Drug Target Review

FEBRUARY 10, 2025

In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.

Therapies 64

Therapies Treatment Engineering Clinical Trials 64

Broad Institute

MARCH 11, 2025

By Count Me In Communications March 11, 2025 Count Me In , a patient-partnered research initiative led by the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute, is launching a new project focused on translocation renal cell carcinoma (tRCC), a rare and aggressive form of kidney cancer.

Research 113

Research Medical Schools Treatment Disease 113

Broad Institute

JANUARY 9, 2025

Scientists engineer CRISPR enzymes that evade the immune system By Allessandra DiCorato January 9, 2025 Breadcrumb Home Scientists engineer CRISPR enzymes that evade the immune system The new genome-editing tools could lead to safer, more efficient gene therapies. Lisa Yang Center for Autism Research at MIT, and the K.

Engineering 140

Engineering Immune Response Therapies DNA 140

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. To explore these challenges further, we spoke with Cheng Wang, a postdoctoral researcher at the University at Buffalo.

Research 52

Research Treatment Therapies Drug Development 52

The Pharma Data

JUNE 25, 2025

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

Therapies 52

Therapies Trials RNA Treatment 52

The Pharma Data

JUNE 26, 2025

FDA Approves Streamlined Monitoring Requirements and REMS Program Removal for Bristol Myers Squibb’s CAR T Cell Therapies Breyanzi and Abecma, Marking Milestone Toward Expanding Access to Cancer Treatment In a significant regulatory development, Bristol Myers Squibb announced that the U.S.

FDA Approval 40

FDA Approval Therapies FDA Treatment 40

Chemical Biology and Drug Design

JULY 3, 2025

Molecular docking and in vitro assays confirm improved binding affinity and cytotoxicity, highlighting CA4's potential as a promising drug candidate for cancer therapy. Computationally guided modifications of centaureidin led to the development of CA4, demonstrating enhanced antioxidant and antitumor activities.

Drug Development 100

Drug Development Drugs Therapies Production 100

Broad Institute

MARCH 25, 2025

Related groups Healthy Engagement in Autism Research Dialogue (HEARD) In recent years, public discussions about autism, autism research, and individuals with autism have revealed a wide range of views, at times becoming contentious. Phan : Im one of the newer perspectives entering into this conversation.

Research 64

Research Science Therapies Production 64

Drug Target Review

APRIL 30, 2025

In the rapidly evolving field of cell and gene therapy (CGT), the ability to manage complex biological data, optimise manufacturing processes, and accelerate drug discovery is crucial. How AI is solving CGTs biggest challenges Cell and gene therapy is inherently complex. Faster time-to-market and reduced costs.

Drugs 90

Drugs Therapies Clinical Trials Trials 90

Drug Target Review

JUNE 16, 2025

Targeting GPCRs to fight inflammatory diseases GPCR-targeting drugs are well known therapies for a range of disease types, including cardio-metabolic, central nervous system and endocrinological disorders. GPCR-targeting therapies such as DT-9046 represent a compelling opportunity in that respect, with major clinical and commercial potential.

Disease 59

Disease Therapies Small Molecule Immune Response 59

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. To date, more than 160 monoclonal antibody therapies targeting nearly 100 disease-related proteins have received regulatory approval globally.

Therapies 40

Therapies Biosimilars Compliance Virus 40

Chemical Biology and Drug Design

MAY 12, 2025

As the most effective gene editing method currently available, the CRISPR/Cas system has proven to be highly valuable in genomic research across a wide range of species since its discovery as a component of the adaptive immune system in bacteria. This goal is achieved through an RNA-guided procedure.

Treatment 100

Treatment Therapies DNA RNA 100

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. Delpassand’s clinical experience plays a key role in shaping the research pipeline.

Therapies 100

Therapies Treatment Drug Development Clinical Development 100

Chemical Biology and Drug Design

JANUARY 22, 2025

The future of treating challenging fungal infections lies in novel therapies targeting new antifungal targets, overcoming resistance mechanisms, and exploring innovative dual inhibitors. ABSTRACT Invasive fungal infections (IFIs) pose significant challenges in clinical settings, particularly due to their high morbidity and mortality rates.

Therapies 100

Therapies Drugs Research 100

Drug Target Review

JULY 7, 2025

Persistent challenges with NHP use Despite their biological relevance, using live NHPs in research poses several major challenges: Ethical concerns NHPs, due to their advanced cognition and social behaviour, are at the centre of ongoing ethical debates. Supply chain disruptions Availability is a critical bottleneck in NHP-based research.

Drug Research 52

Drug Research Research Animal Testing Drugs 52

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

Chemical Biology and Drug Design

JANUARY 20, 2025

This study emphasizes the distinct role of the peptide, linker, and drug for optimal PDC activity and highlights the need to carefully match components when assembling PDCs as targeted therapies.

Small Molecule 187

Small Molecule Drugs International Therapies 187

Broad Institute

JUNE 11, 2025

Broad Institute and Bayer extend their cardiovascular alliance By Leah Eisenstadt June 11, 2025 Breadcrumb Home Broad Institute and Bayer extend their cardiovascular alliance Partnership will continue with renewed focus on developing new cardiovascular therapies.

