Issue 222
Is your company reshaping the technological future of pharma, either in manufacturing or drug development? Or are you aware of a company that’s breaking the mold and developing new tech unlike any other?
If you said yes to one of these questions, I urge you to check out the latest installment of The Medicine Maker Innovation Awards – an annual competition that recognizes the greatest technological advancements our industry has to offer from the past year.
Whether it be new equipment, software, instruments, or formulation technology, we’re looking for admissions across the various fast-moving areas of drug development and manufacture – and that includes cell and gene therapy! All products that have launched this year are eligible, which means there's still half a year of potential innovation ahead of us.
We’re accepting nominations from individuals, groups, or organizations, though vendors are encouraged to submit their latest and greatest products. We would also like to hear from those of you using the technology “in the trenches” in labs and factories.
The nomination form is available here. You simply need to fill out the name of the technology, release date, and why you feel it deserves a place in our Innovation Awards. Nominations will close on October 15.
Until next week,
Jamie Irvine | Associate Editor
Essential Reading
The foam effect
A team of bioengineers from the Fred Hutchinson Cancer Center, Seattle, Washington, recently reported that foaming liquid formulation performed better than a standard liquid formulation at transferring gene therapy components to cells. When they mixed the foam with cells, they noticed the foam significantly boosted gene transfer and outperformed liquid formulations. Next, they injected a small amount of this foam into the abdominal cavity of mice, observing a successful transfer from the foam to the targeted cells without causing significant side effects. In a press release, Mattias Stephan, lead author and professor in the translational and therapeutics division at Fred Hutch, said, "Our gene therapy foam shows for the first time that by taking a small amount of an expensive gene therapy drug, increasing its volume by embedding it in a solution that is mostly made of densely packed air bubbles and then applying it to cells, we can achieve a strong and safe transfer of gene therapy agents to cells."
The SCD bandwagon
BioLineRx has announced a multi-center phase I clinical trial sponsored by St. Jude Children's Research Hospital to evaluate motixafortide for the mobilization of hematopoietic stem cells used in the development of gene therapies for patients with sickle cell disease. Their decision follows the FDA’s recent approvals of Casgevy and Lyfgenia – and considering motixafortide, in combination with filgrastim, received FDA approval for patients with multiple myeloma in late 2023, its developers are optimistic about its prospects. Enrollment is expected to begin in the next few months.
Worth Your Time...
Business
Cytiva unveils new cell therapy manufacturing platform – named Sefia – to improve patient access and wider adoption of autologous CAR T.
Cellular Origins and the Cell and Gene Therapy Catapult collaborate to develop a robotic platform for the universal automation of cell and gene therapy manufacturing.
ScaleReady launches G-Rex Grant Program – a $20 million initiative for academic and commercial institutions engaged in various aspects of cell and gene therapy development.
Research
Researchers introduce an agent-based model to rationalize the outcomes of different CAR T-cells therapies strategies over heterogeneous tumor-derived organoids.
Nature article assesses whether scientists can control the dangerous immune response associated with second dose gene therapies.
Scientists explore the therapeutic potential of AAV-mediated gene therapy for the treatment of sialidosis – a glycoprotein-based storage disease.
