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Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.
The $400,000 prize, to be shared among the four winners, recognizes their role in creating and advancing a technology that has reshaped how physicians treat leukemia, lymphoma, and multiple myeloma, and is now showing promise in treating autoimmune and infectious diseases. In the early 2000s, both teams began preparing for clinical trials.
The failure rate in clinical trials exceeds 90%, often due to insufficient safety data, efficacy concerns, or regulatory non-compliance. Even drugs that complete clinical trials may face delays or rejections if submission documents are incomplete or do not align with regulatory expectations.
Nonclinical safety assessment serves as a cornerstone in drug development, providing essential data to identify early signs of potential toxicity and inform subsequent clinical trial design. CAR T cells) WHAT IS THE BENEFIT OF NONCLINICAL CYTOKINE RELEASE EVALUATION? Image Thumbnail_Blog_The Altascientist issue 44_Cytokine Release Assays-3.jpg
This meant putting the fundamentals of mRNA production directly into the hands of local innovators — a dramatic departure from previous models that kept much of vaccine production within a small number of firms in high-income settings. mRNA Technology Transfer Programme Moves Into Phase 2.0, 2026–2030).
Merck’s ENFLONSIA Receives ACIP Endorsement for RSV Prevention in Infants, Marking a Major Milestone in Pediatric Infectious Disease Prevention U.S. The disease burden is disproportionately high among infants under 1 year of age, and even more so among premature infants or those with underlying heart or lung conditions.
Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. Even after microbiologists discovered the bacterium that causes the illness in 1882, it wasn’t until the 1920s that researchers were able to develop a vaccine for TB.
2] [5] [6] Clinical trials In September 2023, the drug’s developer, Sorrento Therapeutics, announced top-line data that olgotrelvir had met its primary endpoints in a phase III clinical trial that enrolled 1,212 patients with mild or moderate COVID-19. days on average compared to patients in the placebo group.
Ibtrozi was approved based on a pair of trials showing response rates of 90% and 85%, respectively, in patients who hadn’t previously received another tyrosine kinase inhibitor. Among those who had gotten another such therapy, the rates were 52% and 62% in those trials. You can unsubscribe at anytime. Registered in England and Wales.
Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease. Published June 26, 2025 Gwendolyn Wu Senior reporter post share post print email license B cells, like those pictured above, malfunction in autoimmune disease.
The same team also invented several other products that proved useful for treating battlefield injuries, including gamma globulins to inoculate soldiers against diseases and fibrin film to minimize bleeding during brain surgeries. Developing processes at the pilot scale supplied more than enough human albumin for clinical trials, for example.
The Strategy must address appropriately restructuring the Federal Governments response to the childhood chronic disease crisis, including by ending Federal practices that exacerbate the health crisis or unsuccessfully attempt to address it, and by adding powerful new solutions that will end childhood chronic disease.
GSK Licenses Shigella Vaccine Candidate to Bharat Biotech in Push for Global Health Equity GSK today announced a significant step forward in the battle against Shigella a leading cause of diarrhoeal disease and death in children under five by licensing its Shigella vaccine candidate, altSonflex1-2-3 , to Bharat Biotech International Limited (BBIL).
“A big part of my role involves interacting with customers, learning from them on their challenges and providing solutions to expedite their path to clinical trials or commercialisation of their drug,” Galbraith explains. We are driving rapid analytics to support the release of cell and gene therapies and other biologics.
On a conference call held Wednesday afternoon, Sarepta CEO Doug Ingram said that "failure to adapt" to recent setbacks "would risk our long-term viability as an organization and decrease the opportunity to bring the greatest benefit to the greatest number of patients living with rare disease." You can unsubscribe at anytime.
Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. Human constraints also play a role at a later stage, when clinical trials are involved.”
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. Kennedy Jr., Kennedy Jr.
Spencer Platt via Getty Images The first marketed treatment for the insatiable hunger associated with Prader-Willi disease is selling more quickly than Wall Street analysts expected, an early, but encouraging sign of demand. The disease, which affects an estimated 10,000 to 20,000 people in the U.S., You can unsubscribe at anytime.
Innovations like gene therapies are providing hope for diseases that previously had no treatment options, while precision medicine such as cell therapy personalises treatments to fit each patients signature biological characteristics. The COVID-19 pandemic highlighted these issues on a global scale.
While vaccines, therapeutics, and preventive measures have significantly reduced the global burden of the disease, […] The post COVID-19 in 2025: Are Decentralized Clinical Trials the New Standard? The world continues to feel the ripple effects of the COVID-19 pandemic, even into 2025.
Interim results from an early-stage trial last year showed Lexeo’s Friedreich’s ataxia drug, LX2006, reduced signs associated with heart complications among a small group of study participants. Trade Deficit NBC News Outside groups organize to form unbiased, independent vaccine panel View all Events 07 JUL Webinar | 10 a.m.
