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
A scientist in a lab conducting research on cell-based therapeutics and biotechnology.
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.
7) Prime Medicine, Inc. (NASDAQ:PRME)
Number of Hedge Fund Holders: 21
Prime Medicine, Inc. (NASDAQ:PRME) is a biotechnology company, which delivers genetic therapies to address the spectrum of diseases by deploying gene editing technology.
One promising program in Prime Medicine, Inc. (NASDAQ:PRME)’s pipeline focuses on Wilson’s Disease, which is a genetic disorder affecting copper metabolism. Wall Street believes that this program offers significant opportunities because of the large patient population it can potentially serve.
In October, Prime Medicine, Inc. (NASDAQ:PRME) presented the first in vivo preclinical data from its Wilson’s Disease program, demonstrating that Prime Editors can efficiently correct pathogenic mutations, without introducing safety concerns or detectable off-target edits. Notably, Prime Editors are advanced gene-editing tools developed by Prime Medicine, Inc. (NASDAQ:PRME).
These data are meaningful both for the company’s efforts in Wilson’s Disease (a devasting condition that can affect over 20,000 people in the US and Europe and for which there are currently no approved disease-modifying therapies) and in liver disease programs more broadly.
Prime Medicine, Inc. (NASDAQ:PRME)’s Wilson’s Disease program leverages its proprietary, universal LNP platform, and these interim results suggest that its LNP is expected to successfully deliver increased potency, and an improved safety profile and biodistribution as compared to other commonly used LNPs in development. Prime Medicine, Inc. (NASDAQ:PRME) is looking forward to advancing its Wilson’s Disease program toward an investigational new drug (IND) and/or clinical trial application (CTA) filing in H1 2026.
By focusing on Wilson’s Disease, Prime Medicine, Inc. (NASDAQ:PRME) targets to address a significant unmet medical need, potentially capturing a niche market with limited competition.
6) Beam Therapeutics Inc. (NASDAQ:BEAM)
Number of Hedge Fund Holders: 24
Beam Therapeutics Inc. (NASDAQ:BEAM) leverages advanced base editing technology to develop innovative therapies targeting genetic diseases. Its approach allows for precise and permanent modifications of DNA at the single-base level.
Beam Therapeutics Inc. (NASDAQ:BEAM)’s most advanced program, BEAM-101 for sickle cell disease (SCD), demonstrated promising early results. Rapid engraftment and high levels of fetal hemoglobin (HbF) in treated patients were observed, potentially outperforming other gene therapies in development. By reactivating the production of HbF, BEAM-101 focuses on alleviating the symptoms of SCD by reducing the presence of sickled red blood cells and improving oxygen delivery.
Current treatments, like hydroxyurea and chronic transfusions, tend to manage symptoms but do not address the root cause. This provides a significant opportunity for transformative therapies like BEAM-101. By permanently reactivating HbF, BEAM-101 can reduce the need for ongoing medical interventions and improve patients’ quality of life.
Beam Therapeutics Inc. (NASDAQ:BEAM)’s newly developed automated CD34+ cell process for BEAM-101 manufacturing is expected to revolutionize gene therapy. By increasing process capacity threefold, maintaining high consistency and reducing contamination risk simultaneously, Beam Therapeutics Inc. (NASDAQ:BEAM) can produce its therapies more efficiently and cost-effectively than competitors. Some of the competitive advantages include improved scalability, reduced production costs, and enhanced product consistency.
5) Verve Therapeutics, Inc. (NASDAQ:VERV)
Number of Hedge Fund Holders: 25
Verve Therapeutics, Inc. (NASDAQ:VERV) is a clinical-stage genetic medicines company, which is engaged in developing gene-editing medicines for patients to treat cardiovascular diseases in the US.
Verve Therapeutics, Inc. (NASDAQ:VERV) continues to actively develop next-generation PCSK9 therapies, focusing on offering innovative solutions for patients with high cholesterol. Its approach involves using gene editing technology to target PCSK9, a protein that is critical in regulating cholesterol levels in the blood.
