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Many biotech stocks struggled in 2023 despite a robust year for U.S. drug approvals. "We expect this environment to continue but look forward to the prospect of lower interest rates in 2024." When that happens, Canaccord Genuity said to expect a "strong rally across the biotech sector rewarding innovative, but riskier assets." Analysts have said the rich price reflects the opportunity for ImmunoGen's Elahere cancer treatment , which has quickly established itself as the standard of care for types of ovarian cancer. 'Oversold and cheap' In a research note Friday, Jefferies analyst Michael Yee said "a significant short squeeze" was helping biotech stocks in the fourth quarter.
Persons: Dan Lyons, Janus Henderson, John Newman, Canaccord Genuity, Janus, Lyons, Bristol Myers Squibb, Karuna, AstraZeneca, Eli Lilly, AbbVie, Jonathan Miller, Miller, bode, Jonathan Krinsky, Krinsky, BTIG's, Jefferies, Michael Yee, Yee, NASH, Madrigal, we're, David Risinger, — CNBC's Michael Bloom Organizations: Drug, Federal, Bristol Myers, Karuna Therapeutics, CART, Bristol Myers Squibb, Cerevel Therapeutics, Securities and Exchange Commission, pharma, P Biotech, Nasdaq Biotech, Apogee Therapeutics, Madrigal Pharmaceuticals, Madrigal, resmetirom, Vertex Pharmaceuticals, Leerink Partners Locations: ImmunoGen
CNN —The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool. The treatment, made by Vertex Pharmaceuticals, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a lab. “Modifying the stem cells from the bone marrow of the patient avoids the problems associated with immune compatibility, i.e. The release from the MHRA did not say how much the treatment would cost, but it’s likely to be expensive. CRISPR-Cas9 has had a major impact on biomedical research, clinical medicine and agriculture and is widely used in labs around the world.
Persons: , , Julian Beach, , — Emmanuelle Charpentier, Jennifer A, Doudna —, Casgevy, Alena Pance, ” Pance, Cas9, Jiankui Organizations: CNN, country’s Medicines, Healthcare, Agency, Beta, Vertex Pharmaceuticals, US Food and Drug Administration, University of Hertfordshire, Science Media Centre Locations: United Kingdom, South Asian
LONDON (AP) — Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. Casgevy is currently being reviewed by the U.S. Food and Drug Administration; the agency is expected to make a decision early next month, before considering another sickle cell gene therapy. Millions of people around the world, including about 100,000 in the U.S., have sickle cell disease. Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria.
Persons: , Helen O'Neill Organizations: Medicines and Healthcare Regulatory Agency, Vertex Pharmaceuticals, CRISPR Therapeutics, Helen O'Neill of University College London, Medicines, U.S . Food, Drug Administration, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: Europe, CRISPR, South Asian, Britain, U.S, Africa, India, Eastern
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
Biden’s Race Against a Cure
  + stars: | 2023-11-13 | by ( The Editorial Board | ) www.wsj.com   time to read: 1 min
Wonder Land: Citing the president’s age lets Democrats off the hook for the political failure of his economic policies. Images: AP/AFP/EPA/Getty Images Composite: Mark KellyThe Food and Drug Administration may soon approve two gene therapies with the potential to cure more than 100,000 Americans with debilitating sickle-cell disease. Gene therapies fix missing or defective genes. The one-time treatments have the potential to cure inherited disorders with early death sentences. The FDA has approved nine gene therapies for such diseases as Duchenne muscular dystrophy, spinal muscular atrophy and retinal dystrophy.
Persons: Mark Kelly The Organizations: AFP, EPA, Drug Administration, Medicare, Medicaid Services, FDA
The company's research and development (R&D) engine is intact and therefore it is unlikely to do transformative deals soon, Jorgensen said. It was the latest in a series of similarly-sized deals the company has done this year. Last week, when reporting quarterly earnings, it forecast another year of double-digit sales growth for its two most popular drugs: weight-loss drug Wegovy and diabetes Ozempic. To keep up with demand, Novo is racing to increase production of those drugs, which contain the same active pharmaceutical ingredient, semaglutide, and are delivered in self-injection pens. A healthcare banker, speaking on condition of anonymity, told Reuters after the interview there were many companies in the cardiometabolic space with early-stage drugs that Novo Nordisk may be able to buy.
