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☆ New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle
▼ + stars: | 2024-02-23 | by ( Bertha Coombs | ) www.cnbc.com time to read: +2 min

The approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color. Sickle cell has forced him to leave his job and at times taken him away from his family. Still, he's hesitant to try the new one-time gene therapies because they require months of intensive medical preparation, including chemotherapy, to prepare patients' bone marrow stem cells for extraction and gene editing. Vertex Pharmaceuticals ' gene therapy Casgevy lists for $2.2 million, while Bluebird Bio 's treatment Lyfgenia lists for $3.1 million. Kanter said it will take time to ramp up capacity and to set up facilities across the country to treat patients at scale.

Persons: Michael Goodwin, Goodwin, I've, I'm, he's, …, Goodwin's hesitancy, Julie Kanter, Kanter Organizations: Health, Vertex Pharmaceuticals, University of Alabama, National Alliance of Sickle Cell Centers, for Disease Control, National Alliance of Sickle Cell Locations: Birmingham

☆ US FDA to delay decision on Bristol Myers-2seventy bio cancer therapy
▼ + stars: | 2023-11-20 | by ( ) www.reuters.com time to read: +2 min

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. The U.S. Food and Drug Administration (FDA) has not yet confirmed when the advisory panel would meet, they added. Shares of Bristol Myers Squibb fell more than 2% and those of 2seventy bio slid nearly 20% in early morning trade. The therapy was originally co-developed by bluebird bio (BLUE.O) but following its spin-off in 2021 became part of 2seventy's portfolio. Bristol Myers and 2seventy bio, however, have faced manufacturing constraints, and a month-long planned maintenance shutdown of one of Abecma's manufacturing facility in June had also negatively impacted sales.

Persons: Andrew Kelly, 2seventy bio's, Bristol Myers, Johnson, Biotech's, Khushi, Sriraj Kalluvila Organizations: Food and Drug Administration, FDA, REUTERS, Bristol Myers Squibb, U.S . Food, Drug Administration, Bristol, Johnson, Thomson Locations: White Oak , Maryland, U.S, Bengaluru

☆ Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval
▼ + stars: | 2023-11-16 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Regulators in Britain on Thursday approved the first treatment derived from CRISPR, the revolutionary gene-editing method. Called Casgevy, the treatment is intended to cure sickle-cell disease and a related condition, beta thalassemia. The companies anticipate that the Food and Drug Administration will approve Casgevy for sickle-cell patients in the United States in early December. The agency will decide on approval for beta thalassemia next year. That treatment does not rely on gene editing, insteading using a method that inserts new DNA into the genome.

Organizations: Vertex Pharmaceuticals, CRISPR Therapeutics, and Drug, Bluebird Bio Locations: Britain, Boston, Switzerland, United States, Somerville, Mass

☆ These 4 stocks report quarterly results next week. Here's what we hope to see
▼ + stars: | 2023-11-04 | by ( Zev Fima | ) www.cnbc.com time to read: +11 min

The major averages rebounded strongly this week as quarterly earnings came in largely better than expected and interest rates fell on softer economic data. The Dow Jones Industrial Average rose more than 5% for its best week since October 2022. The big theme next week is earnings and the four portfolio companies delivering their quarterly results. Here's the full rundown of all the important domestic data in the week ahead as we consider a number areas of our portfolio for trades next week. ET: Treasury budget statement for October (See here for a full list of the stocks in Jim Cramer's Charitable Trust.)