The development of PCSK9 therapies is the focal point for Verve Therapeutics, Inc. (NASDAQ:VERV), with 2 distinct generations of treatments in the pipeline. The first-generation PCSK9 therapy saw setbacks, which led to a pause in enrollment because of concerns about ALT elevation and thrombocytopenia. These issues were believed to be related to the lipid nanoparticle (LNP) delivery system rather than the gene editor itself.
As a result, Verve Therapeutics, Inc. (NASDAQ:VERV) transitioned its focus to the next-generation PCSK9 therapy. This updated version incorporates improvements like GalNAc for targeted delivery and uses learnings from indirectly validated LNP technology. The next-gen therapy remains focused on enhancing liver tropism, potentially addressing the safety concerns observed in the first-generation treatment.
Verve Therapeutics, Inc. (NASDAQ:VERV)’s gene editing approach to PCSK9 inhibition can revolutionize treatment compliance in cardiovascular disease management. Traditional PCSK9 inhibitors tend to require regular injections, leading to poor adherence over time. Verve Therapeutics, Inc. (NASDAQ:VERV)’s one-time treatment is expected to eliminate the need for ongoing administration, which can improve patient compliance significantly.
4) CRISPR Therapeutics AG (NASDAQ:CRSP)
Number of Hedge Fund Holders: 27
CRISPR Therapeutics AG (NASDAQ:CRSP) is focused on utilizing the revolutionary CRISPR-Cas9 gene-editing technology to develop transformative therapies for genetic diseases.
CRISPR Therapeutics AG (NASDAQ:CRSP) received a significant boost with the approval of a CRISPR-based therapy, Casgevy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia in patients aged 12 years and older. CRISPR Therapeutics AG (NASDAQ:CRSP) partnered with Vertex Pharmaceuticals for the development and commercialization of Casgevy, with strong initial uptake of the therapy. Transfusion-dependent beta-thalassemia (TDT) is a severe form of beta-thalassemia, a hereditary blood disorder caused by mutations in the HBB gene, which encodes the beta-globin component of hemoglobin.
TDT and SCD have high unmet medical needs. On a global basis, millions of patients are impacted, with a significant subset eligible for curative gene-editing treatments. Many patients are dependent on regular blood transfusions and face complications, making a potentially one-time curative therapy like Casgevy highly attractive.
Wall Street analysts expect high per-patient costs for Casgevy, which should significantly boost revenue. Furthermore, as a result of a strategic alliance with Vertex Pharmaceuticals, CRISPR Therapeutics AG (NASDAQ:CRSP) can leverage Vertex’s established commercial infrastructure and expertise, ensuring efficient market entry and scalability.
CRISPR Therapeutics AG (NASDAQ:CRSP)’s pipeline extends beyond Casgevy, with numerous promising candidates in various stages of development. Its allogeneic CAR-T cell therapy programs, mainly CTX112 for B-cell malignancies, demonstrated encouraging results.
3) Intellia Therapeutics, Inc. (NASDAQ:NTLA)
Number of Hedge Fund Holders: 30
Intellia Therapeutics, Inc. (NASDAQ:NTLA) is a genome editing company, which continues to focus on the development of curative therapeutics.
Intellia Therapeutics, Inc. (NASDAQ:NTLA) continues to make strides in treating conditions like hereditary angioedema (HAE) and transthyretin amyloidosis (ATTR), positioning itself as a potential disruptor in the gene editing space. HAE is a rare, genetic disorder characterized by unpredictable and debilitating swelling episodes. The current treatments for HAE focus on frequent injections or infusions to prevent attacks, which can be burdensome for patients.
NTLA-2002 provides a one-time, potentially curative solution by silencing the KLKB1 gene, which reduces kallikrein protein levels responsible for triggering HAE attacks. Furthermore, NTLA-2002’s potential as a single-dose therapy is expected to replace chronic, lifelong treatments. This transformative approach should capture a significant share of the HAE treatment market by addressing the root cause of the disease, providing sustained efficacy, and improving patient quality of life.