Persons: Lars Fruergaard Jorgensen, Wegovy, Jorgensen, Novo, Novo's, Jacob Gronholt, Maggie Fick, Terje Solsvik, Emelia, Barbara Lewis Organizations: Novo, Novo Nordisk, Reuters, KBP Biosciences, Nordisk, Pedersen, Thomson Locations: Novo, COPENHAGEN, Singapore, Copenhagen, London
As the company's fortunes soar on demand for its popular weight-loss medicine Wegovy, Jorgensen said Novo sought to acquire companies working on medicines in the areas where it is already focused. The company's research and development (R&D) engine is intact and therefore it is unlikely to do transformative deals soon, Jorgensen said. It was the latest in a series of similarly-sized deals the company has done this year. Last week, when reporting quarterly earnings, it forecast another year of double-digit sales growth for its two most popular drugs: weight-loss drug Wegovy and diabetes Ozempic. To keep up with demand, Novo is racing to increase production of those drugs, which contain the same active pharmaceutical ingredient, semaglutide, and are delivered in self-injection pens.
Persons: Lars Fruergaard Jorgensen, Wegovy, Jorgensen, Novo, Novo's, Jacob Gronholt, Maggie Fick, Terje Solsvik, Emelia, Barbara Lewis Organizations: Novo, Novo Nordisk, Reuters, KBP Biosciences, Nordisk, Pedersen, Thomson Locations: Novo, COPENHAGEN, Singapore, Copenhagen, London
Family members are affected too — they may need to take time off work during the most intensive phase of the treatment. Additionally, most Americans with sickle cell are Black and may not trust a health care system that has often failed to provide the most basic preventive and therapeutic care for those with the disease. Some with sickle cell are anxious about undergoing a medical treatment that is on the cutting edge of biotechnology. “We are finally at a spot where we can envision broadly available cures for sickle cell disease,” said Dr. John Tisdale, director of the cellular and molecular therapeutics branch at the National Heart, Lung and Blood Institute and a member of the advisory committee. Kyra is now in intensive care as doctors try to control her pain.
Persons: It’s, , John Tisdale, Dana Jones, San Antonio, Kyra Organizations: Blood Institute
[1/2] A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston, Massachusetts, U.S., October 23, 2019. REUTERS/Brian Snyder/File Photo Acquire Licensing RightsOct 31 (Reuters) - A panel of advisers to the U.S. health regulator said on Tuesday Vertex Pharmaceuticals (VRTX.O) and CRISPR Therapeutics (CRSP.BN) could assess potential safety risks of their sickle cell disease gene therapy after approval. If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy. Patients who were treated with the therapy were free from severe vaso-occlusive crisis for 12 months from the infusion of exa-cel. "It was made pretty clear that any theoretical off-target editing concerns do not outweigh the benefits of drug approval," said Salim Syed, analyst at Mizuho.
Persons: Brian Snyder, Scot Wolfe, Salim Syed, Sriparna Roy, Krishna Chandra Organizations: Vertex Pharmaceuticals, REUTERS, CRISPR Therapeutics, Staff, U.S . Food, Drug Administration, UMass Chan Medical, Analysts, FDA, Mizuho, Thomson Locations: Boston , Massachusetts, U.S, Bengaluru
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
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
Pien, Poland Reuters —Archaeologists in Poland have uncovered the remains of a 17th-century child padlocked to his grave to stop him rising from the dead, a discovery that turns the spotlight on beliefs in vampires as Halloween approaches. A woman’s body was also found in the cemetery with a padlock on her leg and a sickle around the neck, suggesting she was believed to be a vampire. “These are people who, if it was done intentionally, were afraid of … contact with these people because they might bite, drink blood,” Polinski said. The child’s grave was desecrated at some point after burial and all bones removed apart from those in the legs. Archaeologists have found other methods used to stop the living dead, with Polinski describing strange practices found in some burials.
