agreement grants DNA Nanobots access to UC Berkeley gene editing technology, which enables genes to be folded into their own delivery devices.
The proof of concept for the technology was published in Nucleic Acids Research in 2022.
Christopher Lucas, Chief Scientific Officer of DNA Nanobots, explains, “Gene and cell therapies offer great promise to treat and potentially cure genetic diseases and cancers, but current viral gene delivery approaches have significant limitations including toxicity and immunogenicity, extremely high manufacturing costs, lack of particle uniformity, limited tissue targeting, and restrictions on gene size. DNA Nanobots plans to combine the licensed UC Berkeley tech with our expertise and existing [intellectual property] to form uniform gene delivery systems with programmable attachment of tissue-specific targeting molecules.”
European facility to accelerate advanced therapies
Thermo Fisher Scientific has inaugurated a new current good manufacturing practice, ultra-cold facility in Bleiswijk, Netherlands to accelerate the development of advanced therapies and address growing demand from cell and gene therapy clinical trials in Europe. The facility will specialize in ambient to cryogenic storage, clinical and commercial packaging, labeling and distribution, and clinical Qualified Person release services.
Molly Flick, vice president and general manager of bioservices and specialty logistics at Thermo Fisher Scientific, stated: “The opening of this cutting-edge facility supporting the development of cell and gene therapies and other biologics, coupled with our proven track record in managing valuable materials, places us in an unmatched position to help our global clients develop innovative therapies.”
Engineered B-cell medicine receives Orphan Drug Designation
A novel engineered B-cell medicine has been granted Orphan Drug Designation by the US FDA. BE-101 is being developed to treat hemophilia B and will enter Phase I/II clinical trial in the second half of this year.
“Despite recent advances, patients with hemophilia B still suffer from bleeding events, joint damage and chronic pain,” said Joanne Smith-Farrell, CEO of Be Biopharma, the company developing BE-101. “BE-101 has the potential to be the first and only Factor IX replacement therapy that is extremely durable, re-dosable and titratable, representing an opportunity to dramatically improve the treatment paradigm in hemophilia B. The Orphan Drug Designation reinforces the potential of this therapy and underscores the need for improved therapeutic options for these patients.”
This designation is granted by the FDA to medications or biologics aimed at treating rare diseases or conditions affecting fewer than 200,000 individuals in the United States. The designation provides several key benefits, including seven years of exclusive marketing rights post-approval, exemption from user fees, and eligibility for tax credits on qualified clinical trials.
