Analysts recommend these gene-editing stocks.
Gene therapy and gene editing are on the cutting edge of modern biotechnology. Gene therapies are used to correct genetic abnormalities by introducing genetic material at a cellular level, and they can often take the form of adding a functioning copy of a gene. Gene editing takes the science to the next level, seeking to directly alter the genome itself as a therapeutic approach. Most gene-editing stocks have taken a big hit in 2022, but analysts at ResearchAndMarkets.com still expect the genome-editing market to grow 53.1% annually and reach $59.5 billion by 2027. Here are seven of the best gene-editing stocks to buy, according to Bank of America.
Crispr Therapeutics AG (ticker: CRSP)
Crispr Therapeutics is a biotech company that uses gene-editing tool CRISPR-Cas9 to treat blood disorders and cancer. Crispr and partner Vertex Pharmaceuticals Inc. (VRTX) are optimistic that the company’s gene-editing treatment will obtain approval from the Food and Drug Administration for treating sickle cell disease and transfusion-dependent beta thalassemia in the near future. Analyst Geoff Meacham says there is a high likelihood of regulatory approval. Additional updates on other key products, including treatments targeting B-cell malignancies in the immune system, could be bullish catalysts for the stock in 2023. Bank of America has a “buy” rating and $116 price target for CRSP stock, which closed at $40.33 on Dec. 29.
Beam Therapeutics Inc. (BEAM)
Beam Therapeutics uses CRISPR technology to change single bases in the genome with a technique called base editing, which only breaks one strand of the DNA and may have higher cell survival rates. The company focuses on hemoglobinopathy and oncology indications but is also researching treatment for ocular and liver disease. Analyst Greg Harrison says Beam’s recent news that it is moving a treatment candidate called BEAM-102 into wave two of its sickle cell disease program is a positive step. Also significant is the company’s initiation of another treatment as a product candidate for a genetic disorder called alpha-1 antitrypsin deficiency. Harrison says the company’s unique platform can create significant value for investors over time. Bank of America has a “buy” rating and $74 price target for BEAM stock, which closed at $38.71 on Dec. 29.
Apellis Pharmaceuticals Inc. (APLS)
Apellis Pharmaceuticals is a biotech company focused on therapies targeting the complement pathway of the immune system, which enhances the immune system’s ability to clear damaged cells. The company’s main product is pegcetacoplan, which is already approved in the U.S. for treating paroxysmal nocturnal hemoglobinuria — a rare acquired blood disease — and is in late-stage development for treating geographic atrophy, a cause of vision loss in old age. In 2021, Apellis announced a new collaboration with Beam Therapeutics to leverage base editing technology to research new precision therapies for the eye, liver and brain. Analyst Tazeen Ahmad says Apellis is ahead of its competition in the race to bring the first geographic atrophy treatment to the market. Bank of America has a “buy” rating and $82 price target for APLS stock, which closed at $51.52 on Dec. 29.
Graphite Bio Inc. (GRPH)
Graphite Bio is a gene-editing technology company that leverages both CRISPR and natural DNA repair processes. Meacham says Graphite has enough cash to fund the initial data readout from its sickle cell disease program and an investigational new drug submission for a product that replaces the mutated beta-globin gene to treat beta-thalassemia. Graphite expects mid-phase, proof-of-concept data in mid-2023. Meacham says Graphite remains in early stage clinical development, so investing in the stock may require patience. Graphite’s advantage is that its gene-editing platform is differentiated from competitors and has the potential to generate significant long-term value. Bank of America has a “buy” rating and $10 price target for GRPH stock, which closed at $3.22 on Dec. 29.
Caribou Biosciences Inc. (CRBU)
Caribou Biosciences is a biotech company developing gene-edited cell therapies to treat cancer. In December, Caribou provided positive updates on two of its trial-phase treatments targeting non-Hodgkin lymphoma and solid tumors. Caribou said a third of the lymphoma patients maintained complete response at 12 months, and the company has enrolled three patients to test the therapy at a higher dose in 2023. The tumor treatment has solid preclinical data, says Meacham, but more data is needed to determine whether it could be a growth catalyst for the company. Meacham says the initial negative market reaction to the data was surprising given the updates didn’t change his bullish thesis. Bank of America has a “buy” rating and $27 price target for CRBU stock, which closed at $6.05 on Dec. 29.
Intellia Therapeutics Inc. (NTLA)
Intellia Therapeutics is a clinical-stage biotech company developing gene-editing products to treat a range of diseases. Its lead program for treating transthyretin amyloidosis, a protein disorder affecting the heart, is in mid-phase trials. Harrison says Intellia’s latest batch of data on a treatment for hereditary angioedema, which causes swelling in the body, demonstrates impressive efficacy, and the company plans to move to the Phase 2 dose expansion stage of that program in early 2023. Harrison says positive data from that program is also good news for Intellia’s lead program, given similarities between the two. Bank of America has a “buy” rating and $92 price target for NTLA stock, which closed at $35.28 on Dec. 29.
Vertex Pharmaceuticals Inc. (VRTX)
Vertex Pharmaceuticals develops small-molecule therapeutics for treating cystic fibrosis and inflammatory conditions. In 2021, Vertex acquired the rights to 60% of the profits from sales of Crispr Therapeutics’ gene-editing therapy CTX001, now called exa-cel, for up to $1.1 billion, pending regulatory approval. In September, Vertex and Crispr announced they are submitting exa-cel to the FDA and European regulators for review in treating sickle cell disease and transfusion-dependent beta-thalassemia. Meacham says Vertex has a differentiated growth profile, and its emerging orphan disease pipeline could generate additional upside for investors. Bank of America has a “buy” rating and $345 price target for VRTX stock, which closed at $289.08 on Dec. 29.
7 top gene-editing stocks to buy for 2023:
— Crispr Therapeutics AG (CRSP)
— Beam Therapeutics Inc. (BEAM)
— Apellis Pharmaceuticals Inc. (APLS)
— Graphite Bio Inc. (GRPH)
— Caribou Biosciences Inc. (CRBU)
— Intellia Therapeutics Inc. (NTLA)
— Vertex Pharmaceuticals Inc. (VRTX)
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Update 12/30/22: This story was previously published at an earlier date and has been updated with new information.