An updated edition of the May 21, 2025, article.

The promising field of genomics has undergone rapid evolution over the past decade. 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.

This genetic information is being used to evaluate individual responses to drugs, leading to the development of targeted therapies. This, in turn, has paved the way for a revolutionary era in genetic medicine. The innovative breakthroughs in this field have grabbed the attention of pharma and biotech companies. Given the constant need for innovative medical treatments, genomics holds the key to the future of the medical world.

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 Intellia Therapeutics (NTLA Quick QuoteNTLA - Free Report) and CRISPR Therapeutics AG (CRSP), hold the 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 may consider adding stocks like MeiraGTx Holdings plc (MGTX Quick QuoteMGTX - Free Report) , Beam Therapeutics (BEAM Quick QuoteBEAM - Free Report) , Krystal Biotech (KRYS Quick QuoteKRYS - Free Report) to their portfolios.

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3 Genomics Stocks in Spotlight

MeiraGTx Holdings plc is a clinical-stage genetic medicine company with a broad pipeline of late-stage clinical programs, including Parkinson's disease, radiation-induced xerostomia and AIPL1-associated retinal dystrophy. These programs use targeted local delivery of small doses of genetic medicines to treat both inherited and more common conditions with severe unmet need.

Efficacy data of rAAV8.hRKp.AIPL1 for the treatment of AIPL1-related retinal dystrophy, or LCA4, demonstrated meaningful responses in 11 out of 11 LCA4 children treated. The company plans to file for Marketing Authorization Approval under exceptional circumstances with the U.K. Medicines and Healthcare products Regulatory Agency and is also in discussions with the FDA regarding a potentially similar pathway to approval in the United States.

The FDA recently granted the Regenerative Medicine Advanced Therapy (“RMAT”) designation to AAV-GAD for the treatment of Parkinson’s disease not adequately controlled with anti-Parkinsonian medications. MGTX plans to initiate a late-stage study of AAV-GAD in the second half of 2025. Earlier, MeiraGTx was granted RMAT designation by the FDA for AAV2-hAQP1 for the treatment of Grade 2/3 Grade 2/3 radiation-induced xerostomia.

The successful development of any of the therapies will be a significant boost for this Zack Rank #3 (Hold) company. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Beam Therapeutics is advancing a broad, diversified portfolio of base editing programs against distinct, genetically validated editing targets. The company’s quiver of gene editing technologies is anchored by its proprietary base editing technology, which potentially enables a differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA.

The company is currently developing BEAM-101 for sickle cell disease (SCD). The FDA recently granted orphan drug designation to BEAM-101 for SCD. BEAM is also looking to advance BEAM-103 and BEAM-104 for development in SCD and beta-thalassemia, potentially building on the same regulatory, manufacturing, clinical and commercial foundations being established with BEAM-101.

BEAM currently carries a Zack Rank #3.

Krystal Biotech received a significant boost with the FDA approval of Vyjuvek, the first-ever revocable gene therapy for treating patients, aged six months or older, with dystrophic epidermolysis bullosa. Vyjuvek was recently approved in the EU as well.

The initial uptake of this therapy is encouraging, and approval in additional geographies should boost sales.

Krystal is also advancing a robust pipeline of investigational genetic medicines in the fields of respiratory, oncology, dermatology, ophthalmology and aesthetics. This Zacks Rank #3 company has a strong balance sheet with cash and investments of $765.3 million (at the end of the first quarter), which should help fund its pipeline development. The successful development of any of these pipeline candidates will diversify the company’s revenue stream.