Drug Target Review

JULY 3, 2023

There is a significant unmet need for effective treatments to alleviate suffering and prevent premature death across the spectrum of genetic diseases. Unfortunately, the majority of genetic diseases lack a cure, leaving limited therapeutic options available.

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FDA Law Blog: Biosimilars

OCTOBER 14, 2024

California Stem Cell Treatment Center, Inc., At the clinics, Defendants offer treatments that consist of extracting fat tissue from patients and, through a multi-step process, turning that tissue into a liquefied mixture of stem cells, other cells and cell debris known as Stromal Vascular Fraction (SVF). By Steven J. 21 U.S.C. §

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Drug Target Review

JANUARY 15, 2024

Cancer is a disease driven by variable genetic mutations. Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Additionally, in evaluating the effectiveness of our initial pipeline asset, we sought tumours suitable for intratumoural injection that present surface-accessible lesions.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The gene presents a complex landscape. million DNA bases.

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ASPET

JANUARY 23, 2024

Patients with sickle cell disease (SCD) display priapism, a prolonged penile erection in the absence of sexual arousal. The current pharmacological treatments for SCD-associated priapism are limited and focused on acute interventions rather than prevention.

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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The Pharma Data

NOVEMBER 9, 2021

– More than 30 Abstracts Across HDV, HCV, HBV, NASH and PSC Reflect Gilead’s Ongoing Commitment to Addressing Challenges Facing People Living With Liver Diseases –. Donations include the impact of treatment with bulevirtide, an investigational treatment for people with habitual hepatitis delta contagion (HDV) in theU.S.

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Drug Target Review

AUGUST 31, 2023

In the pursuit of a remedy for Alzheimer’s disease, a frontier in medical science is illuminating a glimmer of hope. Chronic inflammation involving microglia has been linked to the disease, as the release of inflammatory molecules triggers an increase in β-amyloid production.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Using humans as the model, we use an approach called deep phenotyping to explore the relationships between cells, genes, biological pathways and patterns of disease. At present, we test discarded human livers deemed not good enough for transplant.

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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.

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biobide

FEBRUARY 2, 2024

Pediatric cancers exhibit differences at the genetic level compared to the same form of adult disease, which may influence the selected treatment. Zebrafish Pioneers Advance in Pediatric Cancer Research and Treatment Increasing the survival rate is the primary goal when screening for cancer drugs.

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The Pharma Data

FEBRUARY 24, 2022

Allergan, an AbbVie (NYSE: ABBV) company, today announced new data to be presented from Allergan’s leading portfolio of eye care treatments at the 2022 American Glaucoma Society (AGS) Annual Meeting being held in Nashville, TN and virtually from March 3-6. Robinson, M.D.,

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Conversations in Drug Development Trends

AUGUST 29, 2024

By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.

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DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. –5.9%

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Chemical Biology and Drug Design

SEPTEMBER 20, 2023

Ovarian cancer is the most lethal gynecologic cancer with recurrent or resistant disease observed in 70% of patients. The combined treatment successfully enhanced cisplatin cytotoxicity and helped to overcome cisplatin resistance in CR-Caov3 cells.

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). Importantly, transcriptomics information shows that certain aspects of a disease change the gene expression profile.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? By encapsulating specific self-antigens within our biodegradable nanoparticles, we enable the targeted delivery of the CNPs to antigen-presenting cells (APCs) primarily in the spleen and liver.

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Drug Target Review

AUGUST 29, 2024

However, these treatments will eventually fail for the majority of patients, so there is a strong need for better CAR therapies. How might the CD5 knockout approach impact the treatment of other types of cancers beyond T-cell lymphoma?

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Drug Target Review

JANUARY 24, 2024

Scientists from Northwestern Medicine have demonstrated that RNA interference could have a crucial role in the onset and development of Alzheimer’s disease (AD). Alzheimer’s disease Alzheimer’s is the most common cause of dementia. The team found very short sequences present in some of these sRNAs.

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Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. Preeclampsia may present at any gestation but is more commonly encountered in the third trimester.

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Conversations in Drug Development Trends

APRIL 5, 2024

For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.

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ASPET

JUNE 4, 2024

The goal of the present study was to determine the antiarthritic potential of the minor cannabinoid 8 -THC using the collagen-induced arthritis (CIA) mouse model. The 8 -THC treatment (30 mg/kg) reduced paw swelling and qualitative signs of arthritis.

