An updated edition of the March 28, 2025, article.
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.
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. The primary focus is to evaluate all the genes of an organism rather than individual genes.
Of late, there have been several breakthroughs in this field, attracting the attention of pharma and biotech companies. Genetic information is being used to evaluate individual responses to drugs, leading to the development of targeted therapies.
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 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, a global leader in sequencing and array-based solutions for genetic and genomic analysis. While some companies are utilizing genetic sequencing to develop solutions not only for healthcare but also for other areas, diagnostic companies leverage sequencing data to identify specific genetic variations and map them to a known condition.
Another breakthrough is the development of genome editing approaches, such as CRISPR/Cas9 technology. Gene editing companies, such as BEAM Therapeutics BEAM and CRISPR Therapeutics AG CRSP, 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.
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.
If you're looking to capitalize on this trend, our Genomics and Synthetic Biology screen makes it easy to identify high-potential stocks at any given time. At present, investors can consider adding stocks like Intellia Therapeutics NTLA, uniQure QURE and Verve Therapeutics VERV to their portfolio.
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3 Genomics Stock Picks to Bet On
Intellia Therapeutics is a clinical-stage gene editing company, focused on developing drugs with CRISPR-based therapies. NTLA is leveraging its modular platform to advance in vivo and ex vivo therapies for diseases with high unmet need. The in vivo programs use CRISPR to enable precise editing of disease-causing genes directly inside the human body.
NTLA’s lead in vivo product candidates are nex-z for the treatment of ATTR amyloidosis and NTLA-2002 for the treatment of HAE. NTLA-2002 is a wholly owned, investigational in vivo CRISPR-based therapy designed to knock out the kallikrein B1 gene in the liver, with the goal of achieving lifelong control of HAE attacks after a single dose. Nex-z is an investigational CRISPR-based therapy designed to inactivate the TTR gene in liver cells, thereby preventing the production of TTR protein for the treatment of ATTR amyloidosis. Both are currently in phase III studies. NTLA has collaborated with Regeneron for Nex-z.
This Zacks Rank #2 (Buy) company is also advancing ex vivo applications to address immuno-oncology and autoimmune diseases, where CRISPR is used as the tool to create the engineered cell therapy outside the body. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
uniQure is advancing a focused pipeline of innovative gene therapies for the treatment of Huntington’s disease, amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (“SOD1-ALS”), refractory mesial temporal lobe epilepsy (“mTLE”) and Fabry disease. The company has made good pipeline progress.
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.
The company is also conducting a phase I/IIa study (GenTLE) on AMT-260 for the treatment of MTLE 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.
The successful development of any of these candidates will be a significant boost for this Zacks Rank #2 company.
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. VERV is developing a pipeline of gene editing programs targeting the three lipoprotein pathways — low-density lipoprotein, or LDL, triglyceride-rich lipoproteins, and lipoprotein(a), or Lp(a) — that drive atherosclerotic cardiovascular disease (ASCVD), the most common form of CVD.
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. VERVE-201 is designed to permanently turn off the ANGPTL3 gene in the liver.
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. VERVE-301 is being developed in collaboration with Lilly. The company also carries a Zacks Rank #2.
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