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

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

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

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

PPD

JUNE 20, 2025

The road ahead for idiopathic pulmonary fibrosis treatments The landscape of idiopathic pulmonary fibrosis treatment development is poised to evolve significantly. With the expiration of pirfenidone’s patent exclusivity in 2021, sponsors have renewed their focus on developing new therapies.

Clinical Development

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

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

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

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

PPD

JULY 29, 2025

These elongated timelines are contributing to soaring costs, with the average cost of developing a new drug rising to approximately $2.6 For sponsors, the imperative to streamline development processes and improve clinical development speed and efficiency has never been more critical. billion (versus $1-1.5

Clinical Trials

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

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

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

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

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

The Pharma Data

JUNE 24, 2025

Over the years, the collaboration has matured into one of the most productive academic-pharma partnerships in China. Oncology remains a core priority, reflecting the rising global burden of cancer and the need for more effective, targeted therapies.

Pharma R&D

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

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

DrugBank

JUNE 18, 2025

Drug development is a complex and highly regulated process. Before a therapy can be approved for patient use, it must undergo extensive clinical testing and strictly adhere to regulatory guidelines. This underscores the role of regulatory oversight in ensuring manufacturing consistency and product safety.

Drug Development

The Pharma Data

JUNE 22, 2025

Exelixis Reports Positive Phase 3 Results for Zanzalintinib and Atezolizumab Combo in Metastatic Colorectal Cancer Exelixis , has announced promising topline results from the STELLAR-303 phase 3 pivotal clinical trial, a major milestone in the development of its investigational therapy zanzalintinib. Amy Peterson, M.D.,

Trials

Alta Sciences

JULY 7, 2025

The focus is to ensure that the drug does not have any potential to induce an excessive immune response, particularly cytokine storm, which can lead to severe adverse effects and impede clinical development. CAR T cells) WHAT IS THE BENEFIT OF NONCLINICAL CYTOKINE RELEASE EVALUATION?

Immune Response

The Pharma Data

JUNE 16, 2025

The decision to proceed to Phase III underscores Roche’s continued commitment to developing disease-modifying treatments for Parkinson’s disease — a neurodegenerative condition affecting more than 10 million people globally. This further strengthens the biologicial rationale for proceeding into a larger and more definitive Phase III trial.

Disease

The Pharma Data

JUNE 6, 2025

Over the past five decades, it has grown steadily, earning global recognition for developing therapeutic solutions for some of the most challenging and underserved conditions in medicine. The company actively seeks partnerships with academic institutions, research organizations, and biopharma peers to co-develop transformative therapies.

Pharmaceuticals

The Pharma Data

JULY 2, 2025

Importantly, the treatment effect emerged early in the course of therapy and continued to increase over time. Despite existing therapies, many PAH patients remain at substantial risk. Patients were randomized 1:1 to receive either WINREVAIR or placebo, both in combination with maximum-tolerated background therapy. Source link

Trials

The Pharma Data

JUNE 20, 2025

The new recommendation supports the use of darolutamide in combination with androgen deprivation therapy (ADT) for patients with metastatic hormone-sensitive prostate cancer (mHSPC) in the European Union (EU). The Urgent Need for New Prostate Cancer Therapies The need for innovative therapies in prostate cancer remains critical.

Trials

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

Drug Target Review

MAY 26, 2025

With a particular focus on biologics, cell, and gene therapies, Galbraiths work is centred on equipping drug developers with the tools, insights, and support needed to advance their therapies from concept to clinic. We are driving rapid analytics to support the release of cell and gene therapies and other biologics.

Drug Development

BioPharma Drive: Drug Pricing

JULY 10, 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. Since the drug’s U.S. By Jonathan Gardner • Updated April 9, 2025 Keep up with the story.

Drugs

New Drug Approvals

MAY 15, 2025

A final lipophilicity adjustment led to the discovery of CDZ173 (leniolisib), a potent PI3K selective inhibitor with suitable properties and efficacy for clinical development as an anti-inflammatory therapeutic. New Drug Therapy Approvals 2023 (PDF). Pharming Group N.V. Press release). 24 March 2023. Retrieved 25 March 2023. ^

FDA Approval

New Drug Approvals

JULY 8, 2025

It is currently in Phase III clinical development by the German biopharmaceutical company AiCuris Anti-infective Cures AG. US FDA granted fast track designation for pritelivir in 2017 and breakthrough therapy designation 2020. The product is isolated by filtration and washed with purified water. Crute, J.J.; Tsurumi, T.;

Virus

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

PPD

APRIL 14, 2025

This expansion is creating opportunities for clinical trials related to a range of new therapy areas and their subpopulations. Expertise that accelerates your clinical development success The PPD clinical research business of Thermo Fisher Scientific has significant experience in customized Phase I-IV metabolic clinical trials.

Clinical Trials

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

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

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

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

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