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The Pharma Data

NOVEMBER 23, 2021

Roche moment blazoned that new data from its expansive haematology portfolio will be presented at the American Society of Hematology (ASH) Annual Meeting and Exposition from 11-14 December 2021. Data presented at ASH by Roche span multitudinous blood conditions, including carcinoma, leukaemia, multiple myeloma and haemophilia.

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The Pharma Data

MAY 22, 2023

DUPIXENT® (DUPILUMAB) LATE-BREAKING PHASE 3 COPD RESULTS PRESENTED AT ATS AND SIMULTANEOUSLY PUBLISHED IN THE NEW ENGLAND JOURNAL OF MEDICINE Regeneron Pharmaceuticals, Inc. The Dupixent Phase 3 results were presented in the 2023 American Thoracic Society (ATS) International Conference session “New England Journal of Medicine and JAMA.

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The Pharma Data

AUGUST 7, 2021

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. LOPD symptoms may present at any age.

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The Pharma Data

APRIL 28, 2022

Researchers at Karolinska Institutet in Sweden have now developed a method for identifying the immune cells involved in autoimmune diseases, and have identified four new target molecules of potential significance for future personalised treatment of MS. This way of creating personalised treatment is called precision medicine.

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The Pharma Data

OCTOBER 27, 2020

Cowen Virtual Presentation Series – 2020 IO Next Summit at 1:15 p.m. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. EST on Monday, November 9, 2020. EST on Friday, November 13, 2020. EST ( 12:20 p.m.

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The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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The Pharma Data

JULY 29, 2021

Eisai and Biogen Present Preliminary Assessment of the Clinical Effects of Lecanemab Following 18 Months of Treatment in the Open-Label Extension of the Phase 2 Proof of Concept Study at 2021 Alzheimer’s Association International Conference (AAIC) . United States and virtually from July 26 to 30, 2021 (Presentation No.:

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The Pharma Data

MARCH 28, 2022

Kerendia ™ (10 mg or 20 mg), a non-steroidal, selective mineralocorticoid receptor (MR) antagonist, is approved for the treatment of chronic kidney disease and type 2 diabetes, excluding patients with end-stage renal disease or on dialysis. Finerenone is different to existing CKD in T2D treatments.

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The Premier Consulting Blog

OCTOBER 11, 2022

Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases.

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The Pharma Data

JUNE 7, 2023

The research presented reinforces the efficacy of LUMAKRAS ® /LUMYKRAS ® (sotorasib) in advanced non-small cell lung cancer (NSCLC) and metastatic colorectal cancer (mCRC). This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

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Chemical Biology and Drug Design

AUGUST 7, 2023

Xanthine oxidase (XO) inhibitors are commonly used to treat gout, nephropathy, and renal stone diseases related to hyperuricemia. The present review discusses the many synthetic and natural XO inhibitors that have been developed which are found to have greater potency.

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The Pharma Data

MARCH 8, 2021

The data were presented at the 28th Conference on Retroviruses and Opportunistic Infections ( virtual CROI 2021). No treatment-emergent resistance to any components of Biktarvy occurred in participants treated with Biktarvy. BOXED WARNING: POST TREATMENT ACUTE EXACERBATION OF HEPATITIS B. Study 1489 and Study 1490 are ongoing.

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The Pharma Data

SEPTEMBER 6, 2021

First data to be presented from the phase II coopERA Breast Cancer study evaluating neoadjuvant giredestrant treatment for oestrogen receptor (ER)-positive, HER2-negative breast cancer. Roche’s Chief Medical Officer and Head of Global Product Development. Lung Cancer Highlights.

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Broad Institute

OCTOBER 3, 2024

Largest-ever genetic study of epilepsy finds possible therapeutic targets By Ari Navetta October 3, 2024 Breadcrumb Home Largest-ever genetic study of epilepsy finds possible therapeutic targets Scientists have uncovered new genetic links to different types of epilepsy, which could lead to more tailored treatments.

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The Pharma Data

MARCH 5, 2021

Biogen (Nasdaq: BIIB) today announced it will host webcasts of its pre-recorded presentations and live discussions related to its Alzheimer’s disease investigational therapy, aducanumab, at the upcoming AD/PDTM 2021 Virtual Conference. An archived version of the webinar will be available following the presentation.

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