The emergence of genetic engineering and recombinant DNA technologies in the early 1970s created an inflection point across the therapeutic sector, with an intent to alleviate chronic ailments using gene editing techniques. Gene therapy has been evolving as an innovative & enticing approach to effectively deliver & incorporate therapeutic genes at a cellular level using vectors, thereby treating human genetic disorders. However, the treatment cost is one of the influencing factors determining the extent of accessibility & affordability of cell & gene therapies worldwide. The price range of cell & gene therapy is based on the value of a life saved or the improved quality of life within a specific timeframe, also referred to as quality-adjusted life year (QALY) gains.
By Nitin Jindal
To illustrate, the expected price to genetically treat Metachromatic Leukodystrophy—an autosomal recessive lysosomal storage disorder (LSD)— using AVXS-101 (brand name: Zolgensma) is around 2 million Euros per treatment. In addition, the US-based biopharmaceutical company, CSL Behring, has got approval from FDA for its recently developed one-time gene therapy against hemophilia B, Hemgenix. It would cost USD 3.5 million per treatment. On the contrary, the average healthcare expenses to treat or control hemophilia for a lifetime likely reach USD 20 million due to repetitive courses of treatment for an extended period.
Although, it is only feasible for some to spend hefty amounts on genetic disorders treatment. Hence, various companies in the field of biomedical treatment are innovating cost-effective & easily accessible cell & gene therapies. Industries have explored numerous innovative solutions to enable cell and gene therapies to reach more patients. In the last five recent years, researchers have recognized several cost-effective, time or process-efficient technologies to improve the overall manufacturing process of cell and gene therapies.
The exclusive webinar on “Roadmap Toward Cost-Efficient Cell & Gene Therapies” will be organized by Ingenious e-Brain Solutions on 24th January 2023 at 11:00 AM CEST [Europe] and 25th January 2023 at 08:30 AM PDT [US]. The webinar will focus on various cost-effective upstream technologies, such as the optimization of different viral & non-viral vectors and capsid-engineered viral particles, AI-mediated/in-silico-based vector designing, cell culturing methods, AI-assisted genetic/epigenetic modifications, etc. Further, speakers will discuss recently explored refining technologies such as closed bioreactor systems equipped with tiny, automated sensors; magnetic beads; and novel resins; along with novel packaging/ manufacturing methodologies for therapeutic formulation.
The spokespersons will particularly emphasize how AI contributes to generating effective cell/gene therapies, thereby reducing the overall developmental cost of therapeutic candidates expended on R&D activities. AI can significantly accelerate the pace of CAR-T cell/ vector systems designing, feeding, and control. The speakers will have a word on AI/ML-based systems for in silico screening of thousands of CAR/ genetic constructs to identify candidates with high binding affinity to the target cell for immune system activation. This will also unleash the potential of AI in developing personalized DNA/mRNA/CAR-T-based therapies.
To get acquainted with the advancements in developing
cost-efficient & effective cell & gene therapies, please attend this
live webinar by registering@
https://www.iebrain.com/webinars/roadmap-toward-cost-efficient-cell-and-gene-therapies/
About us: Ingenious e-Brain is a business research & analytics firm that assists global clients in identifying solutions to their R&D challenges and meeting their objectives. The organization enables this by offering world-class research through its network of industry experts, ranging from technological advancements to corporate engagements. Ingenious e-Brain values and walks alongside clients' goals, assisting them in developing novel therapeutics, adopting digital or automated processes, mitigating adverse effects of therapies, staying ahead of the competition, meeting business goals, addressing R&D challenges, and planning their short- and long-term roadmaps.
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