We recently compiled a list of the The 10 Best Gene Therapy Stocks to Buy Right Now. In this article, we’ll look at where Beam Therapeutics Inc. (NASDAQ:BEAM) stands against other gene therapy stocks.
Gene therapy is known as a medical technique that involves modifying or manipulating genes to treat or prevent diseases. The therapy focuses on addressing the underlying genetic cause of a condition by repairing, replacing or altering genes within an individual’s cells. Industry experts believe that this innovative therapy has the potential to treat a variety of genetic disorders, cancers and other diseases.
According to IMARC, healthcare infrastructure in the US has adapted to accommodate gene therapies. According to the IQVIA count, 114 gene therapy trials were initiated in 2023. Of these, a large majority (~77%) were sponsored by the healthcare industry. According to Kella Kapnisi, head of cell and gene therapy at Team Consulting (a medical technology design and development consultancy), there are 38 cell and gene therapies approved by the FDA so far. In particular, many of them have reached commercialization through laboratory manufacturing processes, mainly manual.
According to IMARC, the size of the US gene therapy market has been set at $1,312.2 million in 2024. The market should reach $3,697.8 million. by 2033. This phenomenal growth is expected to stem from the increasing prevalence of genetic disorders, advances in genetic engineering, a strong focus on strategic collaborations, supportive regulatory frameworks and increased investments in R&D.
Since the inception of the Orphan Drug Designation program, the FDA has designated more than 1,000 drugs for rare diseases as orphan drugs, many of which are gene therapies. Furthermore, optimism around the gene therapy industry grew after the regulatory body showed its dedication to promoting the development of these drugs in 2023. IMARC added that the FDA approved three novel gene therapies for conditions such as spinal muscular atrophy and hemophilia.
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According to Kella Kapnisi, as the cell and gene therapy industry has continually evolved, the need and requirement for purpose-built technology is of utmost importance. Customized small-batch manufacturing systems for personalized therapies and decentralized manufacturing, real-time process control with the help of advanced online and offline analytics, and sophisticated data management and analytics systems using ML technology remain important trends that could drive cell growth and gene therapy manufacturing.