BioPharma Drive: Drug Pricing

NOVEMBER 2, 2023

The rare disease drug developer is turning to former Genentech head Alexander Hardy as the launch of its Roctavian gene therapy stumbles.

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Fierce BioTech

AUGUST 20, 2024

Alzheimer’s disease research and drug development is evolving at a rapid pace. Decades of research has led to the approval of the first disease-modifying drugs and new pathological discoveries. |

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BioPharma Drive: Drug Pricing

AUGUST 2, 2024

The one-time drug developer is pivoting to cancer research. Elsewhere, Neurocrine reported a strong quarter and venBio raised half a billion dollars for biotech investing.

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BioPharma Drive: Drug Pricing

NOVEMBER 11, 2024

Innovation-without-borders philosophy seeks to bring forward only the most promising therapies.

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BioPharma Drive: Drug Pricing

MARCH 21, 2024

Though the medicine hit its goal in a Phase 3 study in myasthenia gravis, the results fell short of expectations in a boost to rival drug developers like Argenx and AstraZeneca.

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BioPharma Drive: Drug Pricing

APRIL 14, 2025

The rare disease drug landscape is evolving, with regulatory guidance shaping its future. Learn more.

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Broad Institute

DECEMBER 18, 2023

Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. We asked Hyman, who is also a core institute member at Broad, to talk about the new framework and what he thinks are the biggest opportunities.

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Crown Bioscience

NOVEMBER 11, 2024

Introduction to Biomarkers in Drug Development Biomarkers have become a cornerstone in the advancement of modern drug development and precision medicine. The use of biomarkers has transformed the pharmaceutical industry by allowing researchers to develop therapies that target specific pathways in diseases.

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PPD

DECEMBER 16, 2024

Our annual look at the state of the drug development industry highlights a dual set of challenges complicating progress. Rising costs have become a persistent challenge for drug developers, driven by a combination of internal and external pressures that have intensified in recent years.

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Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. Monitoring biomarkers can help assess changes in a disease, its level of expression, or the extent of its progression.

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Chemical Biology and Drug Design

JANUARY 21, 2025

ABSTRACT Infectious diseases, including bacterial, fungal, and viral, have once again gained urgency in the drug development pipeline after the recent COVID-19 pandemic. Tuberculosis (TB) is an old infectious disease for which eradication has not yet been successful.

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PPD

SEPTEMBER 25, 2023

As the clinical trial landscape evolves, drug developers are faced with novel challenges and changes in study recruitment, trial size and structure, and more. The adoption of new innovations, strategies and technologies offers opportunities to address persistent challenges and develop suitable approaches for the future.

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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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DrugBank

OCTOBER 17, 2024

Data science has emerged as an innovative tool in the biopharmaceutical industry, leveraging the power of machine learning and artificial intelligence to drive innovation and efficiency across the entire drug development lifecycle. This was seen in the case of the BRAF V600E mutation test for melanoma patients receiving vemurafenib.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Broad Institute

SEPTEMBER 6, 2023

This collaboration has the potential not only to accelerate our understanding of the diseases, but also potentially enable scientific advancements in disease-modifying interventions — a true game changer in addressing cardiometabolic diseases.” The collaboration will focus on advancing three programs over the next three years.

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DrugBank

JULY 20, 2023

Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. We're excited to expand our data offering by launching a new Rare Disease add-on to complement our Clinical Trial dataset.

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BioPharma Drive: Drug Pricing

OCTOBER 31, 2024

The immune disease drug developer expects to raise $300 million in a private financing set to close before its combination with the struggling Aerovate.

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FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. Early Direction for the Rare Disease Innovation Hub The Hub, which will be co-chaired by Drs. By Sarah Wicks & James E.

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PPD

JANUARY 15, 2025

As clinical trials become increasingly complex, particularly in decentralized trials and rare disease studies, sponsors experience increased challenges in site selection, forecasting and resourcing, and patient recruitment and enrollment.

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Drug Target Review

JULY 18, 2023

Artificial Intelligence (AI) is poised to transform the field of target discovery in drug development, offering immense potential to enhance efficacy, personalised medicine, and accelerate the development of innovative compounds. This means that we are not doing something right.