Persons: FactSet, we'll, Hulu —, Krystal, Horton, Zimmer, LEV, Armour, Ralph Lauren Corporation, CRON, Patterson, Walt Disney, MARA, Ginkgo, FREYR Battery, FREY, Arcelor Mittal, Wynn, Jim Cramer's, Jim Cramer, Jim Organizations: Dow Jones, Nasdaq, ISM, Services, Coterra Energy, Emerson Electric, Wall, National Instruments, Disney, Hulu, CNBC, Comcast, ESPN —, Wynn Resorts, WYNN, Asia that's, Wynn, Wynn's, Wynn Interactive, United, Brookfield Asset Management, Axsome Therapeutics, Network Corporation, Krystal Biotech, Ceragon Networks, Kosmos Energy Ltd, TreeHouse Foods Inc, Air Lease Corporation, Inc, Eversource Energy, Fennec Pharmaceuticals Inc, Graham Corporation, Aereas Inteligentes, Hilton, Energy, Realty Income Corp, Semiconductors, Vertex Pharmaceuticals Inc, Clover Health, Contura Energy, Diamondback Energy, FS KKR Capital Corp, Celanese Corp, Eos Energy Enterprises, Goodyear Tire & Rubber Company, WEN, Pall Corp, TransMedics, Emerson, Uber Technologies, Holdings, Melco Resorts, Entertainment, Zimmer Biomet Holdings, ACM Research, Lion Electric Company, Emerson Electric Co, Vertex Energy, Waters Corp, Air Products & Chemicals, Esperion Therapeutics, Fidelity National Information Services Inc, Bowlero Corp, Hotels, KKR, Co, Devon Energy Corp, Occidental Petroleum Corp, Dutch Bros Inc, Gilead Sciences, ContextLogic Inc, Akamai Technologies, Viatris Inc, Technologies, Corsair, Enovix Corporation, eBay, EBAY, DaVita Inc, Pan American Silver Corp, Roblox Corporation, Fisker Inc, Warner Bros ., Nuvei Corporation, Farms, Akebia Therapeutics, Biogen Inc, 3D Systems Corporation, Gray Television Inc, BlackSky Technology Inc, Cronos, EVgo Inc, Lumentum Holdings Inc, Nexstar Media Group, UTI Energy, Starwood Property Trust, AMC Entertainment Holdings, Marathon Digital Holdings, IonQ Inc, Digital Turbine, Virgin Galactic Holdings , Inc, Arm Holdings plc, ARM, Ginkgo Bioworks Holdings, Suncor Energy, AppLovin Corporation, B2Gold Corp, Luminar Technologies, MGM Resorts International, MGM, Bloom Energy Corporation, Li Auto Inc, Oatly, Kopin Corp, Fiverr International, YETI Holdings, YPF S.A, Ship Lease, Himax Technologies Inc, Dickinson, CommScope Holding, RBC, Agile Therapeutics, Apyx Medical Corporation, Grab Holdings, Hawaiian Electric Industries, Profire Energy, Flags Inc, Stereotaxis Inc, TransDigm, US Foods Holding Corp, Utz Brands, Valvoline Inc, Weibo Corporation, Wix.com Ltd, Trade, Petroleo Brasileiro SA Petrobras, Growth, Aurora Cannabis Inc, Navitas Semiconductor Corp, Archer Aviation Inc, Semiconductor, Energy Fuels Corp, Illumina Inc, Spike Investment Corp, Algonquin Power & Utilities Corp, AngloGold, Central Puerto S.A, Hudson Global, AirSculpt Technologies, Partners, Soho House, Jim Cramer's Charitable, Handout, Getty Locations: U.S, Macao, Asia, China, Las, Singapore, Wynn's Vegas, Boston, United Arab Emirates, Gilead, VVV, Central, Soho

☆ A New Sickle Cell Treatment Will Change Lives — but How Many? - The New York Times
▼ + stars: | 2023-10-30 | by ( Daniela J. Lamas | ) www.nytimes.com time to read: +3 min

And though comprehensive sickle cell care — at dedicated centers with expert hematologists, social workers and pain management specialists — reduces hospitalizations, and is the standard for diseases like cystic fibrosis and hemophilia, which do not disproportionately affect Black people, these centers are few and far between for sickle cell. Into this complicated landscape enters the possibility of gene therapy. It’s important to note that this isn’t the first cure for sickle cell. is expected to review another gene therapy from the company Bluebird Bio that targets sickle cell disease but does not use CRISPR; this was the therapy Mr. Holmes received as part of the N.I.H. When she was 17 and hospitalized, facing the reality of her chronic illness, she told her mother that she was ready to pursue gene therapy.

Persons: Holmes, Elizabeth Ford, Ford Organizations: Vertex Pharmaceuticals, CRISPR Therapeutics, Bluebird

☆ F.D.A. Experts Will Vote on Safety of a Cure for Sickle Cell Disease
▼ + stars: | 2023-10-30 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +4 min

An estimated 100,000 people in the United States have sickle cell disease, most of whom have African ancestry. will decide on another application for sickle cell gene therapy made by Bluebird Bio. Two other companies and an academic center, Boston Children’s Hospital, are testing their own sickle cell gene therapies. While these therapies could reduce the suffering of sickle cell patients in the United States and other wealthy countries, there is an even greater need for them in some developing countries like Nigeria. One company, Beam, is testing a way to provide gene editing that requires nothing more than a single infusion in a doctor’s office.