Persons: padlocked, , , Dariusz Polinski, Nicolaus, Nicolas, ” Polinski Organizations: Poland Reuters — Archaeologists, Nicolaus Copernicus University, Nicolas Copernicus University Institute of Archaeology Locations: Pien, Poland, Polish, Toruń
CNN —For the past year and a half, medical cannabis company CEO Gary Long has spent a lot of his time reassuring Georgia’s small-town mayors about what will soon be coming to their local pharmacy: medical marijuana. The Georgia Board of Pharmacy is currently processing applications from pharmacies around the state that want to sell low-dose THC products. National chains such as CVS and Walmart won’t be selling THC products in Georgia, but Long said 130 local pharmacies have already agreed to sell his product exclusively. Making medical cannabis so accessible in such a traditionally conservative Deep South state like Georgia caught some by surprise. Californians have been able to buy medical cannabis since 1996 and adults there got the right to use it recreationally in 2016.
Persons: Gary Long, that’s, , Long, Coke, Andrew Turnage, Jimmy Kimmel, “ I’ve, ” Long, Jonathan Marquess, ” Marquess, it’s, Jay Wexler, , Brian Kemp, Marquess ’, Marquess, there’s, ” Turnage, Aaron Smith, ” Smith, Dr, Sanjay Gupta, Turnage, I’ve Organizations: CNN, Botanical Sciences, Georgia, of Pharmacy, CVS, Walmart, Medical Cannabis Commission, Georgia General Assembly, US Drug Enforcement, Boston University School of Law, Justice, Gov, Pharmacy, Georgia Pharmacy Association’s Academy of Independent Pharmacy, National Cannabis Industry Association, Get CNN, CNN Health Locations: Georgia, United States, Connecticut, Massachusetts
Companies Beam Therapeutics Inc FollowOct 19 (Reuters) - Beam Therapeutics (BEAM.O) said on Thursday it will focus on developing its experimental sickle cell disease treatments and reduce about 20% of its current workforce, or 100 employees, as part of a restructuring plan. The gene-editing specialist expects to incur one-time expenses of about $6.6 million related to the workforce reduction, which it plans to complete in the fourth quarter. Its experimental sickle cell disease treatments include early-stage candidate BEAM-101, which activates fetal hemoglobin, the dominant form of hemoglobin present in the fetus during gestation. Beam will pause the development of its hepatitis B virus candidate, currently being evaluated in lab studies, and explore partnership opportunities for it. Reporting by Bhanvi Satija and Christy Santhosh in Bengaluru; Editing by Shilpi MajumdarOur Standards: The Thomson Reuters Trust Principles.
Persons: Beam, David Liu, Feng Zhang, J Keith Joung, Bhanvi Satija, Christy Santhosh, Shilpi Majumdar Organizations: Beam Therapeutics, U.S . Food, Drug Administration, Thomson Locations: Cambridge , Massachusetts, Bengaluru
If the body uses up its stores, iron deficiency can lead to a reduction in hemoglobin and the number of healthy red blood cells, called anemia. So someone with normal hemoglobin levels might still have low levels of iron, Dr. Munro said. There are other forms of anemia, including inherited red blood cell disorders like sickle cell disease, but anemia caused by iron deficiency is the most common type in the U.S. During pregnancy, when the demand for iron in the mother, the placenta and the growing fetus increases, so too does the risk that iron deficiency turns into anemia. Complicating matters, there is debate among medical institutions about what is considered a healthy amount of iron in the blood.
Persons: Munro, Angela Weyand, Michael Georgieff, Weyand Organizations: U.S, University of Michigan Medical, Masonic Institute, University of Minnesota, World Health Organization
mRNA vaccine: 5 things to know
  + stars: | 2023-10-02 | by ( Katie Hunt | ) edition.cnn.com   time to read: +5 min
Here are five things to know about Karikó and Weissman’s game-changing research and mRNA vaccines. What mRNA doesMessenger RNA, or mRNA, is a form of nucleic acid that tells cells what to do based on the information contained in DNA. Messenger RNA-based vaccine technology doesn’t rely on a modified version of a virus to produce an immune response. Potential beyond fighting Covid-19The advent of mRNA vaccine technology has led to safe and strong protection against Covid-19. And mRNA technology is also being checked out as a possible alternative to gene therapy for intractable conditions such as sickle cell disease.
