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Published July 8, 2025 Gwendolyn Wu Senior reporter post share post print email license Proteasomes are cellular machines for breaking down proteins. Love Employee via Getty Images For decades now, scientists have been experimenting with a new way to target and destroy proteins linked to disease. You can unsubscribe at anytime.
Proteinuria reduction is a well-established surrogate marker for the progression of kidney disease, and this level of decline is considered both statistically significant and clinically meaningful in slowing disease progression toward end-stage kidney disease (ESKD). Treatment-emergent adverse events (TEAEs) occurred in 76.3%
Durable Benefit and Positive Clinical Outcomes “Today’s results underscore the promising potential of Intellia’s approach to genome-editing therapy — a one-time treatment that has been well tolerated and offers a highly differentiated, durable effect for patients suffering from a serious disease,” said John Leonard, M.D.,
Primary membranous nephropathy is a rare and serious autoimmune kidney disease characterized by the formation of autoantibodies that damage the glomerular basement membrane, often resulting in nephrotic syndrome —a condition marked by high levels of protein in the urine, severe swelling, and a significant risk of progression to kidney failure.
Improved understanding Understanding metabolism is also crucial for drug discovery and investigating disease mechanisms. The ability to measure reactive oxygen species can provide important information on, for example, cell signaling events including those that take place in bacteria and other cells. and Hinshelwood, C.
By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. “We call this phenomenon ‘event-driven pharmacology.”
As clinical development progresses, pharmaceutical companies must submit periodic updates to regulatory agencies, reporting on patient safety data, adverse events, and protocol modifications. Once a drug completes Phase III trials, companies prepare a New Drug Application or Biologics License Application (BLA) for final review.
“Ziihera offers a targeted monotherapy with a favorable safety profile and compelling efficacy, representing a crucial advance in the fight against this challenging disease.” under license from Zymeworks Inc. , Arndt Vogel , professor of gastroenterology at Hannover Medical School. formerly BeiGene, Ltd.)
1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]
Published June 11, 2025 Ben Fidler Senior Editor post share post print email license The FDA on June 11, 2025 approved Nuvation's Ibtrozi for ROS1-positive non-small cell lung cancer. Ibtrozi has also shown the ability to help people whose cancer has spread to the brain, a leading cause of disease progression, the company said.
Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Vor Biopharma licensed a drug in June 2025 that targets proteins essential to B cell survival. Now, Vor is reestablishing itself as an autoimmune disease company.
Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) and granted it priority review status, seeking to update the U.S. The product was developed under a licensing agreement with Bristol Myers Squibb.
Drug development has also been hampered by serious adverse events, including Grade 3 or higher levels of neutropenia and interstitial lung disease. However, HER3-DXd missed achieving a statistically significant improvement in overall survival, leading to the withdrawal of its U.S. regulatory application in NSCLC.
Published July 16, 2025 • Updated 2 hours ago Ben Fidler Senior Editor post share post print email license Sarepta Therapeutics announced a workforce reduction on July 16, 2025. Among them are treatments for spinocerebellar ataxia, Huntington’s disease and facioscapulohumeral muscular dystrophy. You can unsubscribe at anytime.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Moderna COVID vaccine gets full approval for children The approval comes amid regulatory upheaval under HHS head Robert F. You can unsubscribe at anytime. Kennedy Jr.,
Published July 10, 2025 Ben Fidler Senior Editor post share post print email license Soleno Therapeutics on July 10, 2025 announced preliminary sales for its new Prader-Willi Syndrome drug Vykat. The disease, which affects an estimated 10,000 to 20,000 people in the U.S., You can unsubscribe at anytime.
Published June 24, 2025 Gwendolyn Wu Senior reporter post share post print email license Lexeo Therapeutics and two life sciences investors are working together to launch a startup to develop cardiac RNA therapies. You can unsubscribe at anytime. Earlier this year, it laid off 15% of its workforce.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Dive Brief Organon drug for endometriosis falls short in mid-stage study Company executives had seen the drug as a potential multi-billion dollar opportunity in women’s health.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Merck to buy Verona and its lung drug in $10B deal The New Jersey-based pharma has inked one of the biggest deals of the year in pursuit of a promising new way to treat COPD.
