Issue 199
How many times have you heard phrases like: “gene therapy is a rapidly evolving industry with the potential to transform patients’ lives?” I’m guessing quite a few. But “potential” only gets us part of the way – and there are numerous obstacles standing in the way of progress, with each one (big and small) demanding attention.
We were recently joined by Elpidia Gamez of Thermo Fisher Scientific who told us how even the format choice for cell culture media can make a difference in gene therapy development.
Gamez said that, though liquid formats are convenient for small-scale development, scaling up poses challenges in shipping and storage costs. Dry powder media addresses these issues but requires rehydration, which adds further complexity. In this regard, Gamez suggested that granulated media offers a compromise.
For Gamez, “Granulated options provide a simpler reconstitution process and lower dust generation without the need for pH or osmolality adjustments, increasing efficiency and reducing the risk of inconsistency. Moreover, supplements can be integrated into a granulated format, effectively resulting in a convenient single-component product.”
What other practical solutions have you seen emerging – even small innovations that take us from “potential” to “transformative?” Let us know.
Until next week,
Jamie Irvine | Associate Editor
P.S. As the end of the year approaches, I wanted to take the opportunity to extend warm wishes to you and your loved ones. Whatever your beliefs, I hope you embrace the joy of togetherness and reflection. Happy holidays!
Essential Reading
Every CAR Matters
Early results from a Phase I clinical trial of AT101, a novel CAR T cell therapy targeting CD19, has revealed a 100 percent complete response rate at higher doses. The study, published in Molecular Cancer, took place in South Korea with 12 relapsed or refractory B cell non-Hodgkin's lymphoma patients and employed a unique binding mechanism through a different CD19 epitope – namely, h1218. “We were not expecting such a drastic early difference in this study,” said Marco Ruella, an assistant professor of Hematology-Oncology and Scientific Director of the Lymphoma Program, in a press release. “The CART19 products that are already FDA-approved are very effective, and it’s not easy to do better. While there is not a randomized trial of this product yet, the initial results seem very promising, and we look forward to moving into the planned Phase II portion of the study.” In the future, the researchers hope to include patients who have previously received CAR19 therap
Hope for Hemophilia B
CSL has unveiled three-year results from the HOPE-B study, confirming the efficacy and safety of HEMGENIX, a gene therapy treatment for individuals with hemophilia B. The data reveals that a one-time infusion of HEMGENIX sustains elevated factor IX levels and reduces annual bleeds for years. More specifically, of the 54 participants, HEMGENIX maintained mean factor IX levels and 94 percent of patients remained free of continuous prophylactic therapy. The three-year data also revealed a sustained 64 percent reduction in the mean annualized bleeding rate for all bleeds in alignment with the study's primary endpoint.
Worth Your Time...
Business
University of Chicago Medicine Comer Children’s Hospital among first in country to offer newly approved gene therapy for sickle cell disease to patients 12 years and older.
ProBioGen receives Japanese patent for DirectedLuck technology – a gene delivery system based on active transposase fusion variant.
Governor Hochul releases proposal request for cell and gene therapy hub on Long Island to advance life-saving cancer treatments
Research
Research suggests stem cell treatment using mRNA technology may promote cell-to-hepatocyte conversion in acute and chronic liver diseases.
Cedars-Sinai investigators use therapeutic cells derived from the heart to improve cardiopulmonary function in people in phase I trial.
CERTAINTY initiative aims to develop virtual twin that will improve treatment with personalized cancer immunotherapies, such as CAR-T cell therapy.
