Top NASDAQ Genetics Stocks

1. Enochian Biosciences

Year-to-date gain: 187.8 percent; current share price: US$8.49

The first genetics stock on this list is biopharmaceutical company Enochian Biosciences (NASDAQ:ENOB), which is developing gene-modified cellular and immune therapies. The company’s platforms can potentially be applied to multiple indications, including HIV/AIDS, hepatitis B and all corona and influenza viruses, as well as cancer.

Enochian’s pipeline includes its lead candidate, ENOB-HV-11, which is in preclinical development to treat HIV/AIDS as a vaccine. The company recently completed two US Food and Drug Administration (FDA) pre-investigational new drug requests, one for a potential cure for hepatitis B virus infections and the other for a potential cure for HIV.

2. aTyr Pharma

Year-to-date gain: 100.52 percent; current share price: US$7.78

aTyr Pharma (NASDAQ:LIFE) is developing medicines based on novel biological pathways. According to the biotherapeutics company, its "research and development efforts are focused on a newly discovered area of biology, the extracellular functionality and signaling pathways of tRNA synthetases." The protein compositions in aTyr’s pipeline are derived from 20 tRNA synthetase genes and their extracellular targets.

aTyr’s primary clinical-stage product candidate is ATYR1923, which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in inflammatory lung diseases. In mid-September, the company released positive data from a Phase 1b/2a clinical trial, demonstrating a consistent dose response for ATYR1923 in pulmonary sarcoidosis with no serious adverse effects.

3. Aadi Bioscience

Year-to-date gain: 63.4 percent; current share price: US$24.02

Commercial-stage biopharmaceutical company Aadi Bioscience (NASDAQ:AADI) develops precision therapies for genetically defined cancers with alterations in mTOR pathway genes.

In November, the company received FDA approval for its product candidate FYARRO for the treatment of adult patients with "locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumors."

“The approval of FYARRO, the first approved drug for advanced malignant PEComa, an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease,” said Andrew Wagner, a senior oncologist at the Dana-Farber Cancer Institute and the principal investigator in the registrational trial for FYARRO.

Going forward, Aadi Bioscience plans to initiate the next trial for FYARRO in early 2022.

4. Adial Pharmaceuticals

Year-to-date gain: 52.94 percent; current share price: US$2.60

Clinical-stage biopharma stock Adial Pharmaceuticals (NASDAQ:ADIL) is developing treatments for addiction. AD04, its lead investigational new drug product, is a genetically targeted serotonin-3 receptor antagonist for the treatment of alcohol use disorder. The therapeutic agent is currently being investigated under a pivotal ONWARD Phase 3 clinical trial in patients with certain target genotypes. These genotypes are identified using the company’s proprietary companion diagnostic genetic test. AD04 also has the potential to treat other addictive disorders, ranging from opioid use disorder to gambling and obesity.

The company has had plenty of positive news flow recently, including September’s announcement that more than 50 percent of patients expected to complete Adial’s ONWARD Phase 3 trial of AD04 for the treatment of alcohol use disorder had finished the full 24 week treatment period. The trial is slated to wrap up in Q1 2022.

5. PRoQR Therapeutics

Year-to-date gain: 56.90 percent; current share price: US$6.59

Last on this NASDAQ genetics stocks list is PRoQR Therapeutics (NASDAQ:PRQR), which develops transformative ribonucleic acid (RNA) therapies for the treatment of severe genetic eye diseases.

The company has a growing product pipeline based on its proprietary RNA repair platform technologies. The most advanced of these products is sepofarsen (QR-110), an RNA therapy that aims to restore vision in patients suffering from Leber congenital amaurosis 10 due to a genetic mutation.

PROQR Therapeutics expects to release top-line data from a pivotal Phase 2/3 trial of sepofarsen in late Q1 or early Q2 2022. Sepofarsen has been granted orphan drug designation, fast-track designation and rare pediatric disease designation by the FDA and has received orphan drug designation, as well as access to the PRIME scheme from the European Medicines Agency.