5 Most Promising Gene Therapy Companies to Watch

In this piece, we will take a look at the five most promising gene therapy companies to watch. For more companies, take a look at 10 Most Promising Gene Therapy Companies to Watch.

5. Krystal Biotech, Inc. (NASDAQ:KRYS)

Market Capitalization as of November 12, 2022: $1.97 billion

Krystal Biotech, Inc. (NASDAQ:KRYS) develops treatments for rare diseases such as fibrosis, Netherton syndrome, and skin conditions. The firm is headquartered in Pittsburgh, Pennsylvania.

Krystal Biotech, Inc. (NASDAQ:KRYS)’s latest quarter saw the firm beat analyst estimates, as it posted -$0.17 in earnings per share for its third fiscal quarter. The firm is one of three companies that is developing a treatment for dystrophic epidermolysis bullosa (DEB), and the FDA has accepted the treatment’s license application alongside outlining that it will decide on the details by February next year. Krystal Biotech, Inc. (NASDAQ:KRYS)’s treatment is capable of healing full DEB wounds in six months.

Chardan raised the company’s share price target to $133 from $130 in November 2022, as it stated that several of the firm’s treatments are ready for patient dosing. The firm has a $1.97 billion market capitalization.

Krystal Biotech, Inc. (NASDAQ:KRYS)’s largest investor is Jeremy Green’s Redmile Group which owns 1.8 million shares that are worth $124 million.

4. Prime Medicine, Inc. (NASDAQ:PRME)

Market Capitalization as of November 12, 2022: $2.02 billion

Prime Medicine, Inc. (NASDAQ:PRME) is a biotechnology company that uses gene editing. The firm’s technologies enable treatments to precisely target genomes by blending proteins. It is headquartered in Cambridge, Massachusetts.

Prime Medicine, Inc. (NASDAQ:PRME) is currently valued at $2.02 billion by the stock market. Its technology was formulated at the Harvard University and the Massachusetts Institute of Technology, and the main programs that are currently under development can bring the firm as much as $13 billion in revenue. The firm also just completed its initial public offering (IPO) of 10 million shares in late October, as it sold the shares at $17 each. The current share price has appreciated since then and stands at $21 per share.

3. Beam Therapeutics Inc. (NASDAQ:BEAM)

Market Capitalization as of November 12, 2022: $3.58 billion

Beam Therapeutics Inc. (NASDAQ:BEAM) develops genetic treatments for diseases such as leukemia, sickle cell disease, eye diseases, and central nervous system disorders. It is also based in Cambridge, Massachusetts.

Beam Therapeutics Inc. (NASDAQ:BEAM) announced in November 2022 that it aims to focus on its BEAM-101 editing drug for sickle cell disease instead of filing the BEAM-102 drug. The firm aims to enroll the first patient for BEAM-101 trials later this year, and its third quarter results revealed that revenue nearly doubled annually from $8.6 million to $15.6 million, but at the same time research and development expenditures jumped by 56% annually. The company ended the quarter with $1.1 billion of cash in hand.

Beam Therapeutics Inc. (NASDAQ:BEAM)’s largest investor is Catherine D. Wood’s ARK Investment Management which owns 8.7 million shares that are worth $309 million.

2. Intellia Therapeutics, Inc. (NASDAQ:NTLA)

Market Capitalization as of November 12, 2022: $4.32 billion

Intellia Therapeutics, Inc. (NASDAQ:NTLA) develops treatments for liver diseases, leukemia, cancers, and autoimmune disorders. The firm was set up in 2014 and is headquartered in Cambridge, Massachusetts.

Intellia Therapeutics, Inc. (NASDAQ:NTLA) is aiming to become an all out player in the gene editing sector, and the firm took a bold move earlier this year when it acquired another firm for a whopping $200 million. While acquisitions are common play in the business world, it is highly uncommon for a research focused firm with limited finances to make such a move. The deal will let Intellia Therapeutics, Inc. (NASDAQ:NTLA) expand its treatments beyond diseases of the eyes and liver, and diversify its gene editing portfolio to other areas as well.

Intellia Therapeutics, Inc. (NASDAQ:NTLA) has a $4.32 billion market capitalization.

Catherine D. Wood’s ARK Investment Management is Intellia Therapeutics, Inc. (NASDAQ:NTLA)’s largest investor. It owns 9.5 million shares that are worth $439 million.

1. CRISPR Therapeutics AG (NASDAQ:CRSP)

Market Capitalization as of November 12, 2022: $4.72 billion

CRISPR Therapeutics AG (NASDAQ:CRSP) is a Swiss company that is perhaps the most renowned gene therapy company in the world. The firm’s CRISPR gene editing platform is synonymous with the industry, and this allows researchers to make direct changes to the gene sequence and DNA. CRISPR Therapeutics AG (NASDAQ:CRSP) is headquartered in Zug, Switzerland.

CRISPR Therapeutics AG (NASDAQ:CRSP) is looking to complete the regulatory filings of its CTX001 treatment for sickle cell disease and thalassemia. The firm’s thalassemia treatment aims to have a price of $5 million and the price of its sickle cell treatment can go even higher. Additionally, CRISPR Therapeutics AG (NASDAQ:CRSP)’s thalassemia treatment alone is estimated to be worth a whopping $11 billion, making the math and future potential clear when CRISPR Therapeutics AG (NASDAQ:CRSP)’s market value of $4.72 billion is considered.

CRISPR Therapeutics AG (NASDAQ:CRSP)’s largest investor is Catherine D. Wood’s ARK Investment Management which owns 974 million shares that are worth $564 million.

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