In this article, we will discuss the 10 Best Gene Therapy Stocks to Buy Right Now.

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.

With this in mind, let us now have a look at the 10 Best Gene Therapy Stocks to Buy Right Now

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).

10 Best Gene Therapy Stocks to Buy Right Now

10) 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).

9) MeiraGTx Holdings plc (NASDAQ:MGTX)

Number of Hedge Fund Holders: 14

MeiraGTx Holdings plc (NASDAQ:MGTX) is a clinical-stage gene therapy company, which focuses on developing treatments for patients with serious diseases.

MeiraGTx Holdings plc (NASDAQ:MGTX)’s primary areas of research include treatments for xerostomia (dry mouth), XLRP, and Parkinson’s disease. The company continues to make significant strides in its regulatory strategy. MeiraGTx Holdings plc (NASDAQ:MGTX) has reached an important alignment with the U.S. Food and Drug Administration (FDA) regarding its ongoing placebo-controlled Phase II trial for xerostomia treatment. This alignment might potentially support a Biologics License Application (BLA) as early as 2026. This will mark a significant milestone in the company’s commercialization journey.

The company announced that the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to AAV2-hAQP1 for the treatment of Grade 2/3 radiation-induced xerostomia (RIX). Notably, AAV2-hAQP1 is an investigational gene therapy for the treatment of radiation-induced xerostomia (RIX), a severe dry mouth condition caused by radiation therapy for head and neck cancers.

The RMAT designation is expected to enable MeiraGTx Holdings plc (NASDAQ:MGTX) to benefit from increased interactions with the FDA to further accelerate the development pathway and BLA approval. Apart from an accelerated pathway to approval and reduced regulatory risk, MeiraGTx Holdings plc (NASDAQ:MGTX) will also get a significant first-mover advantage in this therapeutic area.

8) Voyager Therapeutics, Inc. (NASDAQ:VYGR)

Number of Hedge Fund Holders: 15

Voyager Therapeutics, Inc. (NASDAQ:VYGR) is a biotechnology company, which focuses on the treatment of gene therapy and neurology diseases.

Voyager Therapeutics, Inc. (NASDAQ:VYGR)’s revenue growth is expected to stem from its innovative platform, proprietary technologies, and diverse pipeline. Its wholly-owned pipeline programs consist of an anti-tau antibody for Alzheimer’s disease (AD), a superoxide dismutase 1 (SOD1), silencing gene therapy for amyotrophic lateral sclerosis (ALS), and tau silencing gene therapy for AD.

Voyager Therapeutics, Inc. (NASDAQ:VYGR)’s lead product candidate is VY7523, which is an anti-tau antibody for AD. Notably, enrollment and dosing are now completed in the single ascending dose clinical study and top-line safety and pharmacokinetic data is expected in H1 2025.

Voyager Therapeutics, Inc. (NASDAQ:VYGR) plans to initiate a multiple ascending dose (MAD) clinical trial in Alzheimer’s patients in 2025, with initial tau PET imaging data anticipated in H2 2026. Alzheimer’s disease represents a substantial market with high unmet medical needs. An effective anti-tau therapy could capture significant market share, potentially driving substantial revenue upon approval and commercialization.

As per Grand View Research, the global Alzheimer’s disease diagnostics market size was pegged at US$7.54 billion in 2023 and should grow at a CAGR of 11.4% from 2024 to 2030.