Published June 26, 2025 Ned Pagliarulo Lead Editor post share post print email license An Incyte researcher works in a laboratory. The company has had a good deal of success with Jakafi, a multipurpose drug approved to treat rare blood cancers and graft-versus-host disease. You can unsubscribe at anytime. billion in sales.
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Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from News roundup Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules Biocryst is offloading part of its business to an Italian drugmaker for $250 million upfront.
The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) for use in individuals 16 years of age and older. AUTHORIZED USE IN THE U.S.:
Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious hematologic diseases, today announced that the company conducted a Type B Meeting for omidubicel with the U.S. Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases.
Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?
Anderson Professor of Chemical and Biomolecular Engineering, and his colleagues, are reporting in iScience the event of an intranasal subunit vaccine that gives durable local immunity against inhaled pathogens. But now, we’re one step closer. Navin Varadarajan, University of Houston M.D.
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. About Bristol Myers Squibb. About Dragonfly. Dragonfly has a deep pipeline of wholly owned programs developed using its platform. Source link.
Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. You should not place undue reliance on these statements. Source link:[link].
recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Rise of the best-in-class biologics plays: Are you looking to in-license or invest in “de-risked” biology that is already in the clinic? Join the club. A permanent gene edit to produce a difficult-to-titrate supply of GLP1 in the liver?
1, 2020 /PRNewswire/ — Sosei Group Corporation (“the Company”) (TSE: 4565) announces it has entered into a global collaboration and license agreement with Biohaven Pharmaceutical Holding Company Ltd. (“Biohaven”, NYSE: BHVN). .
TOKYO and CAMBRIDGE, England , Dec.
(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,
15, 2020 (GLOBE NEWSWIRE) — Burning Rock Biotech Limited (NASDAQ: BNR, the “Company” or “Burning Rock”) today announced that it entered into an exclusive licensing agreement with Oncocyte Corporation (NYSE American: OCX) to bring DetermaRx , a risk stratification test for early stage lung cancer patients, to China. 1 Kratz et al.,
RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases.
We intend to use PWS as the anchor indication and will further investigate additional orphan disease opportunities in due course.”. Disease Highlights.
“Buprenorphine is the most important medication in our arsenal for treating opioid use disorder, which is currently one of the most lethal diseases for Americans.” As a result, some clinicians are hesitant to pursue this DEA license or even engage in treatment of patients with OUD.
Live Webcast : accessible from the Company’s website at www.eloxxpharma.com under Events and Presentations or with this link: [link]. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. WALTHAM, Mass., 15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc.
The event is held in partnership with Drug Discovery News. Understanding the role of neuroimmune interactions has become increasingly important in tackling complex neurological diseases. Focused on Neuroimmunology in Drug Discovery, the free-to-attend forum is taking place at at MSDs (the tradename of Merck & Co.,
It is also licensed under the brand name Efluelda ® in Europe where it is indicated for adults aged 60 and older. It is also licensed under the brand name Efluelda® in Europe where it is indicated for adults aged 60 and older. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. Since licensed by Ridgeback all funds used for the development of EIDD-2801 by Ridgeback have been provided by Wayne and Wendy Holman and Merck. Source link:[link].
NYSE American: SYN) is a diversified clinical-stage company leveraging the microbiome to develop therapeutics designed to prevent and treat gastrointestinal (GI) diseases in areas of high unmet need. .
ROCKVILLE, Md. , 24, 2020 /PRNewswire/ — Synthetic Biologics, Inc.
Synthetic Biologics, Inc.
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People with type 2 diabetes are at increased risk for heart attacks, stroke, and other forms of cardiovascular disease, and at an earlier age than other people. In fact, the evidence suggests that such drugs might even offer some protection against heart disease.
When a loved one is diagnosed with Alzheimer’s disease , it can be challenging to know what to do or how to cope. You may first want to understand this degenerative disease so that you can best support your loved one after their Alzheimer’s diagnosis. What is Alzheimer’s Disease? . Learn More About Alzheimer’s Disease.
The program was licensed to Allergan in April 2016 , and Allergan was acquired by AbbVie in May 2020. The event reported today has no immediate impact on the consolidated financial results for the accounting period ending December 2021. ” About the License Agreement. TOKYO and CAMBRIDGE, England , Jan.
The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) for use in individuals 12 years of age and older. AUTHORIZED USE IN THE U.S.:
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