Core Insights - The biotech industry presents opportunities for significant returns in short timeframes due to clinical or regulatory advancements, with several companies projected to see substantial share price increases in the next year [1][2] Group 1: CRISPR Therapeutics - CRISPR Therapeutics is a leading gene-editing company that received approval for its therapy Casgevy, the first CRISPR-based medicine, but has struggled with sales post-approval due to complex administration [3][4] - Wall Street's average price target for CRISPR Therapeutics is $84.62, indicating a potential upside of 159%, making it a long-term investment consideration despite short-term volatility [4][5] - The company has a promising pipeline, including potential treatments for type 1 diabetes and cancer, appealing to risk-tolerant investors [5] Group 2: Iovance Biotherapeutics - Iovance Biotherapeutics specializes in cancer therapies using tumor-infiltrating lymphocytes and gained U.S. approval for Amtagvi to treat melanoma, achieving $164.1 million in revenue in 2024 [6][7] - The stock has a price target of $20.91, suggesting a potential upside of 543%, with upcoming regulatory approvals and a large patient base in the U.S. as catalysts for growth [7][8] - Iovance could be an attractive option for patient investors, given its innovative approach and potential for further clinical successes [9] Group 3: Regeneron - Regeneron is a well-established biotech firm facing challenges with its key product Eylea due to competition, but has a price target of $914.55, indicating a 50% upside [10] - A legal battle over Eylea's biosimilar could significantly impact share prices, while the success of Dupixent and a robust pipeline enhance its investment appeal [11][12] - The company has initiated a dividend program and continues share buybacks, reinforcing its position as a strong long-term investment [12] Group 4: Sarepta Therapeutics - Sarepta Therapeutics focuses on gene therapies for rare diseases and recently launched Elevidys, but faced a setback with a patient death linked to liver failure [13][14] - Despite the controversy, the average price target remains at $165.35, suggesting an upside of 182%, contingent on clarifying the cause of the patient's death [14][15] - Given the current uncertainty surrounding Elevidys, caution is advised for potential investors until more information is available [15]

Core Viewpoint - Sarepta Therapeutics' shares fell over 6% following the European Medicines Agency (EMA) placing a clinical hold on studies for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), due to a patient's death post-treatment [1][2]. Company Overview - Elevidys is a one-shot gene therapy developed in collaboration with Roche, and it is the first of its kind for DMD in the United States [4][9]. - The therapy has shown significant sales potential, contributing nearly 60% to Sarepta's total revenues in Q4 2024, with expectations of $2.9-$3.1 billion in net product revenues for 2025, two-thirds of which are anticipated from Elevidys [6]. Clinical Developments - The clinical hold was initiated after a patient's death attributed to acute liver failure, with ongoing investigations into the potential role of a recent CMV infection [2][3]. - Enrollment and dosing in three studies evaluating Elevidys at EU sites have been paused until the cause of death is fully analyzed [3]. Market Impact - Concerns regarding the clinical hold may lead to increased hesitance among doctors to prescribe Elevidys, potentially hindering its market adoption in the U.S. and delaying its launch in the EU [5]. - Year-to-date, Sarepta's shares have decreased nearly 52%, contrasting with a 1% growth in the industry [7]. Product Portfolio - Sarepta's portfolio includes four approved therapies for DMD, with Elevidys being a major contributor to revenue growth, having generated approximately $821 million in 2024 compared to $200 million the previous year [9][10]. - Other therapies in the portfolio, such as Exondys 51, Vyondys 53, and Amondys 45, have also seen strong demand and are expected to address nearly a third of all DMD patients in the U.S. [10].

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NEW YORK, March 26, 2025 (GLOBE NEWSWIRE) -- Pomerantz LLP is investigating claims on behalf of investors of  Sarepta Therapeutics, Inc. (“Sarepta” or the “Company”) (NASDAQ: SRPT). Such investors are advised to contact Danielle Peyton at newaction@pomlaw.com or 646-581-9980, ext. 7980. The investigation concerns whether Sarepta and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices.  [Click here for information about joining the class action] On M ...

