We recently compiled a list of the 10 Best Gene Therapy Stocks to Buy Right Now. In this article, we are going to take a look at where bluebird bio, Inc. (NASDAQ:BLUE) stands against the other gene therapy stocks.
Gene therapy is referred to as a medical technique that involves modifying or manipulating genes to treat or prevent disease. 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 opine that this innovative therapy has the potential to treat a variety of genetic disorders, cancers, and other diseases.
As per IMARC, healthcare infrastructure in the US adapted to accommodate gene therapies. As per IQVIA’s tally, 114 gene therapy trials were initiated in the year 2023. Out of this, a large majority (~77%) were sponsored by the healthcare industry. As per Kella Kapnisi, Head of Cell and Gene Therapy at Team Consulting (a medical technology design and development consultancy), as of now, there are 38 cell and gene therapies approved by the FDA. Notably, many of these have reached commercialization through mostly manual laboratory manufacturing processes.
Growth Drivers of The Gene Therapy Industry
As per IMARC, the US gene therapy market size has been pegged at US$1,312.2 million in 2024. The market should reach US$3,697.8 million by 2033. This phenomenal growth is expected to stem from the increasing prevalence of genetic disorders, genetic engineering advancements, a strong focus on strategic collaborations, supportive regulatory frameworks, and heightened investments in R&D.
Ever since the initiation of the Orphan Drug Designation program, the FDA designated over 1,000 medicines for uncommon diseases as orphan drugs, many of which are gene therapies. Furthermore, optimism around the gene therapy industry grew after the regulatory body showcased its dedication to promoting the development of these medicines in 2023. IMARC added that the FDA approved 3 novel gene therapies for conditions such as spinal muscular atrophy and hemophilia.
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Trends to Drive Growth in 2025
As per Kella Kapnisi, as the cell and gene therapy industry has been continuously evolving, the need and requirement for purpose-built technology is of utmost importance. Small batch manufacturing systems that are customized for personalized therapies and decentralized manufacturing, real-time process control with the help of advanced inline and online analytics, and sophisticated data analysis and management systems using ML technology remain important trends that might drive the growth of cell and gene therapy manufacturing.
As per IMARC, the collaborations between biotech companies, pharmaceutical firms, and university research institutes can pool the funds needed to address challenges related to creating and promoting gene treatments.
IMARC further added that the increased prevalence of genetic disorders and chronic diseases, together with increasing strategic collaborations and partnerships are important trends in the US gene therapy market. The growing cases of cancer and genetic disorders remain one of the key factors in the gene therapy market. Gene therapy is a key component of cancer treatment as it can precisely research and target malignant cells to prevent further damage.
Our Methodology
To list the 10 Best Gene Therapy Stocks to Buy Right Now, we conducted extensive research and scanned through several online rankings. After getting the initial list of 15-20 stocks, we shortlisted the ones having high hedge fund holdings. Finally, the stocks are arranged in ascending order of their hedge fund sentiments, as of Q3 2024.
At Insider Monkey we are obsessed with the stocks that hedge funds pile into. The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 275% since May 2014, beating its benchmark by 150 percentage points (see more details here).
A medical researcher inspecting a petri-dish filled with bacteria in a laboratory setting.
bluebird bio, Inc. (NASDAQ:BLUE)
Number of Hedge Fund Holders: 14
bluebird bio, Inc. (NASDAQ:BLUE) is a biotechnology company, which researches, develops, and commercializes gene therapies for curative severe genetic diseases in the US.
bluebird bio, Inc. (NASDAQ:BLUE)’s product portfolio includes treatments for sickle cell disease (SCD), beta-thalassemia (BT), and cerebral adrenoleukodystrophy (CALD). Its flagship product, LYFGENIA for SCD, is at the forefront of its commercialization efforts. LYFGENIA is a one-time gene therapy to treat sickle cell disease in individuals 12 years of age or older and a history of vaso-occlusive events (VOEs).
Total revenue (Net) came in at $10.6 million in Q3 2024 as compared to $12.3 million for Q3 2023, driven by quarter-to-quarter variability in drug product infusions. Revenue for the third quarter consists of revenue from LYFGENIA, following the completion of the first infusion for SCD. bluebird bio, Inc. (NASDAQ:BLUE) previously guided to an anticipated reduction of net revenue in the third quarter. However, it now expects net revenue of at least $25 million in Q4 2024, as previously reported patient starts are infused.
Despite advancements, the adoption of gene therapies like LYFGENIA faces challenges, including high costs and complex treatment processes. To address these challenges, bluebird bio, Inc. (NASDAQ:BLUE) continues to focus on expanding its network of qualified treatment centers and securing favorable reimbursement policies. As of August 2024, the company has activated 70 qualified treatment centers for LYFGENIA and ZYNTEGLO, and 6 for SKYSONA. This strategic expansion should facilitate patient access and fuel revenue growth. ZYNTEGLO is a gene therapy developed for the treatment of beta-thalassemia and SKYSONA is a gene therapy developed for the treatment of cerebral adrenoleukodystrophy (CALD).
Overall BLUE ranks 10th on our list of the best gene therapy stocks to buy right now. While we acknowledge the potential of BLUE as an investment, our conviction lies in the belief that some deeply undervalued AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for a deeply undervalued AI stock that is more promising than BLUE but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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Disclosure: None. This article is originally published at Insider Monkey.