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Drug Target Review

JULY 13, 2023

Dewpoint Therapeutics, a platform drug discovery company founded in 2018, has been at the forefront of this exciting field. Dr Isaac Klein, Chief Scientific Officer at Dewpoint, brings a wealth of knowledge and expertise to the field of drug discovery. Klein notes the importance of Dewpoint’s disease-agnostic approach.

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Drug Target Review

OCTOBER 30, 2024

Imagine being able to create an in vitro replica of a diseased organ to study the molecular mechanism underlying the illness. Now take a step further: envision testing drugs in these organoids to identify the ones that can treat disease safely and effectively without needing to run expensive clinical trials first.

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Drug Patent Watch

DECEMBER 9, 2024

High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. This approach has led to the discovery of numerous potential drug candidates. Q: How long does a drug patent last?

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Science Daily: Pharmacology News

AUGUST 9, 2023

The drug tecovirimat is currently in use for the treatment of mpox -- the disease caused by monkeypox virus -- that spread worldwide in 2022. Tecovirimat is an anti-poxviral drug, and its use is driving the emergence of drug-resistant variants of the monkeypox virus.

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LifeSciVC

FEBRUARY 27, 2024

By Ivana Magovčević-Liebisch, CEO of Vigil Neuroscience, as part of the From The Trenches feature of LifeSciVC Patients and their care partners are at the center of our work in the life sciences industry – but at what point during the drug development process should companies start to engage these key stakeholders?

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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SCIENMAG: Medicine & Health

OCTOBER 31, 2023

— A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys.

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Quanticate

SEPTEMBER 13, 2024

With the implementation of technology rapidly increasing, stricter regulatory standards, and growing demand for innovative treatments, the trends within the industry continue to evolve, impacting the future of clinical trials both positively and negatively.

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Drug Discovery Today

JULY 29, 2022

Geneva-based event pledges five Swiss francs per visitor to support new drug development project

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Conversations in Drug Development Trends

NOVEMBER 21, 2023

Since the advent of modern medicine, the scientific community has developed more than 3,000 drugs for thousands of diseases. Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. There are so many patients suffering from diseases that have no treatments.

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FDA Law Blog: Drug Discovery

JANUARY 30, 2025

This target completion date is informed by the natural history of the disease, availability of alternative treatment, anticipated recruitment timeline, and the projected timeline for efficacy analysis(es); 2) the sponsors progress and plans for postapproval conduct of the trial provide sufficient assurance to expect timely completion of the trial.

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Reprocell

DECEMBER 11, 2024

Inflammatory bowel disease (IBD) is rising in prevalence across the globe, as the number of individuals diagnosed with the condition has increased by 95% since 1990. Inflammatory bowel disease (IBD) is rising in prevalence across the globe, as the number of individuals diagnosed with the condition has increased by 95% since 1990.

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SCIENMAG: Medicine & Health

JUNE 27, 2023

HOUSTON – (June 27, 2023) – Many of the drugs we use to treat cancer and infectious disease are ⎯ or derive from ⎯ natural products, but it’s difficult to know exactly how nature assembles them. Credit: (Photo by Gustavo Raskosky/Rice University) HOUSTON – (June 27, 2023) – Many of the drugs we use to […]

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BioPharma Drive: Drug Pricing

JULY 20, 2023

The setback cut shares of the once high-flying infectious disease drug developer in half, adding to the challenge facing new CEO Marianne De Backer.

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Conversations in Drug Development Trends

MAY 14, 2024

By: Amy Raymond, Derek Ansel, Nathan Chadwick, & Juliane Mills When choosing a CRO for a rare disease study, what truly sets them apart is their methodology: the CRO’s mindset, their approach to each unique study, and their agility in navigating the inherent complexities of rare disease research. Common goals empower change.

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thought leadership

JUNE 5, 2023

CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, is a gene-editing technology that holds enormous promise for treating genetic diseases. However, developing CRISPR-based drugs is a complex and challenging undertaking that requires careful project management.

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