10 Most Promising Gene Therapy Companies to Watch

Published on November 14, 2022 at 2:31 pm by Ramish Cheema in Lists, News

In this piece, we will take a look at the ten most promising gene therapy companies to watch. For more companies, take a look at 5 Most Promising Gene Therapy Companies to Watch.

Gene therapy is a revolutionary new technology that has followed the growing advances in computing power. The human body is estimated to be made up of 20,000 genes, and these are the basic programming blocks of its structure. They determine the characteristics that make humans unique and are responsible for a variety of traits such as hair and eye color, height, skin color, and others.

The gene therapy sector also has some of the strongest growth estimates that you are likely to come across. For instance, a report from the research firm Grand View Research believes that this industry will grow at a compounded annual growth rate (CAGR) of 20.4% by 2028 for an estimated value of $10 billion. The research firm attributes this growth to the increase of viral vectors in developing treatments and the growing popularity of gene therapy for treating cancers such as melanoma and B-cell lymphoma. Other diseases that are currently on the radar of gene therapy include muscular atrophy and arterial disease.

Transparency Market Research has an equally strong CAGR of 19.8% for the industry, as it believes that the sector was worth $1.3 billion in 2018 and it will grow to $5.3 billion by 2028. T-cell therapy for lymphoma is a popular choice of treatment for B-cell lymphoma among gene therapy firms, and they are knocking on the Federal Drug Administration’s (FDA) doors to grant them the approval to clear their products for the market. These companies are currently focusing their efforts on patients with high income as the costs of research and development the novel technologies is high. Additionally, Europe and the United States are the largest spenders in the gene therapy market.

Finally, Fortune Business Insights is the most optimistic research firm for gene therapy. It has a whopping 33.6% CAGR for the industry, as the firm estimates that from a value of $3.61 billion in 2019, the industry will grow to $35.6 billion by 2027. There are hundreds of different firms that are operating in the market, and as of 2018, 259 different drug candidates were under Phase 1 clinical trials. These trials test the dosage and the method of delivery of treatment, alongside other details. Treatments for neurological disorders make up the largest chunk of the industry, and the U.S. will dominate the industry in the years to come.

Out of the hundreds of different gene therapy firms, we have compiled a list of the largest for you today, and the top players are Intellia Therapeutics, Inc. (NASDAQ:NTLA), CRISPR Therapeutics AG (NASDAQ:CRSP), and Beam Therapeutics Inc. (NASDAQ:BEAM).

Pixabay/Public Domain

Our Methodology

We studied the gene therapy industry and picked the biggest players working on the most promising gene therapies and treatments. These companies are ranked according to their market cap.

Most Promising Gene Therapy Companies to Watch

10. Akouos, Inc. (NASDAQ:AKUS)

Market Capitalization as of November 12, 2022: $500 million

Akouos, Inc. (NASDAQ:AKUS) is an American company that is headquartered in Boston, Massachusetts. It focuses on developing treatments for hearing loss and hearing disorders.

Akouos, Inc. (NASDAQ:AKUS)’s market capitalization sits at $500 million. The firm is in the process of being acquired by the pharmaceutical giant Eli Lilly, with the deal that was announced in October 2022 seeing Eli Lilly pay a total of $610 million for Akouos, Inc. (NASDAQ:AKUS). The company also received the Food and Drug Administration’s (FDA) approval to start testing its AK-OTOF treatment on children.

Akouos, Inc. (NASDAQ:AKUS)’s largest investor in our database of 895 hedge funds is Oleg Nodelman’s EcoR1 Capital which owns 1.9 million shares that are worth $9.2 million.

CRISPR Therapeutics AG (NASDAQ:CRSP), Intellia Therapeutics, Inc. (NASDAQ:NTLA), and Beam Therapeutics Inc. (NASDAQ:BEAM) are met by Akouos, Inc. (NASDAQ:AKUS) in our list of top gene therapy stocks.

9. bluebird bio, Inc. (NASDAQ:BLUE)

Market Capitalization as of November 12, 2022: $620 million

bluebird bio, Inc. (NASDAQ:BLUE) is a biotechnology company that develops gene therapies for diseases and disorders such as sickle cell disease, thalassemia, and SCD. The firm was set up in 1992 and it is headquartered in Cambridge, Massachusetts, the United States.

bluebird bio, Inc. (NASDAQ:BLUE)’s fiscal third quarter results saw the firm beat analyst earnings per share (EPS) estimates by 24 cents as it posted -$0.94 in EPS during the quarter. The firm also partnered up with another company in October 2022 to reduce the cost of its thalassemia drug Zynteglo to ensure that those footing the high cost of the drug can receive a rebate if results are not delivered in 2 years. Zynteglo is the most expensive treatment in the market as it comes with a whopping price tag of $2.8 million. It is the only FDA approved drug for beta-thalassemia, and the agreement will reach roughly 16 million individuals across the United States.

bluebird bio, Inc. (NASDAQ:BLUE)’s market capitalization sits at $620 million.

8. Editas Medicine, Inc. (NASDAQ:EDIT)

Market Capitalization as of November 12, 2022: $900 million

Editas Medicine, Inc. (NASDAQ:EDIT) is a biotechnology company that uses the CRISPR gene editing platform to develop treatments for several diseases. These include Usher syndrome, retinal diseases, sickle cell disease, solid tumors, cancers, and a neurological disease. The firm is headquartered in Cambridge, Massachusetts, the United States.

