Top 4 NASDAQ Genetics Stocks (Updated January 2026)

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). This therapy targets the genes causing rare muscle diseases.

Through its proprietary AOC platform, Avidity has 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, Avidity entered into a definitive agreement to be acquired by Novartis (NYSE:NVS) for US$12 billion. The deal includes the company's late-stage neuromuscular programs (AOC 1001, 1020 and 1044) and the AOC platform.

Avidity's early stage precision cardiology programs will spin off into a new public company prior to closing in H1 2026. The spinoff 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 advanced its PRISM RNA platform across multiple programs in 2025. It is also performing a Phase 1 trial test of its WVE-007 obesity candidate, 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 the first quarter of 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.

The company's 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 the FDA granted 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 biotech firm is also developing STK-002 for the treatment of autosomal dominant optic atrophy, an inherited optic nerve disorder.

Both candidates from the company 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.