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US FDA's top vaccine official Vinay Prasad leaves agency after 3 months
US FDA's top vaccine official Vinay Prasad leaves agency after 3 months

Reuters

time13 hours ago

  • Health
  • Reuters

US FDA's top vaccine official Vinay Prasad leaves agency after 3 months

July 30 (Reuters) - The U.S. Food and Drug Administration's top vaccine official, Vinay Prasad, has left the agency after just three months as Director of the Center for Biologics Evaluation and Research, a government spokesperson said. Prasad, an oncologist who was a fierce critic of U.S. COVID-19 vaccine and mask mandates, had been appointed in May by FDA Commissioner Marty Makary. Together they had promised to speed regulatory action and pilot new review pathways. In recent days, criticism of his tenure has intensified around the agency's handling of a gene therapy for Duchenne muscular dystrophy from Sarepta Therapeutics (SRPT.O), opens new tab. The FDA-approved therapy played a role in the death of two teens who had advanced disease, and after a third death in a separate experimental gene therapy from the company, the FDA on July 18 asked Sarepta to stop all shipments of the approved DMD therapy, saying it had safety concerns. Laura Loomer, a far-right activist and President Donald Trump ally, on July 20 posted a blog in which she called Prasad a "progressive leftist saboteur" who was undermining the agency's work. Loomer critiques earlier this year led to the ouster of national security officials. On Monday, the Wall Street Journal's opinion section ran two pieces criticizing Prasad, one of which called him a Bernie Sanders acolyte in MAHA drag, referring to the Trump administration's "Make America Healthy Again" movement. Both said he was not basing regulatory decisions on whether drugs helped patients, but on price. "When he saw some of these sort of smear pieces, he didn't want to be a distraction," Makary said of Prasad's departure in an interview with CNBC on Wednesday. Prasad, whose departure was announced late on Tuesday, did not immediately respond to a request for comment. The FDA on Monday changed course on Sarepta and said shipments to the main group of patients for the drug could restart. Other changes under Prasad, who was also the agency's chief medical and science officer, included a new policy limiting the approval for COVID-19 vaccines, and his FDA division also rejected therapies from Replimmune (REPL.O), opens new tab and Capricor Therapeutics (CAPR.O), opens new tab. Shares of Replimmune nearly doubled in value, while those of Sarepta and Capricor gained 11% and 18%, respectively on Wednesday. Endpoints and STAT news first reported Prasad's departure. Prasad was a physician who joined the agency from the University of California, San Francisco. He has had stints at the National Cancer Institute and the National Institutes of Health. The Department of Health and Human Services, which oversees the FDA, did not immediately respond to a request for comment on when Prasad's successor could be announced. The FDA and other health agencies have seen multiple shakeups in recent months, under the leadership of U.S. Health Secretary Robert F. Kennedy Jr. Prasad's predecessor and longtime vaccine official at the FDA, Peter Marks, was ousted earlier this year, followed by gene therapy head Nicole Verdun. "While another vacancy and another potential new face add uncertainty, we still think Prasad's departure is a net positive for the sector as many of our conversations have been overwhelmingly focused on regulatory risk and what he may or may not do," said Leerink analyst Joseph Schwartz.

uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress
uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress

Associated Press

time2 days ago

  • Business
  • Associated Press

uniQure Announces Second Quarter 2025 Financial Results and Highlights of Recent Company Progress