Persons: “, Mariah Jacqueline Scott, Scott, ”, Stephan Grupp, What’s Organizations: Institute for Clinical, CRISPR Therapeutics, Children’s Hospital of Philadelphia, Bluebird, Boston Children’s Hospital Locations: United States, Highland Park, N.J, Boston, Nigeria

☆ 2seventy bio to lay off 40% of workforce; CEO to step down
▼ + stars: | 2023-09-12 | by ( ) www.reuters.com time to read: +2 min

Nick Leschly, CEO of bluebird, speaks at the opening of Lonza Houston, the world’s largest dedicated cell and gene therapy facility, in Houston, Texas, U.S., April 10, 2018. REUTERS/Daniel Kramer Acquire Licensing RightsSept 12 (Reuters) - 2seventy bio (TSVT.O) said on Tuesday it plans to lay off about 40% of its workforce to lower costs and focus on the biotech firm's cancer cell therapy Abecma, lifting the company's shares more than 8% in premarket trade. The company added that CEO Nick Leschly will step down and transition to the role of chairman. Leschly was earlier the CEO of bluebird bio (BLUE.O) for 11 years, but changed role after the gene therapy maker's oncology portfolio was spun off from it as 2seventy. The company will now focus on another type of multiple myeloma patients, for which it expects approval from the U.S. Food and Drug Administration in mid-December.

Persons: Nick Leschly, Lonza, Daniel Kramer, Leschly, 2seventy, Abecma, Johnson, Bristol Myers, William Baird, Pratik Jain, Shounak Dasgupta, Maju Samuel Organizations: bluebird, REUTERS, Johnson, Legend Biotech, Bristol, Bristol Myers Squibb, U.S . Food, Drug Administration, Thomson Locations: Lonza Houston, Houston , Texas, U.S, Abecma, Bengaluru

☆ Here are Wednesday's biggest analyst calls: Apple, AT&T, Tesla, Amazon, Meta, Microsoft, Spotify and more
▼ + stars: | 2023-07-19 | by ( Michael Bloom | ) www.cnbc.com time to read: +7 min

JPMorgan adds Qualcomm and HP Inc. to the focus list JPMorgan added HP Inc. to the focus list and said it's a top value idea. The firm also added Qualcomm to the focus list and says it's a top growth idea. JPMorgan adds Charles Schwab to the focus list JPMorgan added the stock to its focus list after its earnings report and said it sees "improving fundamentals." Bank of America reiterates Amazon as buy Bank of America said it's bullish heading into Amazon earnings later this month. Bank of America reiterates Block as buy Bank of America said shares of the company formerly known as Square are undervalued.

Persons: Tesla, it's, Charles Schwab, Schwab, Jefferies, Berkshire Hathaway, Archer, Guggenheim, ServiceNow Organizations: JPMorgan, Qualcomm, HP Inc, Watch, Cisco, " Bank of America, of America, Bank of America, Meta, UBS, Berkshire, Citi, Republic Services, Hollywood, Argus, Street, Aviation, UW, DuPont, Texas, Microsoft, Apple Locations: 2023E, 2025E, Berkshire, ACHR, EE

Against this backdrop, CNBC Pro used FactSet data to screen for stocks most vulnerable to a short squeeze. A short squeeze occurs once a shorted stock climbs higher, forcing investors who have short bets on shares to cover their positions. Novavax , meanwhile, has about 42% short interest from investors. Overall, short interest in the name increased 8.7% between June 15 and June 30. See below for the full rundown of potential short squeeze targets.

Persons: there's, Novavax Organizations: Nasdaq, Dow Jones, JPMorgan, CNBC Pro, New York Stock Exchange, Sunnova Energy Locations: Canada

☆ Stocks making the biggest moves midday: Dollar General, Salesforce, C3.ai, Chewy and more
▼ + stars: | 2023-06-01 | by ( Michelle Fox | ) www.cnbc.com time to read: +4 min

Dollar General — Shares sank nearly 20% after the company reported an earnings and revenue miss for the first quarter. Its second-quarter revenue guidance also beat expectations, per StreetAccount. The company also reduced its full-year revenue guidance in the low single-digits range from the prior midsingle-digit range estimates. The drop in shares came as cost concerns and dwindling demand for consulting deals overshadowed better-than-expected results and an improved full-year earnings outlook. The company also raised its full-year earnings per share guidance.

