Latest news with #Sarepta
Yahoo
4 days ago
- Business
- Yahoo
Sarepta Therapeutics (NasdaqGS:SRPT) SRP-9003 Gene Therapy Earns FDA Platform Technology Designation
Sarepta Therapeutics recently announced that its SRP-9003 gene therapy received a platform technology designation from the FDA, and updated safety protocols for ELEVIDYS in the UK. Despite these developments, the company's shares fell 43% last month. This decline may have been influenced by market volatility amid geopolitical tensions and weak economic data. While these broader market factors likely added weight to the movement, Sarepta's price plunge suggests specific investor concerns, potentially around the challenges and risks disclosed in their recent product updates, affected sentiment. Be aware that Sarepta Therapeutics is showing 2 risks in our investment analysis and 1 of those is significant. Outshine the giants: these 26 early-stage AI stocks could fund your retirement. The recent FDA platform technology designation for SRP-9003 and updated safety protocols for ELEVIDYS in the UK illuminate the company's focus on addressing safety concerns and operational intricacies, potentially positively affecting Sarepta's future revenue and earnings prospects. These actions aim to bolster patient and physician confidence, fostering smoother administrative processes and treatment uptakes. Nevertheless, the broad 43% share price decline last month amid the geopolitical and economic backdrop highlights the market's sensitivity towards perceived risks and uncertainties, as reflected in Sarepta's significant price fluctuations. Over the past three years, Sarepta's total shareholder returns, including dividends, have decreased by 68.73%. This underperformance is further emphasized by a comparison to the one-year return, where Sarepta lagged behind both the US Biotechs industry, which decreased by 9.6%, and the broader US market, which increased by 10.9%. This historical performance context underscores the challenges Sarepta faces in aligning its operational improvements with shareholder value recovery. In terms of future prospects, these strategic updates and anticipated product data releases could influence revenue and earnings forecasts, potentially validating analysts' growth expectations. Analysts project a price target of US$147.33, 68.3% above the current share price of US$46.75. Achieving this target hinges on ramping up administrative efficiencies, expanding therapeutic offerings, and realizing long-term revenue drivers, assuming favorable patient and regulatory responses in the wake of recent developments. The analysis detailed in our Sarepta Therapeutics valuation report hints at an deflated share price compared to its estimated value. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGS:SRPT. This article was originally published by Simply Wall St. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Forbes
4 days ago
- Health
- Forbes
Second Patient Death After Gene Therapy: Implications For The Future
As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics announced a second patient death following treatment with its gene therapy, Elevidys, for Duchenne muscular dystrophy. This news has sent shockwaves through the rare disease and biotech communities. It raises questions about the safety of adeno-associated virus-based gene therapies. The latest fatality, a 15-year-old, died from acute liver failure, a recognized but rare complication of adeno-associated virus-based gene transfer. This death follows a similar case reported in March. These recent tragedies have profound implications. Elevidys, Sarepta's gene therapy, was among the first to receive approval for the treatment of Duchenne muscular dystrophy. Yet, the deaths of two patients treated with Elevidys now force us to confront a difficult question. Do the potential benefits of this therapy truly outweigh the risks, particularly for those who can no longer walk and may be at higher risk of complications? Every advance in medicine brings new challenges, and gene therapy is no exception. The deaths associated with Elevidys have raised the regulatory bar for all investigational gene therapies that use adeno-associated virus vectors. This means more stringent safety monitoring, more careful patient selection, and better reporting of outcomes. This heightened scrutiny is not unwelcome, even if these measures may slow the pace of development in the short term. They are essential for ensuring safety and responsibility. Adeno-associated virus-based vectors have become the workhorse of gene therapy due to their efficiency and relatively low risk of complications. The field has long recognized that high-dose adeno-associated virus-based administration can trigger severe, sometimes fatal, immune-mediated toxicities. These primarily affect the liver. In 1999, Jesse Gelsinger died after receiving an adenoviral gene therapy. His tragic death marked a turning point in the field and led to decades of increased scrutiny and regulation. While adeno-associated viruses are generally less immunogenic than adenoviruses, high doses can activate the body's immune system. This leads to inflammation, liver injury, and, in rare cases, organ failure. Recent studies highlight that immune responses can contribute to toxicity, particularly in the liver. However, not all patients are impacted equally. Recent findings underscore that age and disease status can significantly influence a patient's response to adeno-associated virus-based gene therapy. Clinical studies reveal that individual factors, such as underlying infections, pre-existing liver disease, or genetic predispositions, can amplify the risks. For example, in the first reported Elevidys death, a concurrent infection may have played a role in the patient's rapid decline. Research from the University of Bristol indicates that in ocular gene therapy trials, older patients, particularly females, are more likely to experience severe inflammatory responses. However, in the Elevidys cases, both fatalities involved males who had lost the ability to walk due to advanced disease progression. This is a critical factor linked to heightened immune vulnerability. This underscores that risks are context-specific, depending on therapy type, delivery site, and patient health status. To address the pressing safety concerns, Sarepta is investigating several strategies. One approach under consideration is the addition of sirolimus, an immunosuppressant widely used in organ transplantation. While this aims to reduce immune-mediated liver damage, experience from liver transplant patients warns that sirolimus can nearly triple infection risk, even at low doses. Its use in gene therapy remains experimental and demands careful evaluation before it can be widely adopted. At the same time, non-viral delivery methods are emerging as a promising alternative. Lipid nanoparticles offer a way to bypass many of the immune challenges posed by viral vectors. Their ability to be tailored for precise tissue targeting and the absence of pre-existing immunity in patients make them particularly attractive for conditions like Duchenne muscular dystrophy, where repeated dosing may be necessary. Though still less efficient than viral vectors in some applications, the technology is advancing quickly. The second death following Elevidys for Duchenne muscular dystrophy is a sobering reminder of the risks inherent in adeno-associated virus-based gene transfer, particularly for older or more severely affected patients. Still, these tragedies are not new to the field. We have seen similar inflection points before. The history of gene therapy is marked by moments of triumph and tragedy. Each shapes the path forward. As we reflect on these recent events, it is clear that the field stands at another such crossroads—one that will require careful balancing of hope, caution, and scientific rigor to chart the best course for patients and the future of medicine. For a deeper exploration of these issues, including real-world patient stories and the latest scientific advances, see Destiny's Child No Longer: Rewriting Genetic Fate, which provides a detailed examination of this therapy and other gene therapies.
