Top 5 NASDAQ Genetics Stocks (Updated January 2025)

1. Avidity Biosciences (NASDAQ:RNA)

Year-over-year gain: 149.51 percent
Market cap: US$3.33 billion
Share price: US$27.87

Avidity Biosciences is a biopharma firm developing a new form of RNA therapy called antibody oligonucleotide conjugates (AOC) that targeted the genes causing rare muscle diseases. Through its proprietary AOC platform, Avidity is conducting clinical development 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.

Avidity announced on February 20, 2024, that the US Food and Drug Administration (FDA) granted rare pediatric disease designation to its investigational therapy AOC 1044 for the treatment of Duchenne muscular dystrophy in people with certain mutations. Shares in the company rose more than 43 percent following the news to US$20.11 by March 1.

The FDA awarded breakthrough therapy designation to Avidity’s lead clinical development program, AOC 1001 for the treatment of myotonic dystrophy type 1, in early May.

Avidity’s stock price jumped by nearly US$10 to US$38.36 per share on June 12, the day Avidity shared positive initial data from the Phase 1/2 trial of AOC 1020, which “demonstrat(ed) unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with facioscapulohumeral muscular dystrophy.”

By August 9, shares in the company had risen by a further 22 percent to US$46.95 per share after it announced positive data from its Phase 1/2 trial for AOC 1044 in people living with Duchenne muscular dystrophy, including results showing a significant increase of 25 percent of normal in dystrophin production and a reduction of creatine kinase levels to near normal.

Shares in Avidity reached a yearly peak of US$52.50 on November 13, a day after the company introduced its first two precision cardiology development candidates targeting the root cause of genetic diseases of the heart.

3. UniQure (NASDAQ:QURE)

Year-over-year gain: 127.85 percent
Market cap: US$747.59 million
Share price: US$13.99

UniQure is a gene therapy company focused on patients with severe medical needs. In November 2022, the 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.

UniQure had its first big leap in its share value after the company announced a positive interim data update showing slowing of disease progression in its Phase 1/2 trials of AMT-130 for Huntington’s disease on July 9, 2024. The stock shot up more than 167 percent to US$10.12 per share.

Its next significant move to the upside came on December 10 when shares reached US$15.30 after uniQure notified shareholders it had reached an agreement with the FDA on an accelerated approval pathway for AMT-130.

“This is an important milestone for the Huntington’s disease community as it puts us on the most rapid and efficient pathway to deliver a potentially life-changing therapy to people living with this devastating neurodegenerative disorder,’ said Walid Abi-Saab, chief medical officer of uniQure. “We have initiated BLA readiness activities and look forward to further engaging with the FDA in the first half of 2025 to discuss our statistical analysis plan and the technical CMC requirements.”

Shares in uniQure hit a yearly high of US$18.05 on January 2, 2025.

4. Sangamo Therapeutics (NASDAQ:SGMO)

Year-over-year gain: 114.05 percent
Market cap: US$229.51 million
Share price: US$1.10

Sangamo Therapeutics is a genomic medicine company developing multiple platforms for developing gene therapies, such as gene editing and cell therapy, to address the unmet needs of patients afflicted with serious neurological diseases.

On July 24, 2024, the company reported on positive topline results from the Phase 3 AFFINE trial evaluating giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe hemophilia A. The company was co-developing the therapy with and licensing it to Pfizer (NYSE:PFE). Sangamo’s share value more than doubled from July 23 to reach US$0.92 per share on July 29.

On October 22, Sangamo announced that the FDA has given the company a clear regulatory pathway to accelerated approval for its wholly owned gene therapy product candidate isaralgagene civaparvovec (ST-920), for the treatment of Fabry disease. 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.

However, the company was hit by a surprise at the end of 2024, and announced on December 30 that Pfizer decided to terminate its global collaboration and license agreement with Sangamo for the hemophilia A treatment. The termination is effective April 21, 2025, at which time Pfizer will return full rights to the therapy to Sangamo.

‘We are committed to exploring the optimal path forward for this important treatment, including seeking the right partner with the focus and understanding of the genomic medicine commercial environment to bring this medicine to patients,’ Sangamo CEO Sandy Macrae stated in the release. The news gave the stock a more than 56 percent hair cut to US$1.01 per share.

5. Stoke Therapeutics (NASDAQ:STOK)

Year-over-year gain: 88.67 percent
Market cap: US$502.66 million
Share price: US$9.49

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 genetic epilepsy. The company is also developing STK-002 for the treatment of autosomal dominant optic atrophy, an inherited optic nerve disorder.

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. A few days later, the company’s share price had risen by 118 percent to reach US$14.17 per share.

Shares of Stoke Therapeutics hit a yearly peak of US$17.52 on June 13.

Other good news coming out of Stoke during 2024 included new positive data out of its Phase 1/2a and open-label extension studies for STK-001 on September 10, as well as the FDA granting STK-001 breakthrough therapy designation on December 4 for the treatment of Dravet syndrome ‘with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene.’