PPD

MARCH 11, 2025

Real-world data (RWD) and real-world evidence (RWE) have emerged as transformational tools in addressing these hurdles, advancing rare disease drug development. MDT helps bridge the gap in preference heterogeneity to ensure that rare disease therapies are developed with the diverse needs of patients in mind.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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The Pharma Data

NOVEMBER 29, 2020

Prior to joining Gannex, Dr. Palmer was Head of Liver Disease Clinical Development at Takeda Pharmaceuticals. She has also held senior leadership positions at Shire Pharmaceuticals and Kadmon Corporation, where she was in charge of the global development of NASH and other liver disease programs. ” About Ascletis.

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. This progress in clinical development qualifies Ascentage Pharma for a milestone payment according to the terms of the licensing agreement. SUZHOU, China and ROCKVILLE, Md. ,

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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Plenge Gen

MARCH 10, 2021

In this guest blog, Dr. Kristen Hege, senior vice president of Early Clinical Development, Hematology/Oncology & Cell Therapy at Bristol Myers Squibb (BMS), describes a harrowing tale hiking in the Sierra Mountains and what it taught her about leadership.

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Drug Target Review

DECEMBER 13, 2023

These disease signatures capture both the linear and non-linear effects of genetic and molecular interaction networks and enable the identification of associations including those that are only relevant to a subgroup of patients that influence disease risk, prognosis and/or therapy response.

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Drug Target Review

DECEMBER 28, 2023

In most cancers, the tumour evolves by acquiring mutations that confer growth advantages or resistance to therapies. However, if the therapy targets only the subclonal (branch) mutations, this will result in the mere ‘pruning’ of specific branches rather than elimination of the whole cancer.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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The Pharma Data

DECEMBER 29, 2020

HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the company was officially included into the Shenzhen-Hong Kong Stock Connect program (the “Hong Kong Stock Connect”).

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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Agency IQ

FEBRUARY 2, 2024

EMA finalizes clinical anticancer therapeutic guidance update In a newly finalized revision, the EMA’s guideline covering all aspects of clinical development of anticancer therapeutics has expanded its already broad scope. The EMA started providing guidance on the clinical development of anticancer therapeutics in 1996.

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Drug Target Review

MARCH 17, 2025

However, a deeper understanding of brain function particularly the role of synaptic plasticity is now opening the door to innovative therapies. With over 25 years of experience in the pharmaceutical industry, Donello has been instrumental in drug discovery and development.

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Drug Target Review

JULY 11, 2023

In this article, I spoke to Olivia Cavlan, Chief Corporate Development and Strategy Officer at Alchemab Therapeutics Ltd, about how AI is transforming the landscape of disease research, unlocking a treasure trove of knowledge that may hold the key to a healthier future. Cavlan will explore more of these ends in our next article.

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The Pharma Data

JANUARY 20, 2021

Rouleau has an international reputation in cardiovascular research, particularly in the field of both basic mechanisms and improving the clinical care of patients with heart failure. His publication list includes more than 475 articles and 7 book chapters. Dr. Wells is the author or co-author of over 400 published articles.

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Drug Target Review

DECEMBER 27, 2023

Aside from CD24, there are several other ‘don’t eat me’ signals that have been characterised, and there are multiple programmes currently in clinical development that target these signals. The potential of FcγRIIB in immunotherapy Are there other ‘don’t eat me’ signals that are known to play a role in cancer immune evasion?

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The Pharma Data

NOVEMBER 26, 2020

The EGM resolved on amendment of § 4 (share capital) and § 5 (number of shares) of the Articles of Association for the purpose of enabling the directed issue of shares and warrants, as resolved by the Board of Directors on 27 October 2020 subject to the approval by the EGM. § LUND, Sweden , Nov. LUND, Sweden , Nov. sammanläggning 1:25 ).

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The Pharma Data

JANUARY 4, 2021

Despite the significant advances in therapeutics in recent years, the 5-year survival rate of AML remains at 25%–30%, which still represents a significant unmet medical need for therapies with more durable efficacy and a better safety profile. . SUZHOU, China and ROCKVILLE, Md. , ” Reference.

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Vial

APRIL 24, 2024

In addition to the areas above (aside from DTx), Syneos Health offers biosimilars , cell and gene therapy , endocrine and metabolic, immunology and inflammation, infectious diseases, pediatrics, respiratory, and women’s health services. Digital therapeutics is not explicitly listed as a therapeutic area of expertise for Syneos Health.

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Vial

APRIL 10, 2024

This article provides a comparative overview of Vial and IQVIA. IQVIA’s core strengths lie in its specialized global IT infrastructure tailored for healthcare, analytics-driven clinical development, a robust ecosystem of real-world solutions, and an expanding suite of proprietary clinical and commercial applications.

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Drug Target Review

JUNE 3, 2024

Results from these preclinical studies were published in the peer-reviewed publication, Journal of Immunology in August 2022 as a “Top Read” article. Notably, we anticipate initiating the first clinical study of CNP-103 in late 2024, on the heels of our Series A announced earlier this year on January 30.

