Broad Institute

JULY 29, 2025

Related groups OASIS Consortium Carpenter-Singh Lab Health and Environment Sciences Institute (HESI) Related news A marriage of microscopy and machine learning Roughly a third of drug candidates fail in the first phase of clinical trials, often due to unanticipated toxic effects that weren’t apparent in animal tests.

Animal Testing

Drug Target Review

APRIL 30, 2025

It involves a dynamic and often unpredictable process where every stage, from target identification to clinical trials, generates vast amounts of data. Additionally, AI-driven predictive modelling can shorten the preclinical phase by simulating biological responses, leading to more targeted and efficient clinical trials. The result?

Drugs

Drug Target Review

DECEMBER 11, 2024

Our approach, grounded in Good Laboratory Practice (GLP) and honed through thousands of studies, ensures that even the most subtle toxicological indicators are reliably detected. Such an approach ensures reliable preclinical toxicology data and supports the transition of innovative therapies from the lab to clinical trials.

International

Drug Target Review

JUNE 19, 2025

SRP-001 has already moved beyond the laboratory. South Rampart Pharma has completed its Phase 1 clinical trial, demonstrating strong safety, tolerability and pharmacokinetics. The next milestone is a Phase II trial, aiming to begin in 3Q2025, evaluating SRP-001 in acute pain following third molar extraction (wisdom tooth removal).

Clinical Trials

Broad Institute

JULY 21, 2025

& Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, to correct the root cause of AHC in mice. The technology has already been successfully tested in a clinical trial for another rare genetic disease.

Disease

Drug Target Review

JULY 7, 2025

Their unique suitability has made them valuable for evaluating pharmacokinetics, toxicology and safety in drug candidates before human clinical trials. NHP-C cardiomyocytes offer a more predictive and ethical platform, especially for drugs intended to advance from animal testing to first-in-human trials. National Research Council.

Drug Research

Broad Institute

OCTOBER 29, 2024

Importantly, unlike other MCL1 inhibitors that have raised concerns about cardiovascular side effects in early-stage clinical trials, BRD-810 acts quickly within cancer cells and is eliminated from the body in animal models within a few hours. This rapid clearance minimizes the drug’s potential impact on healthy cells.

Drugs

Alta Sciences

JUNE 6, 2025

Optimizing Tube Labeling for Improved Efficiency The tube labeling process starts with the generation of an Excel file generated from our Laboratory Information Management System (LIMS), which contains a list of unique IDs and sample information. This file is then imported into an MS App interface to create the user request.

Pharmacokinetics

Conversations in Drug Development Trends

JANUARY 21, 2025

Jayaprakash Kotha, MBBS, PhD, ASCP (SH), Vice President, Bioanalytical Laboratory Satish Kumar, MBB, Head of Process Improvement Continuous Innovation is a Cornerstone of Bioanalysis Approximately 80% of drugs that begin the research process fail to reach approval. What is one contributing factor that sets the 20% that do apart from the rest?

Clinical Trials

Broad Institute

APRIL 5, 2025

The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.

Science

Broad Institute

JUNE 24, 2025

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. Of the 19 base editing and prime editing clinical trials underway now, more are taking place outside the U.S.

Treatment

The Pharma Data

JUNE 13, 2025

The two companies have signed a Memorandum of Understanding (MoU) to provide comprehensive support and resources to Japanese drug-discovery startups, helping them bring their innovations from laboratory benches toward eventual global delivery of new medicines.

Research

The Pharma Data

JUNE 9, 2025

Jude Children’s Research Hospital and lead investigator of the CLEVER (MK-1654-004) and SMART (MK-1654-007) clinical trials, emphasized the significance of this development. Key results from the CLEVER trial include: A 60.5% Octavio Ramilo, Chair of the Department of Infectious Diseases at St.

FDA Approval

Advarra

JULY 3, 2025

Designed to better manage risk, patient safety, and data integrity in clinical trials, the updated guideline is expected to be adopted by regulatory authorities worldwide. Read on to explore how ICH E6 (R3) may affect your ongoing and future trials—and use the following 10 critical categories of questions to assess your readiness.

Compliance

The Pharma Data

JULY 2, 2025

The approval, granted on the basis of promising results from the ongoing Phase 1/2 LINKER-MM1 trial, targets a subset of patients who have exhausted at least four prior treatment regimens, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

FDA

The Pharma Data

JUNE 24, 2025

The goal is to transform laboratory discoveries into clinically relevant therapies that can reach patients faster and more efficiently. Notably, over 80% of Bayer’s major global multi-center clinical trials now include study sites in China, spanning early-phase development to late-stage pivotal trials.

Pharma R&D

Conversations in Drug Development Trends

NOVEMBER 12, 2024

Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys. While clinical supply is essential to any successful trial, autologous cell therapy trials occupy the far end of the spectrum regarding risk tolerance.

Therapies

Codon

JULY 28, 2025

For the former, clinicians extract a patient’s cells, engineer them in a laboratory, and then infuse them back into the body. One recent Duchenne muscular dystrophy trial saw such outcomes at high doses, illustrating the trade-off between sufficient gene delivery and immune risk.

