Regeneron and Intellia are also working together to evaluate a CRISPR gene-editing therapy targeting hemophilia. In addition, Intellia is collaborating with multiple partners in researching the use of gene-editing therapies in treating other genetic diseases, autoimmune diseases, and cancer. Several companies are using CRISPR to edit human genomes in an attempt to treat (and even cure) genetic diseases. Their therapies either use an ex vivo approach or an in vivo approach.
This important segment of the life science market is focused on how genes can help treat or prevent serious conditions in patients. This includes the potential for healthcare professionals to implement gene therapy at the cellular level instead of using medication or surgery, replacing “faulty” genes with new ones to potentially cure diseases. The industry is still in its early stages, and many companies are losing money.
Sangamo Therapeutics (NASDAQ:SGMO)
The two companies are now working together to evaluate nexiguran ziclumeran (formerly known as NTLA-2001) in a late-stage study targeting the rare genetic disease transthyretin amyloidosis (ATTR). Verve Therapeutics is a clinical-stage company developing a new class of genetic medicines for cardiovascular disease (CVD). The primary objective is to transition CVD treatment from chronic therapies to single-course, gene-editing medicines. Intellia Therapeutics is a clinical-stage gene editing company, focused on developing drugs with CRISPR-based therapies.
Certain Zacks Rank stocks for which no month-end price was available, pricing information was not collected, or for certain other reasons have been excluded from these return calculations. Zacks may license the Zacks Mutual Fund rating provided herein to third parties, including but not limited to the issuer. Another breakthrough is the development of genome editing approaches, such as CRISPR/Cas9 technology. Gene editing companies, such as BEAM Therapeutics (BEAM Quick QuoteBEAM – Free Report) and CRISPR Therapeutics AG (CRSP Quick QuoteCRSP – Free Report) , hold potential to treat and cure diseases caused by genetic variants. As the name suggests, they make changes or correct defects in the organism’s DNA.
- Each ChatGPT query, each model update, each robotic breakthrough consumes massive amounts of energy.
- The primary focus is to evaluate all the genes of an organism rather than individual genes.
- NTLA is leveraging its modular platform to advance in vivo and ex vivo therapies for diseases with high unmet need.
Top Genomics Stocks to Add to Your Portfolio in 2025
Invitae’s sales increased to $116 million in the second quarter of 2022, up 152 percent from the previous year. The company is expected to generate between $475 and $500 million in revenue in 2022. The genomics industry is growing rapidly, and investors are taking notice. In recent years, several high-profile IPOs in the space, including Illumina (ILMN) and 23andMe (DNA). The future is powered by artificial intelligence, and the time to invest is NOW.
Do gene-editing companies typically pay dividends?
The fields of gene editing and cell and gene therapies (CGTs) are also developing quickly. The UK approved Casgevy for sickle cell disease and β-thalassemia, pointing out the potential of CRISPR gene editing. According to Deloitte’s research report, the growing market for CGTs, from US$5.3 billion in 2022 to $19.9 billion in 2027, signals a shift towards customized advanced medicine despite high costs leading innovative business models.
Alternatively, other approaches, such as long-read sequencing, map longer pieces of DNA essential to understanding structurally complex regions where many genetic variants are located. Recent improvements in technology, chemistry, throughput, and costs are driving new and hybrid approaches, including integrated long-read and short-read sequencing. One of the genetic sector’s major contributions is the discovery of new genetic drivers of diseases.
Health insurance is a necessity today, which means it’s a great addition to your portfolio. The successful development of any of these candidates will be a significant boost for this Zacks Rank #2 company. The FDA recently granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease. In March 2025, QURE held a Type B meeting with the FDA to discuss chemistry, manufacturing and controls requirements for AMT-130, in support of a planned biologics license application (“BLA”) submission. An additional Type B meeting (focused on the pivotal statistical analysis plan) was also held. Explore 30 cutting-edge investment themes with Zacks Thematic Screens and uncover your next big opportunity.
