Drug Target Review

APRIL 30, 2025

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Kosten Digital was established to bridge this gap.

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PPD

SEPTEMBER 16, 2024

ML is also used to identify predictors of response to a therapy or risk for adverse events (AEs) that can be used to inform value-based contracting or treatment strategies intended to minimize “wasted” use of a drug. Ready to learn more about convergence of real-world data and technology for clinical trials?

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The Pharma Data

JUNE 13, 2025

Standard single-target CD19-directed CAR T-cell therapies typically enable long-term remissions in roughly 40% of patients, emphasizing a significant unmet need for new and more effective treatment strategies. Among patients who had previously undergone two or more lines of therapy (n = 12), the ORR was 92% and the CRR was 75%.

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BioPharma Drive: Drug Pricing

AUGUST 6, 2024

The company plans to limit sales of the hemophilia gene therapy to the U.S., Italy and Germany, while ending most clinical development work.

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SugarCone Biotech

MARCH 2, 2025

The critical question then becomes one of clinical translation finding tumor types that are EGFR-positive and are believed to be immunosuppressed by TGF-. Bicara has focused ficerafusp alfa clinical development on head and neck squamous cell carcinoma (HNSCC), adding pembrolizumab to separately block the PD-1 pathway.

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Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

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Covalent Modifiers

MAY 9, 2024

BIIB129 (25) demonstrated efficacy in disease-relevant preclinical in vivo models of B cell proliferation in the CNS, exhibits a favorable safety profile suitable for clinical development as an immunomodulating therapy for MS, and has a low projected total human daily dose.

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thought leadership

AUGUST 26, 2024

In the dynamic world of pharmaceuticals and biotechnology, small Sponsor companies are often the unsung heroes driving innovative therapies through the complex journey from concept to clinic.

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Clinical Development Therapies Pharmaceuticals Compliance 71

PPD

NOVEMBER 11, 2024

As a result, biopharma and biotech companies working to bring their drug pipelines to the market require deep expertise from trusted partners to help deliver critical therapies for their patients. According to the United Nation’s 2024 World Population Prospects data, the global population of those 65 years old and over will grow from 0.8

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Drug Target Review

FEBRUARY 1, 2024

Historically, what has limited investor interest in funding neurological and psychiatric therapies in development? Investors have historically taken a ‘guilty until proven innocent’ approach to investing in neuroscience therapies. All stakeholders have benefited from these innovations.

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Drug Target Review

MAY 23, 2025

In the ever-evolving landscape of immuno-oncology, managing the side effects of advanced therapies has become just as important as enhancing their efficacy. Existing therapies, such as tocilizumab, work by blocking specific inflammatory pathways but often come with limitations.

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Cytel

AUGUST 28, 2023

A clinical development plan — a comprehensive strategy for developing an investigational product through regulatory submission — is a critical component of drug development and helps ensure that new therapies are safe, effective, and of high quality.

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FDA Law Blog: Drug Discovery

DECEMBER 15, 2024

Valentine We recently published the first part of our review of FDAs draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. If you make a tissue product you may produce one lot per patient where a gene therapy may only need a handful of lots through Phase 3.

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PPD

JANUARY 15, 2025

As a result, drug developers make better decisions more quickly (removing 50% of study timeline whitespace) to bring new therapies to market faster. Ready to learn more about how to leverage AI and machine learning to maximize your clinical trial efficiency?

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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance describes various methods to assess either cell distribution for cell therapy products or vector biodistribution for gene therapy products.

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LifeSciVC

OCTOBER 10, 2024

In spite of somewhat arduous clinical development pathways, these data sets are now providing some cautious optimism for each mechanism’s potential to drive benefits for NSCLC patients. The post ESMO Reflections: Glimmers of Hope with the Next Wave of I-O Therapies? appeared first on LifeSciVC.

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Drug Discovery Today

JANUARY 20, 2022

Cambridge and Wetherby, UK, 18 January 2022: Avacta Group plc (AIM: AVCT), a clinical stage biopharmaceutical company developing innovative cancer therapies and powerful diagnostics based on its proprietary Affimer® and pre|CISION™ platforms announces that the next pre|CISION™ drug candidate, AVA3996, has been selected for pre-clinical development (..)

