Food and Drug Administration (FDA) approval the following month for Casgevy in treating transfusion-dependent beta-thalassemia. The company’s mission is “developing transformative gene-based medicines for serious human diseases.” In addition, Regeneron is evaluating a hemophilia B therapy licensed from Intellia in phase 2 clinical testing. Intellia will be eligible for milestone payments and royalties if the experimental therapy advances through clinical development and ultimately wins regulatory approval. Genomics stocks can be boom or bust depending on the results of their clinical trials. Ionis investors got some great news in early September 2025, when the company announced positive top-line results from the phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG).

Conclusion: Genomics Stocks

Poseida Therapeutics is a clinical-stage biotech company that employs cutting-edge gene-editing systems to develop cellular and gene therapies. PSTX stock, which closed at $7.60 on Nov. 22, has received a “buy” rating and a $20 price target from Bank of America analyst Wamsi Mohan. The recent spotlight on companies in the evolving genomics sector can be attributed to a drastic reduction in the cost, accuracy, and time required to map an individual’s entire genome. A major player in this field is Illumina (ILMN Quick QuoteILMN – Free Report) , a global leader in sequencing and array-based solutions for genetic and genomic analysis. Illumina gained its dominant market position by championing short-read sequencing technology, which breaks DNA into short segments to aid in analysis for genetic research.

Investing in gene-editing stocks

• However, the US Food and Drug Administration has placed the firm on hold.
• Data for this list of genetics stocks on the NASDAQ was collected on January 15, 2024, using TradingView’s stock screener, and stocks with market caps above US$50 million were considered.
• This entails companies dedicated to finding cures for diseases caused by genetic variations.

This stock is so off-the-radar, so absurdly undervalued, that some of the most secretive hedge fund managers in the world have begun pitching it at closed-door investment summits. While the world is distracted by flashy AI tickers, a few smart investors are quietly scooping up shares of the one company powering it all from behind the scenes. Ionis Pharmaceuticals develops RNA-targeted medicine to treat serious diseases. Along with Biogen (BIIB -0.54%), it developed Qalsody, the first medicine for the genetic cause of amyotrophic lateral sclerosis (ALS).

The company estimates its serviceable addressable market today stands at around $13 billion, with $6 billion of the total in single-cell and $7 billion in spatial. 10x has captured only a sliver of that market, though, with 2024 revenue of $610.8 million. Stoke Therapeutics is another biotech company with a focus on developing RNA medicine. With its proprietary research platform TANGO, which stands for targeted augmentation of nuclear gene output, the company is developing antisense oligonucleotides to selectively restore protein levels. However, the genomics stocks company was hit by a surprise at the end of 2024, and announced on December 30 that Pfizer decided to terminate its global collaboration and license agreement with Sangamo for the hemophilia A treatment.

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In 2025, it launched Oncodetect, a molecular residual disease test, and Cancerguard, a first-of-its-kind multicancer early detection blood test. It’s a diagnostics company with about 8,000 patient access points, the most extensive network in the U.S., and 2,000 Quest patient service centers. CRISPR Therapeutics isn’t the only CRISPR-focused biotech company with a big partner. Intellia Therapeutics has teamed up with Regeneron Pharmaceuticals (REGN -1.48%). Although all CRISPR stocks are risky, CRISPR Therapeutics is arguably less risky than most.

Breeding cycles are being accelerated, especially for perennial crops, by methods like transgenics, genome editing, and marker-assisted breeding. The global effect is nonetheless constrained by regulatory complexity, particularly by uneven policies between regions such as the EU. Remember that progress is frequently sporadic with genomics companies. A buy-and-hold strategy is a must to give your holdings time to pay off. Cologuard has been a big revenue driver for this company, but it has been expanding its product lineup.

How to buy gene-editing stocks

Pacific Biosciences of California (also known as PacBio) is pioneering long-read sequencing technology, which uses longer DNA segments to map the whole genome. Long-read sequencing can uncover parts of the genome missed by short-read sequencing. Although the Human Genome Project was declared “complete” in 2003, it missed about 8% of the genome.

• But the real story isn’t Nvidia — it’s a much smaller company quietly improving the critical technology that makes this entire revolution possible.
• Using long sections of DNA, the PacBio platform allows researchers to sequence an entire genome with high fidelity (HiFi).
• The UK approved Casgevy for sickle cell disease and β-thalassemia, pointing out the potential of CRISPR gene editing.
• Their therapies either use an ex vivo approach or an in vivo approach.

He believes today’s trends are tomorrow’s cultural history—and loves helping readers make sense of it all. PacBio moved to acquire Omniome Inc. to further its Sequencing by Blinding (SBB) chemistry. PacBio also reported a 33% increase in revenue year over year, amounting to $36 million in 2022. The commercial launch of Casgevy continues to gain momentum, with Vertex leading the charge.

In July, Invitae and PacBio agreed that PacBio’s HiFi sequencing and SBB chemistry would be used in whole-genome testing. For example, you can invest in companies that are doing cutting-edge research in the field, in companies that are developing new genomics applications, or in the infrastructure necessary to support the field’s growth. In March 2025, the company announced positive initial results from a phase 1/2 study evaluating BEAM-302 in treating alpha-1 antitrypsin deficiency (AATD). AATD is a genetic disorder that affects the lungs and/or liver and can lead to emphysema and liver disease. Gene-editing stocks are shares of biotechnology companies that focus on modifying DNA.

NASDAQ: BEAM

The 32nd Plant and Animal Genomics Conference, held in San Diego from January 10-15, 2025, brought attention to how quickly agricultural genomics technology is developing. What took the original Human Genome Project 13 years and $3 billion can now be performed for less than $200 in a matter of days, with new platforms promising $100 per human genome. VERV’s lead clinical-stage programs target the PCSK9 and ANGPTL3 genes, which have been extensively validated as targets for lowering LDL cholesterol, or LDL-C. VERVE-102 targets PCSK9 and is designed to permanently turn off the PCSK9 gene in the liver. The candidate is being evaluated in the open-label phase Ib Heart-2 study. The study is designed to evaluate the safety and tolerability of VERVE-102 in adult patients with heterozygous familial hypercholesterolemia, or HeFH, and/or premature coronary artery disease.

The stocks are ranked in ascending order of the upside potential, as of November 14. Melissa Pistilli has been reporting on the markets and educating investors since 2006. She has covered a wide variety of industries in the investment space including mining, cannabis, tech and pharmaceuticals.

It also returns some extra cash to investors in the form of a dividend. Interestingly, genomics has ushered in innovative concepts, such as synthetic biology, which applies engineering principles to biology. Synthetic biology products serve life sciences researchers across a variety of healthcare applications, including drug discovery, disease detection, enzyme engineering, gene editing and basic academic research. The rapid evolution of genomics, the study of genes and their functions, in recent times has paved the way for a revolutionary era in genetic medicine. Given the constant need for innovative medical treatments, genomics holds the key to the future of the medical world. It’s evaluating two experimental CAR T-cell therapies in clinical trials.