Advanced biotechnology includes gene treatment and editing. The addition of a functional copy of a gene is a common method of treatment in gene therapies, which are intended to rectify genetic defects by injecting genetic matter at the cell level. The therapeutic potential of gene editing is groundbreaking because it seeks to directly modify the DNA. The majority of gene-editing shares saw significant losses in 2022, but researchers at ResearchAndMarkets.com currently forecast that the global market for genome editing would increase by 53.1% yearly and reach $59.8 billion by 2027. Below are the top gene editing stocks to invest in.
Top Gene Editing Stock Investments
CRISPR Therapeutics AG
CRISPR Therapeutics is a biotechnology business that treats blood diseases and cancer using the gene-editing technology CRISPR-Cas9. Crispr and its collaborator, Vertex Pharmaceuticals Inc., are hopeful that their gene-editing therapy for sickle cell disorder and transfusion-based beta-thalassemia will be approved by the Food & Drug Authority (FDA) soon. According to expert Geoff Meacham, the chance of clearance from regulators is rather strong. Potential catalysts for price appreciation in the company in 2023 include the release of new information on other major items, such as therapies for B-cell abnormalities in the immune system.
Beam Therapeutics Inc.
Base editing, which is used by Beam Therapeutics and employs CRISPR innovation, involves altering a single base in the genome. This method only damages one strand of DNA, which might lead to improved cell longevity. Although the corporation's primary emphasis is on treating hemoglobinopathy and cancer, it is also doing research into potential treatments for ophthalmic and liver diseases. Beam's recent announcement that it would advance BEAM-102, a therapy candidate, through wave two of its sickle cell disorder study was met with praise from expert Greg Harrison. The business has also started developing a therapy for alpha-1 antitrypsin deficiency, which is a serious hereditary condition. According to Harrison, the corporation's novel platform has the potential to provide considerable long-term returns for investors.
Apellis Pharmaceuticals Inc.
The medicines developed by Apellis Pharmaceuticals, a biotech firm, improve the defense system's capacity to eliminate injured cells by focusing on the complement pathway. Pegcetacoplan, the corporation's principal medication, is now in late-stage research to cure geographic atrophy, a condition that results in age-related vision loss. It is already authorized in the U.S. for curing the uncommon acquired blood disorder paroxysmal nocturnal hemoglobinuria. Apellis & Beam Therapeutics formed a partnership in 2021 to use base-altering tech to study novel precision medicines for the eye, kidneys, and brain. According to expert Tazeen Ahmad, Apellis is currently in the lead to be the first company to market with a cure for geographic atrophy.
Graphite Bio Inc.
The gene-editing method developed by Graphite Bio makes use of both CRISPR and endogenous DNA-repair mechanisms. Meacham claims that Graphite has sufficient cash on hand to cover the cost of both the first data report from its beta-thalassemia treatment trial and an experimental new medication application. Around the middle of the year 2023, Graphite hopes to have proof-of-concept information. According to Meacham, Graphite is still in the clinical trial phase, so investors may need to be patient. Graphite has the edge over its rivals because its gene-editing platform stands out from the crowd and has the capacity to produce substantial long-term value.
Caribou Biosciences Inc.
Caribou Biosciences develops gene-edited cancer treatments. Caribou Biosciences announced encouraging developments in December about two of its investigational medicines for cancers other than Hodgkin's lymphoma & solid tumors. Approximately one-third of the lymphoma patients remained in full remission after 12 months, according to Caribou; the firm has recruited three patients to evaluate the treatment at a higher dosage in 2023. Meacham believes the tumor therapy has strong preclinical results, but more information is required to assess whether it can serve as a growth driver for the firm. Given that the data updates didn't cause him to rethink his optimistic thesis, Meacham found the first negative market response to the data unexpected.
Intellia Therapeutics Inc.
Intellia Therapeutics is a biotechnology business in the clinical research and development stage, working on gene-editing medicines for the treatment of various disorders. Transthyretin amyloidosis is indeed a protein condition of the heart, and this company's flagship treatment program is now in the middle of clinical studies. According to Intellia CEO Michael Harrison, the business's recent results on therapy for hereditary angioedema, which induces soreness in the body, indicate outstanding effectiveness, and the firm aims to advance to the Phase 2 dosage expansion stage of that program in early 2023. Given their commonalities, Harrison believes that the program's favorable statistics are also excellent news for Intellia's flagship program.
Vertex Pharmaceuticals Inc.
For diseases including cystic fibrosis & inflammatory disorders, Vertex Pharmaceuticals creates new small-molecule medicines. For up to $1.2 billion in 2021, awaiting regulatory clearance, Vertex purchased the rights to 60 percent of the income from sales of CTX001, now branded exa-cel, a gene-editing medicine developed by Crispr Therapeutics. Earlier this month, Vertex & Crispr made public their plans to submit exa-cel to the Food and Drug Administration and European authorities for evaluation as a treatment for sickle cell disorder and transfusion-based beta-thalassemia. According to Meacham, Vertex has a distinct growth profile, and its expanding orphan disease portfolio might provide investors with more potential. It's included in the top gene editing stocks 2022.
