Several significant developments have aided and accelerated the growth of markets in biotechnology, in the period of review. Notable recent advancements in biotechnology are CRISPR, Spheroids for drug development, Quantitative Cell Based Assay using high content analysis platform and Luciferase based high-throughput screening assay. Such technologies assure the wide adoption of cell line technologies for drug development, simplify the gene editing process making it cost-effective.
Companies operating in cell line market are investing huge amount in setting up manufacturing units with advanced state of art facility and expanded production facilities. At the same time, the genetic sequencing technology is advancing at a great pace especially in the healthcare sector in diagnosis of various cancers.
Gene therapy technology has opened new avenues which places emphasis on curing disease that do not have an effective therapeutic availability. Johnson & Johnson, together with the University of Pennsylvania, is developing a third-generation AAV-based gene therapy for Alzheimer’s disease. The goal is to use AAV viral delivery to initiate the expression of therapeutic anti-Alzheimer’s antibodies in the brain. Novartis launched Zolgensma for the treatment of spinal muscular atrophy. The research represents a novel way to get biologics into the brain to treat Alzheimer’s disease and other neurological diseases. This is expected to pave way for further innovations in AAV-based gene therapy.
The sector continuously faces some challenges related to marketing, paying for innovation, pricing pressures from the government organizations.
Paying for development costs is always most critical part for the manufacturers. In 2019, gene therapies and many cell biology technologies are developed to address the needs of some important diseases and proven to be effective. However, this comes for a high cost. For example, Zolgensma, a viral vector based gene therapy for the treatment of spinal muscular atrophy costs around $5 million. It is important to figure out how to pay for the innovation which is quite challenging.
The pricing and paying for getting the products to market are important challenges. The innovation may take several years from clinical trial phase to get launched in the market. The investors may be disappointed if the company doesn't show expected profits and loses the hope while stock prices fall. This means that companies must carefully manage expectations and maintain balance.