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The Pharma Data

DECEMBER 18, 2020

Roxadustat is also in clinical development for anemia associated with MDS and for chemotherapy-induced anemia. AstraZeneca in CVRM Cardiovascular, Renal and Metabolism (CVRM) together forms one of AstraZeneca’s three therapy areas and is a key growth driver for the Company. Related Articles: Roxadustat FDA Approval History.

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New Drug Approvals

JANUARY 3, 2023

1] It is being developed by Mirati Therapeutics. [1] Identification of the Clinical Development Candidate MRTX849, a Covalent KRASG12C Inhibitor for the Treatment of Cancer.” 2] The FDA granted the application for adagrasib fast-track , breakthrough therapy , and orphan drug designations. [2] 1] It is taken by mouth. [1]

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The Pharma Data

NOVEMBER 12, 2020

As part of an extensive clinical development programme, AstraZeneca and Acerta Pharma are currently evaluating Calquence in more than 20 company-sponsored clinical trials. Related Articles: Calquence (acalabrutinib) FDA Approval History. Calquence binds covalently to BTK, thereby inhibiting its activity.4,5 2016;9(21).

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The Premier Consulting Blog

OCTOBER 11, 2022

However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases.

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The Pharma Data

OCTOBER 27, 2020

Clynes developed several novel therapeutic approaches in cancer and autoimmunity, including DC targeting for cancer vaccines and Syk and JAK mediated inhibition of DCs and T cells in autoimmunity therapy.

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Vial

MARCH 25, 2024

In the past three years, Worldwide has collaborated on 105 oncology studies supported by a global team of oncology clinical development experts. Other therapeutic areas include cell and gene therapy. With more than 30 years of experience in cardiovascular research, Worldwide has global trial experience in all phases.

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LifeSciVC

MARCH 1, 2023

Such is the story of targeting casitas B-lineage lymphoma-B (CBL-B) that was highlighted in a recent scientific review article published in the Journal for ImmunoTherapy of Cancer. Even good ideas are met with healthy skepticism for the practical and technical limitations of being able to effectively test a hypothesis.

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Molecular Design

JUNE 8, 2023

Taking into account changes in their physicochemical properties over time, drugs are analyzed according to their target class, therapy area, and route of administration. Using ChEMBL version 26, a data set of 643 drugs acting on 271 targets was assembled, comprising 1104 drug−target pairs having ≥100 published compounds per target.

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The Pharma Data

OCTOBER 26, 2020

This additional extension of the collaboration will allow us to choose the best CDX antibody for pre-clinical and clinical development.” ” Market Abuse Regulation (MAR) Disclosure. Enquiries: Hemogenyx Pharmaceuticals plc. [link]. .

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The Pharma Data

NOVEMBER 20, 2020

Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. Many Progeria patients have received continuous Zokinvy therapy for more than 10 years. PALO ALTO, Calif., 20, 2020 /PRNewswire/ — Eiger BioPharmaceuticals, Inc.

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The Pharma Data

JANUARY 15, 2021

Enhertu previously received Priority Review and Breakthrough Therapy Designation (BTD) in the U.S. About the Enhertu Clinical Development Program. for the treatment of patients with metastatic non-small cell lung cancer whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy.

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Vial

MARCH 1, 2024

Leading Global CROs To advance new therapies, pharma, biotech, and medical device sponsors engage CROs to help navigate the complex landscape of drug development and regulatory pathways and to manage clinical trials efficiently, ethically, and in compliance with good clinical practice ( GCP ) standards.

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The Pharma Data

JANUARY 20, 2021

“The approval of Verquvo provides doctors, health care professionals, and patients with a welcome new option to current available therapies.”. This approval builds upon Merck’s proud history of developing therapies for the treatment of patients with cardiovascular disease.”. Therapy was initiated at Verquvo 2.5

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The Pharma Data

JANUARY 18, 2021

This pioneering research led to the clinical development of a humanized anti-VEGF Fab (Ranibizumab, Lucentis ® ), which has also been approved as a therapy for neovascular age-related macular degeneration (AMD), retinal vein occlusion and diabetic macular edema. Enquiries: OKYO Pharma Limited. .

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The Pharma Data

NOVEMBER 10, 2020

This clinical study will evaluate the safety, tolerability, and clinical activity of escalating doses of orally administered capsules of Foralumab. “The This announcement contains inside information for the purposes of Article 7 of EU Regulation 596/2014. NEW YORK and LONDON, Nov.

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Metabolite Tales Blog

JULY 5, 2023

With metabolic stability in mind, we take a look at the metabolism of some FDA approved synthetic macrocyclic drugs, and a few of those that were/are in clinical development where metabolism data was available. A combination therapy of glecaprevir and pibrentasvir has recently been implicated in a case of liver injury.

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