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.
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.
Permission granted by Incyte Dive Brief: Incyte has named veteran pharmaceutical executive Bill Meury as its new CEO, replacing longtime head Hervé Hoppenot, who led the cancer and blood disease drugmaker for the past 11 years. says US won’t donate to global vaccine effort View all Events 07 JUL Webinar | 10 a.m. billion in sales.
And some of the remedies are still practiced today: the treatment for Guinea-worm disease was, and still is , to wrap the emerging end of the worm around a stick and slowly pull it out. Dreser had the right to veto any drug going to clinical trials, so this is where the story of aspirin could have ended. Credit: Wellcome Collection.
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.
Dr. Makary noted that he doesnt believe that the traditional playbook works for everythingparticularly in the case of life-threatening diseases where randomized controlled trials are neither feasible nor humane. He also specifically mentioned rare diseases here , in addition to his example of late-stage illnesses.
In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.
The COVID-19 pandemic brought mRNA to global prominence by developing highly effective vaccines by Moderna and Pfizer-BioNTech. However, mRNA technology is not limited to infectious diseases. However, mRNA technology has introduced new possibilities, particularly in developing personalized cancer vaccines and combination therapies.
Peer-reviewed / Randomised Controlled Trial / People Study of healthy US adults found that a single dose of the VLA1553 vaccine candidate was generally safe, well tolerated and provokes an immune response.
A trial of a new vaccine that appears to train the immune system to fight coronavirus has begun in the UK. The trial on 10,000 people will now see if the vaccine can prevent people getting ill. Some of the vaccines being developed for Covid-19 use either completely new or barely proven technologies. .
Enrollment in a Phase 1 trial of a new investigational universal influenza vaccine candidate has begun at the National Institutes of Health’s Clinical Center in Bethesda, Maryland.
8, 2020 — AstraZeneca’s COVID-19 vaccine is safe and effective, new data from late-stage trials shows. Overall, the vaccine protected against symptomatic disease in 70% of cases, according to a team led by researchers from Oxford University in England. TUESDAY, Dec. 8 in The Lancet.
Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021.
COVID-19 vaccine developers are faced with an ethical quandary — whether to let phase 3 trial participants become “unblinded” and to receive authorized vaccines as they become available, which could hinder the collection of meaningful trial data. Pfizer, which has received emergency approvals in the U.S.
AstraZeneca has started a Phase 3 trial of its experimental coronavirus vaccines in the US, making it the third company to start late-stage trials of a vaccine. . The trial will see participants receive either two doses of the vaccine or a placebo. Photo by Daniel Paquet from Regina, Canada.
November 16, 2020 — An independent data and safety monitoring board (DSMB) overseeing the Phase 3 trial of the investigational COVID-19 vaccine known as mRNA-1273 reviewed trial data and shared its interim analysis with the trial oversight group on Nov.
Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. The Companies expect interim results from this trial in the third quarter of 2021. “Our About the Phase 1/2 clinical trial.
Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc. Valneva SE (Nasdaq: VALN; Euronext Paris: VLA), a specialty vaccine company, and Pfizer Inc. NYSE: PFE) today reported positive Phase 2 pediatric data for their Lyme diseasevaccine candidate, VLA15.
Food and Drug Administration to expand the enrollment of their Phase 3 pivotal COVID-19 vaccinetrial to up to approximately 44,000 participants which also allows for the enrollment of new populations. The pivotal trial is event-based and there are many variables that will ultimately impact read-out timing.
Moderna has completed enrollment of 30,000 participants for its phase 3 study evaluating COVID-19 vaccine candidate mRNA-1273. The randomized, placebo-controlled trial is being conducted in partnership with the NIH’s National Institute of Allergy and Infectious Diseases and HHS’ Biomedical Advanced Research and Development Authority.
Johnson & Johnson (the Company) has begun vaccinating adolescent participants in the ongoing Phase 2a clinical trial for its COVID-19 vaccine candidate, developed by the Janssen Pharmaceutical Companies of Johnson & Johnson. Janssen’s COVID-19 Vaccine Candidate.
Valneva SE (“Valneva”), a specialty vaccine company focused on prevention of diseases with major unmet needs, and Pfizer Inc. It will compare the three-dose vaccination schedule (Month 0-2-6) with a two-dose schedule (Month 0-6). “We We are excited to be part of the Lyme diseasevaccine development program with Valneva.
Medicago, a biopharmaceutical company headquartered in Quebec City, and GlaxoSmithKline (GSK) are pleased to announce the start of Phase 3 clinical testing of Medicago’s plant-derived COVID-19 vaccine candidate in combination with GSK’s pandemic adjuvant, as part of the ongoing Phase 2/3 study. g of CoVLP are administered 21 days apart.
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