Drug Target Review

JUNE 6, 2025

At the forefront of this transformation is Dr Ebrahim Delpassand, a nuclear medicine physician and the driving force behind RadioMedix (RMX), a radiopharmaceutical company focused on developing targeted diagnostics and therapies. These measures ensure both the efficacy and safety of the final product.

Therapies 100

Therapies Treatment Drug Development Clinical Development 100

PPD

MARCH 13, 2024

This significance and focus are ever more apparent when the starting cellular material is imperative to drug product success. Within cell therapy clinical trials, apheresis collections contribute to the specific constituents of the given therapy.

Clinical Development 98

Clinical Development Clinical Trials Therapies Trials 98

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%.

Therapies 40

Therapies Treatment Clinical Development Trials 40

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. As risks are identified, customized action plans are created and provided to clinical teams to keep studies on track.

Clinical Trials 95

Clinical Trials Trials Clinical Research Drug Development 95

SugarCone Biotech

MARCH 2, 2025

Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. We recently viewed quite positive updates from Scholar Rock who have a TGF--1 antagonist in the clinic. So, you cannot link antagonist targeting these two pathways together, at least in the TME.

Therapies 67

Therapies Clinical Trials Trials Clinical Development 67

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.

Therapies 97

Therapies Clinical Trials Clinical Research Trials 97

LifeSciVC

APRIL 24, 2024

Additionally, for illustrative reasons this is geared towards a single target / product focus vs. broader platform diligence, though many of these mental models will apply for selecting targets and indications for a platform. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)?

Production 132

Production Small Molecule Regulations Trials 132

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.

Therapies 52

Therapies FDA Clinical Development Production 52

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 states that only one 90-minute pre-BLA meeting will typically be granted for a specific product or indication planned for an original marketing application.

Therapies 59

Therapies FDA Production Disease 59

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.

Production 52

Production Therapies Clinical Development 52

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.

Clinical Development 52

Clinical Development Drug Development Therapies Production 52

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.

Production 52

Production Therapies DNA Virus 52

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.

Immune Response 52

Immune Response Therapies Treatment Clinical Trials 52

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.

Treatment 105

Treatment Packaging Clinical Research Therapies 105

Conversations in Drug Development Trends

JULY 3, 2025

Approximately two in 10 patients with hematologic malignancies are estimated to be eligible and able to receive cell therapy. The REMS Burden Explained Once deemed necessary for managing the risks associated with CAR T therapies, the REMS requirements imposed significant operational burdens on treatment centers.

Therapies 40

Therapies Clinical Trials Regulations FDA 40

The Pharma Data

JUNE 24, 2025

The partnership represents a major financial and strategic milestone for Revolution Medicines, which has built a robust pipeline of targeted therapies aimed at cancers driven by mutations in the RAS family of genes—some of the most prevalent and historically difficult-to-drug oncogenes in human cancer.

Drug Development 40

Drug Development Clinical Trials Therapies Drugs 40

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.

Clinical Development 52

Clinical Development Therapies Clinical Trials Treatment 52

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.

Trials 98

Trials Clinical Trials Clinical Development Clinical Research 98

The Pharma Data

JUNE 24, 2025

Royalty Pharma and Revolution Medicines Forge $2 Billion Strategic Funding Pact to Accelerate Global Development of RAS Cancer Therapies Royalty Pharma plc, a prominent funder of innovation in the biopharmaceutical industry, announced a transformative agreement with Revolution Medicines, Inc.,

Clinical Development 40

Clinical Development Packaging Therapies Treatment 40

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.

Therapies 52

Therapies Clinical Trials FDA Production 52

BioPharma Drive: Drug Pricing

JUNE 23, 2025

Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Thus, these drugs best complement, rather than replace, healthy lifestyle interventions.

Treatment 130

Treatment Drugs Therapies FDA 130

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. Developing effective ECs requires more than just matching clinical trial inclusion and exclusion criteria within the RWD source.