Persons: Katalin Karikó, Drew Weissman, Peggy Peterson, Robin Shattock, ” Shattock, , Karikó, Weissman, , Roberts, Thomas Perlmann, it’s Organizations: CNN, University of Pennsylvania, Penn Medicine, Imperial College London, Covid, Penn’s Perelman, of Medicine, Vaccine, Perelman School of Medicine, Nobel Assembly Locations: Hungary
From mobile banking and online bill pay to direct deposit and digital payment apps, the waves of cashless options seem to swell by the day leaving a steep learning curve in their wake. Digital payment apps are the new norm for teensI talked to Ruben Salazar Genovez, global head of Visa Direct, about the survey. According to the survey, digital payment apps like Venmo, PayPal, and Zelle are the second most popular form of payment, trailing behind cash, for transactions between parents and teens. As digital payment apps gain steam, that means financial literacy is more important than ever. Payment apps are creating new avenues for fraudEmerging technologies have given rise to new forms of fraud, and digital payment apps are no exception.
Persons: Xer, Ruben Salazar Genovez, I've, Genovez, it's Organizations: Visa, PayPal, Monopoly, Football, Banking
Max Gomez, an award-winning medical and science journalist who delivered informed reports for more than 40 years on TV stations in New York and Philadelphia, most recently during the Covid-19 pandemic, died on Sept. 2 at his home in Manhattan. His partner, Amy Levin, said the cause was head and neck cancer, with which he had been diagnosed four years ago. One of his reports on Alzheimer’s disease focused on his father, a physician, who was swindled as his memory abandoned him. Dr. Gomez had been chief medical correspondent at WCBS, Channel 2, in New York City since 2007 and made his last appearance there in March 2022. He also worked at WNBC, Channel 4, and WNEW, Channel 5 (now WNYW), as well as KYW, Channel 3, in Philadelphia.
Persons: Max Gomez, Amy Levin, Dr, Max, , Gomez Organizations: WCBS, WNBC, WNEW, KYW Locations: New York, Philadelphia, Manhattan, New York City
But in recent years, DNA analysis has helped scientists uncover the tale behind the "vampire's" remains. An annotated image shows the placement of the "Connecticut vampire" skull and bones in the grave. The "Connecticut vampire" likely died of tuberculosisFor decades, the "Connecticut vampire" was known only as "JB55," after the initial "JB" that had been carved into the brass tacks used to close the coffin. A 3D-scan of Barber's skull was combined with DNA analysis to estimate John Barber's features, shown here in an artist's illustration. Not everybody agrees, however, that this burial site qualifies as a bona fide "vampire" burial site.
Persons: John Barber, , Jolly Roger, I'd, Nick Bellantoni, he'd, Michael Ross, Daniels, Ellen Greytak, John Baker, Baker, It's, Parabon NanoLabs, John Barber's, Parabon, Barber's, sickles, Scott, Matteo Borrini Organizations: Service, Smithsonian, Smithsonian Magazine, Armed Forces DNA, Inc, Liverpool John Moores University Locations: Connecticut, Wall, Silicon, Griswold , Connecticut, Virginia, New England
Researchers have unearthed the skeletal remains of a "vampire child" in a Polish graveyard. The child was buried face down with a triangular padlock on its foot. The skeletal remains of the child, who anthropologists believe was 5 to 7 years old, were discovered in an unmarked, mass cemetery in the Polish village of Pień, near Ostromecko. Triangular padlocks were attached to people’s feet to keep them tethered to the ground once buried, Poliński said. Courtesy of Dariusz PolińskiThere are several reasons a person may have been buried in such a cemetery, Poliński said.
Persons: Dariusz Poliński, Nicolaus, Poliński, Dariusz, Dariusz Poliński Matteo Borrini, Insider's Katherine Tangalakis, Marianne Guenot, Borrini Organizations: Christian Europe, Service, Privacy, Nicolaus Copernicus University, Liverpool John Moore University Locations: Wall, Silicon, Polish, Pień, Ostromecko
Share Share Article via Facebook Share Article via Twitter Share Article via LinkedIn Share Article via EmailUkraine removes Soviet symbols from Motherland Monument in KyivOn the largest monument in Ukraine's capital city of Kyiv, a Soviet-era hammer and sickle has been replaced with a Ukrainian coat of arms.