Persons: Chewy, Hormel, Wednesday's, CrowdStrike, Salesforce, Okta, JPMorgan Chase, Veeva, Samantha Subin, Yun Li, Alex Harring, Tanaya Macheel Organizations: StreetAccount, Revenue, Refinitiv, JPMorgan, Veeva Systems, Saudi Arabia's Public Investment Fund, Barclays Locations: Vallejo , California

☆ Here are Thursday's biggest analyst calls: Target, Tesla, Toast, Chevron, Meta, Toll Brothers and more
▼ + stars: | 2023-06-01 | by ( Michael Bloom | ) www.cnbc.com time to read: +8 min

JPMorgan downgrades Okta to neutral from overweight JPMorgan Chase said it sees too many macroeconomic pressures for the company. " JPMorgan downgrades Target to neutral from overweight JPMorgan Chase said in its downgrade of the stock it sees a "weakening" consumer. Bank of America initiates Toast as buy Bank of America said the restaurant tech company is "best in class." JPMorgan upgrades Domino's Pizza to overweight from neutral JPMorgan Chase said in its upgrade of Domino's that it's too cheap to ignore. Bank of America reiterates Dick's as buy Bank of America said it's bullish on the company's new store format.

Persons: Goldman Sachs, Goldman, Johnson, it's, Salesforce, JPMorgan downgrades, JPMorgan Chase, we've, Horton, PulteGroup, TOST, BorgWarner, Morgan Stanley, Tesla, XOM, Cowen, Phillips, Wedbush, Macquarie, Dick's, it's bullish, DKS Organizations: AAP, Barclays, JCI, RBC, Chevron, JPMorgan, JPMorgan downgrades Target, Deutsche Bank, " Bank of America, Bank of America, Restaurant Association, BorgWarner Deutsche Bank, Citi, Meta, ExxonMobil, UBS, CSX, Garden Entertainment, Knicks, Rangers, Networks Locations: Europe, New York

☆ Here are Friday's biggest analyst calls: Amazon, Apple, Tesla, Royal Caribbean, Intel, Hasbro & more
▼ + stars: | 2023-04-28 | by ( Michael Bloom | ) www.cnbc.com time to read: +5 min

Here are Friday's biggest calls on Wall Street: Bank of America upgrades Hasbro to neutral from underperform Bank of America said it sees resilient demand for Hasbro . Goldman Sachs reiterates Amazon as buy Goldman said it's standing by the stock after its robust earnings report Thursday. " Benchmark upgrades Intel to buy from hold Benchmark said in its upgrade of Intel that the worst is already baked in. Bank of America upgrades Pilgrim's Pride to buy from neutral Bank of America said in its upgrade of the poultry company that the worst is behind it. Morgan Stanley reiterates Apple as overweight Morgan Stanley said it's still bullish on Apple shares heading into earnings next week.

Short interest in Novavax increased more than 13% to 37.8 million shares, or about 45% of the total float, between March 15-31, according to data compiled by FactSet. Other biotech companies that saw growing short interest are Cassava Sciences , with a 10% increase, and Fate Therapeutics , which saw a 26% boost. Meanwhile, bluebird bio had a 29% increase in its short interest to 26% of float. Cinemark and AMC Entertainment both saw increases in their short position to 28% and 27% of their respective floats. All names trade on the NYSE or Nasdaq, have short positions amounting to at least 25% of their total float and have market values of at least $100 million.

☆ Shares of this under-the-radar cell therapy stock are set to double, says Baird
▼ + stars: | 2023-03-07 | by ( Michelle Fox | ) www.cnbc.com time to read: +2 min

Shares of biotech company bluebird bio should double in the coming year, thanks to its treatments for sickle cell disease, a genetic blood disease and a rare genetic brain condition, according to Baird. The Wall Street firm initiated coverage of the stock on Monday with an outperform rating and $10 price target, which implies 104% upside from Monday's close. The company's management said in January that 40 patients had initiated benefits verification for Zynteglo during the last three months of 2022. Lastly, Baird expects the use of bluebird's Skysona treatment for CALD, which was approved in September 2022, to increase significantly in the coming years. The under-the-radar stock, which has a market cap of over $500 million, is down about 29% this year, as of Monday's close.

☆ Here are Tuesday's biggest analyst calls: Apple, Amazon, Arista, Costco, Joby, Netflix & more
▼ + stars: | 2023-03-07 | by ( Michael Bloom | ) www.cnbc.com time to read: +6 min

Here are Tuesday's biggest calls on Wall Street: Northcoast upgrades Costco to buy from hold Northcoast said it sees more upside for the wholesale retailer. " Goldman Sachs initiates Arista Networks and Juniper as buy Goldman initiated several networking equipment stocks, noting they are attractively valued. Deutsche Bank downgrades Joby to sell from hold Deutsche said it sees too many risks for the aviation company. Morgan Stanley reiterates Walmart as overweight Morgan Stanley said its standing by shares of Walmart as the retail giant continues to sign up new members for its Walmart+. Loop reiterates Netflix as hold Loop said its survey checks show that password sharing charging is increasing revenue for Netflix.