Editas Medicine, Inc. (NASDAQ:EDIT) develops the EDIT-301 treatment for sickle cell anemia and beta thalassemia. The firm uses a proprietary technology in this treatment and it expects to release the results from the phase 1/2 trial by the end of this year. Editas Medicine, Inc. (NASDAQ:EDIT)’s latest earnings, for its third quarter, saw the firm post -$0.81 in GAAP EPS, which ended up beating analyst estimates. The company had $479 million in cash by the end of the third quarter, while its research and development expenses stood at $41.3 million, indicating that it has sufficient reserves for research for several quarters.

Editas Medicine, Inc. (NASDAQ:EDIT)’s market capitalization is $900 million and Chardan reduced its share price target to $43 from $60 in November 2022 but remained optimistic for the upcoming EDIT-301 data release.

Editas Medicine, Inc. (NASDAQ:EDIT)’s largest investor is Ken Griffin’s Citadel Investment Group. It owns 1.2 million shares that are worth $14 million.

7. uniQure N.V. (NASDAQ:QURE)

Market Capitalization as of November 12, 2022: $1.03 billion

uniQure N.V. (NASDAQ:QURE) develops treatments for hemophilia, Huntington’s disease, Parkinson’s disease, temporal lobe epilepsy, Alzheimer’s disease, and lateral sclerosis. The firm was founded in 1998 and is headquartered in Amsterdam, the Netherlands.

uniQure N.V. (NASDAQ:QURE) has a market capitalization of $1.03 billion and its shares have jumped by 2.79% year to date, at a time when risky stocks such as biotechnology have generally seen investors flee away from them for safe haven stocks and U.S dollar. The bulk of this growth has come during the second half of this year when it released the trial data for its Huntington’s disease treatment. The study showed that after 12 months, the mutant HTT gene in patients had dropped by 54%, and the next data release from the trials is slated for the first half of next year.

H.C. Wainwright kept a massive $90 share price target (current share price is $22) for uniQure N.V. (NASDAQ:QURE) in August 2022 as it stated that a recent selloff is nothing but an attractive buying opportunity.

uniQure N.V. (NASDAQ:QURE)’s largest investor in our database is Steven Cohen’s Point72 Asset Management which owns 3.8 million shares that are worth $71 million.

6. Verve Therapeutics, Inc. (NASDAQ:VERV)

Market Capitalization as of November 12, 2022: $1.66 billion

Verve Therapeutics, Inc. (NASDAQ:VERV) is a gene editing firm that develops treatments exclusively for heart diseases. The company’s treatments also target the PCKS9 gene in the liver, and it has agreements and partnerships with several companies and organizations, including Harvard University. Verve Therapeutics, Inc. (NASDAQ:VERV) is headquartered in Cambridge, Massachusetts.

Verve Therapeutics, Inc. (NASDAQ:VERV)’s shares soared in July this year after BMO Capital penned that the firm is one of the best gene editing stocks and stated that it has significant long term growth potential. The firm set a $48 share price target for the company and an Outperform rating as it initiated coverage, mentioning that Verve Therapeutics, Inc. (NASDAQ:VERV)’s gene editing technologies have stood the test of clinical testing and passed with flying colors. Verve Therapeutics, Inc. (NASDAQ:VERV)’s treatment for cholesterol can reduce malignant genes by up to 98% according to data released in August 2022.

Verve Therapeutics, Inc. (NASDAQ:VERV) has $1.66 billion in market capitalization and its shares jumped by another 15% on November 11, 2022.

Eli Casidin’s Casdin Capital is Verve Therapeutics, Inc. (NASDAQ:VERV)’s largest investor through a $47 million stake that comes via 3.1 million shares.

Along with Intellia Therapeutics, Inc. (NASDAQ:NTLA), CRISPR Therapeutics AG (NASDAQ:CRSP), and Beam Therapeutics Inc. (NASDAQ:BEAM), Verve Therapeutics, Inc. (NASDAQ:VERV) is a hot gene therapy stock.

Click to continue reading and see 5 Most Promising Gene Therapy Companies to Watch.

AI, Tariffs, Nuclear Power: One Undervalued Stock Connects ALL the Dots (Before It Explodes!)

Published on May 7, 2025 at by Inan Dogan, PhD

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10 Most Promising Gene Therapy Companies to Watch

Published on November 14, 2022 at 2:31 pm by Ramish Cheema in Lists, News

Most Promising Gene Therapy Companies to Watch

10. Akouos, Inc. (NASDAQ:AKUS)

9. bluebird bio, Inc. (NASDAQ:BLUE)

8. Editas Medicine, Inc. (NASDAQ:EDIT)

7. uniQure N.V. (NASDAQ:QURE)

6. Verve Therapeutics, Inc. (NASDAQ:VERV)

AI, Tariffs, Nuclear Power: One Undervalued Stock Connects ALL the Dots (Before It Explodes!)

Published on May 7, 2025 at by Inan Dogan, PhD

Here’s why this is a deal you can’t afford to pass up:

A New Dawn is Coming to U.S. Stocks

Published on January 7, 2025 at by Insider Monkey Staff

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10 Most Promising Gene Therapy Companies to Watch

Published on November 14, 2022 at 2:31 pm by Ramish Cheema in Lists, News

Share

Most Promising Gene Therapy Companies to Watch

10. Akouos, Inc. (NASDAQ:AKUS)

9. bluebird bio, Inc. (NASDAQ:BLUE)

8. Editas Medicine, Inc. (NASDAQ:EDIT)

7. uniQure N.V. (NASDAQ:QURE)

6. Verve Therapeutics, Inc. (NASDAQ:VERV)

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AI, Tariffs, Nuclear Power: One Undervalued Stock Connects ALL the Dots (Before It Explodes!)

Published on May 7, 2025 at by Inan Dogan, PhD

Here’s why this is a deal you can’t afford to pass up:

A New Dawn is Coming to U.S. Stocks

Published on January 7, 2025 at by Insider Monkey Staff