Industry Overview - Companies in synthetic biology engineer biological systems for applications in pharmaceuticals and agriculture, utilizing genetic engineering and biotechnology techniques to create novel organisms and processes, with significant growth potential in curing genetic diseases and extending human lifespan [1] - The genomics and synthetic biology sectors are attracting substantial interest from venture capitalists and institutional investors due to their potential for innovation and tailored treatments based on genetic makeup [8][21] Company Highlights - CRISPR Therapeutics is leveraging its CRISPR/Cas9 gene-editing platform to develop therapies for hemoglobinopathies, cancer, and diabetes, achieving a milestone with the first-ever approval for a CRISPR/Cas9 gene-edited therapy, Casgevy, in the UK for treating sickle cell disease and transfusion-dependent beta-thalassemia [3][12][23] - Monte Rosa Therapeutics is focused on developing small-molecule precision medicines that utilize the body's mechanisms to selectively degrade proteins relevant to various diseases, including MYC-driven tumors and autoimmune diseases [5][11] - Immunocore Holdings is developing a novel class of T cell receptor bispecific immunotherapies for cancer and autoimmune diseases, with its product KIMMTRAK already in use for treating unresectable or metastatic uveal melanoma [13][25] Price Target and Upside Potential - The average short-term price target for CRISPR Therapeutics indicates a potential increase of 136.7% from the last closing price of $28.32, with a maximum upside of 253.1% and a downside of 15.3% [4] - Monte Rosa Therapeutics has a projected price target increase of 161.1% from a closing price of $6.93, suggesting a maximum upside of 188.6% with no downside [14] - Rigel Pharmaceuticals shows a potential price increase of 82.1% from a closing price of $17.09, with a maximum upside of 233.5% and a downside of 18.1% [18] - Castle Biosciences has a projected price target increase of 51.4% from a closing price of $27.53, indicating a maximum upside of 81.6% with no downside [20] Investment Recommendations - Five small and mid-sized genomics and synthetic biology stocks are recommended for investment, including CRISPR Therapeutics, Immunocore Holdings, Monte Rosa Therapeutics, Rigel Pharmaceuticals, and Castle Biosciences, all carrying favorable Zacks Ranks [7][9]

Core Viewpoint - CRISPR Therapeutics has faced a significant decline in stock value, down 23% year to date, despite recent regulatory approvals for its gene-editing medicine, Casgevy [1] Regulatory Approvals - Since November 2023, CRISPR Therapeutics has received approvals for Casgevy in multiple regions, including the U.S., U.K., European Union, Saudi Arabia, and Bahrain [2] Market Potential and Pricing - Casgevy is priced at $2.2 million in the U.S., targeting a market of approximately 35,000 patients in the U.S. and Europe, but high costs may limit accessibility [3] - In the U.S., around 100,000 people have sickle cell disease (SCD), with an estimated 50% to 60% on Medicaid, which may enhance access to Casgevy through government initiatives [4][11] - The potential market for Casgevy also includes about 23,000 patients in Saudi Arabia and Bahrain [20] Financial Outlook - Analysts project that Casgevy's peak annual sales could exceed $2.2 billion, with CRISPR entitled to 40% of the profits from the medicine [18] - In the first nine months of the year, CRISPR reported revenue of approximately $1.6 million, indicating slow uptake of Casgevy [21] Competitive Landscape - CRISPR Therapeutics faces competition from Bluebird Bio, which has alternative treatments for SCD and transfusion-dependent beta-thalassemia (TDT), but CRISPR and Vertex have more financial resources [7] - The company has less competition in Europe and the Middle East, presenting a multibillion-dollar revenue opportunity for Casgevy [15] Future Developments - The Biden administration's initiative to assist Medicaid patients in affording Casgevy is expected to improve access and could facilitate future approvals for other gene-editing therapies [5][11][13] - CRISPR Therapeutics is also developing additional gene-editing programs, including investigational cancer treatments and a potential cure for type 1 diabetes [13] Innovation and Validation - The development of Casgevy has validated CRISPR Therapeutics' gene-editing platform, suggesting that the company may achieve more clinical and regulatory successes in the next five years [8]

CRISPR Therapeutics (CRSP -1.64%) isn't a stock that's wanting for reasons to invest. Between the solid launch of its first commercialized therapy and a promising pipeline that's continuing to deliver, this biotech is on track for growth. Plus, investors just got another big green flag to invest in its stock.Here's what you need to know.This new development augurs a bright futurePer the company's presentation at the American Society of Hematology's (ASH) annual meeting on Dec. 9, the Food and Drug Administr ...

Innovation holds the key in the ever-evolving biotech sector, and most companies in this sector are striving to develop a drug or treatment using a breakthrough technology.Biotech companies with promising gene therapies in their portfolio/pipeline have garnered investors’ attention in 2024. Interest in this space renewed following the FDA approval of a few gene therapies toward the end of 2023.Consequently, some pharma/biotech bigwigs scurried for a lucrative catch in the space, either through licensing dea ...

Investors looking for stocks that can make dramatic gains can find some in the biopharmaceutical industry. Right now, there are a pair of biopharma stocks that Wall Street expects to rocket higher in 2025.The Food and Drug Administration (FDA) approved CRISPR Therapeutics' (CRSP 1.05%) first treatment last December, and its successful launch could push the stock much higher.  Investment bank analysts also expect Iovance Biotherapeutics (IOVA 0.13%) to soar in 2025.Before chasing exciting price targets, thou ...

CRISPR Therapeutics (CRSP -3.57%) is recognized as a pioneer in the field of gene therapy. This biotechnology has the potential to revolutionize medicine through precise modifications of a person's DNA to treat and cure genetic diseases. In 2023, the company's Casgevy product for sickle cell disease became the first-ever CRISPR-based therapy approved by the Food and Drug Administration (FDA) as an important company milestone.Nevertheless, commercialization has been slow, with the market already waiting for ...

Shares of CRISPR Therapeutics AG (CRSP) have gained 0.8% over the past four weeks to close the last trading session at $51.17, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $81 indicates a potential upside of 58.3%.The mean estimate comprises 24 short-term price targets with a standard deviation of $34.31. While the lowest estimate of $30 indicates a 41.4% decline from the cur ...

Ark Invest chief Cathie Wood is known for scooping up shares of innovators early in their growth stories and holding on for the long term. The idea is to get in on these players for a bargain price, and then benefit as they launch products and revenue gains momentum. This takes time, but patient investors could score a major win by following Wood's strategy -- and by picking up a couple of her favorite stocks. Over the past several weeks, Wood has added to one of her key positions, a stock that's lost about ...

On Nov. 21, shares of CRISPR Therapeutics (CRSP 3.44%) were down 47% from a peak they reached in March. This might be a little surprising to folks who have been following this developer of gene therapies. After all, it's been less than a year since regulators in the U.S. and E.U. approved its first therapy, Casgevy, to treat two blood-based disorders.Casgevy's initial launch hasn't been as exciting as investors and its partner, Vertex Pharmaceuticals (VRTX 0.13%), had hoped. Less than a year into the launch ...

Godzilla and King Kong surely weren't born big. They began small and grew over time to their gargantuan sizes even if the movies don't tell their childhood stories.Likewise, huge companies of today were once much smaller (unless perhaps they were spin-offs). Investors who spotted them early had opportunities to make fortunes.Can you still find such monsters in the making? Three Motley Fool contributors think so. Here's why they think biotech stocks CRISPR Therapeutics (CRSP 3.44%), Summit Therapeutics (SMMT ...