Business Wire
4 days ago
- Business
- Business Wire
SRPT BREAKING NEWS: Sarepta Therapeutics, Inc. is being Investigated for Securities Fraud after Second Elevidys Death; Investors with Losses are Notified to Contact BFA Law
NEW YORK--(BUSINESS WIRE)--Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Leading securities law firm Bleichmar Fonti & Auld LLP announces an investigation into Sarepta Therapeutics, Inc. (NASDAQ: SRPT) for potential violations of the federal securities laws. Share If you invested in Sarepta, you are encouraged to obtain additional information by visiting: Why is Sarepta being Investigated? Sarepta is a biopharmaceutical company focused on developing treatments for rare diseases. Sarepta's most important product is Elevidys, a therapy for the treatment of Duchenne muscular dystrophy. During the relevant period, Sarepta repeatedly touted the safety profile of Elevidys and told investors that the benefits of the treatment outweighed its risks. In truth, Elevidys causes fatal acute liver failure in some patients. The Stock Declines as the Truth is Revealed On March 18, 2025, Sarepta announced that a patient that had been treated with Elevidys died after suffering acute liver failure. On this news, the price of Sarepta stock fell $27.81 per share, or over 27%, from $101.35 per share on March 17, 2025, to $73.54 per share on March 18, 2025. Nevertheless, on the same day, Sarepta assured investors that 'the benefit-risk of ELEVIDYS remains positive.' Next, on June 15, 2025, Sarepta announced that a second patient treated with Elevidys had died from acute liver failure. The company further announced that because of the second death, it was suspending certain shipments of Elevidys and paused dosing in an ongoing clinical trial of the treatment. On this news, the price of Sarepta stock fell $15.24 per share, or more than 42%, from $36.18 per share on June 13, 2025, to $20.94 per share on June 16, 2025. Click here for more information: . What Can You Do? If you invested in Sarepta you may have legal options and are encouraged to submit your information to the firm. All representation is on a contingency fee basis, there is no cost to you. Shareholders are not responsible for any court costs or expenses of litigation. The firm will seek court approval for any potential fees and expenses. Why Bleichmar Fonti & Auld LLP? Bleichmar Fonti & Auld LLP is a leading international law firm representing plaintiffs in securities class actions and shareholder litigation. It was named among the Top 5 plaintiff law firms by ISS SCAS in 2023 and its attorneys have been named Titans of the Plaintiffs' Bar by Law360 and SuperLawyers by Thompson Reuters. Among its recent notable successes, BFA recovered over $900 million in value from Tesla, Inc.'s Board of Directors, as well as $420 million from Teva Pharmaceutical Ind. Ltd. For more information about BFA and its attorneys, please visit Attorney advertising. Past results do not guarantee future outcomes.
Time of India
5 days ago
- Health
- Time of India
Second patient death reported with gene therapy for muscular dystrophy
Washington: Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy . Sarepta reported the death over the weekend and provided additional details about its response, which includes pausing shipments of the therapy, Elevidys, for older patients who are no longer able to walk. The one-time treatment is approved for children with a genetic variant of Duchenne's muscular dystrophy, which causes weakness, loss of mobility and early death in males. Elevidys is the first gene therapy approved in the U.S. for the rare muscle-wasting condition, but it has faced scrutiny since its accelerated approval in 2023. The second death, like an earlier one reported in March, occurred in a teenage boy who suffered a fatal case of acute liver injury, a known side effect of the therapy. Older patients receive a larger dose of the therapy. Sarepta said it would pause a study in those patients and assemble an expert panel to recommend new safety protocols for taking the drug. Those changes are expected to include increased use of immune-system suppressing drugs, company executives said Monday. The liver injury associated with the therapy is thought to be connected to the immune system's response. Sarepta said it was cooperating with the Food and Drug Administration, which would have to sign off on any changes to the product's use. Elevidys received expedited approval despite concerns from some FDA scientists about its effectiveness in treating Duchenne's. The FDA granted full approval last year and expanded the therapy's use to patients 4 years and older, regardless of whether they are still able to walk. Previously it was only available for younger patients who were still walking. Shares of the Cambridge, Massachusetts-based company fell more than 42% to close at about $21 in trading. Wall Street analysts speculated that FDA officials, including new vaccine chief Dr. Vinay Prasad, might impose more restrictions on the drug or even block its use. Prasad has been highly critical of the therapy since its approval under the agency's previous leadership. "Now with two deaths reported in this segment of the market, it seems incrementally more possible that the FDA could step in and remove the therapy from the market in non-ambulatory patients," said Leerink Partners analyst Joseph Schwartz, in a Sunday research note. Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time infusion.
Business Insider
5 days ago
- Business
- Business Insider
Sarepta initiated with a Peer Perform at Wolfe Research
Wolfe Research initiated coverage of Sarepta (SRPT) with a Peer Perform rating. Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