Core Viewpoint - Sarepta Therapeutics is facing significant challenges, particularly following a 20% drop in share price due to concerns surrounding its key product, Elevidys, after a patient suffered fatal liver failure [1][5]. Group 1: Company Overview - Sarepta Therapeutics specializes in gene therapies for Duchenne muscular dystrophy (DMD), with Elevidys being its most important product targeting the genetic causes of the disorder [3]. - Elevidys received full U.S. approval for ambulatory DMD patients aged 4 and older, but is under accelerated approval for non-ambulatory patients, requiring further clinical trials to confirm efficacy [4]. Group 2: Recent Developments - A young patient treated with Elevidys experienced fatal acute liver failure, raising concerns about the treatment's safety, despite liver injury being a known risk [5]. - In Q4, Sarepta reported revenue of $658.4 million, a 66% increase year-over-year, with Elevidys contributing $384.2 million, accounting for over 50% of total revenue [5]. Group 3: Market Reaction and Future Outlook - The market reacted negatively to the news of the patient death, creating uncertainty around Elevidys' future sales and prescribing trends [5][7]. - Despite the incident, the company noted that the patient had a recent cytomegalovirus infection, which could have contributed to the liver failure, suggesting that the situation may not be solely attributable to Elevidys [6]. - The worst-case scenario would involve regulators pulling Elevidys from the market, but this is considered unlikely due to the lack of alternative effective treatments for DMD [7]. - Sarepta has other DMD treatments in development, but Elevidys was expected to be the primary growth driver, making the current uncertainty a risk for the company's stock [8].

Analyst's Disclosure: I/we have no stock, option or similar derivative position in any of the companies mentioned, but may initiate a beneficial Long position through a purchase of the stock, or the purchase of call options or similar derivatives in SRPT over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article. Seeking ...

Sarepta Therapeutics (SRPT) shares soared 8.7% in the last trading session to close at $79.97. The move was backed by solid volume with far more shares changing hands than in a normal session. This compares to the stock's 30.2% loss over the past four weeks.This rise in share price can be attributed to the recovery in Sarepta’s share price after it tanked earlier this week on reports of a patient’s death following treatment with Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy.This biopha ...

Core Viewpoint - Sarepta Therapeutics' stock experienced a significant decline of over 20% following the report of a patient's death after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy (DMD) [1][4]. Company Overview - The patient who died was reported to have suffered from acute liver failure, a known risk associated with Elevidys. This incident marks the first death linked to the therapy, although the patient also had a recent CMV infection that may have contributed to the liver damage [2][3]. - Sarepta has communicated the event to health authorities and plans to update the prescribing information for Elevidys. The company asserts that the overall benefit-risk profile of the therapy remains positive, with over 800 patients treated [3]. Financial Impact - The decline in Sarepta's stock is attributed to concerns that this incident may deter doctors from prescribing Elevidys, potentially slowing its market adoption. Elevidys accounted for nearly 60% of the company's total revenues in Q4 2024, with projected net product revenues for the year between $2.9 billion and $3.1 billion, two-thirds of which are expected from Elevidys sales [4]. - Year-to-date, Sarepta's shares have decreased by nearly 40%, contrasting with a 6% growth in the industry [5]. Product Portfolio - Sarepta's portfolio includes four approved therapies for DMD, with Elevidys being the first one-shot gene therapy for the condition in the U.S. Since its launch in June 2023, Elevidys has shown significant sales potential, generating approximately $821 million in 2024 compared to $200 million in the previous year [7]. - The company has a partnership with Roche for the development of Elevidys, which has exclusive rights to market the therapy outside the U.S. [8]. - Other therapies in Sarepta's portfolio include Exondys 51, Vyondys 53, and Amondys 45, which collectively have the potential to address nearly one-third of all DMD patients in the U.S. [9].

Sarepta Therapeutics Inc (NASDAQ:SRPT) is down 23.2% to trade at $77.86 at last check, and earlier hit a 52-week low of $75.06. The security is pacing for its worst single-day percentage loss since October 2023 after the gene-editing company revealed a 16-year-old boy died from acute liver failure after receiving its gene therapy for a rare muscular dystrophy. Year to date, the equity is down 37.5%. Beam Therapeutics Inc (NASDAQ:BEAM) is down 6.8% to trade at $22.85 at last glance, brushing off a price-targ ...

About this content About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, ...