Drug Target Review

JULY 21, 2025

Disorders such as Alzheimer’s disease , primary glioblastoma and amyotrophic lateral sclerosis (ALS) all affect the CNS and are considered fatal, while more common conditions, including depression , strokes and epilepsy , require long-term treatments. This makes it difficult to identify relevant and effective treatments.

Drug Development

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. Safety biomarkers account for adverse effects of a therapy under study. There are several types of biomarkers to consider.

Clinical Development

Drug Target Review

DECEMBER 23, 2024

While these therapies hold great promise for improving cancer treatment outcomes, their development presents significant challenges, especially in achieving the optimal balance between efficacy and safety. Properly managing these toxicities is crucial to enhancing the safety and therapeutic effectiveness of ADC treatments.

Research

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. The founding of RadioMedix was born from a recognition of unmet needs in cancer treatment.

Therapies

Chemical Biology and Drug Design

MAY 10, 2025

Although it is currently only used in veterinary therapy, carprofen proves to be a molecule with a versatile therapeutic potential for human pharmacotherapy. The literature review was conducted using four databases: Web of Science, ScienceDirect, Scopus, Embase, and Reaxys.

Small Molecule

Drug Target Review

JULY 8, 2025

Her team sits right at the edge of innovation, where discovery science meets clinical execution. Not only are they chasing new targets, they are also working to understand the biology behind resistance, discover biomarkers that matter and build smarter therapies from the ground up.

Therapies

Drug Target Review

MARCH 4, 2025

Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Obesity is rising worldwide, driven by lifestyle, diet, and genetics.

Therapies

Drug Target Review

OCTOBER 30, 2024

8 With the widespread use of induced pluripotent stem cells (iPSCs), organoids can now be generated from the skin or blood cells of both healthy volunteers and disease patients, opening the door to studying various diseases and creating individually customised potential treatments. 2017;23(1):49-59. Assembling human brain organoids.

Treatment

The Pharma Data

JUNE 24, 2025

This alliance aims to address mounting challenges in the development and commercialization of mAb therapies and gene therapies, particularly those involving AAV vectors. Biologic drugs, including monoclonal antibodies, have become essential in the treatment of a wide array of conditions, from autoimmune diseases to various cancers.

Therapies

Drug Target Review

JULY 15, 2025

Several strategic partnerships formed by Beckman Coulter Life Sciences illustrate how cooperative models and an open‐chemistry approach facilitating advances in genomics research. The findings underscored that automation improves data consistency and quality, thereby supporting more accurate diagnostics and personalised treatment strategies.

RNA

Drug Target Review

JULY 3, 2025

Given macro healthcare influences (eg, economic uncertainty, environmental changes) and the numerous available treatments for major diseases, drug developers may need to reassess their therapeutic strategies. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

Drug Development

Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Adapted from an Allen Institute press release.

Disease

Drug Target Review

DECEMBER 6, 2024

In cases where control or treatment groups show large variability, this could reflect true biological responses to a treatment, and removing such data could reduce the accuracy of the analysis. It is crucial during this phase to strike a balance between removing erroneous data and preserving meaningful biological variability.

RNA

Crown Bioscience

JULY 24, 2025

For many patients, resistance implies enduring multiple lines of therapy, translating into relapses and diminishing hope. But with this challenge comes an urgent call to refine our approach to treatment.

Drug Development

Drug Target Review

JUNE 17, 2025

For example, a tumour’s resistance to therapy may not stem from its mutational profile alone but from its interaction with surrounding stroma or immune cells – invisible interactions in dissociated, bulk sequencing data. Conclusion The scientific community has made remarkable progress in mapping the genome.

RNA

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

Drug Patent Watch

DECEMBER 9, 2024

In the vast realm of pharmaceutical research and development, there’s a fascinating intersection between ancient wisdom and modern science. “Pharmacognosy is the bridge between traditional medicine and modern pharmaceutical science, offering a treasure trove of potential new drugs waiting to be discovered.”

Drugs

The Pharma Data

JUNE 9, 2025

The acceptance, which follows the CDE’s grant of Priority Review status in May, represents an accelerated regulatory pathway for what may become the first approved systemic therapy for TGCT in China. Pimicotinib: A Promising CSF-1R Inhibitor Pimicotinib is an investigational small molecule developed by Abbisko Therapeutics Co.,

Treatment

The Pharma Data

JUNE 25, 2025

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

RNA

The Pharma Data

JUNE 18, 2025

The company’s lead candidate, NCX-L2 , is specifically designed to slow or even halt the progression of Parkinson’s disease—a neurodegenerative condition that currently has no disease-modifying therapies on the market. While current treatments like levodopa temporarily alleviate symptoms, none are capable of slowing or stopping neuronal loss.