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The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.

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Conversations in Drug Development Trends

DECEMBER 7, 2023

These survey results also highlight the importance of evaluating a variety of potential CRO partners of different sizes to choose the best match for your clinical development goals and corporate culture. We are acutely aware that turnover in key project team personnel can lengthen project timelines and inflate clinical trial costs.

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Alta Sciences

FEBRUARY 19, 2025

Regulatory Considerations for Oligonucleotide Drug Development and Safety In 2024, the U.S. It also outlines recommendations for drug-drug interaction assessments during clinical development, such as the assessment of appropriate biomarkers that reflect modulation of the target protein.

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Drug Target Review

JUNE 9, 2023

This exclusive interview with Dr Sharon Benzeno, Chief Commercial Officer, Immune Medicine at Adaptive Biotechnologies, unveils some ground-breaking research on T- cell therapy for cancer , which has seen the first TCR-based therapeutic candidate progress to clinical development, offering promising advancements in innovative cancer treatments.

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The Pharma Data

JUNE 16, 2025

Teva and Fosun Pharma Forge Strategic Partnership to Develop and Commercialize Innovative Anti-PD1-IL2 Therapy (TEV-56278) in Immuno-Oncology Teva Pharmaceutical Industries Ltd. TEV-56278 is a first-in-class anti-PD1-IL2 ATTENUKINE therapy designed by Teva’s internal team of innovative scientists.

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PPD

NOVEMBER 21, 2023

To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinical development.

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The Pharma Data

JUNE 12, 2025

This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy. We look forward to further evaluating rilzabrutinib in subsequent phases of its clinical development.” Fast Track and Orphan Drug Designations from FDA The U.S.

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DS in Pharmatics

JANUARY 7, 2023

Cell and gene therapies for treating multiple myeloma, such as CAR-T, have resulted in breakthrough health outcomes for patients. In the last decade, approval of cell and gene therapy (CGT) products has increased significantly.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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PPD

OCTOBER 8, 2024

The highly dynamic biotech industry has a core need to remain as flexible and agile as possible across the spectrum of clinical development activities. One primary way these companies create a nimble and adaptive environment is by outsourcing some portion of clinical development functions.

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Drug Target Review

JUNE 11, 2025

Drug development faces significant challenges: long timelines, high costs , complex processes and low probabilities of success (PoS), exacerbated by the shift towards more complex molecules, biologics and cell and gene therapies, hindering patient access to vital treatments.

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Cytel

NOVEMBER 10, 2023

Independent data monitoring committees review unblinded clinical trial data and issue recommendations to designated sponsor liaisons. Characteristics of cell and gene therapy trials often require slightly different approaches to IDMC conduct in comparison to typical pivotal trials.

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The Pharma Data

JUNE 6, 2025

Commitment to Innovation in Rare Blood Disorders Sanofi’s pipeline, centered on the complex biological mechanisms that drive rare blood diseases, reflects a strategic investment in therapies that aim to address persistent unmet medical needs. and EU, in late 2024 as the first therapy of its kind. In the U.S.,

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The Pharma Data

JUNE 11, 2025

(Nasdaq: IONS) has announced the dosing of the first patient in the Phase 3 REVEAL clinical trial, marking a significant milestone in the development of ION582, an investigational therapy for Angelman syndrome (AS).

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The Pharma Data

JUNE 12, 2025

Among the 85 relapsed or refractory patients, 36% had previously received a single line of treatment, 42% had undergone two lines, and 12% had gone through as many as three lines of prior therapy. Vice President of Early Clinical Development and Translational Research at Johnson & Johnson Innovative Medicine.

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FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” It finalized a draft guidance published in 2022.

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The Pharma Data

JUNE 2, 2023

Gilead and Kite Oncology Demonstrate Growing Hematology Pipeline and Strength of Leading Cell Therapy Portfolio at EHA FOSTER CITY, Calif. & & SANTA MONICA, Calif.–(BUSINESS –(BUSINESS WIRE)– Gilead Sciences, Inc.

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FDA Law Blog: Drug Discovery

JANUARY 4, 2023

Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and contr­­ols (CMC).

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