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Here are three noteworthy news developments over the past two days, which contain useful insights about our stocks. For the full year 2023, Novo now expects sales growth in the range of 24% and 30%, compared to prior estimates of 13% to 19%. In addition, operating profit growth is expected to be in the range of 28% and 34%, up from a prior range of 13% to 19%. As a subscriber to the CNBC Investing Club with Jim Cramer, you will receive a trade alert before Jim makes a trade. Jim waits 45 minutes after sending a trade alert before buying or selling a stock in his charitable trust's portfolio.
Deutsche Bank is more bullish on Catalent after the New Jersey-based company expanded its manufacturing partnership with Moderna . Catalent shares were upgraded to buy from hold on Monday, with a price target of $88 per share, Deutsche Bank said. CTLT YTD mountain Catalent received an upgrade from Deutsche Bank on Monday from hold to buy with a price target of $88 per share. Catalent shares were upgraded to buy from hold on Monday, with a price target of $88 per share, Deutsche Bank said. Stock Chart Icon Stock chart icon Catalent received an upgrade from Deutsche Bank on Monday from hold to buy with a price target of $88 per share.
The recent selloff in shares of Sarepta Therapeutics looks "overdone," according to Citi. Shares of Sarepta Therapeutics pulled back about 20% one day last month after regulators said they would hold an advisory committee meeting for SRP-9001, walking back previous plans to assess it without one. Despite these fears, Bitritto-Garg thinks an advisory committee was always in the cards for the treatment, but difficult to obtain as the FDA's Office of Therapeutic Products grappled with a restructuring and lack of resources. SRPT YTD mountain Shares so far this year She added that an advisory committee "may not be indicative of a meaningful change in FDA's position on the filing." Along with the buy rating, Bitritto-Garg slapped a $179 price target on shares, implying 30% upside from Monday's close.
March 20 (Reuters) - The U.S. Food and Drug Administration is moving to optimize the development of gene therapies, including encouraging the use of biomarkers to help get accelerated approval of therapies for serious conditions, an agency official said. The FDA will support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help clinch "accelerated approval", the agency's Peter Marks said on Monday. The U.S. health regulator grants the so-called accelerated approval mainly for drugs and therapies targeted at rare diseases or small patient populations that have had no effective treatments available to them. "The FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases," said Marks, who heads the FDA's Center for Biologics Evaluation and Research, in a statement. The company is seeking approval for its gene therapy under the FDA's accelerated pathway.
[1/2] Sign is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File PhotoMarch 17 (Reuters) - Shares of Sarepta Therapeutics Inc (SRPT.O) slumped as much as 21% on Friday on uncertainty over its gene therapy for Duchenne muscular dystrophy after the U.S. health regulator reversed its decision on a panel meeting. The U.S. Food and Drug Administration planned to hold a meeting of its independent experts to review the gene therapy, the company said late on Thursday, less than a month after saying it would not do so. Sarepta is developing SRP-9001 as the first gene therapy for the treatment of Duchenne muscular dystrophy, an inherited disorder of progressive muscular weakness typically seen in boys. Gene therapies such as BioMarin's (BMRN.O) bleeding disorder therapy have faced setbacks and tough scrutiny in the past, but the FDA approved multiple such products in 2022.
Investors breathed a sigh of relief after the Swiss National Bank said it would provide a liquidity backstop for Credit Suisse. First Republic — The regional bank shares shed over 20% even after the company is set to receive aid from other financial institutions. Credit Suisse — U.S.-listed shares of the Swiss bank fell nearly 11% on Friday, a day after soaring on news the bank will borrow up to 50 billion Swiss francs ($54 billion) from the Swiss National Bank. Warner Bros Discovery — The media company gained 2% after Wells Fargo upgraded the stock to overweight from equal weight. FedEx — The shipping company saw its stock jump over 8% after the company's fiscal third-quarter earnings topped analysts expectations.
A former Twitter exec told i News that Elon Musk was behaving "like the local drunk." "The more Musk behaves like the local drunk – getting into slanging matches with disabled ex-employees – the less current employees will be proud to say they work there," Daisley told i News. Just a week after taking control of Twitter, Musk laid off around half of its workers. "It's a little bit like the character in the cartoon who runs off the cliff but doesn't fall straight away," Daisley told i News. Under Musk, Twitter has become "a pressure cooker," an employee who still works at the company and who spoke on the condition of anonymity told i News.
Elon Musk apologized on Tuesday for mocking a former Twitter employee who has muscular dystrophy. Musk had accused the employee of not doing "actual work" and told him to justify why he should keep his job. Haraldur Thorleifsson, an Iceland-based entrepreneur and former Twitter employee, ended up in a public exchange with CEO Elon Musk. But on Tuesday, Musk publicly trolled Thorleifsson in a series of tweets questioning his value at the company. Musk then said Thorleifsson used his disability as an excuse to work less.
Ex-Twitter manager Esther Crawford tweeted "cruelty is the worst" in an apparent dig at Elon Musk. She was seen as one of Musk's most loyal workers. Musk had publicly clashed with Haraldur Thorleifsson, a former Twitter director, on Monday. After Thorleifsson tweeted that he was unsure of his employment status, noting that he had been locked out of company systems, Musk accused him of using his disability as an "excuse" to do "no actual work." Crawford tweeted on Tuesday that "cruelty is the worst," without providing further context.
Elon Musk accused a former employee of using their disability as an "excuse" to do "no actual work." Musk and Thorleifsson engaged in a heated exchange on Twitter on Monday. The accusation was aimed at Haraldur Thorleifsson, a former Twitter director who engaged in a public spat with Musk on Monday. Thorleifsson, who is based in Reykjavik, Iceland, joined Twitter as a full-time employee in 2021 after Twitter acquired Ueno, a creative agency he founded. Amid his spat with Musk on Monday, Thorleifsson posted a picture of himself in a wheelchair.