Drug Development 52

Drug Development Clinical Trials Disease Trials 52

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).

Therapies 59

Therapies Engineering Production FDA 59

The Pharma Data

JULY 2, 2025

per share and brings Inozyme’s promising late-stage therapeutic candidate, INZ-701, into BioMarin’s growing pipeline of innovative enzyme therapies. BioMarin, a recognized leader in the development of therapies for genetic conditions, believes that INZ-701 has the potential to become a first-in-class treatment for this serious disease.

Therapies 40

Therapies Disease Clinical Development Treatment 40

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.” A considerable roadblock in the development of RMT products is a lack of regulatory predictability.

Therapies 59

Therapies FDA Production Regulations 59

Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

Science 145

Science Disease FDA Approval Pharmaceutical Companies 145

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.

Drugs 64

Drugs Clinical Trials Drug Development Pharmaceuticals 64

The Pharma Data

JUNE 22, 2025

Importantly, the therapy demonstrated a favorable safety profile, with no thromboembolic events reported so far. No thromboembolic events—an important safety consideration for all coagulation therapies—were reported. Engineered by Chugai Pharmaceutical Co., Engineered by Chugai Pharmaceutical Co.,

Clinical Trials 40

Clinical Trials Therapies Treatment Trials 40

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).

Trials 40

Trials Therapies Treatment Disease 40

The Pharma Data

JUNE 26, 2025

From our innovative discovery engine to one of the most comprehensive pipelines in the oncology space, we are uniquely positioned to deliver transformative therapies with speed, precision, and purpose.” BeOne Medicines’ rise has been fueled by a strategic commitment to innovation at every level of the drug development continuum.

Research 40

Research Clinical Development Clinical Trials Engineering 40

The Pharma Data

JUNE 25, 2025

Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.

FDA 40

FDA FDA Approval Therapies Drugs 40

The Pharma Data

JUNE 19, 2025

Grifols’ BT524 Fibrinogen Therapy Shows Promise in Phase 3 Trial Published in The Lancet’s eClinicalMedicine Grifols , a globally recognized healthcare company and a leader in the production of plasma-derived therapies, has marked a significant advancement in the treatment of acquired fibrinogen deficiency (AFD).

Therapies 40

Therapies Trials Clinical Trials Medical Schools 40

PPD

MARCH 26, 2024

A large majority of drug developers utilize FSP or hybrid FSP/FSO models, half are “heavy users” Heavy users: Those that use FSP or hybrid FSP/FSO models for most of their clinical development outsourcing. Heavy users leverage these models for nearly three-quarters of their outsourcing.

Clinical Research 98

Clinical Research Pharmaceutical Companies Clinical Development Drug Development 98

The Pharma Data

DECEMBER 22, 2020

Merck will assist with preclinical development and has the option to exclusively develop, manufacture and commercialize the candidate following Phase 1. Under the agreement A2 will receive an upfront payment, and will be eligible for opt-in and milestone payments, plus royalties on sales of any approved product.

Therapies 52

Therapies Research Laboratories Engineering Laboratories 52

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.

Clinical Development 40

Clinical Development Disease Clinical Trials Therapies 40

DrugBank

SEPTEMBER 26, 2024

This reduction in cost does not equate to a compromise in quality or efficacy, as biosimilars undergo rigorous analytical and functional characterization to demonstrate biosimilarity to the reference product. biosimilars are often priced slightly lower than their reference products, limiting their potential for cost savings.

Biosimilars 98

Biosimilars Clinical Trials Production Marketing 98

The Pharma Data

OCTOBER 14, 2020

15, 2020 /PRNewswire/ — Thermo Fisher Scientific announced the launch of its Gibco CTS Rotea Counterflow Centrifugation System, a modular, closed cell therapy processing system that enables scalable, cost-effective cell therapy development and manufacturing. CARLSBAD, Calif. ,

Therapies 52

Therapies Clinical Trials Science Laboratories 52