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

Science Daily: Pharmacology News

MARCH 7, 2024

Four children have remained free of detectable HIV for more than one year after their antiretroviral therapy (ART) was paused to see if they could achieve HIV remission, according to new research.

Treatment 318

Treatment Clinical Trials Therapies Trials 318

Science Daily: Pharmacology News

FEBRUARY 22, 2024

A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.

Therapies 222

Therapies Clinical Trials Treatment Disease 222

Science Daily: Pharmacology News

OCTOBER 18, 2023

Glioblastoma (GBM), an aggressive brain cancer, is notoriously resistant to treatment, with recurrent GBM associated with survival of less than 10 months.

Therapies 217

Therapies Immune Response Research Virus 217

Science Daily: Pharmacology News

SEPTEMBER 4, 2024

Mice given the treatment had improved recovery from injuries, demonstrating potential for developing the therapy for people. Researchers have designed, in mice, an approach to minimizing the damage from a spinal cord injury through the use of engineered immune cells.

Engineering 341

Engineering Therapies Treatment Research 341

Science Daily: Pharmacology News

FEBRUARY 10, 2025

A revolutionary new treatment called Targeted Thermal Therapy (Tripel T) offers a safer, faster alternative to surgery for patients with high blood pressure caused by Primary Aldosteronism, a hormonal disorder.

Treatment 190

Treatment Therapies 190

Science Daily: Pharmacology News

JULY 26, 2024

New therapy uses synthetic nanofibers to mimic the natural signaling of a protein that is crucial for cartilage formation and maintenance. After just four hours, the treatment activated the gene expression necessary to generate cartilage.

Therapies 322

Therapies Treatment Research 322

Science Daily: Pharmacology News

APRIL 9, 2025

Researchers have discovered a way to get anti-inflammatory medicine across the blood-brain barrier, opening the door to potential new therapies for a range of conditions, including Alzheimer's disease, multiple sclerosis, Parkinson's disease and cancer cachexia.

Treatment 261

Treatment Therapies Disease Drugs 261

Science Daily: Pharmacology News

JULY 31, 2023

Pain experts suggest that scrambler therapy, a noninvasive pain treatment, can yield significant relief for approximately 80% -- 90% of patients with chronic pain, and it may be more effective than another noninvasive therapy: transcutaneous electrical nerve stimulation (TENS).

Therapies 190

Therapies Treatment 190

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

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 52

Therapies Treatment Clinical Trials Science 52

Science Daily: Pharmacology News

AUGUST 28, 2024

Preliminary trials show the therapy relieves pain after a single treatment. Engineers have developed a device that noninvasively stimulates deep brain regions, potentially disrupting the faulty signals that lead to chronic pain.

Engineering 298

Engineering Therapies Trials Treatment 298

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 100

Therapies Treatment Drug Development Clinical Development 100

Drug Target Review

DECEMBER 20, 2024

Smith identifies three key obstacles preventing broader acceptance of AI: Communication gaps : Pharmaceutical and computational science communities often struggle to understand each others technical languages, making collaboration difficult. As a result, 2025 could be the year AI truly transforms the pharmaceutical sector.

Clinical Trials 128

Clinical Trials Trials Pharmaceuticals Drug Development 128

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

Science Daily: Pharmacology News

AUGUST 18, 2023

Novel stem cell therapy called a CALEC transplant was performed safely in four patients with serious chemical burns tracked in a phase I clinical trial for 12 months. The patients experienced early clinical gains: two were able to undergo a corneal transplant and two reported significant improvements in vision without additional treatment.

Therapies 188

Therapies Trials Clinical Trials Treatment 188

Science Daily: Pharmacology News

JUNE 5, 2024

Blood stem cells -- key to transplants that are used as life-saving treatments for blood cancers and blood and immune disorders -- have the capacity to self-renew, but quickly lose their ability to do so in a lab dish.

Therapies 330

Therapies Treatment 330

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. Zylberberg has built an impressive career at the intersection of science, technology, and business.

Drugs 90

Drugs Therapies Clinical Trials Trials 90

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 100

Small Molecule Drug Development Science Therapies 100

Science Daily: Pharmacology News

FEBRUARY 14, 2024

A groundbreaking scientific study has unveiled a remarkable discovery that may have far-reaching implications for the treatment of heart disease. The potential for developing novel therapies that leverage the body's innate regenerative capacity holds great promise for millions of individuals affected by heart disease worldwide.

Disease 298

Disease Therapies Treatment 298

Science Daily: Pharmacology News

MAY 29, 2024

Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments. Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix.

RNA 293

RNA DNA Therapies Treatment 293

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 52

Disease Treatment Therapies Nurses 52

Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies 137

Therapies FDA Approval Engineering Disease 137

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

PPD

JUNE 20, 2025

While two approved treatments, pirfenidone and nintedanib, slow disease progression, there currently is no treatment that reverses the effects or offers a cure for IPF. However, recent advancements and strategic approaches in clinical trials offer hope that additional treatments are on the way. It’s not for lack of trying.

Clinical Development 52

Clinical Development Treatment Clinical Trials Trials 52

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 52

Research Treatment Therapies Drug Development 52

Science Daily: Pharmacology News

FEBRUARY 2, 2024

A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher.

Therapies 259

Therapies Treatment Research 259

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 52

Therapies Science Drug Development Treatment 52

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 52

Treatment Licensing Licensing Science 52

Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

Therapies 116

Therapies Engineering Treatment Molecular Biology 116

Drug Target Review

NOVEMBER 17, 2023

They do not induce ‘graft versus host’ disease when transplanted for allogeneic therapy and there appears little sign of immune rejection. Peripheral blood NK cells have been used in allogeneic cancer therapies and shown to be safe. Only three of the CAR-NK studies were for the treatment of solid tumours.

Therapies 118

Therapies Clinical Trials Cell Biology Trials 118

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

Covalent Modifiers

SEPTEMBER 7, 2023

Science 2023 , 380, 1349-1356 DOI: 10.1126/science.adh0614 Millions who live in Latin America and sub-Saharan Africa are at risk of trypanosomatid infections, which cause Chagas disease and human African trypanosomiasis (HAT). These findings suggest a potential approach toward successful therapeutics for the treatment of Chagas disease.

Treatment 173

Treatment Disease Science Therapies 173

Covalent Modifiers

FEBRUARY 6, 2024

Proceedings of the National Academy of Sciences 2024 , 121 (6), e2317756121. The most common adverse effects are hyperphosphatemia caused by FGFR1 inhibition and diarrhea due to FGFR4 inhibition, as current therapies are not selective among the FGFRs. .; Chen, R.; Watters, J.; Bergstrom, D.

Small Molecule 223

Small Molecule Treatment Science Therapies 223

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 40

Therapies Biosimilars Compliance Virus 40

Perficient: Drug Development

JANUARY 14, 2025

Kristen Quick, Director at Perficient, shared her insights on the transformative potential of Salesforce Life Sciences Cloud for pharmaceutical and med tech organizations, highlighting its ability to drive innovation and improve outcomes. Future Advancements and Innovations The power of Life Sciences Cloud is just beginning.

Science 52

Science Compliance Clinical Trials Therapies 52

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 40

Treatment Therapies Small Molecule Marketing 40