Discoveries made by companies in the genetics sector help support every other life science industry in a variety of ways.
One of the genetic sector’s major contributions is the discovery of new genetic drivers of diseases. Genetic testing has grown substantially over the last few years, thanks to advances in technology; growth has also been spurred by an increase in chronic diseases and the continuing development of test kits for therapeutic areas with unmet medical needs.
Gene therapy is also a huge driver of growth in the overarching genetics market. 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.
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.
Data for this list of genetics stocks on the NASDAQ was collected on December 31, 2025, using TradingView’s stock screener, and stocks with market caps above US$50 million were considered.
1. Avidity Biosciences (NASDAQ:RNA)
Year-over-year gain: 143.8 percent
Market cap: US$10.87 billion
Share price: US$72.14
Avidity Bioscience is a biopharma firm developing a new form of RNA therapy called antibody oligonucleotide conjugates (AOC) that target the genes causing rare muscle diseases.
Through its proprietary AOC platform, Avidity developed programs for three rare muscle diseases: AOC 1001 for myotonic dystrophy type 1, AOC 1044 for Duchenne muscular dystrophy and AOC 1020 for facioscapulohumeral muscular dystrophy. The company is also working to expand its pipeline into cardiology and immunology.
In October 2025, Avidity entered into a definitive agreement to be acquired by Novartis (NYSE:NVS), which will include the company’s late-stage neuromuscular programs (AOC 1001, 1020, 1044) and the AOC platform, for US$12 billion.
Avidity’s early-stage precision cardiology programs will spin off into a new public company prior to closing in H1 2026. The spin-off will also have rights to use and develop the AOC platform for cardiology applications.
2. Wave Life Sciences (NASDAQ:WVE)
Year-over-year gain: 36.52 percent
Market cap: US$3.13 billion
Share price: US$17.12
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 for WVE-007 in obesity.
Wave Life Sciences advanced its PRISM RNA platform across multiple programs in 2025. It is also performing a Phase 1 trial testing its WVE-007 obesity candidate, which is an investigational INHBE GalNAc-siRNA using Wave’s proprietary SpiNA design.
In December, the company reported positive interim data from the WVE-007 trial, which showed that a single dose resulted in sustained Activin E reduction, supporting infrequent dosing. Target engagement updates and body composition readouts are planned for Q1 2026.
3. UniQure (NASDAQ:QURE)
Year-over-year gain: 33.15 percent
Market cap: US$1.47 billion
Share price: US$23.86
UniQure is a gene therapy company focused on patients with severe medical needs. In November 2022, the US Food and Drug Administration (FDA) approved the company’s gene therapy Hemgenix (etranacogene dezaparvovec), which is the world’s first gene therapy for hemophilia B.
Today, uniQure’s proprietary gene therapy pipeline includes treatments for patients with Huntington’s disease, refractory temporal lobe epilepsy, ALS and Fabry disease.
Its gene therapy pipeline advanced in 2025, with positive Phase I/II topline data for Huntington’s disease candidate AMT-130 showing 75 percent slowing of disease progression at three years via cUHDRS, alongside 60 percent functional capacity preservation.
While data from the Phase I/II study led the FDA to grant AMT-130 breakthrough therapy designation in April, in December the agency told UniQure it believes the data may not be adequate to support a pre-biologics license application under the accelerated approval pathway. The company is pursuing a follow-up meeting.
4. Stoke Therapeutics (NASDAQ:STOK)
Year-over-year gain: 186.96 percent
Market cap: US$1.81 billion
Share price: US$31.74
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.
Stoke’s first product candidate, zorevunersen (STK-001), is in clinical testing for the treatment of Dravet syndrome, a severe form of genetic epilepsy. The company is also developing STK-002 for the treatment of autosomal dominant optic atrophy, an inherited optic nerve disorder.
Both candidates advanced in 2025, with STK-001 enrolling patients in Phase 3 after positive long-term data showed seizure reductions and cognitive gains. Likewise, STK-002’s clinical development program is being informed by results, presented in October, of a Phase 1 two year natural history study on the disease progression of autosomal dominant optic atrophy.
Securities Disclosure: I, Meagen Seatter, hold no direct investment interest in any company mentioned in this article.