~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up ~ ~ Appointment of Kylie O'Keefe as Chief Customer and Strategy Officer ~ ~ Cash, cash equivalents and current investment securities of approximately $377.0 million as of June 30, 2025 are expected to fund operations into second half of 2027 ~ ~ uniQure to host earnings call at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, July 29, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the second quarter of 2025 and highlighted recent progress across its business. 'We delivered tremendous progress across our business in the first half of 2025, setting the stage for a transformative period ahead for uniQure,' said Matt Kapusta, chief executive officer of uniQure. 'With alignment from the FDA on a biological license application (BLA) pathway and pivotal topline data expected in September, AMT-130 is well-positioned to potentially become the first disease-modifying therapy for people living with Huntington's disease.' 'At the same time, we're encouraged by early clinical signals from the first patient treated with AMT-260 in refractory mesial temporal lobe epilepsy and the continued advancement of our Fabry and ALS programs,' continued Mr. Kapusta. 'Backed by a strong balance sheet and an exceptional team, we believe that we are well prepared to execute on the opportunities ahead, including the planned commercialization of AMT-130, and delivering lasting value to patients and shareholders.' Recent Company Developments and Updates Financial Highlights Cash position: As of June 30, 2025, the Company held cash, cash equivalents and current investment securities of $377.0 million, compared to $367.5 million as of December 31, 2024. The net increase in cash was primarily related to net proceeds of $80.5 million from the follow-on offering in January and February 2025. Based on the Company's current operating plan, including the planned U.S. launch of AMT-130, the Company expects cash, cash equivalents and current investment securities will be sufficient to fund operations into the second half of 2027. Revenues: Revenue for the three months ended June 30, 2025 was $5.3 million, compared to $11.1 million in the same period in 2024. The decrease of $5.8 million in revenue resulted from a $3.4 million increase in license revenue, a decrease of $7.1 million from collaboration revenue, and a decrease of $2.1 million from contract manufacturing of HEMGENIX® for CSL Behring. Following the divestment of the Lexington facility in July 2024, revenue from contract manufacturing is recorded net of cost within other expenses. Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were nil for the three months ended June 30, 2025, compared to $7.2 million for the same period in 2024. Following the divestment of the Lexington facility in July 2024, cost of contract manufacturing is recorded net of revenue within other expenses. R&D expenses: Research and development expenses were $35.4 million for the three months ended June 30, 2025, compared to $33.7 million during the same period in 2024. The $1.7 million increase was related to a net increase of $6.3 million in external program spend and $4.0 million higher expenses related to an increase in the fair value of contingent consideration. This was offset by a decrease of $4.7 million in employee-related expenses, a decrease of $2.1 million in facility expenses and a $1.8 million decrease in costs related to preclinical supplies and other expenses. SG&A expenses: Selling, general and administrative expenses were $13.5 million for the three months ended June 30, 2025, compared to $15.8 million during the same period in 2024. The $2.3 million decrease was primarily related to a $1.6 million decrease in employee-related expenses and a $0.6 million decrease in professional fees compared to the prior year period. Other expense: Other expense was $2.2 million for the three months ended June 30, 2025, compared to $0.2 million during the same period in 2024. The increase was primarily related to $1.4 million of non-cash expense recognized to amortize the right to purchase HEMGENIX® from Genezen on favorable terms. Other non-operating items, net: Other non-operating items, net was an income of $6.6 million for the three months ended June 30, 2025, compared to an expense of $11.3 million for the same period in 2024. The $17.9 million increase in other non-operating items, net was primarily related to an increase in net foreign currency gains of $19.6 million, a decrease in interest income of $2.3 million and a reduction of interest expense of $0.6 million due to the $50.0 million repayment of Hercules debt in July 2024. Net loss: The net loss for the three months ending June 30, 2025, was $37.7 million, or $0.69 basic and diluted loss per ordinary share, compared to a $56.3 million net loss for the same period in 2024, or $1.16 basic and diluted loss per ordinary share. Investor Conference Call and Webcast Information uniQure management will host an investor conference call and webcast today, Tuesday, July 29 at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at and following the event a replay will be archived for 90 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. If you are joining the conference call, please dial in 15 minutes before the start time. About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure's gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as 'anticipate,' 'believe,' 'could,' 'establish,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look forward to', 'may,' 'plan,' 'potential,' 'predict,' 'project,' 'seek,' 'should,' 'will,' 'would' and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company's cash runway and its ability to fund its operations into the second half of 2027; the timing and outcome of regulatory interactions with respect to the AMT-130 program, FDA's support for the program and the Company's plans to provide further regulatory updates, including the timing of its planned BLA submission and anticipated request for priority review designation; the Company's plans to incorporate a course of steroids as an immunosuppression strategy for patients receiving AMT-130; the Company's plans to announce AMT-130 topline three-year data in September 2025; the initiation of a fourth cohort evaluating high-dose AMT-130; the Company's commercialization plans, including with respect to AMT-130; and the Company's plans for further clinical updates and plans to announce initial data in its AMT-191 and AMT-162 programs. The Company's actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company's programs; the Company's interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways and timing for regulatory approval; the Company's ability to continue to build and maintain the Company infrastructure and personnel needed to achieve its goals; the Company's effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company's ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company's ability to obtain, maintain and protect intellectual property; and the Company's ability to fund its operations and to raise additional capital as needed. These risks and uncertainties are more fully described under the heading 'Risk Factors' in the Company's periodic filings with the U.S. Securities & Exchange Commission ('SEC'), including its Annual Report on Form 10-K filed with the SEC on February 27, 2025, its Quarterly Report on Form 10-Q filed with the SEC on May 9, 2025, and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.

EMA Says No to Duchenne Gene Therapy Elevidys
EMA Says No to Duchenne Gene Therapy Elevidys