☆ Drug Prices Reach New High—in the Millions
▼ + stars: | 2022-12-26 | by ( Peter Loftus | ) www.wsj.com time to read: 1 min

The most recent gene therapy approved in the U.S. set a price record: $3.5 million for CSL’s Hemgenix, a treatment for hemophilia B. A new era of expensive drugs has arrived: medicines priced in the millions of dollars a patient. Since August, U.S. or European health regulators have approved four new products intended as one-time treatments for rare genetic diseases that carry list prices of at least $2 million a patient, including two from Bluebird Bio Inc.

☆ The most expensive drug in the world has a $3.5 million price tag. 2 potential patients share their hopes and fears for the one-time treatment.
▼ + stars: | 2022-12-05 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +7 min

The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. The drugmaker CSL Behring set a $3.5 million price for the one-time treatment. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. But a critical unknown in judging CSL's $3.5 million price is the durability of the treatment. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.

☆ The most expensive drug in the world just got approved at $3.5 million, and hemophilia patients are conflicted: 'Compared to what we pay, it doesn't seem too terrible.'
▼ + stars: | 2022-12-01 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +7 min

The FDA has approved a new gene therapy to treat hemophilia B, a genetic bleeding disorder. Hemophilia patients told Insider they're excited about the new drug but worried about the price. Lackey is learning to live with a severe case of hemophilia B, a rare genetic condition that keeps blood from clotting properly. While the treatment breaks ground as the first approved gene therapy for hemophilia, its price is also unprecedented. Lojewski said the $3.5 million price wasn't guided by other gene-therapy prices.

☆ Regulators have approved the world's most expensive medicine that costs $3.5 million per patient
▼ + stars: | 2022-11-24 | by ( Ryan Hogg | ) www.businessinsider.com time to read: +3 min

The FDA approved a treatment costing $3.5 million, making it the world's most expensive medicine. Hemgenix effectively treated several patients with the blood condition Hemophilia B in trials. An independent study said a fair price for the drug would be about $2.9 million. download the app Email address By clicking ‘Sign up’, you agree to receive marketing emails from Insider as well as other partner offers and accept our Terms of Service and Privacy PolicyUS regulators have approved a hemophilia drug that will cost $3.5 million per patient, making it the world's most expensive medicine. However, the drug will have a list price of $3.5 million per dose, Managed Healthcare Executive reported, making it the world's most expensive medicine by some distance.

Nov 22 (Reuters) - Australian drugmaker CSL Ltd (CSL.AX) on Tuesday set the list price of its one-time gene therapy for hemophilia B at $3.5 million, making it the world's most expensive treatment, following its approval by the U.S. health regulator. The first gene therapy for the rare genetic blood clotting disorder offers a long-term solution for patients as against current treatments from Biogen (BIIB.O), Pfizer (PFE.N) and others that focus on regular infusions. The gene therapy consists of an engineered virus carrying a gene expressed in the liver to produce clotting factor IX. While Zynteglo was priced at $2.8 million, Skysona had a wholesale cost of $3.0 million. CSL shares were up 1% at A$300.62 on the Australian stock exchange, while UniQure stock edged up 0.9% in extended trading.

☆ Stocks making the biggest moves in the premarket: AutoZone, Take-Two Interactive, bluebird bio and more
▼ + stars: | 2022-09-19 | by ( Peter Schacknow | ) www.cnbc.com time to read: +2 min

Take-Two Interactive (TTWO) – Take-Two shares slid 5.8% in the premarket after Bloomberg reported that a hacker released gameplay from its upcoming Grand Theft Auto IV game online. bluebird bio (BLUE) – bluebird bio rallied 7.3% in premarket trading after the Food and Drug Administration approved the company's gene therapy for a rare and lethal brain disease in children. Wix (WIX) – Wix shares jumped 4.5% in premarket action after activist investor Starboard Value revealed a 9% stake in the web development platform company. NCR (NCR) – NCR slid another 1.3% in the premarket on top of a 20.3% plunge Friday after Morgan Stanley downgraded the stock to "equal-weight" from "overweight." Adobe fell 1.3% in premarket trading after falling 16.8% last Thursday and another 3.1% on Friday.

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