Immune Response

The Pharma Data

JUNE 26, 2025

Food and Drug Administration (FDA) granted approval to ENFLONSIA in early June 2025 based on compelling results from two pivotal clinical trials: the Phase 2b/3 CLEVER trial and the Phase 3 SMART trial. In these trials, ENFLONSIA not only met its primary efficacy endpoints but also showed a favorable safety profile.

Vaccine

Codon

NOVEMBER 3, 2024

Still, while these developments provide cause for hope, each drug or vaccine faces a variety of challenges, ranging from financial incentives for clinical trials to the difficulty of discovering new antimicrobials that pass safety and efficacy tests. A phase 3 clinical trial for M72/AS01E, funded by the Gates Foundation, began this year.

Vaccine

Codon

JUNE 5, 2025

Dozens more clinical trials, based upon similar gene-editing technologies, are now underway. Cas9 is also being used in a number of ongoing clinical trials for everything from HIV to protein folding disease and lymphoma. Therapeutics The first Cas13-based clinical trial was approved in 2024.

DNA

The Pharma Data

JULY 2, 2025

FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.

Trials

Codon

APRIL 14, 2025

Inside the FutureHouse laboratory. Learning requires trial, error, and revision. Outside of purely intellectual domains, the more trial and error cycles we can perform in a given time, and the more accurate the error signals, the better we'll learn. Photo by Xiaofan Fang. Intuitively, this makes sense.

Protein Expression

Codon

JANUARY 5, 2025

This skillset, shared by Oncley and several others who later become key fixtures in Cohn’s laboratory, proved invaluable to wartime efforts when it became clear that many protein purification experiments would require extensive modification of instruments to work at scale. Curious researchers from external laboratories often attended.

Laboratories

LifeSciVC

JUNE 12, 2025

Josh’s work at the Marine Biological Laboratory unlocked the potential of utilizing an endogenous protein to make a single alphabet change on RNA (RNA editing), a process that was precise, reversible and had druglike characteristics. (Academic specializing in RNA editing).

RNA

Drug Patent Watch

JANUARY 9, 2025

Are you in early-stage clinical trials, or are you gearing up for commercial launch? A CDMO should have a mature data integrity program in place, especially in the laboratory. How do you approach the CDMO selection process to ensure the best outcome for your startup? Your timeline will significantly impact your CDMO requirements.

Compliance

The Pharma Data

JUNE 13, 2025

The approval is supported by data from KEYNOTE-689, a pivotal Phase 3 trial that evaluated KEYTRUDA in this patient population. Based on these trial results, KEYTRUDA as a key component of this regimen shows tremendous potential to enable patients to live longer without disease recurrence and hopefully improve their overall prognosis.”

FDA Approval

Codon

NOVEMBER 1, 2024

Although Amodei does acknowledge some real-world issues limiting scientific progress — such as the slow growth of organisms and tedious clinical trials — he mostly passes over the more general tools that will be required to accelerate research in the near term. ” Nobody really knows without trying it out in the laboratory.

DNA

Conversations in Drug Development Trends

DECEMBER 19, 2024

At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.

Clinical Trials

Reprocell

NOVEMBER 14, 2024

To address the issues of time and cost, pharmaceutical companies rely on central laboratories (CLs) for consistency, organization, and efficiency during the clinical trial phase, which is essential for drug approval.

Marketing

Fierce BioTech

JUNE 17, 2025

Whether you're navigating your first trial or scaling a growing pipeline, see how a purpose-built lab model can help you move smarter and faster. Whether you're navigating your first trial or scaling a growing pipeline, see how a purpose-built lab model can help you move smarter and faster.

Laboratories

New Drug Approvals

JUNE 9, 2025

Clinical trials: Clinical trials have demonstrated the safety and potential efficacy of hexasodium phytate in reducing hydroxyapatite crystallization in patients undergoing hemodialysis, providing a basis for its use in treating calciphylaxis.

Clinical Trials

PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. The SELECT trial set out to understand whether the drug has similar effect on patients without diabetes. As of mid-2024, this includes at least 650 Phase I-IV trials, with about 430 of those already ongoing.

Clinical Trials

Broad Institute

JULY 8, 2025

AI/ML methods Thus far we have constructed, trained, assessed, and implemented more than 40 clinical AI models for care quality assessments, clinical trial enrollment, information retrieval from EHR systems, risk stratification, and more.

Clinical Trials

BioPharma Drive: Drug Pricing

JUNE 26, 2025

Published June 26, 2025 Ned Pagliarulo Lead Editor post share post print email license An Incyte researcher works in a laboratory. Incyte expects multiple pivotal trial readouts this year, along with proof-of-concept data for several pipeline candidates. You can unsubscribe at anytime. Registered in England and Wales. TechTarget, Inc.s

Therapies

National Institute on Drug Abuse: Nora's Blog

JUNE 12, 2025

Over two decades ago, as a NIDA grantee, I and my colleagues at Brookhaven National Laboratory and SUNY-Stony Brook used PET neuroimaging to show the recovery of lost dopamine transporters in the striatum of people with methamphetamine use disorder after prolonged abstinence.

Research

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

Despite including guidance on confirmatory trials and FDAs authority to require them, the Agency decided to publish an additional guidance focused solely on how it is interpreting the authority to require that such trials be underway prior to accelerated approval or within a specified time period after the date of accelerated approval.

FDA