Intellia Therapeutics, Inc. (NASDAQ:NTLA)’s CRISPR technology and delivery system provide a healthy foundation for clinical success and market adoption.
In October, Intellia Therapeutics, Inc. (NASDAQ:NTLA) presented positive Phase 2 data from the ongoing Phase 1/2 study, with results continuing to support the potential of NTLA-2002 to be a functional cure for HAE. As per Coherent Market Insights, HAE market size is expected to touch US$5.36 billion by 2031, demonstrating a strong unmet opportunity for Intellia Therapeutics, Inc. (NASDAQ:NTLA).
2) Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT)
Number of Hedge Fund Holders: 31
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases.
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT)’s lead program, RP-A501 for Danon disease, received significant attention from the medical community. Danon disease is a rare, X-linked cardiomyopathy caused by a deficiency of LAMP2 protein, that often leads to severe cardiac issues and early mortality. RP-A501 is being developed as a potential one-time gene therapy and the results of the long-term Phase 1 study demonstrate the promise of gene therapy across cardiac diseases, including PKP2-ACM, BAG3-DCM, and others.
Long-term follow-up data from the Phase 1 trial of RP-A501 demonstrated sustained improvement in LAMP2 expression and left ventricular mass index (LVMI), together with a favorable tolerability profile. The pivotal Phase 2 trial is fully enrolled and ongoing, with primary endpoints consisting of improved LAMP2B expression and reduced LVMI.
Wall Street analysts believe that medicine possesses a high 65-75% chance to succeed in a pivotal Phase II study next year and can be a $1 billion opportunity for Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT). Despite being a rare disease, the severity of Danon disease and the lack of effective treatments ensure strong demand.
Baron Funds, an investment management company, released its Q2 2024 investor letter. Here is what the fund said:
“Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) specializes in the development of gene therapies for rare genetic diseases outside of oncology. Currently these include Danon disease, Fanconi anemia, lysosomal acid lipase deficiency, and pyruvate kinase deficiency. The first three drug treatments are slated for commercial launch by 2025, which should generate substantial revenue. Shares detracted from performance after the FDA extended the priority review period by three months for the Kresladi gene therapy for leukocyte adhesion deficiency, potentially influenced by sluggish competitive gene therapy launches from bluebird bio in sickle cell disease and BioMarin in hemophilia B. Given the lifesaving nature of Rocket’s therapies and the high unmet need for each of these life ending diseases, we retain conviction in our investment.”
1) Sarepta Therapeutics, Inc. (NASDAQ:SRPT)
Number of Hedge Fund Holders: 50
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company, which focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. The company’s primary focus is on developing genetic-based treatments for rare diseases, particularly those caused by genetic mutations.
The cornerstone of Sarepta Therapeutics, Inc. (NASDAQ:SRPT)’s recent success has been ELEVIDYS, a gene therapy approved by the FDA in June 2023, and later expanded in 2024 to treat ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) patients aged 4 and above. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It mainly affects boys and is caused by mutations in the DMD gene, which encodes the protein dystrophin. Dystrophin is important for maintaining the structural integrity of muscle cells.
ELEVIDYS focuses on addressing a broader patient population using a truncated dystrophin gene delivered via an AAV vector. The one-time treatment demonstrates a meaningful advance. It provides improved functional outcomes in clinical trials. The therapy’s premium pricing, which stands at $3.2 million per patient, places it among the most expensive treatments in the U.S. Despite this, it comes out to be cost-effective over time as compared to recurring exon-skipping therapies, which cost up to $1.5 million on an annual basis.
As per Wall Street, ELEVIDYS approval demonstrates a pivotal shift in Sarepta Therapeutics, Inc. (NASDAQ:SRPT)’s business model, pivoting from a predominantly recurring revenue structure to a business model that relies more on high-value, one-time therapies. This strategic shift enables Sarepta Therapeutics, Inc. (NASDAQ:SRPT) to generate substantial cash flow to fuel innovation while maintaining a balanced pipeline.
While we acknowledge the potential of SRPT 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 SRPT but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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