Organizations: Ukraine Locations: Kyiv, Ukraine's, Soviet, Ukrainian
Ukraine has replaced the Soviet emblem on one of Kyiv’s most prominent monuments with its own coat of arms, part of a broader push since the full-scale invasion to stamp out tributes to Russian power. The Motherland Monument — a 335-foot-tall stainless steel behemoth towering over Kyiv, Ukraine’s capital — was designed to assert Soviet invincibility. Unveiled in 1981, the monument is a figure of a woman raising a sword in her right hand and a shield in her left. The shield was emblazoned with the Soviet hammer and sickle.
Locations: Ukraine, Kyiv, Ukraine’s, , Soviet
Kyiv last week replaced the Soviet hammer and sickle symbol with a trident – the Ukrainian coat of arms – on the shield of the Motherland Monument, which dominates the capital’s skyline. Its construction began in 1979, and it depicted a woman holding a sword and a shield emblazoned with the Soviet hammer and sickle symbol. Workers remove the Soviet-era emblem from the motherland monument in Kyiv on August 1, 2023. And they don’t know how.”Putin’s desire to undermine Ukrainian national identity and autonomy has remained a key motivation for the conflict. Kudrya, meanwhile, was a Soviet spy and a leader of a sabotage group in Kyiv during World War II.
Persons: Vladimir Putin’s, Valentyn Ogirenko, Pilipey, ” “, , Maria Zakharova, Putin, Andrei Ivanov, Andriy Ivanov, Ivan Kudrya, John McCain, Andreyi, , McCain, Kudrya, ” “ McCain, Ukraine …, Pobigay Organizations: CNN, Getty, Russian Foreign, Kremlin, UNESCO, Locations: Ukraine, Kyiv, Soviet, Ukrainian, Kyiv's, AFP, Kiev, Russian Kiev, Moscow, Latvian, Pavlenko, Russia
[1/5] Workers mount a Ukrainian national emblem to the shield of the 'Motherland' monument replacing the Soviet one, amid Russia's attack on Ukraine, at a compound of the World War II museum in Kyiv, Ukraine August 6, 2023. REUTERS/Valentyn OgirenkoKYIV, Aug 6 (Reuters) - Workers installed Ukraine's national trident on an iconic monument depicting the Motherland in Kyiv on Sunday, replacing old Soviet symbols in one of the most visible examples of breaking away from the past and Moscow's influence. Originally, the shield bore the Soviet Union's coat of arms - a crossed hammer and sickle surrounded by ears of wheat. Kyiv says the invasion appears to be an imperial mission to recreate the Soviet Union. Ukraine outlawed Soviet symbols in 2015, the year after Russia annexed Crimea and backed separatist proxies in the country’s east.
Persons: decommunize, Vladimir Putin, Vladimir Lenin, John McCain, Olena Harmash, Yurii, Pavel Polityuk, Frances Kerry Organizations: Workers, REUTERS, KYIV, Soviet Union, European Union, Soviet, Thomson Locations: Ukrainian, Soviet, Ukraine, Kyiv, Valentyn, Dnipro, Russian, Soviet Union, Russia, Crimea, U.S
A view of the site of the Catoctin Furnace, an iron forge where enslaved people of African descent once worked, in Cunningham Falls State Park in Maryland, U.S., in this undated photograph. The site now also is providing unique insight into African American history thanks to research involving DNA obtained from the remains of 27 individuals buried in a cemetery for enslaved people at Catoctin Furnace. For African American and United States history, revealing these stories and family legacies is important to understanding and acknowledging who we are, where we came from and how we are connected to each other today," Bruwelheide added. Enslaved people of African descent were forced to work in agricultural, industrial and domestic settings in parts of the United States. In a first-of-its-kind analysis, the researchers examined historical DNA alongside genetic testing company 23andMe's personal ancestry database to identify 41,799 Americans related to the 27 individuals, including 2,975 close relatives.
Persons: Aneta, Camp David, Kari Bruwelheide, Bruwelheide, Éadaoin Harney, Andy Kill, enslavers, Kathryn Barca, Barca, Will Dunham, Rosalba O'Brien Organizations: REUTERS, U.S, Smithsonian Institution, Democratic, Smithsonian's National, of, United, Workers, Catoctin, Smithsonian, Thomson Locations: Cunningham Falls, Park, Maryland, U.S, Handout, REUTERS WASHINGTON, Camp, Catoctin, West, Central Africa's, Senegal, Gambia, Angola, Democratic Republic of Congo, Africa, Americas, Washington, United States, Civil
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