New York CNN —Elon Musk publicly scoffed at a Twitter employee’s uncertainty about whether he had been laid off in a recent round of cuts and spoke dismissively of the employee’s disability in a series of tweets Monday night. Haraldur Thorleifsson, an Iceland-based Twitter senior director, tweeted at Musk that access to his computer had been cut off nine days earlier, when Twitter reportedly laid off some 200 employees. It’s not the first time Musk, one of the richest men in the world, has publicly mocked employees at Twitter, the company he bought for $44 billion last year. Hundreds of former Twitter employees are now taking legal action against the company, alleging broken severance promises and, in some cases, discrimination, including against disabled employees. Following the exchange with Musk, Thorleifsson said in a tweet that Twitter’s HR head had confirmed he is no longer employed at the company.
Morgan Stanley thinks that one of Sarepta Therapeutics ' drugs will receive accelerated approval from the Food and Drug Administration — creating a promising investing opportunity. Analyst Matthew Harrison upgraded Sarepta shares to overweight from equal weight, saying that the path for SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy (DMD), now appears "de-risked." Morgan Stanley said that with the accelerated launch of SRP-9001, Sarepta has potential for over $5 billion in peak sales. To be sure, Harrison said potential risks to a downside include updates to the DMD gene therapy program, as well as manufacturing and regulatory risks. Data from competitors could also negatively impact Sarepta's position in the DMD market, the analyst noted.
Revenue for the quarter came in at $663 million, falling well below analysts' estimates of $742 million, according to Refinitiv. Sarepta Therapeutics — The biotech stock soared by 17% after Morgan Stanley upgraded Sarepta shares to overweight from equal weight. Kohl's reported a loss of $2.49 per share on $5.78 billion of revenue. Analysts surveyed by Refinitiv had expected positive earnings of 98 cents per share on $5.99 billion of revenue. Monster reported $1.51 billion in revenue, falling short of analysts' expectations of $1.6 billion.
Rivian also shared an electric vehicle production outlook that fell short of analysts' expectations. First Solar — Shares of First Solar gained 15% after the solar company issued full-year guidance on Tuesday that came out ahead of expectations on per-share earnings and revenue. First Solar reported a fourth-quarter loss of 7 cents per share compared with a 17 cent per-share loss expected by analysts surveyed by FactSet. The company's revenue was in line with analysts' expectations of $1 billion. Ambarella gave first-quarter guidance that missed analysts' expectations on Tuesday, according to FactSet.
Pfizer's hemophilia B gene therapy succeeds in late-stage study
  + stars: | 2022-12-29 | by ( ) www.reuters.com   time to read: +1 min
Dec 29 (Reuters) - U.S. drugmaker Pfizer Inc (PFE.N) said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study. The drugmaker licensed its hemophilia B gene therapy from Roche's (ROG.S) Spark Therapeutics unit in 2014 for a $20-million upfront payment. Pfizer plans to discuss the late-stage data with regulatory authorities in Europe and the United States and share additional data for the experimental therapy at a scientific conference in early 2023. According to government data, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 males for hemophilia A and 3.7 cases per 100,000 males for hemophilia B. In November, the U.S. health regulator approved the first gene therapy, CSL Ltd and uniQure's Hemgenix, to treat hemophilia B.Pfizer is also testing other experimental gene therapies in late-stage trials as potential treatments for the bleeding disorder hemophilia A and muscular disorder Duchenne muscular dystrophy.
It may be time for investors to jump into little-known biotech stock Fulcrum Therapeutics , according to Goldman Sachs. "We believe p38α/β MAPK inhibitor losmapimod has the potential to be a novel first-in-class therapy in FSHD," Kumar said in a note to clients. He has assumed a 40% probability of success and 40% peak market share for the FSHD drug. About 5% of the world's population carries the trait for hemoglobin disorders like sickle cell disease, according to the World Health Organization . Kumar said its FTX-6058 experimental drug for sickle cell disease, which is called SCD in short, has been promising in early testing.
A mosaic of human cells inside Vertex Pharmaceuticals' cell and gene-therapy laboratory. The four-story, 267,000-square-foot space is home to Vertex's cell and gene-therapy unit, with about 375 people now residing in the modern-industrial building. That has led to going after unrelated diseases like sickle cell, diabetes, muscular dystrophy, kidney disease, and pain. For instance, Vertex's approach to sickle cell is led by its CRISPR gene-editing program. Vertex PharmaceuticalsThe next test of Vertex's strategy will be an experimental treatment called exa-cel, its CRISPR gene-editing program for sickle cell.
The welfare funds in question were part of the $86 million Mississippi is given each year by the federal government to lift families out of poverty. The state auditor uncovered $77 million in misspent welfare funds in February 2020. The text messages, which were part of a filing by New's attorney, do not establish that Favre knew the public funds discussed were welfare money. The newly released text messages indicate Bryant, a Republican, was much more involved in the project as governor than previously known. She would pay Favre $1.1 million in state funds directly and he would do a few radio ads.
Washington, DC CNN —Tesla’s price for the feature it calls “full self-driving” has increased again. CNN Business spoke with 13 people who have cars with the “full self-driving” beta and the overwhelming majority, 11 people, said they felt it wasn’t worth $15,000. Moody said he expects consumers to pay for the monthly subscription of “full self-driving” rather than pay $15,000 for the promise of the feature’s future potential. Burke spoke with CNN Business during a multi-month road trip around the United States. Burke said that if he was in the market again for a Tesla, the $15,000 price for “full self-driving” would be worth it to him.
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