Disease 93

Disease Therapies Treatment Research 93

Broad Institute

FEBRUARY 27, 2025

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

RNA 66

RNA Therapies DNA Virus 66

Drug Target Review

JUNE 17, 2025

Combining genomics with transcriptomics, proteomics, metabolomics, spatial profiling and cellular imaging, will enable researchers to move beyond single-variable analysis and begin to see the biological system as a whole. Enter multiomics Multiomics is designed to solve this problem by observing multiple molecular layers simultaneously.

RNA 71

RNA Engineering DNA Disease 71

Alta Sciences

JULY 9, 2025

Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig aasimakopoulos Thu, 07/10/2025 - 08:00 I recently returned from attending the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. WHO WAS AT ASGCT 2025?

Therapies 52

Therapies Molecular Biology Science RNA 52

Drug Target Review

DECEMBER 20, 2024

These twins simulate how a patients condition might evolve without treatment, enabling researchers to compare the real-world effects of an experimental therapy against predicted outcomes. He was a postdoctoral researcher in mathematics at the University of Waterloo. Aaron received his Ph.D.

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

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. Ben-Simon et al.) and striatum ( Hunker et al.

Disease 72

Disease Medical Schools DNA Therapies 72

Drug Target Review

DECEMBER 6, 2024

In drug discovery and biological research, the scientists workflow often follows a structured and iterative approach to ensure accuracy, reproducibility and scientific integrity. At the heart of any research is the scientific question. This acts as a safeguard against false positives and misinterpretation of the data.

RNA 52

RNA Drugs Research Science 52

Crown Bioscience

JUNE 23, 2025

As researchers look to understand and overcome drug resistance, they need precise, controllable, and scalable models that allow them to isolate and study resistance mechanisms effectively. Drug resistance remains one of the most pressing challenges in oncology drug development, as it is the leading cause of treatment failure.

Drugs 52

Drugs Drug Development Therapies Treatment 52

Drug Target Review

JULY 8, 2025

With a background spanning drug development, clinical strategy and translational research, Eleni brings more than two decades of experience to the role. 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 52

Therapies Science Drug Development Treatment 52

FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Background on the Grants Program Launched as part of the Orphan Drug Act of 1983, this program aims to encourage research and development of drugs, biologics, medical devices, and medical foods for rare diseases, defined as conditions that affect fewer than 200,000 people in the U.S. The seven awardees included six early-stage trials (e.g.,

Clinical Development 69

Clinical Development Production Clinical Trials Treatment 69

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. Exciting new research is exploring carprofen's broader therapeutic possibilities, extending beyond its original anti-inflammatory role.

Small Molecule 100

Small Molecule Drug Development Science Therapies 100

Drug Target Review

JUNE 9, 2025

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.”

Disease 80

Disease Treatment Engineering Protein Production 80

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 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

Drug Target Review

OCTOBER 30, 2024

Creating replicas of organs in vitro has been a goal of researchers for over a century. 1 This early work laid the foundation for a century of research on finding the optimal conditions for growing parts of organs in in vitro cultures, testing their functions and even mimicking many diseases.

Treatment 105

Treatment Disease Clinical Trials Research 105

Crown Bioscience

JULY 1, 2025

These “ biological missiles ” represent an exciting new advance in anti-cancer therapies and are one of the fastest-growing segments in oncology drug development. This article introduces key assays and models available to research teams looking to investigate ADC mechanisms and develop the next generation of this exciting new drug category.

Drugs 52

Drugs Drug Development Therapies Research 52

Drug Target Review

JULY 15, 2025

Research by Markets and Markets projects the market to grow from $12.13 1 Emergen Research attributes this to the advantages of high-throughput sequencing technologies and declining sequencing costs​. 2 This trend reflects the increasing demand for genomic sequencing in research, clinical diagnostics and other applications.

RNA 91

RNA DNA Laboratories Science 91

Drug Target Review

JANUARY 14, 2025

Operating a workflow, such as the one described in Figure 1, involves several key considerations that affect both the accuracy of results and the efficiency of the research process. Defining the research question A well-defined research question is the cornerstone of an effective scientific workflow in drug discovery.

Drugs 69

Drugs Protein Expression Science Research 69

Drug Target Review

MARCH 4, 2025

Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach. The research demonstrated that obesity is not one disease, but many; each of which should be treated with different interventions, says Bagnall. These individuals respond four times better to GLP-1 medications.

Therapies 52

Therapies Treatment Clinical Trials Science 52

The Pharma Data

JUNE 23, 2025

Study Overview and Population The HYPERION study was designed to assess the efficacy and safety of WINREVAIR when used in combination with standard background therapy, compared to placebo, in adult patients recently diagnosed with pulmonary arterial hypertension. ZENITH, the second pivotal Phase 3 trial, presented at ACC.25

Trials 40

Trials Therapies Treatment Disease 40

Chemical Biology and Drug Design

MARCH 13, 2025

These findings position PTB derivatives as promising candidates for targeted breast cancer therapy. Compounds B8 , B14 , and B18 exhibit ~20-fold lower IC 50 values than tamoxifen, induce early-stage apoptosis, and arrest the cell cycle at the G1 phase via caspase-9 activation.

Therapies 100

Therapies Research 100