DrugBank

JUNE 6, 2024

In the rapidly evolving landscape of oncology drug development, artificial intelligence (AI) has emerged as a revolutionary force, altering not just the pace but the very methodology of discovery and development.   What sets RADR ® apart is its robust data-driven approach.

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Conversations in Drug Development Trends

AUGUST 14, 2024

By: Simran Padam, Medical Director, Medical Affairs Personalized treatment approaches have emerged as pivotal in improving outcomes for hematological cancers. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

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Alta Sciences

AUGUST 29, 2024

Engaging at the outset with a fully integrated and experienced drug development partner can ensure safety, with timely data sharing at every step of the drug development plan, and facilitate agile, flexible decision-making and planning. billion USD in 2023 to $166.53 billion USD by 2028.

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Drug Target Review

AUGUST 7, 2024

The pharmaceutical industry is undergoing a major shift towards the development of breakthrough medicines and advanced therapies. This continued innovation highlights the complexity of the drug development process, particularly as the field is highly regulated by health authorities around the world.

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

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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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Fierce BioTech

APRIL 3, 2024

Mini-Organs, Major Breakthroughs: How Hub Organoids Are Accelerating Oncology Drug Development dwunderlin Wed, 04/03/2024 - 17:28 Tue, 05/14/2024 - 09:30 Resource Type Webinar Dr. Sylvia F. png Listing Introduction Predict patient response to treatment with patient-derived organoids. Click here to login.

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Could you give us an overview of the Lineage platform?

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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Bauer, Senior Regulatory Drug Expert & James E. Valentine — Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). There have been challenges in gene therapy development.

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, S.p.A., By Charles G. Raver & James E.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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PPD

JUNE 27, 2024

As the search for treatments for alopecia areata continues, it’s important for drug developers to understand the key pillars of several dermatology indications: the consistency of endpoints and the intrasite and intersite variability for raters’ assessed endpoints. Accelerate your alopecia areata treatments.

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BioPharma Drive: Drug Pricing

APRIL 10, 2024

The biotech is working on a preventive therapy for the immune disorder, known as FNAIT for short, while J&J is studying its drug nipocalimab as a treatment.

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The Premier Consulting Blog

JUNE 18, 2023

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

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Fierce BioTech

OCTOBER 19, 2023

How cell and gene therapies are forcing sponsors to rethink safety and efficacy monitoring In this whitepaper, we look at how the industry is rising to the challenge, focusing on how cell and gene therapies are radically improving health outcomes and the technologies and techniques that are facilitating their development.

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ASPET

OCTOBER 12, 2023

To maintain cadence with looming threats in a prolonged field care environment, the broader medical countermeasure (MCM) enterprise must adopt new strategies for CBRN-addressing drug development. Repurposing is one such method. CBRN MCMs).

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Conversations in Drug Development Trends

JULY 9, 2024

The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Most rare diseases do not have treatments. many of which are life-threatening.

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The Pharma Data

MARCH 20, 2021

Precision imaging analytics technology known as radiomics will see increased adoption in 2021 and will be incorporated into drug development strategies and clinical trials management, according to Rose Higgins, CEO of HealthMyne. Source link: [link].

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?

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Drug Target Review

SEPTEMBER 25, 2024

While these approaches often produce encouraging initial results, the development of drug resistance remains a major obstacle for long-term patient survival. Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades.

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Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.

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FDA Law Blog: Drug Discovery

MARCH 19, 2023

s Mark Tobolowsky co-authored the peer-reviewed article “ Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials ” in the recently published edition of Human Gene Therapy. Hyman, Phelps & McNamara, P.C.’s

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Vial

DECEMBER 11, 2023

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection.

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ASPET

JULY 15, 2024

XELOX therapy, which comprises capecitabine and oxaliplatin, is the standard first-line chemotherapeutic regimen for colorectal cancer. The PK-TD model successfully predicted the time-course profiles of hematological responses in rats following monotherapy and XELOX combination treatment.

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Alta Sciences

SEPTEMBER 13, 2023

Four Pain Models Altasciences Uses to Assess Treatments During Clinical Trials pmjackson Wed, 09/13/2023 - 17:01 September is pain awareness month, and the importance of pain management and continued research into effective analgesics is integral to helping patients suffering from various pain conditions.

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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

JANUARY 4, 2021

5, 2021 /RPNewswire/ — Last December 25 , the anti-tumor drug candidate Chiauranib, developed by Shenzhen Chipscreen Biosciences Co., Treatment is dominated by chemoradiotherapy, characterized by short-term efficacy but frequent relapse, making the long-term survival rate far less than that of non-small cell lung cancer.

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PLOS: DNA Science

SEPTEMBER 5, 2024

Targeted cancer drugs developed to treat one type of cancer often expand, with further testing, to treat other types, based on molecular similarities. Diagnosis began to shift from a body-part basis to a molecular one, which I wrote about in “Mutation and location important in cancer treatment” for The Lancet in 2015.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

What was the biggest development in rare disease in 2024, and how has that impacted clinical research or patient outcomes? NC: I think the continued growth in cell and gene therapy research and the approvals we’re seeing for genetically-driven rare diseases. There’s now a focus on expediting drug development, which is quite exciting.

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Alta Sciences

AUGUST 28, 2023

Altasciences Completes Successful Phase I Trial of Ischemix’ Novel Compound for Treatment of Traumatic Brain Injury (TBI) pmjackson Mon, 08/28/2023 - 14:04 Laval, Québec, August 30, 2023 – Altasciences is pleased to have completed a Phase I trial on Ischemix, Inc.’s We are proud to support Ischemix in advancing this vital new therapy.”

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