Medscape

time6 days ago

  • Health
  • Medscape

EMA Says No to Duchenne Gene Therapy Elevidys

At its July 2025 meeting, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use recommended not granting marketing authorization for the Duchenne muscular dystrophy (DMD) gene therapy treatment delandistrogene moxeparvovec (Elevidys, Sarepta Therapeutics). Patients with DMD lack normal dystrophin, found primarily in skeletal and cardiac muscle cells, such that their muscles become progressively weaker and eventually stop working. Elevidys is an adeno-associated virus vector-based gene therapy product for ambulatory patients aged 4 years and older with a confirmed mutation in the DMD gene. It contains the active substance delandistrogene moxeparvovec, made of a virus containing genetic material, to produce a truncated version of dystrophin and thereby slow down disease progression. It is given as a single infusion as a one-time treatment designed to treat the underlying cause of DMD. In making their decision, the EMA said that a study had failed to show that Elevidys had an effect on movement abilities after 12 months. No Significant Improvement The study involved 125 children aged between 4 and 7 years with DMD who were able to walk and who received one infusion of either delandistrogene moxeparvovec or placebo. The main measure of effectiveness was an effect on movement abilities over 12 months, assessed using the North Star Ambulatory Assessment (NSAA). The scale ranges from 0 to 34, with higher scores indicating better movement abilities. Improvements in NSAA scores were observed in patients who received delandistrogene moxeparvovec and placebo. The difference in the change in scores between the two groups was 0.65, which was not statistically significant. In addition, although many patients treated with delandistrogene moxeparvovec were shown to produce a shorter form of the dystrophin protein, the levels of dystrophin could not be linked to an improvement in movement abilities, the EMA said. Acute Liver Failure Deaths In March this year, a 16-year-old boy died from acute liver failure after receiving treatment with Elevidys in December the previous year. In the wake of this — and at the request of the EMA — in April this year, the company temporarily halted clinical studies of the treatment in the EU. Patients who had previously been treated with Elevidys in a clinical trial continued to be monitored. In June, a second patient, aged 15, who had been treated with the drug also died from acute liver failure. Earlier this month, the FDA asked the company to include a "black-box" warning for the risk of acute liver injury and liver failure in patients with DMD who can walk. The EMA said that the company that had applied for a marketing authorization may ask for re-examination of the opinion within 15 days of receiving the agency's opinion. Rob Hicks is a retired National Health Service doctor. A well-known TV and radio broadcaster, he has written several books and has regularly contributed to national newspapers, magazines, and online publications. He is based in the United Kingdom.

Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union
Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union

Yahoo

time6 days ago

  • Business
  • Yahoo

Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union

Partner Roche will continue its dialogue with the European Medicines Agency to explore a potential path forward to make ELEVIDYS available to individuals living with Duchenne muscular dystrophy in the EU ELEVIDYS is the first and only disease-modifying gene therapy for Duchenne CAMBRIDGE, Mass., July 25, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD). "While we are disappointed by the CHMP's negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood," said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. "Following the initial FDA approval of ELEVIDYS on June 22, 2023, the therapy has subsequently received regulatory approval in several other countries. In the U.S., we are actively working with the FDA to address recent safety questions. We remain committed to working with regulators to address outstanding questions on safety so that people living with Duchenne have access to this important therapy." ELEVIDYS is the first and only approved gene therapy targeting the underlying cause of disease that has consistently demonstrated stabilization or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health. While the primary endpoint was not met in EMBARK after one year, ELEVIDYS showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other ELEVIDYS studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024, and results from year two were shared at this year's Muscular Dystrophy Association Clinical & Scientific Conference in Dallas. Quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Regulatory approval and commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche. About ELEVIDYS (delandistrogene moxeparvovec-rokl)ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated in U.S. for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval in the U.S. based on expression of ELEVIDYS micro-dystrophin (noted hereafter as "micro-dystrophin") in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). U.S. IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking StatementsThis statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "will," "may," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, discussions with regulators and the prospects for approvals or continued approvals, as applicable, of ELEVIDYS and the potential benefits and risks of ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Source: Sarepta Therapeutics, Inc. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@ 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

Sarepta Therapeutics (SRPT): 'They Should Be Prosecuted,' Says Jim Cramer
Sarepta Therapeutics (SRPT): 'They Should Be Prosecuted,' Says Jim Cramer

Yahoo

time7 days ago

  • Business
  • Yahoo

Sarepta Therapeutics (SRPT): 'They Should Be Prosecuted,' Says Jim Cramer

We recently published . Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is one of the stocks Jim Cramer recently discussed. Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is a biotechnology company that focuses on gene-based therapies. It is one of the most controversial stocks right now due to a massive 40% share price drop in July. Sarepta Therapeutics, Inc. (NASDAQ:SRPT)'s stock was crushed after the FDA demanded that the firm stop shipments of its muscular dystrophy treatment due to patient deaths. Cramer wholeheartedly agreed with the FDA: Photo by National Cancer Institute on Unsplash '[On firm announcing to not comply with the FDA request to stop shipping] Okay, if RFK Jr. agrees with that I guess they don't need to. I mean this is part of the new government. And the new government, some people would say, is not in keeping what keeping the government of Hamilton and Jefferson, the founding fathers. And I would come back and say, they too have become irrelevant right now. They could be relevant later on but I'm not auditioning for the show of Hamilton here. While we acknowledge the potential of SRPT as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an extremely cheap AI stock that is also a major beneficiary of Trump tariffs and onshoring, see our free report on the . READ NEXT: 30 Stocks That Should Double in 3 Years and 11 Hidden AI Stocks to Buy Right Now. Disclosure: None. This article is originally published at Insider Monkey. 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

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