Therapies

Drug Target Review

JUNE 5, 2025

Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.

Disease

Drug Target Review

JULY 30, 2025

The journey of cell and gene therapies from preclinical discovery to clinical trials is complex and challenging, impacting every team member involved, from researchers in the lab to patients receiving treatment. IRT systems dynamically generate and track expiration dates, ensuring treatments reach patients within their viable windows.

Clinical Development

Drug Target Review

JUNE 28, 2025

Amelie Croset, Senior Director of Alliances and Partnering at the therapeutics company Molecular Partners, spoke with Drug Target Review about the science behind the presentation posters, its therapeutic potential and what is next for the platform. ” And if AACR 2025 was any indication, those treatments are closer than we think.

Science

The Pharma Data

JUNE 18, 2025

The investigational ADC is built using Daiichi Sankyo’s proprietary DXd technology and represents one of the newest generation of targeted cancer therapies. Current treatment strategies for mCRPC typically begin with ARPIs such as enzalutamide or abiraterone, followed by taxane-based chemotherapy , most commonly docetaxel.

Trials

The Pharma Data

JUNE 22, 2025

Importantly, the therapy demonstrated a favorable safety profile, with no thromboembolic events reported so far. However, NXT007 takes the science a step further with refined molecular engineering aimed at achieving enhanced efficacy, longer dosing intervals, and a more predictable bleeding protection profile.

Clinical Trials

Drug Target Review

MAY 21, 2025

In an era where biotherapeutics are transforming the healthcare landscape, the organisation, Vitalant , is uniquely positioned to bridge the gap between cutting-edge therapies and community access. Building new services and treatment options for patients are a passion for me.

Therapies

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

The Pharma Data

JUNE 16, 2025

FDA Approves ANDEMBRY (garadacimab-gxii) The First Once-Monthly Prophylactic HAE Therapy Targeting Factor XIIa CSL a leading biotechnology company with a strong track record of developing innovative medicines for patients with rare and serious disorders, today announced that the U.S. when directly comparing ANDEMBRY to placebo.

FDA Approval

Drug Target Review

MARCH 17, 2025

Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.

Treatment

Drug Target Review

JUNE 6, 2025

With an extensive background in small molecule chemistry, bioconjugation and protein science, these expert teams are leading efforts to place payload development at the centre of ADC innovation. Across Sygnature Discovery , multidisciplinary teams in Canada and the UK are actively working to address these challenges.

Small Molecule

The Pharma Data

JUNE 18, 2025

This leadership transition arrives at a pivotal moment in Alnylam’s evolution, as the company builds upon the recent commercial success of AMVUTTRA® (vutrisiran) —a landmark treatment for transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

RNA

Drug Target Review

DECEMBER 16, 2024

Despite challenges as a woman in the field, Dr Hingorani built a career focused on advancing treatments for pediatric and adult cancers. I opted for medicine due to my love for biological sciences. My inclination toward biological sciences led me to medical school to pursue a future in STEM.

Medical Schools

The Pharma Data

JUNE 9, 2025

The COACH study is the first clinical investigation to assess the combined effects of two once-weekly treatments—TransCon® CNP (navepegritide) and TransCon® hGH (lonapegsomatropin)—in children with achondroplasia. TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia,” Dr. Shu stated.

Trials

Drug Target Review

JUNE 19, 2025

As the limitations of current treatments become increasingly clear, researchers are exploring new, safer approaches. His focus, however, is on addressing a critical gap in medicine: the lack of safe, non-addictive pain treatments – a challenge that has driven the development of SRP-001. “As Dr Bazan holds a B.S.

Clinical Trials

Drug Target Review

JULY 18, 2025

Immuno-oncology has transformed cancer treatment, but for many patients, tumours remain resistant to even the most advanced immune checkpoint inhibitors. This opens the door for a disease-modifying approach both as a monotherapy and in combination, expanding treatment options and offering new hope to millions of patients.”

Medical Chemistry

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 6, 2025

The data underscored the significant clinical benefits associated with cystic fibrosis transmembrane conductance regulator (CFTR) modulators—particularly its next-generation triple therapy, ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor).

Therapies