On the eToro’s Digest & Invest podcast on October 21, 2024, Wood emphasized that concentrating only on the most prominent indexes may restrict exposure to ground-breaking innovation. She stated that reduced AI training costs have boosted genomics productivity, opening the door to important breakthroughs like gene editing that targets diseases. She is nevertheless optimistic about these stocks’ long-term worth, despite the present market reluctance and cash flow-driven tendencies made worse by ongoing high interest rates. According to Wood, avoiding this industry may result in missed opportunities as it progresses from development that relies heavily on investments to future profitability.
NASDAQ: NTLA
Sequencing volumes continue to increase in research and are growing rapidly in clinical applications, with the key drivers including whole-genome sequencing, cancer testing, and recurrence monitoring. Wave Life Sciences is another clinical-stage firm focused on unlocking insights from human genetics to deliver RNA-based medicines. The company’s PRISM platform is targeting both rare and prevalent disorders. Its pipeline includes clinical programs for Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency and Huntington’s disease, as well as a preclinical program in obesity.
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Based on its recent results, Exact Sciences looks to be on the road to profitability, no small feat in the genomics market. Year-over-year revenue has been increasing, while net losses have been declining. For the 12-month period ended June 30, 2025, it has pulled in $2.9 billion in revenue. VERVE-201 is being evaluated in the open-label phase Ib study designed to evaluate the safety and tolerability in adult patients with refractory hypercholesterolemia. The company is also conducting a phase I/IIa study (GenTLE) on AMT-260 for the treatment of MTLE genomics stocks in the United States. A phase I/IIa study on AMT-191 for the treatment of Fabry disease is also ongoing.
AMT-162 is being evaluated for the treatment of SOD1-ALS in a phase I/II study. Per estimates, the genomics market will reach $157.47 billion by 2033. Per Grandview research, the global synthetic biology market size was valued at $16.22 billion in 2024 and is projected to witness a CAGR of 17.30% from 2025 to 2030. In simpler terms, genomics includes the study of a complete set of genes, their work process and way of interacting with each other and the environment.
CRISPR Therapeutics
Caribou’s pipeline features two clinical-stage programs, both of which are experimental allogeneic (or “off-the-shelf”) chimeric antigen receptor T-cell (CAR-T) therapies being evaluated in phase 1 clinical trials. CB-010 targets the treatment of B-cell non-Hodgkin lymphoma, while CB-011 targets the treatment of multiple myeloma. On March 25, 2024, Stoke announced “landmark new data” supporting the potential for its STK-001 product candidate to become the first disease-modifying medicine for the treatment of patients with Dravet syndrome.
- According to expert Jason Gerberry, a 75% chance of the suspension being lifted by mid-December is a significant bullish stock trigger.
- Below, we’ll take a closer look at some of the most promising categories of genomics companies.
- From the resultant dataset, we chose 11 stocks with the highest number of hedge fund investors, using Insider Monkey’s database of 1000 hedge funds in Q to gauge hedge fund sentiment for stocks.
- CRISPR Therapeutics is evaluating two in vivo gene-editing therapies (where gene editing is performed inside the body) in early stage clinical studies.
Pharma and biotech companies often dabble in genetics along with their core disciplines, meaning that some firms may also have operations in other areas. The top NASDAQ genetics stocks listed below have products related to gene therapy, genetic testing, genetically defined cancers and rare genetic diseases. In 2021, Intellia and its partner, Regeneron Pharmaceuticals (REGN -1.48%), announced the first clinical data supporting the use of in vivo (in the body) CRISPR-Cas9 gene editing in humans.
Which company is leading CRISPR?
It’s been more than 20 years since the completion of the Human Genome Project. However, we are in the early innings of a genomic revolution that could create many companies with multibillion-dollar market capitalizations. This won’t happen overnight, but for patient buy-and-hold investors, genetic sequencing company stocks could be a healthy addition to your portfolio. Many rapidly growing companies are emerging in the genomics field as technological advances substantially reduce the cost, accuracy, and time required to map a human being’s genome. Sangamo said it expects a potential biologics license application submission in the second half of 2025. Shares in the genetic stock rose more than 69 percent in one day to US$1.54, and continued climbing over the following weeks to its highest yearly peak of US$2.87 on November 9.